Page 20«..10..19202122..3040..»

BioVie Interview to Air on Bloomberg TV in the US on the RedChip Money Report®

By Dr. Matthew Watson

SANTA MONICA, Calif., Feb. 19, 2021 (GLOBE NEWSWIRE) -- BioVie Inc. (NASDAQ: BIVI) ("BioVie" or "Company"), a clinical-stage company developing innovative drug therapies for liver disease, today announced an interview with Terren Peizer, Chairman and Chief Executive Officer, will air on The RedChip Money Report® on Bloomberg TV in the US on Saturday, February 20, at 7 p.m. local time in 73M homes across the United States. The RedChip Money Report® also airs on Bloomberg International in Europe in 100M homes at 6 pm local time on Sundays.

See original here:
BioVie Interview to Air on Bloomberg TV in the US on the RedChip Money Report®

To Read More: BioVie Interview to Air on Bloomberg TV in the US on the RedChip Money Report®
categoriaGlobal News Feed commentoComments Off on BioVie Interview to Air on Bloomberg TV in the US on the RedChip Money Report® | dataFebruary 19th, 2021
Read All

Clene Nanomedicine to Present Updated Interim Data from Phase 2 Multiple Sclerosis Programs at ACTRIMS Forum 2021

By Dr. Matthew Watson

SALT LAKE CITY, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Clene Inc. (NASDAQ: CLNN) (along with its subsidiaries, “Clene”) and its wholly owned subsidiary Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company, today announced that it will present updated interim data from its REPAIR-MS study and updated blinded interim data from its VISIONARY-MS study in poster presentations at the Americas Committee for Treatment and Research in Multiple Sclerosis’ ACTRIMS Forum 2021 (“ACTRIMS”) taking place virtually from February 25-27, 2021.

Go here to read the rest:
Clene Nanomedicine to Present Updated Interim Data from Phase 2 Multiple Sclerosis Programs at ACTRIMS Forum 2021

To Read More: Clene Nanomedicine to Present Updated Interim Data from Phase 2 Multiple Sclerosis Programs at ACTRIMS Forum 2021
categoriaGlobal News Feed commentoComments Off on Clene Nanomedicine to Present Updated Interim Data from Phase 2 Multiple Sclerosis Programs at ACTRIMS Forum 2021 | dataFebruary 19th, 2021
Read All

Precision Announces 3-Year Pre-clinical Study Results Showing Long-term Durability and Safety of ARCUS In Vivo Gene Editing to Cut LDL Cholesterol…

By Dr. Matthew Watson

Study Led by Gene Therapy Program at the University of Pennsylvania Published in Molecular Therapy Study Led by Gene Therapy Program at the University of Pennsylvania Published in Molecular Therapy

Read more:
Precision Announces 3-Year Pre-clinical Study Results Showing Long-term Durability and Safety of ARCUS In Vivo Gene Editing to Cut LDL Cholesterol...

To Read More: Precision Announces 3-Year Pre-clinical Study Results Showing Long-term Durability and Safety of ARCUS In Vivo Gene Editing to Cut LDL Cholesterol…
categoriaGlobal News Feed commentoComments Off on Precision Announces 3-Year Pre-clinical Study Results Showing Long-term Durability and Safety of ARCUS In Vivo Gene Editing to Cut LDL Cholesterol… | dataFebruary 19th, 2021
Read All

Imara to Present at SVB Leerink 10th Annual Global Healthcare Conference

By Dr. Matthew Watson

BOSTON, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Imara Inc. (Nasdaq: IMRA), a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin, today announced that Rahul Ballal, Ph.D., President and Chief Executive Officer, will participate in a Fireside Chat at the SVB Leerink 10th Annual Global Healthcare Conference on Friday, February 26, from 1:40-2:10 p.m. ET.

