Medical skin care products are used for beautifying or to address some other skin care problems. The cosmetic industry is booming and skin care forms a very huge part of this industry. The aesthetic appearance is so important that people spend a lot on skin care products and treatment. People being more technologically aware of the various new skin care products trending in the market. In addition to the aesthetic application, the medical skin care products are also used to address issues such as acne, pimples or scars.

Get Sample Copy of Report @https://www.persistencemarketresearch.com/samples/18469

Medical Skin Care Products Market: Drivers and Restraints

The medical skin care products is primarily driven by the need of natural based active ingredients products which are now trending in the market. Consumers demand medical skin care products which favor health and environment. Moreover, the consumers are updated with the trends so that various companies end up providing such products to satisfy the customers. For instance, a single product face mask has thousands of different variants. This offers consumers different options to select the product depending on the skin type. Moreover, the market players catering to the medical skin care products are offering products with advanced technologies. For instance, Santinov launched the CICABEL mask using stem cell material based on advanced technologies. The stem cells used in the skin care product helps to to protect and activate the cells and promote the proliferation of skin epidermal cells and the anagenesis of skin fibrosis.

Medical Skin Care Products Market: Segmentation

On the basis of product type the medical skin care products market can be segmented as:

On the basis of application, the medical skin care products market can be segment as:

On the basis of distribution channel, the medical skin care products market can be segment as:

Medical Skin Care Products Market: Overview

Medical skin care products are used to address basic skin problems ranging from acne to scars. There are various advancements in the ingredients used to offer skin care products to the consumers. For instance, the use of hyaluronic acid and retinoids is the latest development in the industry. The anti-aging creams are at the forefront as the help treating issues such as wrinkles, scars, acne, and sun damage. Another, product in demand is the probiotic skincare which include lactobacillus and bifidobacterium.

Request Customization of this Report @https://www.persistencemarketresearch.com/request-customization/18469

Medical Skin Care Products Market: Region-wise Outlook

In terms of geography, medical skin care products market has been divided into five regions including North- America, Asia- Pacific, Middle-East & Africa, Latin America and Europe. North America dominated the global medical skin care products market as international players are acquiring domestic companies to make their hold strong in the U.S. LOral is accelerating its U.S. market by signing a definitive agreement with Valeant Pharmaceuticals International Inc. to acquire CeraVe, AcneFree and Ambi skin-care brands for US$ 1.3 billion. The acquisition is expected LOreal to get hold of the brands in the price-accessible segment. Asia Pacific is expected to be the fastest growing region owing to the increasing disposable income and rising awareness towards the skin care products.

Medical Skin Care Products Market: Key Market Participants

Some of the medical skin care products market participants are Avon Products Inc., Beiersdorf AG, Colgate-Palmolive Company, Kao Corporation, LOral S.A., Procter & Gamble, Shiseido Company, The Estee Lauder Companies Inc., Unilever PLC, Revlon, Clinique Laboratories, llc., Murad, LLC., SkinCeuticals, RMS Beauty, J.R. Watkins and 100% PURE.

Read the original here:
Medical Skin Care Products Market to Witness a Pronounce Growth During 2017 2025 - Zebvo

Skin Stem Cells Comments Off on Medical Skin Care Products Market to Witness a Pronounce Growth During 2017 2025 – Zebvo | November 8th, 2019

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (NASDAQ: AVRO) (the Company), a Phase 2 clinical-stage gene therapy company, today reported financial results for the third quarter ended September 30, 2019 and provided a business update.

We are thrilled with the progress across our pipeline, including the dosing of the first patient in our cystinosis program and receipt of orphan drug designation for our investigational gene therapy for Gaucher disease, commented Geoff MacKay, President and Chief Executive Officer of AVROBIO. In our Fabry program, we have now dosed eight patients across two clinical trials and we are on track to use our optimized lentiviral vector and a conditioning regimen utilizing therapeutic drug monitoring for the first time to dose a patient in our Phase 2 clinical trial for Fabry disease by the end of 2019. While our rapid expansion and early data have been exciting, we are humbled by the needs of the rare disease communities with whom we engage. They impress a sense of urgency on our work to deliver a new paradigm that we believe can supersede current treatment options and potentially provide patients freedom from a lifetime of disease.

Program Updates and Milestones

Third Quarter 2019 Financial Results

AVROBIO reported a net loss of $17.1 million for the third quarter of 2019 as compared to a net loss of $11.6 million for the comparable period in 2018. This increase was due to increased research and development expenses, as well as increased general and administrative expenses.

Research and development expenses were $13.0 million for the third quarter of 2019 as compared to $9.2 million for the comparable period in 2018. This increase was driven by increased program development activities related to the advancement of the Companys pipeline, as well as increased personnel-related costs resulting from an increase in employee headcount.

General and administrative expenses were $5.0 million for the third quarter of 2019 as compared to $3.0 million for the comparable period in 2018. This increase was primarily due to an increase in employee headcount, expenses associated with being a publicly traded company, including consulting expenses, and the impact of non-cash stock-based compensation.

As of September 30, 2019, AVROBIO had $206.4 million in cash and cash equivalents, as compared to $126.3 million in cash and cash equivalents as of December 31, 2018. The cash balance as of September 30, 2019 reflects the receipt of net proceeds of $129.5 million from the Companys July 2019 follow-on equity offering. Based on the Companys current operating plan, AVROBIO expects its cash and cash equivalents as of September 30, 2019 will enable the Company to fund its operating expenses and capital expenditure requirements into the second half of 2021.

About AVROBIO, Inc.

AVROBIO, Inc. is a leading, Phase 2 gene therapy company focused on the development of its investigational gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. The Companys plato platform includes a proprietary vector system, automated cell manufacturing solution and refined conditioning regimen deploying therapeutic drug monitoring. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit http://www.avrobio.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as aims, anticipates, believes, could, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy, prospective products and goals, the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, the intended incentives conferred by orphan-drug designation, potential regulatory approvals and the timing thereof, expected benefits from the appointment of Ms. Verdin to the position of Chief Human Resources Officer and Ms. May to the position of Chief Commercial Officer, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, plans and objectives of management for future operations, future results of anticipated products, and the market opportunity for and anticipated commercial activities relating to our product candidates, and statements regarding the Companys financial and cash position and expected cash runway. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs Quarterly Report on Form 10-Q for the quarter ended June 30, 2019, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

CONDENSED CONSOLIDATED BALANCE SHEETS

(In thousands)

(Unaudited)

September 30,

December 31,

2019

2018

Cash and cash equivalents

$

206,362

$

126,302

Prepaid expenses and other current assets

7,345

3,718

Property and equipment, net

2,673

2,634

Other assets

825

825

Total assets

$

217,205

$

133,479

Accounts payable

$

1,408

$

2,784

Accrued expenses and other current liabilities

8,502

7,822

Deferred rent, net of current portion

535

689

Total liabilities

10,445

11,295

Total stockholders equity

206,760

122,184

Total liabilities and stockholders equity

$

217,205

$

133,479

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

(In thousands, except share and per share data)

(Unaudited)

Three Months Ended September 30,

Nine Months Ended September 30,

2019

2018

2019

2018

Operating expenses:

Research and development

$

13,042

$

9,232

$

37,755

$

22,286

General and administrative

5,022

Continued here:
AVROBIO, Inc. Reports Third Quarter 2019 Financial Results and Provides Business Update - Business Wire

Skin Stem Cells Comments Off on AVROBIO, Inc. Reports Third Quarter 2019 Financial Results and Provides Business Update – Business Wire | November 8th, 2019

Arizona has been called "the wild, wild west" of regenerative medicine.

The Valley is one of the most popular places in the country for stem cell clinics. The new and controversial therapy is being marketed and practiced all over Phoenix and Scottsdale.

The less invasive procedure promises to heal pain, nearly anywhere in their body. It is advertised as effective, safe, and ethical, but outside experts and industry insiders say consumers need to do their research to avoid being exploited, and potentially spending thousands in cash on a worthless injection.

"IT HAS GREAT POTENTIAL"

The world of regenerative medicine is still being explored and developed.

"It actually gives you really good results," explained Dr. Matthew Hernandez, a naturopathic physician with Ethos.

"There's a lot of hope and promise, generally around the prospects for stem cells," said ASU Professor Emma Frow.

"Were still in the developmental stage. Stem cell therapy has been around for less than ten years. Thats new in medicine," said Dr. Steven Sorr, a naturopathic physician who runs Source of Health in Scottsdale.

"It encourages your own body to heal itself," said Janet McConnell, a 63-year-old bodybuilder who "had cartilage damage several years ago."

Instead of a surgery that would have derailed her competition training for months, she opted for injections.

"Three years ago, instead of the surgery, I had a PRP treatment," said McConnell. "It was very effective."

Years later, she returned to Dr. Hernandez for another round.

For most, Stem Cell and Platelet Rich Plasma (PRP) therapy is a mystery. "It's kind of controversial and experimental," said Matthew Riddle, Director of Sales for Celling Biosciences.

The treatments concentrate platelets or stem cells, usually from the patient's own blood. Experts say it is important to always ask the doctor or provider where the "growth factors" are coming from, because in order to ensure they are alive they should be coming from the patient's own blood, fat, or bone marrow. Otherwise, patients can receive "dead" stem cells, which are not nearly as effective.

"We are very adamant to use the patient's own cells," said Riddle, who uses a centrifuge to separate out the blood, saline and growth factors that will be re-injected. "When we inject that into an area, we are telling your body to go heal that spot," said Dr. Hernandez.

"Stem cell treatment is really about trying to take the stem cells out of your body and...inject them back into another part of your body, in order to try and heal whatever part of the body is suffering," said Professor Frow.

"IT'S THE NEW WAVE"

According to researchers, Scottsdale and Phoenix are two of the seven "hot spot" cities in the country.

Arizona State University professors Emma Frow and Dave Brafman spent years studying the industry , and mapping out dozens of clinics in the Valley. They believe there are many more, as some intentionally practice under the radar. "I don't believe right now that there is enough evidence to suggest that they work," said Professor Frow.

"They are unregulated, unproven and for-profit," added Professor Brafman.

The profits are plentiful. "There's cash involved, so this isn't covered by insurance," said Dr. Hernandez.

"PREYING ON PEOPLE'S PAIN"

The thousands in cash is one of many reasons the burgeoning industry is ripe for exploitation.

"The other piece too, it is it is new and upcoming," said Dr. Hernandez.

Many potential patients do not know the first thing about the procedure they are being sold, and doctors say many fall for sales tactics that are practiced at traveling seminars.

"They are preying on people's pain," said Dr. Sorr. "I think its really unethical and it upsets me."

Dr. Sorr believes the seminars are "a scam" that specifically targets an elderly clientele.

"They wine you and dine you. They go through a little dinner presentation and it is not the doctor, it's a marketing agency," he said.

The doctor told ABC15 he has had clients who have been duped, even after he told them they were not ideal candidates for stem cell or PRP therapy.

"It really broke my heart that he spent thousands upon thousands of dollars for something that was worthless.

"I don't agree with how they are done," said Dr. Hernandez. "They inject people and they get money. That's not practicing medicine, that is selling."

Both naturopathic physicians told ABC15 that some patients do not need the treatment, or will get subpar results from the injections. They say it is well known in the industry that some practices will continue to sell in order to reap the thousands in cash.

"ALL OF IT FALLS ON THE PATIENT"

Right now, there is little regulation or oversight of the industry in Arizona.

"Really all of it the falls on the patient, with very little recourse if things go wrong," said Dr. Emma Frow.

During the course of our investigation, ABC15 discovered the Arizona Medical Board and County Health Department do not take complaints or oversee the people performing injections. The federal government has also been slow to implement widespread regulation.