Follow this link:
Imara to Present at SVB Leerink 10th Annual Global Healthcare Conference

To Read More: Imara to Present at SVB Leerink 10th Annual Global Healthcare Conference
categoriaGlobal News Feed commentoComments Off on Imara to Present at SVB Leerink 10th Annual Global Healthcare Conference | dataFebruary 19th, 2021
Read All

Pfizer and BioNTech Submit COVID-19 Vaccine Stability Data at Standard Freezer Temperature to the U.S. FDA

By Dr. Matthew Watson

NEW YORK and MAINZ, GERMANY, February 19, 2021 (GLOBE NEWSWIRE) — Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) today announced the submission of new data to the U.S. Food and Drug Administration (FDA) demonstrating the stability of their COVID-19 vaccine when stored at -25°C to -15°C (-13°F to 5°F), temperatures more commonly found in pharmaceutical freezers and refrigerators. The data have been submitted to the FDA to support a proposed update to the U.S. Emergency Use Authorization (EUA) Prescribing Information, which would allow for vaccine vials to be stored at these temperatures for a total of two weeks as an alternative or complement to storage in an ultra-low temperature freezer.

Visit link:
Pfizer and BioNTech Submit COVID-19 Vaccine Stability Data at Standard Freezer Temperature to the U.S. FDA

To Read More: Pfizer and BioNTech Submit COVID-19 Vaccine Stability Data at Standard Freezer Temperature to the U.S. FDA
categoriaGlobal News Feed commentoComments Off on Pfizer and BioNTech Submit COVID-19 Vaccine Stability Data at Standard Freezer Temperature to the U.S. FDA | dataFebruary 19th, 2021
Read All

BridgeBio Pharma and Affiliate ML Bio Solutions Announce Dosing of First Patient in Phase 2 Trial of BBP-418 in Limb Girdle Muscular Dystrophy Type 2i…

By Dr. Matthew Watson

SAN FRANCISCO, Feb. 19, 2021 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO),?a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers,?and affiliate ML Bio Solutions today announced that the first patient has been dosed in a Phase 2 trial of BBP-418 in patients with LGMD2i. BridgeBio and ML Bio’s BBP-418 is the first-ever oral disease-modifying investigational treatment for LGMD2i. BBP-418 was granted Orphan Drug Designation for LGMD2i by the US Food and Drug Administration (FDA) in 2019, and for LGMD by the European Medicines Agency (EMA) in 2020.

Original post:
BridgeBio Pharma and Affiliate ML Bio Solutions Announce Dosing of First Patient in Phase 2 Trial of BBP-418 in Limb Girdle Muscular Dystrophy Type 2i...

To Read More: BridgeBio Pharma and Affiliate ML Bio Solutions Announce Dosing of First Patient in Phase 2 Trial of BBP-418 in Limb Girdle Muscular Dystrophy Type 2i…
categoriaGlobal News Feed commentoComments Off on BridgeBio Pharma and Affiliate ML Bio Solutions Announce Dosing of First Patient in Phase 2 Trial of BBP-418 in Limb Girdle Muscular Dystrophy Type 2i… | dataFebruary 19th, 2021
Read All

Vaccinex Announces Signing of Two Multi-Project Deals with Leading Pharmaceutical Companies Focused on Leveraging Its ActivMAb® Platform

By Dr. Matthew Watson

Partnerships will use Vaccinex’s ActivMAb® platform for viral display of complex antigens to enable antibody discovery Partnerships will use Vaccinex’s ActivMAb® platform for viral display of complex antigens to enable antibody discovery

See the rest here:
Vaccinex Announces Signing of Two Multi-Project Deals with Leading Pharmaceutical Companies Focused on Leveraging Its ActivMAb® Platform

To Read More: Vaccinex Announces Signing of Two Multi-Project Deals with Leading Pharmaceutical Companies Focused on Leveraging Its ActivMAb® Platform
categoriaGlobal News Feed commentoComments Off on Vaccinex Announces Signing of Two Multi-Project Deals with Leading Pharmaceutical Companies Focused on Leveraging Its ActivMAb® Platform | dataFebruary 19th, 2021
Read All

Mersana Therapeutics to Host Conference Call Announcing Fourth Quarter and Year End 2020 Financial Results and Business Updates

By Dr. Matthew Watson

CAMBRIDGE, Mass., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Mersana Therapeutics, Inc. (NASDAQ:MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, today announced that it will host a conference call and webcast on Friday, February 26, 2021 at 8:00 a.m. ET to report financial results for the fourth quarter and year ended December 31, 2020 and provide business updates.