"The FDA has their hands tied," said Dr. Sorr. "There are too many people out there that are doing this that havent had the proper training, they dont have the right experience, the right tools and all that."

There are some larger regulations in Arizona, governing who can handle a needle and perform injections.

Unlike other industries though, including massage therapy, there is no board that checks on licensing or investigates complaints involving botched procedures or alleged fraud.

"The state medical boards, need to become a little bit more involved in sort of identifying, or responding to claims," said Professor Brafman.

"I don't think it would hurt to have it, for sure. At the end of the day it's about protecting the public," said Dr. Hernandez.

For thousands of Arizonans, like Janet McConnell, regenerative medicine has helped heal chronic pain. Before spending thousands thousands though, do your research. "Always get a second opinion," said Dr. Sorr.

"I think this is really a case of buyer beware, or consumer beware," said Professor Frow.

If you are planning on undergoing a stem cell or PRP treatment, click here for questions experts say you should always ask ahead of time.

Read more:
Arizona the "wild west" of stem cell therapy; experts say promising therapy ripe for exploitation - ABC15 Arizona

Bone Marrow Stem Cells Comments Off on Arizona the "wild west" of stem cell therapy; experts say promising therapy ripe for exploitation – ABC15 Arizona | November 6th, 2019

MONTREAL, Nov. 06, 2019 (GLOBE NEWSWIRE) -- ExCellThera Inc., an advanced biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, announced today the publication of full data from the first clinical trial using ECT-001 (single UM171-expanded cord blood) in patients with haematological malignancies. The data were published in the peer-reviewed medical journal, The Lancet Haematology.

The clinical trial findings indicate that ECT-001 cell therapy is feasible, safe (as suggested by the low transplant-related mortality, low incidence of severe acute graft-vs-host disease (GVHD), and absence of moderate to severe chronic GVHD) and allows for the use of small cords without compromising engraftment. In addition, ECT-001 has shown potential to overcome the disadvantages of unexpanded cord blood transplants while maintaining their benefits of low risk of chronic GVHD and relapse. The Lancet Haematology paper provides the first detailed analysis of the study results presented at the 60th American Society of Hematology Annual Meeting (ASH 2018) in December 2018 and supports the recent advancement of the ECT-001 clinical program.

Were pleased that these important results from the first clinical trial using ECT-001 in haematological malignancies are now fully available to the broader bone marrow transplant community, said Dr. Guy Sauvageau, CEO and founder of ExCellThera, and co-senior author of the paper. These results indicate that ECT-001 transplants combine the advantages of conventional grafts using bone marrow (low treatment-related mortality), peripheral blood (fast engraftment) and cord blood (greater accessibility, low relapse and chronic GvHD) in a single, low cost, easy to produce 7-day culture product, which could lead to a paradigm shift in bone marrow transplantation.

The FDA granted ECT-001 Orphan Drug Designation for the prevention of graft-versus-host disease in 2018 and Regenerative Medicine Advanced Therapy Designation in the treatment of hematologic malignancies in 2019. ECT-001 is currently being used for the treatment of blood disorders in other ongoing and approved clinical trials in the United States and Canada. ExCellThera also plans to initiate a European clinical trial as well as a pivotal, multi-centre clinical trial in the coming months.

About ExCellThera Inc.

ExCellThera is an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for use in novel one-time curative therapies for patients with hematologic malignancies, autoimmune and other diseases. ExCellTheras lead solution combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials. excellthera.com

Read more from the original source:
ExCellThera announces publication in The Lancet Haematology highlighting excellent clinical results of ECT-001 in patients with haematological...

Bone Marrow Stem Cells Comments Off on ExCellThera announces publication in The Lancet Haematology highlighting excellent clinical results of ECT-001 in patients with haematological… | November 6th, 2019

As Biotechnology companies, Sesen Bio Inc. (NASDAQ:SESN) and BioTime Inc. (:) are our subject to contrast. And more specifically their risk, institutional ownership, analyst recommendations, profitability, dividends, earnings and valuation.

Earnings & Valuation

Table 1 shows top-line revenue, earnings per share and valuation of the two companies.

Profitability

Table 2 shows Sesen Bio Inc. and BioTime Inc.s return on assets, return on equity and net margins.

Volatility & Risk

Sesen Bio Inc. has a beta of 0.65 and its 35.00% less volatile than S&P 500. From a competition point of view, BioTime Inc. has a 2.81 beta which is 181.00% more volatile compared to S&P 500.

Liquidity

The Current Ratio and a Quick Ratio of Sesen Bio Inc. are 6.4 and 6.4. Competitively, BioTime Inc. has 3.5 and 3.5 for Current and Quick Ratio. Sesen Bio Inc.s better ability to pay short and long-term obligations than BioTime Inc.

Institutional and Insider Ownership

Institutional investors held 31.6% of Sesen Bio Inc. shares and 43.7% of BioTime Inc. shares. 6.56% are Sesen Bio Inc.s share held by insiders. Insiders Competitively, held 3.9% of BioTime Inc. shares.

Performance

In this table we provide the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Sesen Bio Inc. has -13.38% weaker performance while BioTime Inc. has 20.48% stronger performance.

Summary

BioTime Inc. beats on 5 of the 9 factors Sesen Bio Inc.

Sesen Bio, Inc., a late-stage clinical company, develops next-generation antibody-drug conjugate therapies for patients with cancer. It develops its products based on its Targeted Protein Therapeutics (TPTs) platform. The company's lead product candidate is Vicinium, a fusion protein that is in Phase III clinical trial for the treatment of high-grade non-muscle invasive bladder cancer. It also develops Vicinium in combination with Durvalumab, which is in Phase I clinical trial for the treatment of high-grade non-muscle invasive bladder cancer; and Vicinium in combination with AstraZeneca's checkpoint inhibitor for the treatment of squamous cell carcinoma of the head and neck. In addition, the company is developing systemically-administered TPTs, including VB6-845d for the treatment of solid tumors. The company was formerly known as Eleven Biotherapeutics, Inc. and changed its name to Sesen Bio, Inc. in May 2018. Sesen Bio, Inc. was founded in 2008 and is based in Cambridge, Massachusetts.

BioTime, Inc., a clinical-stage biotechnology company, focuses on developing and commercializing products addressing degenerative diseases based on pluripotent stem cells and HyStem cell/drug delivery platform technologies. Its product candidates include Renevia, a facial aesthetics product that is in pivotal clinical trial for the treatment of HIV related facial lipoatrophy; OpRegen, which is in Phase I/IIa clinical trial for the treatment of the dry form of age-related macular degeneration; HyStem-BDNF, a preclinical development program for the delivery of recombinant human brain-derived neurotrophic factor (BDNF) directly into the stroke cavity of patients for aiding in tissue repair and functional recovery; and ReGlyde that is in preclinical development as a device for viscosupplementation and a combination product for drug delivery in osteoarthritis. The company also develops AST-OPC1, a therapy derived from pluripotent stem cells that is in a Phase I/IIa clinical trial for spinal cord injuries; AST-VAC1, a patient-specific cancer immunotherapy that is in Phase II clinical trial for acute myeloid leukemia; and AST-VAC2, a non-patient specific cancer immunotherapy, which is in Phase I/IIa clinical trial to treat non-small cell lung cancer. In addition, it offers liquid biopsy tests for diagnosis of cancer; bone grafting products to treat orthopedic disorders; and mobile health software products. Further, it markets GeneCards, a human gene database; LifeMap Discovery, a database of embryonic development, stem cell research, and regenerative medicine; MalaCards, a human disease database; VarElect, an application for prioritizing gene variants; and GeneAnalytics, a novel gene set analysis tool. Additionally, the company develops and markets Hextend, a blood plasma volume expander used for the treatment of hypovolemia. BioTime, Inc. was founded in 1990 and is based in Alameda, California.

Receive News & Ratings Via Email - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings with our FREE daily email newsletter.

Continue reading here:
Sesen Bio Inc. (SESN) and BioTime Inc. (:) Contrasting side by side - FinanceMercury

Spinal Cord Stem Cells Comments Off on Sesen Bio Inc. (SESN) and BioTime Inc. (:) Contrasting side by side – FinanceMercury | November 6th, 2019

Please enable Javascript to watch this video

A breakthrough medical procedure using stem cell therapy is transforming the lives of disabled pets, and their owners are documenting these transformations in astounding before and after videos some featuring dogs barely able to walk that are suddenly able to run.

Dr. Carmen Petti of the Avon Lake Animal Clinic said while stem cells are proven to help things like arthritis and skin allergies, it is still in the early stages for many diseases and ailments. It's also expensive, costing anywhere between $1,000 to $2,500.

The procedure requires sedation and a minimally invasive surgery where doctors remove two to three tablespoons of fat loaded with stem cells. In one study, 99% of patients saw improvements that lasted up to two years.

Doctors and patients say they are amazed at the improvement they've seen in their patients and pets. They also say that as the procedure becomes more common, it could also become cheaper and more affordable.

More:
Stem cell therapy giving disabled, elderly pets a second chance at life - CW39

Skin Stem Cells Comments Off on Stem cell therapy giving disabled, elderly pets a second chance at life – CW39 | November 6th, 2019

Hematopoietic stem cells (HSCs) are responsible for the constant replenishment of all blood cells throughout life. One of the major challenges in regenerative medicine is to produce tailor-made HSCs to replace the defective ones in patients suffering from blood related diseases. This would circumvent the shortage of donor HSCs available for the clinic. To achieve the controlled production of bona fide HSCs in vitro, in a dish, a better understanding is required of where, when and how HSCs are physiologically produced in vivo, in the living body. Researchers from the groups of Catherine Robin(Hubrecht Institute) and Thierry Jaffredo (UPMC, LBD IBPS, Paris) have found a previously unappreciated hematopoietic wave taking place in the bone marrow of late fetuses and young adults and producing HSCs from resident hemogenic endothelial cells of somite origin. This transient hematopoietic wave fills the gap between the completion of embryonic blood production and the beginning of adult bone marrow hematopoietic production in both chicken and mice.

Endothelial origin of hematopoietic stem cells

The constant production of short-lived blood cells, needed for proper oxygenation of tissues and protection against pathogens throughout life, relies on a small cohort of HSCs. The first HSCs derive from specialized endothelial cells, named hemogenic endothelial (HE) cells, via an endothelial to hematopoietic transition (EHT). EHT transiently occurs in the main arteries, such as the aorta, during the embryonic development of vertebrates. The pool of HSCs is then amplified before migrating to the bone marrow where HSCs will reside during adult life. Whether EHT occurs past the embryonic stage and in other organs, such as the bone marrow, was unknown until now.

Hemogenic endothelial cells in the bone marrow

To find out whether EHT occurs past the embryonic stage and in the bone marrow, the researchers used a combination of experimental embryology, genetic, transcriptomic and functional approaches on chicken and mouse models. By tracing bone marrow-forming endothelial cells through fluorescent genetic labelling and live imaging analyses, they found that the entire vascular network of the bone marrow derives from the somites. The somites are segments of the body that will progressively form important tissues of the organism as the embryo develops, including bones, muscles and skin. Unexpectedly, the researchers found that some somite-derived endothelial cells produce HSCs and multipotent progenitors in the late fetus and young adult bone marrow, through the same EHT process that was thus far only seen in the embryo. These cells are molecularly very similar to the cells undergoing EHT or recently emerged HSCs in the embryonic aorta, with a prominent Notch pathway, endothelial-specific genes and transcription factors involved in EHT. The results therefore demonstrate that HSCs are newly generated past embryonic stages, from hemogenic endothelial cells from somitic origin and via EHT, the same mechanism that occurs in the embryo.

A new wave of blood cell production

The yolk sac of the embryo produces two partially overlapping waves of hematopoiesis. The first (primitive) wave gives rise to hematopoietic cells that last only during embryonic development. The second (definitive) wave produces various progenitors that migrate to the fetal liver to produce the immediately needed blood cells. These progenitors are sufficient for the embryo to survive until birth, when the aorta-derived HSC-dependent wave will take over. The transient hematopoietic production discovered in the present study fills the gap between the end of the yolk sac hematopoiesis and the bone marrow HSC-dependent production of blood cells. Indeed, the pool of HSCs that expanded in the fetal liver starts to colonize the bone marrow only just before birth. HSCs are present in very low numbers and time is most likely required before they find their final adult-type niches and start to differentiate and proliferate into more committed progenitors and mature blood cells. The transient hematopoietic wave that the researchers describe in late fetal and young adult stages might also prepare the bone marrow niches for the HSCs coming from the fetal liver.

Stem cell therapies

Defects in HSCs lead to various blood-related disorders and cancers that are partly treated by HSC transplantations. The controlled production of bona fide HSCs from pluripotent precursors remains very difficult to achieve in vitro, in a petri dish, and therefore requires a better understanding of the HSC production as it occurs physiologically in vivo, in the living body. Identifying all steps of hematopoietic production and the molecular events controlling this process is of fundamental interest and should help to devise innovative stem cell therapies for hematopoietic disorders in the future.

Reference:Yvernogeau, L., Gautier, R., Petit, L., Khoury, H., Relaix, F., Ribes, V., Jaffredo, T. (2019). In vivo generation of haematopoietic stem/progenitor cells from bone marrow-derived haemogenic endothelium. Nature Cell Biology. https://doi.org/10.1038/s41556-019-0410-6

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Follow this link:
Transient Wave of Hematopoietic Stem Cell Production in Late Fetuses and Young Adults - Technology Networks

Skin Stem Cells Comments Off on Transient Wave of Hematopoietic Stem Cell Production in Late Fetuses and Young Adults – Technology Networks | November 6th, 2019

Beauty/6 November 2019, 2:00pm/LIZ HOGGARD

London - On the run up to her 50th birthday, Charlotte Vhtz noticed her skin was changing.

"When youre a child, skin renewal takes place every three to five days," she says. "But when you get into your 50s, it takes two to three months. Even if you apply the most amazing expensive cream, its going on top of dead skin cells. It cant penetrate, so it wont work."

An organic beauty pioneer, Charlotte didnt want to use chemical "tweakments", so she started researching cutting-edge, scientifically proven botanicals that could delay ageing.

"I knew I needed to find a way to exfoliate the skin without being too harsh. I love wrinkles, but I dont like dry, sagging skin."

A former nurse, Charlotte has a background in pharmaceuticals and is fascinated by science. But she also believes passionately in the power of nature.

After six years of research, Charlotte, now 60, has just launched her signature range, Age Defy+ by Cha Vhtz, a natural skincare regime that uses a blend of innovative plant-based alternatives aimed at skin aged 30, 40, 50 and beyond.

Here, she explains how anti-ageing plant extracts, among them pomegranate, hibiscus, neroli and sea holly, can be just as effective.

Skin-plumper

SWAP: Botox for hibiscus. With its magical reputation for increasing skin elasticity, its no wonder hibiscus is called the Botox plant.

It has an incredible ability to inhibit the activity of the enzyme elastase, which is responsible for breaking down our skins precious elastin.

Hibiscus actively combats the ageing process by firming and lifting your skin, allowing it to snap back.

Because of the slightly exfoliating effect of the organic acids found in the plant, hibiscus also helps speed up cell turnover, resulting in a more even-looking skin tone.

It can even help to control acne breakouts, bringing your skin back in balance for a gorgeous, glowing complexion.

Hibiscus also enhances the skins ability to retain moisture, a key factor in keeping a youthful complexion.

Wrinkler-buster

SWAP: Hyaluronic acid (a much-used additive in anti-ageing creams) for beech bud extract.

Beech bud extract is an exceptional ingredient, rich in a range of substances that boost the metabolism, smooth the skins surface and restore hydration.

Perhaps its no surprise that the beech is known as the Everlasting Youth Tree.

Exfoliator

SWAP: Alpha Hydroxy Acids, or AHA (chemical compounds used in abrasive cosmetic exfoliators) for pineapple extract.

Exfoliate dead skin cells by rubbing a thin slice of pineapple or papaya over your face. Leave for five minutes, then rinse off with tepid water.

Pineapple extract is rich in skin-boosting vitamins C and E and bromelain, a protein-digesting enzyme.

Rejuvenator

SWAP: Butylene Glycol (a type of alcohol used as a solvent in anti-ageing serums) for sea holly.

A beautiful, purple, thistle-like plant, sea holly is amazing because it has evolved to survive in the harshest conditions.

An extract is obtained from cultured plant stem cells, which contain all the attributes of the whole plant and, as a result, have powerful regenerative and rejuvenating properties.

Sea holly stimulates and protects the skins natural elastin and collagen, both of which decrease as you age, resulting in greater skin radiance and luminosity.

Anti-ager

SWAP: Dermal fillers for extract of the herb baikal skullcap.

Much-used in Chinese medicine, the extract baicalin comes from the roots of baikal skullcap, a herb in the mint and sage family native to East Asia.

Its an ingredient with remarkable anti-ageing properties in adults aged 30 or over.

Hydrator

SWAP: Retinol (a popular chemical skincare ingredient) for squalane.

Squalane is the saturated, or stable, form of the compound squalene, originally obtained for commercial purposes from shark liver oil, but today extracted from olive oil.

Adult skin is lubricated and protected against external aggressors by the sebum our skin produces. In healthy skin, sebum contains 10 percent to 13 percent squalene. This level drops as we get older.

Excerpt from:
Natural anti-ageing alternatives that actually work - IOL

Skin Stem Cells Comments Off on Natural anti-ageing alternatives that actually work – IOL | November 6th, 2019

For pear butter, you'll want a variety that breaks down in cooking. Bartlett pears will do well.(Photo: Abel Uribe, TNS)

Apples may get all of autumns accolades, but its time for pears to muscle in on the action.

Understanding which pear varieties are best for which uses will help you choose wisely from the fruit youll see at farmers markets, farm stands and grocery stores.

You can eat any pear raw, from juicy Bartletts to crisp Asian pears. But in cooking, you may want the pear to retain its shape, or you may want it to melt into a concentrated sauce. I remember pear varieties that hold their shape for poached pears, and for the pear tart we offer here with a simple mnemonic of ABC: Anjou, Bosc and Comice.

Some varieties are more grainy or gritty than others but peeling any pear will help reduce that graininess. As pears ripen on the tree, they develop stone cells, and most of these lie just under the skin. Most pears are harvested before theyre fully ripe for this reason. While the skin is full of nutrients, sometimes you just want that grittiness to go away.

Like apples, cut pears will brown when exposed to air. For salads and other raw uses where appearance is important, place the pears in water acidulated with lemon juice for a quick bath to prevent browning.

These are the varieties youre likely to see this season, with a bit of information about them and their best uses.

A ripe Bartlett, the juiciest of all the pears, will leave your chin dripping when you eat it out of hand. (Terrence Antonio James/Chicago Tribune/TNS)(Photo: Terrence Antonio James, TNS)

Anjou: Firm and mild flavored, Anjous are good for cooking where you want the pear to pick up the flavors of its cooking companions. Red and green Anjous have the same flavor.

Asian: As crisp as a ripe apple, Asian pears are very mild in flavor. Theyre the outlier in the pear family, more apple than pear.

Bartlett: The juiciest of all the pears, a ripe Bartlett will leave your chin dripping when you eat it out of hand. Choose red or green Bartletts when you want the fruit to cook into a sauce, as we do in the vanilla-cardamom pear butter recipe here.

Bosc: Crisp and mildly sweet, Boscs are the classic choice for poached pears. Theyre easy to recognize because of their cinnamon-colored russeted skin. They tend to be a nice size as well.

Comice: Brightly flavored with the quintessential pear taste, Comice pears are less grainy than many other varieties.

Concorde: A favorite in Europe, the Concorde has a long neck that makes it immediately identifiable. Its distinctively vanilla flavor makes it a favorite for roasting and grilling, but its also great out of hand.

Forelle: A pretty speckled pear thats popular in Europe, this small pear is best for snacking. Its name comes from the German word for trout, because its colors echo the flashing brilliance of the fish. Grown in small quantities in the Pacific Northwest, Forelle tells you its ripe when the skin under its red speckles turns from green to yellow.

French butter: Small with concentrated flavors, make sure French butter pears are fully ripe before use. Underripe fruit has a sharp, tannic flavor. Good for snacking, or in salads.

Seckel: Just as with French butter pears, make sure the little Seckel pears are fully ripe before eating to avoid a tannic hit. Best out of hand, or in salads.

Robin Mather is a longtime food journalist and the author of The Feast Nearby, a collection of essays and recipes from a year of eating locally on a budget. Follow her as she writes her third book at thefeastofthedove.com.

A pastry shell of ground almond meal, butter and sugar holds a pastry cream and poached pears. Sliced almonds finish off the dessert. (Terrence Antonio James/Chicago Tribune/TNS)(Photo: Terrence Antonio James, TNS)

PEAR-ALMOND TART

This simple tart will look and taste more impressive than its simple ingredients might suggest. Remember that you want pears that will hold their shape for this tart. If you cant find creme fraiche, substitute lightly sweetened sour cream as a garnish at serving time.

Prep: 30 minutes

Cook: 40 minutes

Makes: about 12 servings

Crust:

2 1/4 cups ground almond meal

4 1/2 tablespoons sugar

8 tablespoons melted salted butter

Filling:

2 cups sugar, divided use (plus more for browning)

3 Anjou, Bosc or Comice pears, peeled, sliced in half

1 1/2 cups milk

2 teaspoons vanilla

3 eggs, lightly beaten

1/4 cup flour

1/4 cup sliced toasted almonds

Creme fraiche, sweetened sour cream or whipped cream

1. For the crust: Heat the oven to 350 degrees. Combine almond meal, sugar and melted butter in a medium bowl. Stir to combine. Pat the crust mixture into the bottom and up the sides of a 12-inch tart pan and press into place with the bottom of a drinking glass. Bake the crust until just colored, 10 to 15 minutes. Remove and allow to cool completely before filling.

2. For the filling: Heat 4 cups water and 1 1/2 cups sugar to a boil in a large saucepan over medium-high heat. Reduce heat to low. Add the pears; poach until tender, 20-25 minutes. Remove pears from the syrup. Allow to cool, then cut out cores. Cut the pears into fans by slicing into 1/4-inch slices that remain attached by about 1/2 inch at the stem end. Set aside.

3. Combine milk and vanilla in a small saucepan and bring it to just a simmer over medium heat. (Dont let it boil over.) Combine eggs, remaining 1/2 cup sugar and the flour in a large saucepan. Temper the mixture by slowly whisking in a little of the hot milk. Then gradually whisk in the rest. Cook, whisking continuously, over medium heat. At the first sign of a boil, 3 to 6 minutes, remove pan from the heat while continuing to whisk until mixture begins to thicken. Allow the custard to cool.

4. Spoon cooled custard into the tart shell. Lay the fanned-out pears, stem end inward, in the custard. Scatter the sliced almonds over top. Sprinkle with 1 to 2 tablespoons sugar. Heat the broiler in the oven. Place the tart on the middle rack, 4 to 5 inches from the broil. Allow to broil until pears and custard are golden, about 5 minutes, watching carefully.