Read more:
Mersana Therapeutics to Host Conference Call Announcing Fourth Quarter and Year End 2020 Financial Results and Business Updates

To Read More: Mersana Therapeutics to Host Conference Call Announcing Fourth Quarter and Year End 2020 Financial Results and Business Updates
categoriaGlobal News Feed commentoComments Off on Mersana Therapeutics to Host Conference Call Announcing Fourth Quarter and Year End 2020 Financial Results and Business Updates | dataFebruary 19th, 2021
Read All

Intercept to Present at Upcoming Investor Conferences

By Dr. Matthew Watson

NEW YORK, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that Jerry Durso, President and Chief Executive Officer, and Sandip Kapadia, Chief Financial Officer of Intercept, will present at the SVB Leerink 10th Annual Global Healthcare Conference on Friday, February 26, 2021 at 1:00 p.m. ET, and at the Cowen 41st Annual Healthcare Conference on Tuesday, March 2, 2021 at 10:30 a.m. ET.

See the original post:
Intercept to Present at Upcoming Investor Conferences

To Read More: Intercept to Present at Upcoming Investor Conferences
categoriaGlobal News Feed commentoComments Off on Intercept to Present at Upcoming Investor Conferences | dataFebruary 19th, 2021
Read All

RAPT Therapeutics to Present at the 10th Annual SVB Leerink Global Healthcare Conference

By Dr. Matthew Watson

SOUTH SAN FRANCISCO, Calif., Feb. 19, 2021 (GLOBE NEWSWIRE) -- RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in oncology and inflammatory diseases, today announced that Brian Wong, M.D., Ph.D., President and Chief Executive Officer, will present at the 10th Annual SVB Leerink Global Healthcare Conference on Friday, February 26, 2021 at 12:00 pm Eastern Time.

See more here:
RAPT Therapeutics to Present at the 10th Annual SVB Leerink Global Healthcare Conference

To Read More: RAPT Therapeutics to Present at the 10th Annual SVB Leerink Global Healthcare Conference
categoriaGlobal News Feed commentoComments Off on RAPT Therapeutics to Present at the 10th Annual SVB Leerink Global Healthcare Conference | dataFebruary 19th, 2021
Read All

Assembly Bio Announces Upcoming Investor Events

By Dr. Matthew Watson

SOUTH SAN FRANCISCO, Calif., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a clinical-stage biotechnology company developing innovative therapeutics targeting hepatitis B virus (HBV), today announced two upcoming investor events:

Read the original post:
Assembly Bio Announces Upcoming Investor Events

To Read More: Assembly Bio Announces Upcoming Investor Events
categoriaGlobal News Feed commentoComments Off on Assembly Bio Announces Upcoming Investor Events | dataFebruary 19th, 2021
Read All

Yield10 Bioscience Announces Warrant Exercises for Proceeds of $3.8 million

By Dr. Matthew Watson

WOBURN, Mass., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Yield10 Bioscience, Inc. (Nasdaq:YTEN), an agricultural bioscience company, today announced that it has received proceeds of approximately $3.8 million from the exercise of 472,099 warrants exercised between January 1, 2021 and February 16, 2021, and the Company has issued to investors 472,099 shares of common stock in exchange for the exercised warrants. The warrants were issued to investors pursuant to a public offering that closed on November 19, 2019. Since November 19, 2019, a total of 679,395 warrants issued in the public offering have been exercised for total proceeds of $5.4 million. The Series A and Series B warrants issued in the November 2019 public offering are exercisable at $8.00 per share and have expiration dates of May 19, 2022 and May 19, 2027, respectively.