5. Serve warm with creme fraiche, sweetened sour cream or whipped cream.

Nutrition information per serving: 428 calories, 22 g fat, 7 g saturated fat, 69 mg cholesterol, 54 g carbohydrates, 45 g sugar, 8 g protein, 101 mg sodium, 4 g fiber

VANILLA-CARDAMOM PEAR BUTTER

Prep: 35 minutes

Cook: 8-10 hours

Makes: about 7 half-pints

Youll definitely want to use ripe Bartlett pears for this fruit butter because they cook into a silky puree. Making this pear butter in the slow cooker means you dont have to stand over it while it cooks. Weve given directions to both can and freeze this sumptuous delight.

6 1/2 pounds Bartlett pears, peeled, cored and cut into 1/2-inch cubes

Juice of 1 large lemon

1/2 cup sugar

1/4 teaspoon coarse salt

2 teaspoons vanilla

1 teaspoon ground cardamom

4 tablespoons unsalted butter

1. Tumble all ingredients except butter into a slow cooker. Stir to blend, then cover and cook on low until the pear butter is very thick and mounds on a spoon, 8 to 10 hours. Test its readiness by placing a spoonful on a plate; if no liquid escapes around the edges, the pear butter is ready. If it weeps, continue to cook with the lid crosswise to allow excess liquid to evaporate.

2. Stir in the butter until it is fully melted. Ladle the hot pear butter into sterile half-pint jars, leaving 1/4-inch headspace. To can, apply lids and rings just until finger tight; process in a boiling water bath for 10 minutes. To freeze, allow the pear butter to cool to room temperature, then freeze without lids. Once pear butter is frozen, add lids and freeze for up to six months.

Nutrition information per tablespoon: 21 calories, 0 g fat, 0 g saturated fat, 0 mg cholesterol, 5 g carbohydrates, 3 g sugar, 0 g protein, 5 mg sodium, 1 g fiber

Read or Share this story: https://www.detroitnews.com/story/life/food/2019/11/07/what-pears-pick-your-fall-recipes/4156370002/

Continue reading here:
What pears to pick for your fall recipes starting with a pear tart and pear butter - The Detroit News

Skin Stem Cells Comments Off on What pears to pick for your fall recipes starting with a pear tart and pear butter – The Detroit News | November 6th, 2019

This Stem Cell Therapy report has several aspects of marketing research and analysis which includes market size estimations, market dynamics, company & market best practices, entry level marketing strategies, positioning and segmentations, competitive landscaping, opportunity analysis, economic forecasting, industry-specific technology solutions, roadmap analysis, targeting key buying criteria, and in-depth benchmarking of vendor offerings. This Stem Cell Therapy market report offers all-inclusive study about production capacity, consumption, import and export for all the major regions across the world.

Stem Cell Therapy Market is expected to reach USD 15.63 billion by 2025, from USD 7.72 billion in 2017 growing at a CAGR of 9.2% during the forecast period of 2018 to 2025. The Stem Cell Therapy market report contains data for historic year 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025 (Updated values listed in sample report).

Get Sample Analysis of Global Market Information: https://databridgemarketresearch.com/request-a-sample/?dbmr=global-stem-cell-therapy-market

Stem cell therapy is the therapy which uses stem cells for the treatment or prevention of a disease. Bone marrow transplant is the widely applicable therapy which is followed by umbilical cord blood. Research is going on to develop various sources (such as cord blood cells, bone marrow and skin) to use these cells for treatment of various disorders like neurodegenerative diseases and conditions such as heart disease, diabetes and other conditions. Some of the major players operating in the global stem cell therapy market are

Others: ViaCyte, Inc, AbbVie, Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc,, International Stem Cell Corporation, Aastrom Biosciences, Inc., Advanced Cell Technology, Cryo Cell International, Cytori Therapeutics, Inc., Geron Corporation, and Invitrogen and others. The global stem cell therapy market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of the global stem cell therapy market for global, Europe, North America, Asia Pacific and South America.

Get TOC For Full Analysis Of Report:https://databridgemarketresearch.com/toc/?dbmr=global-stem-cell-therapy-market

Major Market Drivers and Restraints:

Drivers:

Restraints:

Segmentation:

The global stem cell therapy market is segmented based on

Potential held by the report

Want Full Report? Enquire Here:https://databridgemarketresearch.com/inquire-before-buying/?dbmr=global-stem-cell-therapy-market

About Us

Data Bridge Market Research is a versatile market research and consulting firm with over 500 analysts working in different industries. We have catered more than 40% of the fortune 500 companies globally and have a network of more than 5000+ clientele around the globe. Our coverage of industries include Medical Devices, Pharmaceuticals, Biotechnology, Semiconductors, Machinery, Information and Communication Technology, Automobiles and Automotive, Chemical and Material, Packaging, Food and Beverages, Cosmetics, Specialty Chemicals, Fast Moving Consumer Goods, Robotics, among many others.

Data Bridge adepts in creating satisfied clients who reckon upon our services and rely on our hard work with certitude. Get Customization and Discount on Report by emailing Corporatesales@databridgemarketresearch.com . We are content with our glorious 99.9 % client satisfying rate.

Contact Us

Data Bridge Market Research

US: +1 888 387 2818

UK: +44 208 089 1725

Hong Kong: +852 8192 7475Mail:Corporatesales@databridgemarketresearch.comhttps://databridgemarketresearch.com

View original post here:
Industry Research On Stem Cell Therapy Market : Report With Pluristem Therapeutics, Cytori Therapeutics Inc, Geron Corporation, Vericel Corporation,...

Skin Stem Cells Comments Off on Industry Research On Stem Cell Therapy Market : Report With Pluristem Therapeutics, Cytori Therapeutics Inc, Geron Corporation, Vericel Corporation,… | November 6th, 2019

SUMMIT, N.J.--(BUSINESS WIRE)--Celgene Corporation (NASDAQ: CELG) today announced data from nearly 70 Company-sponsored, global alliance and investigator-initiated clinical studies evaluating Celgenes investigational and approved therapies will be presented at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition, December 7-10, in Orlando, Fla.

Celgene has a deep and ongoing commitment to innovative research and development in treatments for serious blood disorders, with the potential to transform patient outcomes, said Alise Reicin, M.D., President, Global Clinical Development for Celgene. We look forward to ASH as an opportunity to highlight our commitment and leadership in the research and development of novel therapies for the treatment of blood cancers through new insights in both CD19 and BCMA targeted cell therapies and important progress in our myeloid pipeline.

In leukemia and lymphoma, highlighted studies this year include safety and efficacy results from the pivotal TRANSCEND NHL-001 study of an investigational CD-19 targeted chimeric antigen receptor (CAR) T cell therapy lisocabtagene maraleucel (liso-cel) in relapsed/refractory large B-cell non-Hodgkin lymphoma. Additional liso-cel data from three ongoing studies will evaluate the use of the therapy in an outpatient setting, as well as in transplant noneligible patients with relapsed/refractory large B-cell non-Hodgkin lymphoma (PILOT) and in patients with relapsed/refractory chronic lymphocytic leukemia (TRANSCEND CLL-004).

In multiple myeloma, other notable investigational cell therapy abstracts include the first phase 1 clinical data from the bi-specific T-Cell Engager (TCE) CC-93269 and updated phase 1 clinical data from CAR T program, bb21217, both targeting the B-cell maturation antigen (BCMA) in relapsed/refractory disease.

Several abstracts focusing on data in myeloid diseases including longer-term response data from the phase 3 MEDALIST study of luspatercept to treat anemia in patients with IPSS-R very low-, low-, or intermediate-risk myelodysplastic syndromes with ring sideroblasts who require red-blood-cell (RBC) transfusions will be presented. Additionally, the first data from a phase 2 study of luspatercept in myelofibrosis-associated anemia, results from a study of fedratinib in myelofibrosis patients with low platelet counts, and the first data from CELMoD agent CC-90009, a GSPT1 degrader in relapsed or refractory acute myeloid leukemia (AML) will be presented.

Selected abstracts include*:

Lymphoma & Chronic Lymphocytic Leukemia

Multiple Myeloma

Myeloid Diseases

Beta thalassemia

A complete listing of abstracts can be found at https://www.hematology.org/Annual-Meeting/abstracts/

The safety and efficacy of investigational agents and/or investigational uses of approved marketed products have not been established. There is no guarantee that the agents will receive health authority approval or become commercially available in any country for the uses being investigated.

*All times Eastern Time

About REVLIMID

REVLIMID (lenalidomide) in combination with dexamethasone (dex) is indicated for the treatment of adult patients with multiple myeloma (MM)

REVLIMID is indicated as maintenance therapy in adult patients with MM following autologous hematopoietic stem cell transplantation (auto-HSCT)

REVLIMID is indicated for the treatment of adult patients with transfusion-dependent anemia due to low-or intermediate-1risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities

REVLIMID is indicated for the treatment of adult patients with mantle cell lymphoma (MCL) whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib

REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated follicular lymphoma (FL)

REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated marginal zone lymphoma (MZL)

REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials

REVLIMID is only available through a restricted distribution program, REVLIMID REMS.

Important Safety Information

WARNING: EMBRYO-FETAL TOXICITY, HEMATOLOGIC TOXICITY, and VENOUS and ARTERIAL THROMBOEMBOLISM

Embryo-Fetal Toxicity

Do not use REVLIMID during pregnancy. Lenalidomide, a thalidomide analogue, caused limb abnormalities in a developmental monkey study. Thalidomide is a known human teratogen that causes severe life-threatening human birth defects. If lenalidomide is used during pregnancy, it may cause birth defects or embryo-fetal death. In females of reproductive potential, obtain 2 negative pregnancy tests before starting REVLIMID treatment. Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during and for 4 weeks after REVLIMID treatment. To avoid embryo-fetal exposure to lenalidomide, REVLIMID is only available through a restricted distribution program, the REVLIMID REMS program.

Information about the REVLIMID REMS program is available at http://www.celgeneriskmanagement.com or by calling the manufacturers toll-free number 1-888-423-5436.

Hematologic Toxicity (Neutropenia and Thrombocytopenia)

REVLIMID can cause significant neutropenia and thrombocytopenia. Eighty percent of patients with del 5q MDS had to have a dose delay/reduction during the major study. Thirty-four percent of patients had to have a second dose delay/reduction. Grade 3 or 4 hematologic toxicity was seen in 80% of patients enrolled in the study. Patients on therapy for del 5q MDS should have their complete blood counts monitored weekly for the first 8 weeks of therapy and at least monthly thereafter. Patients may require dose interruption and/or reduction. Patients may require use of blood product support and/or growth factors.

Venous and Arterial Thromboembolism

REVLIMID has demonstrated a significantly increased risk of deep vein thrombosis (DVT) and pulmonary embolism (PE), as well as risk of myocardial infarction and stroke in patients with MM who were treated with REVLIMID and dexamethasone therapy. Monitor for and advise patients about signs and symptoms of thromboembolism. Advise patients to seek immediate medical care if they develop symptoms such as shortness of breath, chest pain, or arm or leg swelling. Thromboprophylaxis is recommended and the choice of regimen should be based on an assessment of the patients underlying risks.

CONTRAINDICATIONS

Pregnancy: REVLIMID can cause fetal harm when administered to a pregnant female and is contraindicated in females who are pregnant. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential risk to the fetus

Severe Hypersensitivity Reactions: REVLIMID is contraindicated in patients who have demonstrated severe hypersensitivity (e.g., angioedema, Stevens-Johnson syndrome, toxic epidermal necrolysis) to lenalidomide

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity: See Boxed WARNINGS

REVLIMID REMS Program: See Boxed WARNINGS: Prescribers and pharmacies must be certified with the REVLIMID REMS program by enrolling and complying with the REMS requirements; pharmacies must only dispense to patients who are authorized to receive REVLIMID. Patients must sign a Patient-Physician Agreement Form and comply with REMS requirements; female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements and males must comply with contraception requirements

Hematologic Toxicity: REVLIMID can cause significant neutropenia and thrombocytopenia. Monitor patients with neutropenia for signs of infection. Advise patients to observe for bleeding or bruising, especially with use of concomitant medications that may increase risk of bleeding. Patients may require dose interruption and/or dose reduction. MM: Monitor complete blood counts (CBC) in patients taking REVLIMID + dexamethasone or REVLIMID as maintenance therapy, every 7 days for the first 2 cycles, on days 1 and 15 of cycle 3, and every 28 days thereafter. MDS: Monitor CBC in patients on therapy for del 5q MDS, weekly for the first 8 weeks of therapy and at least monthly thereafter. See Boxed WARNINGS for further information. MCL: Monitor CBC in patients taking REVLIMID for MCL weekly for the first cycle (28 days), every 2 weeks during cycles 2-4, and then monthly thereafter. FL/MZL: Monitor CBC in patients taking REVLIMID for FL or MZL weekly for the first 3 weeks of Cycle 1 (28 days), every 2 weeks during Cycles 2-4, and then monthly thereafter

Venous and Arterial Thromboembolism: See Boxed WARNINGS: Venous thromboembolic events (DVT and PE) and arterial thromboses (MI and CVA) are increased in patients treated with REVLIMID. Patients with known risk factors, including prior thrombosis, may be at greater risk and actions should be taken to try to minimize all modifiable factors (e.g., hyperlipidemia, hypertension, smoking). Thromboprophylaxis is recommended and the regimen should be based on the patients underlying risks. ESAs and estrogens may further increase the risk of thrombosis and their use should be based on a benefit-risk decision

Increased Mortality in Patients With CLL: In a clinical trial in the first-line treatment of patients with CLL, single-agent REVLIMID therapy increased the risk of death as compared to single-agent chlorambucil. Serious adverse cardiovascular reactions, including atrial fibrillation, myocardial infarction, and cardiac failure, occurred more frequently in the REVLIMID arm. REVLIMID is not indicated and not recommended for use in CLL outside of controlled clinical trials

Second Primary Malignancies (SPM): In clinical trials in patients with MM receiving REVLIMID and in patients with FL or MZL receiving REVLIMID + rituximab therapy, an increase of hematologic plus solid tumor SPM, notably AML, have been observed. In MM patients, MDS was also observed. Monitor patients for the development of SPM. Take into account both the potential benefit of REVLIMID and risk of SPM when considering treatment

Increased Mortality with Pembrolizumab: In clinical trials in patients with MM, the addition of pembrolizumab to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with MM with a PD-1-or PD-L1- blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials

Hepatotoxicity: Hepatic failure, including fatal cases, has occurred in patients treated with REVLIMID + dexamethasone. Pre-existing viral liver disease, elevated baseline liver enzymes, and concomitant medications may be risk factors. Monitor liver enzymes periodically. Stop REVLIMID upon elevation of liver enzymes. After return to baseline values, treatment at a lower dose may be considered

Severe Cutaneous Reactions Including Hypersensitivity Reactions: Angioedema and severe cutaneous reactions including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug reaction with eosinophilia and systemic symptoms (DRESS) have been reported. These events can be fatal. Patients with a prior history of Grade 4 rash associated with thalidomide treatment should not receive REVLIMID. REVLIMID interruption or discontinuation should be considered for Grade 2-3 skin rash. REVLIMID must be discontinued for angioedema, Grade 4 rash, exfoliative or bullous rash, or if SJS, TEN, or DRESS is suspected and should not be resumed following discontinuation for these reactions

Tumor Lysis Syndrome (TLS): Fatal instances of TLS have been reported during treatment with REVLIMID. The patients at risk of TLS are those with high tumor burden prior to treatment. Closely monitor patients at risk and take appropriate preventive approaches

Tumor Flare Reaction (TFR): TFR has occurred during investigational use of REVLIMID for CLL and lymphoma. Monitoring and evaluation for TFR is recommended in patients with MCL, FL, or MZL. Tumor flare may mimic the progression of disease (PD). In patients with Grade 3 or 4 TFR, it is recommended to withhold treatment with REVLIMID until TFR resolves to Grade 1. REVLIMID may be continued in patients with Grade 1 and 2 TFR without interruption or modification, at the physicians discretion

Impaired Stem Cell Mobilization: A decrease in the number of CD34+ cells collected after treatment (>4 cycles) with REVLIMID has been reported. Consider early referral to transplant center to optimize timing of the stem cell collection

Thyroid Disorders: Both hypothyroidism and hyperthyroidism have been reported. Measure thyroid function before starting REVLIMID treatment and during therapy

Early Mortality in Patients With MCL: In another MCL study, there was an increase in early deaths (within 20 weeks); 12.9% in the REVLIMID arm versus 7.1% in the control arm. Risk factors for early deaths include high tumor burden, MIPI score at diagnosis, and high WBC at baseline (10 x 109/L)

ADVERSE REACTIONS

Multiple Myeloma

Myelodysplastic Syndromes

Mantle Cell Lymphoma

Follicular Lymphoma/Marginal Zone Lymphoma

DRUG INTERACTIONS

Periodically monitor digoxin plasma levels due to increased Cmax and AUC with concomitant REVLIMID therapy. Patients taking concomitant therapies such as erythropoietin-stimulating agents or estrogen-containing therapies may have an increased risk of thrombosis. It is not known whether there is an interaction between dexamethasone and warfarin. Close monitoring of PT and INR is recommended in patients with MM taking concomitant warfarin

USE IN SPECIFIC POPULATIONS

Please see full Prescribing Information, including Boxed WARNINGS, for REVLIMID.

About INREBIC

INREBIC (fedratinib) is indicated for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF).

IMPORTANT SAFETY INFORMATION

WARNING: ENCEPHALOPATHY INCLUDING WERNICKES

Serious and fatal encephalopathy, including Wernickes, has occurred in patients treated with INREBIC. Wernickes encephalopathy is a neurologic emergency. Assess thiamine levels in all patients prior to starting INREBIC, periodically during treatment, and as clinically indicated. Do not start INREBIC in patients with thiamine deficiency; replete thiamine prior to treatment initiation. If encephalopathy is suspected, immediately discontinue INREBIC and initiate parenteral thiamine. Monitor until symptoms resolve or improve and thiamine levels normalize.

WARNINGS AND PRECAUTIONS

Encephalopathy, including Wernickes: Serious and fatal encephalopathy, including Wernickes encephalopathy, has occurred in INREBIC-treated patients. Serious cases were reported in 1.3% (8/608) of patients treated with INREBIC in clinical trials and 0.16% (1/608) of cases were fatal.

Wernickes encephalopathy is a neurologic emergency resulting from thiamine (Vitamin B1) deficiency. Signs and symptoms of Wernickes encephalopathy may include ataxia, mental status changes, and ophthalmoplegia (e.g., nystagmus, diplopia). Any change in mental status, confusion, or memory impairment should raise concern for potential encephalopathy, including Wernickes, and prompt a full evaluation including a neurologic examination, assessment of thiamine levels, and imaging. Assess thiamine levels in all patients prior to starting INREBIC, periodically during treatment, and as clinically indicated. Do not start INREBIC in patients with thiamine deficiency; replete thiamine prior to treatment initiation. If encephalopathy is suspected, immediately discontinue INREBIC and initiate parenteral thiamine. Monitor until symptoms resolve or improve and thiamine levels normalize.

Anemia: New or worsening Grade 3 anemia occurred in 34% of INREBIC-treated patients. The median time to onset of the first Grade 3 anemia was approximately 2 months, with 75% of cases occurring within 3 months. Mean hemoglobin levels reached nadir after 12 to 16 weeks with partial recovery and stabilization after 16 weeks. Red blood cell transfusions were received by 51% of INREBIC-treated patients and permanent discontinuation of INREBIC occurred due to anemia in 1% of patients. Consider dose reduction for patients who become red blood cell transfusion dependent

Thrombocytopenia: New or worsening Grade 3 thrombocytopenia during the randomized treatment period occurred in 12% of INREBIC-treated patients. The median time to onset of the first Grade 3 thrombocytopenia was approximately 1 month; with 75% of cases occurring within 4 months. Platelet transfusions were received by 3.1% INREBIC-treated patients. Permanent discontinuation of treatment due to thrombocytopenia and bleeding that required clinical intervention both occurred in 2.1% of INREBIC-treated patients. Obtain a complete blood count (CBC) at baseline, periodically during treatment, and as clinically indicated. For Grade 3 thrombocytopenia with active bleeding or Grade 4 thrombocytopenia, interrupt INREBIC until resolved to less than or equal to Grade 2 or baseline. Restart dose at 100 mg daily below the last given dose and monitor platelets as clinically indicated.

Gastrointestinal Toxicity: Gastrointestinal toxicities are the most frequent adverse reactions in INREBIC-treated patients. During the randomized treatment period, diarrhea occurred in 66% of patients, nausea in 62% of patient and vomiting in 39% of patients. Grade 3 diarrhea 5% and vomiting 3.1% occurred. The median time to onset of any grade nausea, vomiting, and diarrhea was 1 day, with 75% of cases occurring within 2 weeks of treatment. Consider providing appropriate prophylactic anti-emetic therapy (e.g., 5-HT3 receptor antagonists) during INREBIC treatment. Treat diarrhea with anti-diarrheal medications promptly at the first onset of symptoms. Grade 3 or higher nausea, vomiting, or diarrhea not responsive to supportive measures within 48 hours, interrupt INREBIC until resolved to Grade 1 or less or baseline. Restart dose at 100 mg daily below the last given dose. Monitor thiamine levels and replete as needed.

Hepatic Toxicity: Elevations of ALT and AST (all grades) during the randomized treatment period occurred in 43% and 40%, respectively, with Grade 3 or 4 in 1% and 0%, respectively, of INREBIC-treated patients. The median time to onset of any grade transaminase elevation was approximately 1 month, with 75% of cases occurring within 3 months. Monitor hepatic function at baseline, periodically during treatment, and as clinically indicated. For Grade 3 or higher ALT and/or AST elevations (greater than 5 ULN), interrupt INREBIC dose until resolved to Grade 1 or less or to baseline. Restart dose at 100 mg daily below the last given dose. If re-occurrence of a Grade 3 or higher elevation of ALT/AST, discontinue treatment with INREBIC.

Amylase and Lipase Elevation: Grade 3 or higher amylase 2% and/or lipase 10% elevations developed in INREBIC-treated patients. The median time to onset of any grade amylase or lipase elevation was 15 days, with 75% of cases occurring within 1 month of starting treatment. One patient developed pancreatitis in the fedratinib clinical development program (n=608) and pancreatitis resolved with treatment discontinuation. Monitor amylase and lipase at baseline, periodically during treatment, and as clinically indicated. For Grade 3 or higher amylase and/or lipase elevations, interrupt INREBIC until resolved to Grade 1 or less or to baseline. Restart dose at 100 mg daily below the last given dose.