View original post here:
Yield10 Bioscience Announces Warrant Exercises for Proceeds of $3.8 million

To Read More: Yield10 Bioscience Announces Warrant Exercises for Proceeds of $3.8 million
categoriaGlobal News Feed commentoComments Off on Yield10 Bioscience Announces Warrant Exercises for Proceeds of $3.8 million | dataFebruary 19th, 2021
Read All

Atea Pharmaceuticals Announces Chugai In-License of AT-527 from Roche for the Treatment of COVID-19 in Japan

By Dr. Matthew Watson

Chugai obtains exclusive development and marketing rights for AT-527 in Japan from Roche, who has ex-US rights for the treatment of COVID-19 Chugai obtains exclusive development and marketing rights for AT-527 in Japan from Roche, who has ex-US rights for the treatment of COVID-19

See original here:
Atea Pharmaceuticals Announces Chugai In-License of AT-527 from Roche for the Treatment of COVID-19 in Japan

To Read More: Atea Pharmaceuticals Announces Chugai In-License of AT-527 from Roche for the Treatment of COVID-19 in Japan
categoriaGlobal News Feed commentoComments Off on Atea Pharmaceuticals Announces Chugai In-License of AT-527 from Roche for the Treatment of COVID-19 in Japan | dataFebruary 19th, 2021
Read All

CEO of Longeveron Inc. to Present at the Intercontinental Summit on Aging & Gerontology

By Dr. Matthew Watson

MIAMI, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and life-threatening conditions, today announced that Geoff Green, Chief Executive Officer of Longeveron, has been invited to give a presentation titled “A Regenerative Medicine Approach to Aging Frailty” at the Intercontinental Summit on Aging & Gerontology being held virtually on March 8, 2021. He will discuss Longeveron’s ongoing clinical research program in Aging Frailty and Alzheimer’s disease, in addition to the regenerative medicine investigational clinical research field in general for diseases and conditions associated with aging.

See the original post here:
CEO of Longeveron Inc. to Present at the Intercontinental Summit on Aging & Gerontology

To Read More: CEO of Longeveron Inc. to Present at the Intercontinental Summit on Aging & Gerontology
categoriaGlobal News Feed commentoComments Off on CEO of Longeveron Inc. to Present at the Intercontinental Summit on Aging & Gerontology | dataFebruary 19th, 2021
Read All

Agile Therapeutics to Host Fourth Quarter and Full Year 2020 Financial Results Conference Call and Provide Corporate and Twirla Commercial Updates on…

By Dr. Matthew Watson

Live Conference Call and Webcast at 4:30 p.m. ET

View original post here:
Agile Therapeutics to Host Fourth Quarter and Full Year 2020 Financial Results Conference Call and Provide Corporate and Twirla Commercial Updates on...

To Read More: Agile Therapeutics to Host Fourth Quarter and Full Year 2020 Financial Results Conference Call and Provide Corporate and Twirla Commercial Updates on…
categoriaGlobal News Feed commentoComments Off on Agile Therapeutics to Host Fourth Quarter and Full Year 2020 Financial Results Conference Call and Provide Corporate and Twirla Commercial Updates on… | dataFebruary 19th, 2021
Read All

Information expressed in the public domain by the Repharm Group is deceptive and contradictory

By Dr. Matthew Watson

Today, on February 19, information has arrived in the public domain that the company from Repharm Group will acquire the shares of JSC “Olainfarm” directly and indirectly owned by Anna Emilija Maligina.