ADVERSE REACTIONS: The most common adverse reactions for INREBIC treated vs. placebo were diarrhea (66% vs. 16%), nausea (62% vs. 15%), anemia (40% vs. 14%), and vomiting (39% vs. 5%). Dosage interruptions due to an adverse reaction during the randomized treatment period occurred in 21% of patients who received INREBIC. Adverse reactions requiring dosage interruption in >3% of patients who received INREBIC included diarrhea and nausea. Dosage reductions due to an adverse reaction during the randomized treatment period occurred in 19% of patients who received INREBIC. Adverse reactions requiring dosage reduction in >2% of patients who received INREBIC included anemia (6%), diarrhea (3%), vomiting (3%), and thrombocytopenia (2%).

DRUG INTERACTIONS: Coadministration of INREBIC with a strong CYP3A4 inhibitor increases fedratinib exposure. Increased exposure may increase the risk of adverse reactions. Consider alternative therapies that do not strongly inhibit CYP3A4 activity. Alternatively, reduce the dose of INREBIC when administering with a strong CYP3A4 inhibitor. Avoid INREBIC with strong and moderate CYP3A4 inducers. Avoid INREBIC with dual CYP3A4 and CYP2C19 inhibitor. Coadministration of INREBIC with drugs that are CYP3A4 substrates, CYP2C19 substrates, or CYP2D6 substrates increases the concentrations of these drugs, which may increase the risk of adverse reactions of these drugs. Monitor for adverse reactions and adjust the dose of drugs that are CYP3A4, CYP2C19, or CYP2D6 substrates as necessary when coadministered with INREBIC.

PREGNANCY/LACTATION: Consider the benefits and risks of INREBIC for the mother and possible risks to the fetus when prescribing INREBIC to a pregnant woman. Due to the potential for serious adverse reactions in a breastfed child, advise patients not to breastfeed during treatment with INREBIC, and for at least 1 month after the last dose.

RENAL IMPAIRMENT: Reduce INREBIC dose when administered to patients with severe renal impairment. No modification of the starting dose is recommended for patients with mild to moderate renal impairment. Due to potential increase of exposure, patients with preexisting moderate renal impairment require more intensive safety monitoring, and if necessary, dose modifications based on adverse reactions.

HEPATIC IMPAIRMENT: Avoid use of INREBIC in patients with severe hepatic impairment.

Please see full Prescribing Information, including Boxed WARNING.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit http://www.celgene.com. Follow Celgene on Social Media: Twitter, Pinterest, LinkedIn, Facebook and YouTube.

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the U.S. Securities and Exchange Commission, including factors related to the proposed transaction between Bristol-Myers Squibb and Celgene, such as, but not limited to, the risks that: management's time and attention is diverted on transaction related issues, including the planned divestiture of OTEZLA; disruption from the proposed transaction makes it more difficult to maintain business, contractual and operational relationships; legal proceedings are instituted against Bristol-Myers Squibb, Celgene or the combined company; and Bristol-Myers Squibb, Celgene or the combined company is unable to retain key personnel.

Hyperlinks are provided as a convenience and for informational purposes only. Celgene bears no responsibility for the security or content of external websites.

All trademarks are the property of their respective owners.

The rest is here:
Celgene to Present New and Updated Data on Key Hematology Pipeline Therapies at American Society of Hematology (ASH) 2019 Annual Meeting - Business...

Skin Stem Cells Comments Off on Celgene to Present New and Updated Data on Key Hematology Pipeline Therapies at American Society of Hematology (ASH) 2019 Annual Meeting – Business… | November 6th, 2019

Updated safety and efficacy results from ongoing Phase 1 CRB-402 study of bb21217 in relapsed/refractory multiple myeloma

Updated results from ongoing Phase 1/2 (HGB-206) study of LentiGlobin gene therapy for patients with sickle cell disease

New data from ongoing Phase 3 studies of LentiGlobin gene therapy for -thalassemia in pediatric, adolescent and adult patients

CAMBRIDGE, Mass. bluebird bio, Inc. (Nasdaq: BLUE) announced today that new and updated data from its investigational gene and cell therapy programs for multiple myeloma, sickle cell disease (SCD) and transfusion-dependent -thalassemia (TDT) will be presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida, December 7 10.

bluebird bio will present updated safety and efficacy data from the ongoing Phase 1 clinical study (CRB-402) of bb21217. bb21217 is an investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapy being studied, in partnership with Celgene, in patients with relapsed/refractory multiple myeloma (RRMM).

In addition, data from clinical studies of LentiGlobin gene therapy for -thalassemia, including results up to 61 months from the long-term follow-up study (LTF-303) and updated results from the completed Phase 1/2 Northstar (HGB-204) study, will be presented at ASH. The company will also present new data from the ongoing Phase 3 Northstar-2 (HGB-207) study in pediatric, adolescent and adult patients who do not have a 0/0 genotype and from the ongoing Phase 3 Northstar-3 (HGB-212) study in pediatric, adolescent and adult patients who have 0/0 genotype or an IVS-I-110 mutation at both alleles of the -globin gene.

New data from the companys Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD will include additional patients treated in the study and updated data for those previously reported. The company will also present data from exploratory assays designed to assess the relationship between drug product characteristics and red blood cell physiology in patients treated with LentiGlobin for SCD.

Updated Data from Ongoing Phase 1 Clinical Study (CRB-402) of bb21217

Updated Results from an Ongoing Phase 1 Clinical Study of bb21217 Anti-BCMA CAR T Cell Therapy Presenting Author: Jesus G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville, Tenn. Date & Time: Oral #927, Monday, December 9, 2019, 6:45 p.m. ET

bb21217, an investigational BCMA-targeted CAR T cell therapy being developed in partnership with Celgene, is one of bluebird bios lead oncology programs. bb21217 uses the idecabtagene vicleucel CAR molecule (formerly referred to as bb2121) and is manufactured with a process intended to increase the in vivo persistence of CAR T cells.

This presentation will include updated data from the Phase 1 CRB-402 study, the first-in-human study of bb21217 in patients with RRMM, designed to assess the primary endpoint of safety as well as other pre-defined endpoints including efficacy and pharmacokinetics measurements. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM with a projected final enrollment of 74 patients.

Data in the abstract include results as of the data cutoff date of April 20, 2019 for 22 patients who have received bb21217 at three dose levels (12 at 150 x 106 CAR+ T cells; six at 300 x 106 CAR+ T cells; and four at 450 x 106 CAR+ T cells). These patients had a median of seven prior lines of therapy (min-max: 4 17 lines), 18 patients had a prior autologous stem cell transplant, 19 patients received daratumumab and 13 patients received prior treatment with bortezomib, lenalidomide, carfilzomib, pomalidomide and daratumumab.

As of the data cutoff, the adverse events observed were consistent with known toxicities of CAR T therapies. Thirteen of 22 patients developed cytokine release syndrome (CRS); five Grade 1, seven Grade 2, and one Grade 3 case. All 13 patients responded to supportive care, tocilizumab and/or corticosteroids. Five of 22 patients developed neurotoxicity; one Grade 1, two Grade 2, one Grade 3 (vertigo/dizziness), and one Grade 4 (encephalopathy, previously reported). For the one patient previously reported with Grade 4 neurotoxicity, Grade 3 CRS was also reported, and both have resolved.

Eighteen patients were evaluable for clinical response with > two months of follow-up or progressive disease within two months. Eighty-three percent (n=15/18) of evaluable patients demonstrated clinical response per the International Myeloma Working Group Uniform Response Criteria for multiple myeloma. As of the data cutoff, with the median follow-up after bb21217 infusion of five months (min-max: <1 18 months), nine patients remained in response, including two patients with ongoing response at 15 and 18 months.

Evidence of myeloma in the bone marrow, known as minimal residual disease, was undetectable by next-generation sequencing at a sensitivity level of 10-5 or better in all responders who had evaluable bone marrow samples (n=10) at Month 1. CAR T cell persistence was observed in six of eight patients evaluable at six months and in two of two patients evaluable at 12 months.

This study is ongoing to evaluate the potential safety and efficacy of treatment with bb21217, and updated results, including early clinical and CAR T cell persistence data, will be shared at the ASH conference.

Multiple Myeloma Presentations at ASH

Markers of Initial and Long-Term Responses to Idecabtagene Vicleucel (Ide-Cel; bb2121) in the CRB-401 Study in Relapsed/Refractory Multiple Myeloma Presenting Author: Ethan G. Thompson, Ph.D., Celgene, Seattle, Wash. Date & Time: Poster #4328, Monday, December 9, 2019, 6:00 8:00 p.m. ET

Updated Results from an Ongoing Phase 1 Clinical Study of bb21217 anti-BCMA CAR T Cell Therapy Presenting Author: Jesus G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville, Tenn. Date & Time: Oral #927, Monday, December 9, 2019, 6:45 p.m. ET

SCD Presentations at ASH

The Relationships Between Target Gene Transduction, Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene Therapy Presenting Author: Melissa Bonner, Ph.D., bluebird bio, Cambridge, Mass. Date & Time: Oral #206, Saturday, December 7, 2019, 2:15 p.m.

Exploring the Drivers of Clinical Benefit in Initial Patients Treated in the HGB-206 Study of LentiGlobin for Sickle Cell Disease (SCD) Gene Therapy Presenting Author: Mark Walters, M.D., Benioff Childrens Hospital, Oakland, Calif. Date & Time: Poster #2061, Saturday, December 7, 2019, 5:30 7:30 p.m.

Resolution of Sickle Cell Disease Manifestations in Patients Treated with LentiGlobin Gene Therapy: Updated Results from the Phase 1/2 HGB-206 Group C Study Presenting Author: Julie Kanter, M.D., University of Alabama at Birmingham, Birmingham, Ala. Date & Time: Poster #990, Saturday, December 7, 2019, 5:30 7:30 p.m.

TDT Presentations at ASH

Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Transfusion-Dependent -Thalassemia Treated at the Bambino Ges Childrens Hospital, Rome, Italy Presenting Author: Pietro Merli, M.D., IRCCS Ospedale Pediatrico Bambino Ges, Rome, Italy Date & Time: Poster #969, Saturday, December 7, 2019, 5:30 7:30 p.m.

Northstar-3: Interim Results from a Phase 3 Study Evaluating LentiGlobin Gene Therapy in Patients with Transfusion-Dependent -Thalassemia and Either a 0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene Presenting Author: Ashutosh Lal, M.D., UCSF Benioff Childrens Hospital, Oakland, Calif. Date & Time: Oral #815, Monday, December 9, 2019, 5:30 p.m.

Northstar-2: Updated Safety and Efficacy Analysis of LentiGlobin Gene Therapy in Patients with Transfusion-Dependent -Thalassemia and Non-0/0Genotypes Presenting Author: Alexis Thompson, M.D., MPH, Ann & Robert H. Lurie Childrens Hospital of Chicago, Chicago, Ill. Date & Time: Poster #3543, Monday, December 9, 2019, 6:00 8:00 p.m.

Long-Term Clinical Outcomes of LentiGlobin Gene Therapy for Transfusion-Dependent -Thalassemia in the Northstar (HGB-204) Study Presenting Author: Janet Kwiatkowski, M.D., MSCE, Childrens Hospital of Philadelphia, Philadelphia, Pa. Date & Time: Poster #4628, Monday, December 9, 2019, 6:00 8:00 p.m.

Routine Management, Healthcare Resource Use and Patient/Caregiver-Reported Outcomes of Patients with Transfusion-Dependent -Thalassaemia in the United Kingdom: A Mixed Methods Observational Study Presenting Author: Farrukh Shah, MBBS, FRCP, FRCPath, M.D., Whittington Hospital, London, U.K. Date & Time: Poster #3550, Monday, December 9, 2019, 6:00 8:00 p.m.