Visit link:
Information expressed in the public domain by the Repharm Group is deceptive and contradictory

To Read More: Information expressed in the public domain by the Repharm Group is deceptive and contradictory
categoriaGlobal News Feed commentoComments Off on Information expressed in the public domain by the Repharm Group is deceptive and contradictory | dataFebruary 19th, 2021
Read All

Y-mAbs to Announce 2020 Financial and Operating Results on February 25, 2021

By Dr. Matthew Watson

NEW YORK, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB) today announced that it will report its financial results for the fiscal year ended December 31, 2020 on Thursday, February 25, 2021, after the close of the U.S. financial markets. The announcement will be followed by a conference call and webcast with the investment community on Friday, February 26, 2021, at 9 a.m. ET. Participating on the call from Y-mAbs will be Thomas Gad, founder, Chairman and President; Dr. Claus Moller, Chief Executive Officer; and Bo Kruse, Chief Financial Officer.

Here is the original post:
Y-mAbs to Announce 2020 Financial and Operating Results on February 25, 2021

To Read More: Y-mAbs to Announce 2020 Financial and Operating Results on February 25, 2021
categoriaGlobal News Feed commentoComments Off on Y-mAbs to Announce 2020 Financial and Operating Results on February 25, 2021 | dataFebruary 19th, 2021
Read All

Ayala Pharmaceuticals Announces $25 Million Strategic Financing

By Dr. Matthew Watson

Funding Extends Cash Runway through Multiple Expected Value Drivers Into 2023

Read more:
Ayala Pharmaceuticals Announces $25 Million Strategic Financing

To Read More: Ayala Pharmaceuticals Announces $25 Million Strategic Financing
categoriaGlobal News Feed commentoComments Off on Ayala Pharmaceuticals Announces $25 Million Strategic Financing | dataFebruary 19th, 2021
Read All

Stem Cells Market to Inspire a Growth up to US$ 18289.9 Million at a 9.1% CAGR by 2027 – PharmiWeb.com

By daniellenierenberg

Stem cells are undifferentiated cells which are capable of differentiating into any type of cell that make-up the human body and thus, are capable of producing non-regenerative cells such as neural and myocardial cells.

Statistics:

The global stem cells market is estimated to account forUS$ 9,941.2 Mnin terms of value in2020and is expected to reachUS$ 18,289.9 Mnby the end of2027.

For More Information, Request a sample copy of the Business Report: https://www.coherentmarketinsights.com/insight/request-sample/4222

GlobalStem CellsMarket: Drivers

Approval and launch of new products is expected to propel growth of the global stem cells market over the forecast period. For instance, in December 2019, BioRestorative Therapies, Inc. received a Notice of Allowance on its patent application for a method of generating brown fat stem cells from Israeli Patent Office.

Moreover, increasing number of stem cell banking resource centers is also expected to aid in growth of the market. For instance, in March 2020, Stemlife Berhad, a cord blood bank in Malaysia, started a Stem Cell Banking Resource Center in Jerudong Park Medical Center, Brunei.

Quick Buy Stem Cells Market Research Report: https://www.coherentmarketinsights.com/insight/buy-now/4222

Statistics:

Adult stem cells held dominant position in the global stem cells market in 2019, accounting for81.2%share in terms of value, followed by Human Embryonic Stem Cells and Induced Pluripotent Stem Cells, respectively

Figure 1. GlobalStem CellsMarket Share (%), by Value, by Cell Type, 2019.

GlobalStem CellsMarket: Restraints

High cost of stem cell therapy is expected to hinder growth of the global stem cells market. For instance, Bioinformant a research firm engaged in stem cell research, reported that the cost of stem cell therapy ranges between US$ 5,000-8,000 per patient and in some cases it may rise as much as US$ 25,000 or more depending on the complexity of the procedure.

Moreover, restrictions on research activities related to stem cells had hampered the growth of embryonic stem cells historically and resulted in its meager share in the total market in spite of its advantages over adult stem cells.