SCD and TDT Presentation at ASH

Results from the Completed HGB-205 Trial of LentiGlobin for -Thalassemia and LentiGlobin for Sickle Cell Disease Gene Therapy Presenting Author: Elisa Magrin, Ph.D., Necker Childrens Hospital, Assistance Publique-Hpitaux de Paris, Paris, France Date & Time: Poster #3358, Sunday, December 8, 2019, 6:00 8:00 p.m.

Abstracts outlining bluebird bios accepted data at ASH will be available on the ASH conference website at 9 a.m. EST today.

About ide-cel and bb21217 for Multiple Myeloma

bluebird bios lead oncology programs, idecabtagene vicleucel (ide-cel, formerly referred to as bb2121) and bb21217, are investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapies being studied in a broad clinical development program for patients with multiple myeloma. ide-cel and bb21217 are being developed in partnership with Celgene.

KarMMa is a registration-enabling, open-label, single-arm, multi-center Phase 2 study evaluating the efficacy and safety of ide-cel in patients with relapsed/refractory multiple myeloma. In November 2018, bluebird bio announced completion of enrollment in the trial. ide-cel was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration and Priority Medicines (PRIME) eligibility by the European Medicines Agency in November 2017 based on preliminary clinical data from the Phase 1 CRB-401 study.

bluebird bios clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with RRMM, designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM with a projected final enrollment of 74 patients. For more information visit: clinicaltrials.gov using identifier NCT03274219.

ide-cel and bb21217 are not approved for any indication in any geography.

About LentiGlobin for Sickle Cell Disease

LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.

The U.S. Food and Drug Administration granted Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/ or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About LentiGlobin for -Thalassemia

The European Commission granted conditional marketing authorization for LentiGlobin for TDT, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or absent hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

LentiGlobin adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived-hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to LentiGlobin for TDT were hot flush, dyspnoea, abdominal pain, pain in extremities and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted LentiGlobin for -thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

LentiGlobin for -thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for TDT. For more information visit: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/ or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Companys views with respect to the potential for LentiGlobin to treat transfusion-dependent -thalassemia and sickle cell disease, the potential for the bb21217 product candidate to treat relapsed/ refractory multiple myeloma. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of our product candidates will not continue or be repeated in our ongoing or planned clinical trials or in the commercial context, risks that the current or planned clinical trials of our product candidates will be insufficient to support future regulatory submissions or to support marketing approval in the US and EU, and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191106005511/en/

Contacts

Investors: Elizabeth Pingpank, 617-914-8736 epingpank@bluebirdbio.com or Media: Catherine Falcetti, 339-499-9436 cfalcetti@bluebirdbio.com

Continue reading here:
bluebird bio to Present New Data from Gene and Cell Therapy Programs at 61st American Society of Hematology Annual Meeting and Exposition - Financial...

Cardiac Stem Cells Comments Off on bluebird bio to Present New Data from Gene and Cell Therapy Programs at 61st American Society of Hematology Annual Meeting and Exposition – Financial… | November 6th, 2019

This is therefore a comparing of the institutional ownership, earnings and valuation, profitability, risk, dividends, analyst recommendations in Capricor Therapeutics Inc. (NASDAQ:CAPR) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI). The two are both Biotechnology companies that compete with one another.

Earnings and Valuation

Demonstrates Capricor Therapeutics Inc. and Brainstorm Cell Therapeutics Inc. earnings per share, top-line revenue and valuation.

Profitability

Table 2 represents Capricor Therapeutics Inc. (NASDAQ:CAPR) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)s return on assets, return on equity and net margins.

Volatility and Risk

A beta of 1.8 shows that Capricor Therapeutics Inc. is 80.00% more volatile than Standard & Poors 500. Brainstorm Cell Therapeutics Inc. has a 1.19 beta and it is 19.00% more volatile than Standard & Poors 500.

Liquidity

Capricor Therapeutics Inc.s Current Ratio and Quick Ratio are 5.3 and 5.3 respectively. The Current Ratio and Quick Ratio of its competitor Brainstorm Cell Therapeutics Inc. are 1 and 1 respectively. Capricor Therapeutics Inc. therefore has a better chance of paying off short and long-term obligations compared to Brainstorm Cell Therapeutics Inc.

Analyst Ratings

The following table given below contains the ratings and recommendations for Capricor Therapeutics Inc. and Brainstorm Cell Therapeutics Inc.

$11.5 is Capricor Therapeutics Inc.s average target price while its potential upside is 342.31%. Competitively Brainstorm Cell Therapeutics Inc. has a consensus target price of $9, with potential upside of 134.99%. The data from earlier shows that analysts belief suggest that Capricor Therapeutics Inc. seems more appealing than Brainstorm Cell Therapeutics Inc.

Institutional and Insider Ownership

Capricor Therapeutics Inc. and Brainstorm Cell Therapeutics Inc. has shares owned by institutional investors as follows: 5.1% and 11.4%. Insiders owned 32.93% of Capricor Therapeutics Inc. shares. On the other hand, insiders owned about 0.6% of Brainstorm Cell Therapeutics Inc.s shares.

Performance

Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Capricor Therapeutics Inc. has weaker performance than Brainstorm Cell Therapeutics Inc.

Summary

Brainstorm Cell Therapeutics Inc. beats on 7 of the 10 factors Capricor Therapeutics Inc.

Capricor Therapeutics, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of novel therapeutics primarily for the treatment of cardiovascular diseases. The companys development stage drug candidates for cardiovascular diseases include CAP-1002 that is in Phase II clinical trials; and CAP-2003, which is in pre-clinical development for the treatment of certain cardiac and inflammatory conditions. The company was founded in 2005 and is headquartered in Beverly Hills, California.

Brainstorm Cell Therapeutics Inc., a biotechnology company, develops adult stem cell therapies for neurodegenerative disorders that include amyotrophic lateral sclerosis, multiple sclerosis, Parkinsons disease, and others. The company holds rights to develop and commercialize its NurOwn technology through a licensing agreement with Ramot of Tel Aviv University Ltd. Its NurOwn technology is based on a novel differentiation protocol, which induces differentiation of the bone marrow-derived mesenchymal stem cells into neuron-supporting cells and secreting cells that release various neurotrophic factors, including glial-derived neurotrophic factor, brain-derived neurotrophic factor, vascular endothelial growth factor, and hepatocyte growth factor for the growth, survival, and differentiation of developing neurons. The company was formerly known as Golden Hand Resources Inc. and changed its name to Brainstorm Cell Therapeutics Inc. in November 2004 to reflect its new line of business in the development of novel cell therapies for neurodegenerative diseases. Brainstorm Cell Therapeutics Inc. was founded in 2000 and is headquartered in Hackensack, New Jersey.

Receive News & Ratings Via Email - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings with our FREE daily email newsletter.

Read more:
Reviewing Capricor Therapeutics Inc. (CAPR)'s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)'s results - FinanceMercury

Cardiac Stem Cells Comments Off on Reviewing Capricor Therapeutics Inc. (CAPR)’s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)’s results – FinanceMercury | November 6th, 2019

A potentially life shattering diagnosis turned into motivation to help others. A Peoria woman is hosting a Be the Match event at the Greater Peoria YMCA on Wednesday.

While many might say Why me? when diagnosed with a rare bone marrow cancer, Marsha Krone continued to fight on, choosing to make the most of the life she has been given.

A mother of three, grandmother of two, lover of sewing, and teacher for over 30 years, Marsha Krone had a lot to smile about. Until an existing blood disorder threatened to steal her joy.

I went to the Mayo Clinic and they did a bone marrow biopsy and they told me that yes, you have progressed to Myelofibrosis. And so what that means is my bone marrow is essentially turning hard. said Marsha Krone, diagnosed in 2016

Krone needs a stem cell transplant. Her sisters were not a match.Despite millions of donors being listed on the registry she is still waiting for the perfect stranger. However of the ten markers needed to match, two are extremely rare, making it difficult, but not impossible to find one.

Her hope comes from faith.

I cant write my story because he writes my story and I just can choose how Im going to live it. So I choose joy. said Krone

As an ambassador for Be the Match she has put on numerous events, including the one coming up at the Greater Peoria YMCA where shes been a member for many years.

We talked about how could we help in this way and she said well lets hold a match event and try to get as many people engaged as we possibly can. Shes not doing this for herself, shes doing this for everyone out there that needs to find a match said President and CEO of the Greater Peoria YMCA, Andy Thornton

The process is simple. You swab your cheek for DNA, provide your contact information, and join the registry where you could match anyone in the world for stem cells and bone marrow.

If someone else was matched from a drive we promoted, that would be really exciting for me to know that I was the go between to helping someones life be saved. said Krone.

The event takes place Wednesday 11/6 from 3 7 P.M. at The Greater Peoria YMCA

Read more here:
Using her diagnosis to help others, Peoria woman to host 'Be the Match' event - week.com

Bone Marrow Stem Cells Comments Off on Using her diagnosis to help others, Peoria woman to host ‘Be the Match’ event – week.com | November 5th, 2019

Shahid Akhter, editor, ETHealthworld spoke to Dr Na'eem Sadiq, Medical Director, PLEXUS NEURO and STEM CELL RESEARCH CENTER, Bengaluru to know more about stem cell therapy and the challenges associated with it.

STEM CELL : TRENDSStem cell is a word which evokes lot of responses both positive and negative. Few people know that the origin was in the 1800s. The very first bone marrow transplant happened in the year 1968 and then subsequently stem cells have been used for various diseases and much more in blood cancer. They have also been used in chronic neurological disorders, autoimmune disorders and sports injuries.Globally, its all over the world such as in the US, Canada, Germany, China, Ukraine and of course in India as well. In India there are lots of centers and states who have been practicing stem cell technology for quite some time.

STEM CELLS : MARKETThe market is growing since stems cells promise hope for those who have lost hope, where there is no viable treatment and proper cure available for lots of diseases. Stem cells is emerging as a champion for all these people. It was much more available internationally and in the last decade India has taken up.

PLEXUS NEURO AND STEM CELL RESEARCH CENTER- JOURNEYI have been practicing in the field of neuroscience for the last 30 years. Neurosciences is a field where you see patients suffering from chronic diseases. I have been in this field right from early 90s and have been seeing trends changing, but when it comes to neurodegenerative disorders such as Parkinsons, ALS, Multiple Sclerosis, billions of dollars have been spent, new treatment modalities have been found, but nothing has been found to be successful.

We have very strict and rigid eligibility criteria. Once the patient approaches us, we subject the patient to a thorough clinical examination, which lasts anywhere between 2- 3 hours. Once we find that the patient is clinically treatable, or that the patient can be helped, then we subject the patient to other investigations.

The other major difference we have at Plexus is that we do not do only stem cells. Stem cell therapy is a part of our complete regenerative rehabilitation. The program starts after we do the transplant. The patient undergoes rigorous rehabilitation, which includes the entire gamete of practices such as physical therapy, occupational therapy, hand splinting, cognitive rehabilitation therapy, cognitive behavior therapy, speech therapy etc.

We customize and provide a tailor made program as per the patient's needs, with a goal once the patient joins the program and almost all the patients who are in the program get more than what we had aimed at achieving. At the end of the program we evaluate the goals and find that every single patient achieves them. We train the patients as to what they need to do once they finish the program and insist on regular follow up.

We have a team of learned scientists who are all qualified from the UK and our research is ongoing. We are working exclusively in the field of neurosciences to get the best quality of cells and to make it very affordable. Research is on and our data is huge, we will be publishing the results very soon.

PLEXUS : FUTURE PLANSWe have a complete state of the art rehabilitation center where we have some of the best therapists in the world working with us. In fact a few months back we launched one of its kind, Sensory Gym at Plexus and now we have started virtual and augmented reality.