Got any Query? Feel free to ask us at : https://www.coherentmarketinsights.com/insight/talk-to-analyst/4222

GlobalStem CellsMarket: Opportunities

R&D in stem cell donation is expected to offer lucrative growth opportunities for players in the global stem cells market. For instance, in March 2020, researchers from Dankook University and Catholic University, South Korea, reported investigation of the types and degrees of physical and psychological discomfort experienced by hematopoietic stem cell donors before, during, and after the donation process.

Moreover, adoption of online distribution channel is also expected to aid in growth of the global stem cells market. For instance, The US Direct-to-Consumer Marketplace for Autologous Stem Cell Interventions, published in the journal Perspectives in Biology and Medicine, in 2018, the number of new stem cell businesses with websites doubled on average every year between 2009 and 2014, in the U.S.

The global stem cells market was valued atUS$ 9,112.0 Mnin2019and is forecast to reach a value ofUS$ 18,289.9 Mnby2027at aCAGR of 9.1%between2020 and 2027.

Figure 2. GlobalStem CellsMarket Value (US$ Mn), and Y-o-Y Growth (%), 2019-2027

Market Trends/Key Takeaways

Adoption of stem cells for the treatment of various diseases is expected to propel growth of the global stem cells market. For instance, in January 2020, researchers at University of Houston developed biologic cardiac pacemaker-like cells by taking fat stem cells and reprogramming them as an alternative treatment for heart conditions such as conduction system disorders and heart attacks.

Moreover, increasing investment in stem cell therapies is also expected to aid in growth of the market. For instance, in July 2018, the Emory Orthopaedics & Spine Center, in collaboration with Sanford Health, Duke University, Andrews Institute, and Georgia Institute of Technology, received US$ 13 million grant from the Marcus Foundation for a multicenter clinical trial studying stem cell options for treating osteoarthritis. The Phase 3 trial was initiated in March 2019, and is expected to complete by December 2021.

GlobalStem CellsMarket: Competitive Landscape

Major players operating in the global stem cells market include Advanced Cell Technology, Inc., Angel Biotechnology Holdings PLC, Bioheart Inc., Lineage Cell Therapeutics., BrainStorm Cell Therapeutics, Inc., California Stem Cell Inc., Celgene Corporation, Takara Bio Europe AB, Cellular Engineering Technologies, Cytori Therapeutics Inc., Osiris Therapeutics, and STEMCELL Technologies Inc.

View Press Release For More Information @ https://www.coherentmarketinsights.com/press-release/stem-cells-market-3491

GlobalStem CellsMarket: Key Developments

Major players in the market are focused on adopting collaboration and partnership strategies to expand their product portfolio. For instance, in September 2018, STEMCELL Technologies signed an exclusive license agreement with Brigham and Womens Hospital for rights to commercialize technologies for the generation of human pluripotent stem cell-derived kidney organoids.

Coherent Market Insights is a global market intelligence and consulting organization focused on assisting our plethora of clients achieve transformational growth by helping them make critical business decisions. We are headquartered in India, having office at global financial capital in the U.S. and sales consultants in United Kingdom and Japan. Our client base includes players from across various business verticals in over 150 countries worldwide. We pride ourselves in catering to clients across the length and width of the horizon, from Fortune 500 enlisted companies, to not-for-profit organization, and startups looking to establish a foothold in the market. We excel in offering unmatched actionable market intelligence across various industry verticals, including chemicals and materials, healthcare, and food & beverages, consumer goods, packaging, semiconductors, software and services, Telecom, and Automotive. We offer syndicated market intelligence reports, customized research solutions, and consulting services.

To know more about us, please visit our website http://www.coherentmarketinsights.com

Contact:

Coherent Market Insights1001 4th Ave, #3200 Seattle, WA 98154, U.S.Email:sales@coherentmarketinsights.comUnited States of America: +1-206-701-6702United Kingdom: +44-020-8133-4027Japan: +050-5539-1737India: +91-848-285-0837

Follow this link:
Stem Cells Market to Inspire a Growth up to US$ 18289.9 Million at a 9.1% CAGR by 2027 - PharmiWeb.com

To Read More: Stem Cells Market to Inspire a Growth up to US$ 18289.9 Million at a 9.1% CAGR by 2027 – PharmiWeb.com
categoriaCardiac Stem Cells commentoComments Off on Stem Cells Market to Inspire a Growth up to US$ 18289.9 Million at a 9.1% CAGR by 2027 – PharmiWeb.com | dataFebruary 19th, 2021
Read All

The Untapped Potential of Cell and Gene Therapy – AJMC.com Managed Markets Network

By daniellenierenberg

We can absolutely cut the number of cancer deaths down so that one day in our lifetimes it can be a rare thing for people to die of cancer, said Patrick Hwu, MD, president and CEO of Moffitt Cancer Center in Florida and among gene therapys pioneers. It still may happen here and there, but itll be kind of like people dying of pneumonia. Its like, He died of pneumonia? Thats kind of weird. I think cancer can be the same way.

The excitement returned in spades in 2017 when the FDA signed off on a gene-therapy drug for the first time, approving the chimeric antigen receptor (CAR) T-cell treatment tisagenlecleucel (Kymriah; Novartis) for the treatment of B-cell precursor acute lymphoblastic leukemia. At last, scientists had devised a way to reprogram a persons own T cells to attack tumor cells.

Were entering a new frontier, said Scott Gottlieb, MD, then the FDA Commissioner, in announcing the groundbreaking approval.

Gottlieb wasnt exaggerating. The growth in CAR T-cell treatments is exploding. Although only a handful of cell and gene therapies are on the market, FDA officials predicted in 2019 that the agency will receive more than 200 investigational new drug applications per year for cell and gene therapies, and that by 2025, it expects to have accelerated to 10 to 20 cell and gene therapy approvals per year.1

Essentially, you can kill any cancer cell that has an antigen that is recognized by the immune cell, Hwu said. The key to curing every single cancer, which is our goal, is to have receptors that can recognize the tumor but dont recognize the normal cells. Receptors recognizing and then attacking normal cells is what can cause toxicity.

Cell therapy involves cultivating or modifying immune cells outside the body before injecting them into the patient. Cells may be autologous (self-provided) or allogeneic (donor-provided); they include hematopoietic stem cells and adult and embryonic stem cells. Gene therapy modifies or manipulates cell expression. There is considerable overlap between the 2 disciplines.

Juliette Hordeaux, PhD, senior director of translational research for the University of Pennsylvanias gene therapy program, is cautious about the FDAs predictions, saying shed be thrilled with 5 cell and/or gene therapy approvals annually.

For monogenic diseases, there are only a certain number of mutations, and then well plateau until we reach a stage where we can go after more common diseases, Hordeaux said.

Safety has been the main brake around adeno-associated virus vector (AAV) gene therapy, added Hordeaux, whose hospitals program has the institutional memory of both Jesse Gelsingers tragic death during a 1999 gene therapy trial as well as breakthroughs by Carl June, MD, and others in CAR T-cell therapy.

Sometimes there are unexpected toxicity [events] in trials.I think figuring out ways to make gene therapy safer is going to be the next goal for the field before we can even envision many more drugs approved.

In total, 3 CAR T-cell therapies are now on the market, all targeting the CD19 antigen. Tisagenlecleucel was the first. Gilead Sciences received approval in October 2017 for axicabtagene ciloleucel (axi-cel; Yescarta), a CAR T-cell therapy for adults with large B-cell non-Hodgkin lymphoma. Kite Pharma, a subsidiary of Gilead, received an accelerated approval in July 2020 for brexucabtagene autoleucel (Tecartus) for adults with relapsed or refractory mantle cell lymphoma.

On February 5, 2021, the FDA approved another CD19-directed therapy for relapsed/refractory large B-cell lymphoma, lisocabtagene maraleucel (liso-cel; JCAR017; Bristol Myers Squibb). The original approval date was missed due to a delay in inspecting a manufacturing facility (see related article).