In the last 4-5 years we have received more than 75 national and international awards and we stand as one of the leading regenerative rehabilitation centers not only in India, but in Asia.Our endeavor here is to make the treatment the best possible, to make the cells much more advanced, affordable to also provide the treatment in the shortest possible time.

See more here:
Cost is a major challenge in stem cells therapy: Dr Na'eem Sadiq - ETHealthworld.com

Bone Marrow Stem Cells Comments Off on Cost is a major challenge in stem cells therapy: Dr Na’eem Sadiq – ETHealthworld.com | November 5th, 2019

Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.

The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.

Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.

Read more:
Three UCLA scientists receive grants totaling more than $18 million - UCLA Newsroom

Bone Marrow Stem Cells Comments Off on Three UCLA scientists receive grants totaling more than $18 million – UCLA Newsroom | November 5th, 2019

NEW YORK, Nov. 05, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Company will hold a conference call to update shareholders on financial results for the third quarter ended September 30, 2019, and provide a corporate update, at 8:00 a.m., Eastern Standard Time, on Thursday, November 14, 2019.

BrainStorms President & CEO, Chaim Lebovits, will present a corporate update, after which, participant questions will be answered. Joining Mr. Lebovits to answer investment community questions will be Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer, and Preetam Shah, PhD, Chief Financial Officer.

Participants are encouraged to submit their questions prior to the call by sending them to: q@brainstorm-cell.com; Questions should be submitted by 5:00 p.m., Eastern Standard Time, Tuesday, November 12.

The investment community may participate in the conference call by dialing the following numbers:

Those interested in listening to the conference call live via the internet may do so by visiting the Investors & Media page of BrainStorms website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.

A webcast replay of the conference call will be available for 30 days on the Investors & Media page of BrainStorms website:

About NurOwnNurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

Story continues

Read the rest here:
BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Comprehensive Corporate Update - Yahoo Finance

Bone Marrow Stem Cells Comments Off on BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Comprehensive Corporate Update – Yahoo Finance | November 5th, 2019

According to results of a study published in Blood, children and young adults with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) who had minimal residual disease (MRD) prior to treatment and received high-dose preconditioning chemotherapy were most likely to respond to a second-generation CD-19 chimeric antigen receptor-T cell (CAR-T) therapy.1

Although it has been estimated that 90% or more of pediatric patients witha diagnosis of ALL will respond to multi-agent chemotherapy, the prognosis forthose with relapsed/refractory disease remains poor. One CD19-directed CAR-Ttherapy, tisagenlecleucel, is approved by the US Food and Drug Administration inpatients up to age 25 years with B-cell precursor ALL who either have refractorydisease or have experienced a second or later relapse.2

This open label, nonrandomized, phase 1 study (Clinical Trial Identifier: NCT01860937), evaluated the toxicity, feasibility, and response of 19-28z CAR-T therapy, a second-generation CD19-directed CAR-T therapy involving T cells expressing a chimeric receptor composed of an anti-CD19 antibody binding site and intracellular domains from the T-cell coactivating receptors, CD28 and the CD3-zeta chain3 in children and young adults up to 25 years of age with very high-risk ALL.1 Inclusion criteria included at least 2 relapses, early bone marrow relapse following complete response (CR), intermediate/late CR with poor response to re-induction therapy, or those with refractory disease, or ineligibility for allogeneic hematopoietic stem cell transplantation (allo-HSCT) or additional chemotherapy.1

The age range of the 25 patients treated with 19-28z CAR-T therapy onstudy was 1 to 22.5 years, with a median age of 13.5 years. Preconditioningchemotherapy involved high-dose cyclophosphamide (15 patients) and low-dosecyclophosphamide (8 patients), with 3 patients in each subgroup also receivingfludarabine.1

Regarding the feasibility of this approach, the prespecified CAR-Tcell dose was achieved for all patients for whom the 19-28z CAR-T therapyprocedure was undertaken.1

With respect to treatment toxicity,approximately one-third of patients experienced a grade 3/4 adverse event,including cytokine release syndrome (CRS) and neurotoxicity in 16% and 28% ofpatients, respectively. With the exception of 1 patient with grade 4 CRS andneurotoxicity who died following refractory Stenotrophomonas septic shock,these adverse events were reversible.1

Of the 24 patients includedin the response analysis, 75% achieved either a CR or a CR with incompletecount recovery (CRi). In the subsets of patients receiving preconditioningchemotherapy with either high- or low-dose cyclophosphamide, the CR/CRi rateswere 94% and 38%, respectively. Furthermore, treatment response was influencedby disease burden as evidenced by the considerably higher CR/CRi rate inpatients with baseline minimal residual disease (ie, less than 5% bone marrowblasts; 93%) compared with morphological evidence of disease at baseline (5% orhigher bone marrow blasts; 50%).1

The CR/CRi rate for thesubset of patients with pretreatment MRD treated with high-dose cytarabine was100%.1

Consolidation allo-HSCT was performed in 83% (15) of the patientsresponding to CAR-T therapy, with a median time from CAR-T infusion toallo-HSCT of 57 days. At a median follow-up of 28.6 months for respondingpatients, over half of these patients (8) were alive and had no evidence ofdisease.1

In their concluding remarks, the study authorscommented that thisanalysis has allowed us to determine the toxicity profile, confirm feasibility,evaluate response of this approach, and provide a direct comparison of the sameCD19-specific CAR T cell product that was previously published[3] inadult patients for the same indication.

The authors went on to highlight the findingof a reversible toxicity profile in the patients within their study as well asthe impact of preconditioning chemotherapy dose intensity and minimal pretreatmentdisease burden on response.

They further noted that within this cohort,the long-term persistence of response is encouraging, and in our primarilytransplant-naive patient population, the ability to proceed to allo-HSCT hasdemonstrated a favorable overall survival, manageable toxicity, and limitedincidence of relapse.

References

Read the original:
19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results - Cancer Therapy Advisor

Bone Marrow Stem Cells Comments Off on 19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results – Cancer Therapy Advisor | November 5th, 2019

CHICAGO, Nov.5, 2019 /PRNewswire/ -- Scientists from Dystrogen Therapeutics Corp. published data supporting cardioprotective effects of the Company's therapy for muscular dystrophy disorders. Cardiomyopathy is the most devastating cause of morbidity and mortality in Duchenne Muscular Dystrophy (DMD) patients and affects 30% of patients by 14years of age and 50% of patients by 18years of age. Heart failure in these patients is the result of cardiac myocyte death and fibrosis, leading to both diastolic and systolic dysfunction. Dystrogen Therapeutics Corp has developed an engineered chimeric cell therapy which has been previously shown to restore muscle function in pre-clinical studies. For Duchenne's muscular dystrophy, the company has developed dystrophin expressing chimeras "DECs." Using the company's proprietary technology, DECs are created by an ex vivo fusion of allogeneic human myoblast from a healthy donor with autologous human myoblast received from DMD patient. DECs have been shown to maintain the ability to express normal dystrophin protein in previously published pre-clinical studies. The new study published in the October 15th, 2019 online edition of the journal Stem Cell Reports and Reviewsconfirmed the protective effect of DEC on cardiac function after intraosseous delivery shown by increased values of both ejection fraction and fractional shortening, which at 90days revealed a rebound effect when compared to the vehicle injected controls and mice receiving not-chimeric cell therapy. Moreover, these functional improvements correlated with restoration of dystrophin expression in cardiac muscle at 90days post-DEC treatment.

"These findings are potentially significant for the treatment of DMD," said Dr. Maria Siemionow, MD, PhD Dystrogen Therapeutics Corp chief scientific officer and the therapy's inventor. "This study establishes DEC as a promising new option for cardiac protection and potential amelioration of DMD related cardiac pathology."

"These data add to the growing body of literature supporting the potential of our chimeric cell platform to restore systemic muscle function, with less potential side effects then gene therapy-based approaches," said Dr. Kris Siemionow, MD, PhD Dystrogen Therapeutics Corp CEO. "We are very pleased to have these data published in a highly relevant journal for the field and look forward to further exploring this opportunity."

About Dystrogen Therapeutics

Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing personalized therapies for rare diseases. The company has developed a chimeric cell therapy platform. Dystrophin expressing chimeras "DEC" are based on ex vivo fusion of allogeneic human myoblast derived from donors with autologous human myoblast received from the DMD patient, where chimeric cells maintain the ability to express normal dystrophin protein. DEC cells increase the number/pool of normal myoblasts and reduce inflammation. DEC cells induce replacement of fibrotic tissue, thus significantly improving muscle strength and function in DMD pre-clinical studies. The therapy minimizes immune response effects and the need for immunosuppression. This new approach will be based on delivery and restoration of dystrophin in affected muscles preventing the premature loss of mobility and early mortality of DMD patients. The company is planning on enrolling patients for its DEC chimeric cell therapy Duchenne muscular dystrophy trial. This therapy offers a unique advantage and allows the patient's body and immune system to accept the chimeric cells without rejection. Pre-clinical results have demonstrated that increased dystrophin levels correlate with improved functional outcomes. First clinical results from DEC therapy are expected in late 2020.

Contact: info@dystrogen.com

View original content:http://www.prnewswire.com/news-releases/dystrogen-therapeutics-announces-that-treatment-with-dystrophin-expressing-chimeric-dec-cells-improves-cardiac-function-in-preclinical-duchennes-study-300951205.html

SOURCE Dystrogen Therapeutics Corp

Visit link:
Dystrogen Therapeutics Announces That Treatment With Dystrophin Expressing Chimeric (DEC) Cells Improves Cardiac Function in Preclinical Duchenne's...

Cardiac Stem Cells Comments Off on Dystrogen Therapeutics Announces That Treatment With Dystrophin Expressing Chimeric (DEC) Cells Improves Cardiac Function in Preclinical Duchenne’s… | November 5th, 2019

A Qualitative Research Study done by Crystal Market Research on Global Autologous Stem Cell Based Therapies Market report provides current and future trends are outlined to determine the overall attractiveness and to single out profitable Autologous Stem Cell Based Therapies trends to gain a stronger position in Industry anticipated to reflect a positive growth trend in Upcoming years as well. Global Autologous Stem Cell Based Therapies market provide in depth coverage from various aspects and scenario to future trends and opportunities. This Autologous Stem Cell Based Therapies report provide latest customized and syndicated research along with consulting services.

Get FREE Research Sample Copy @https://www.crystalmarketresearch.com/report-sample/HC022437

The detailed study of global Autologous Stem Cell Based Therapies market report portraying the current landscape and important projections for market forecast, growth trend. This report is an whole guide for new aspirant to understand the trending values and Autologous Stem Cell Based Therapies future trend. The report arranged dependent on a top to bottom market examination with contributions from industry expert.

Continue

Get Discount of This Precious Report @ https://www.crystalmarketresearch.com/check-discount/HC022437

Autologous Stem Cell Based Therapies report studies market share and growth opportunity covering regions:

Get Customised Research Report: https://www.crystalmarketresearch.com/customization/HC022437

Contacts Us:

Judy | 304 South Jones Blvd, Suite 1896

Las Vegas NV 89107

United States

E-mail: sales@crystalmarketresearch.com | Ph: +1-888-213-4282

Excerpt from:
Future Outlook: Autologous Stem Cell Based Therapies Market Prediction and Analysis Offered By New Study 2019 2025: Leading Key Players: JCR...

Cardiac Stem Cells Comments Off on Future Outlook: Autologous Stem Cell Based Therapies Market Prediction and Analysis Offered By New Study 2019 2025: Leading Key Players: JCR… | November 5th, 2019