Idecabtagene vicleucel (ide-cel; bb2121; Bristol Myers Squibb) is under priority FDA review, with a decision expected by March 31, 2021. The biologics license application seeks approval for ide-cel, a B-cell maturation antigendirected CAR therapy, to treat adult patients with multiple myeloma who have received at least 3 prior therapies.2

The number of clinical trials evaluating CAR T-cell therapies has risen sharply since 2015, when investigators counted a total of 78 studies registered on the ClinicalTrials.gov website. In June 2020, the site listed 671 trials, including 357 registered in China, 256 in the United States, and 58 in other countries.3

Natural killer (NK) cells are the research focus of Dean Lee, MD, PhD, a physician in the Division of Hematology and Oncology at Nationwide Childrens Hospital. He developed a method for consistent, robust expansion of highly active clinical-grade NK cells that enables repeated delivery of large cell doses for improved efficacy. This finding led to several first-in-human clinical trials evaluating adoptive immunotherapy with expanded NK cells under an FDA Investigational New Drug application. He is developing both genetic and nongenetic methods to improve tumor targeting and tissue homing of NK cells. His eff orts are geared toward pediatric sarcomas.

The biggest emphasis over the past 20 to 25 years has been cell therapy for cancer, talking about trying to transfer a specific part of the immune system for cells, said Lee, who is also director of the Cellular Therapy and Cancer Immunology Program at Nationwide Childrens Hospital, at The Ohio State University Comprehensive Cancer Center Arthur G. James Cancer Hospital, and at the Richard J. Solove Research Institute.

The Pivot Toward Treating COVID-19 and Other Diseases

However, Lee said, NKs have wider potential. This is kind of a natural swing back. Now that we know we can grow them, we can reengineer them against infectious disease targets and use them in that [space], he said.

Lee is part of a coronavirus disease 2019 (COVID-19) clinical trial, partnering with Kiadis, for off-the-shelf K-NK cells using Kiadis proprietary platforms. Such treatment would be a postexposure preemptive therapy for treating COVID-19. Lee said the pivot toward treating COVID-19 with cell therapy was because some of the very early reports on immune responses to coronavirus, both original [SARS-CoV-2] and the new [mutation], seem to implicate that those who did poorly [overall] had poorly functioning NK cells.

The revolutionary gene editing tool CRISPR is making its initial impact in clinical trials outside the cancer area. Its developers, Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD, won the Nobel Prize in Chemistry 2020.

For patients with sickle cell disease (SCD), CRISPR was used to reengineer bone marrow cells to produce fetal hemoglobin, with the hope that the protein would turn deformed red blood cells into healthy ones. National Public Radio did a story on one patient who, so far, thanks to CRISPR, has been liberated from the attacks of SCD that typically have sent her to the hospital, as well from the need for blood transfusions.4

Its a miracle, you know? the patient, Victoria Gray of Forest, Mississippi, told NPR.

She was among 10 patients with SCD or transfusion-dependent beta-thalassemia treated with promising results, as reported by the New England Journal of Medicine.5 Two different groups, one based in Nashville, which treated Gray,5 and another based at Dana-Farber Cancer Institute in Boston,6 have reported on this technology.

Stephen Gottschalk, MD, chair of the department of bone marrow transplantation and cellular therapy at St Jude Childrens Research Hospital, said, Theres a lot of activity to really explore these therapies with diseases that are much more common than cancer.

Animal models use T cells to reverse cardiac fibrosis, for instance, Gottschalk said. Using T cells to reverse pathologies associated with senescence, such as conditions associated with inflammatory clots, are also being studied.

Hordeaux said she foresees AAV being used more widely to transmit neurons to attack neurodegenerative diseases.

The neurons are easily transduced by AAV naturally, she said. AAV naturally goes into neurons very efficiently, and neurons are long lived. Once we inject genetic matter, its good for life, because you dont renew neurons.

Logistical Issues

Speed is of the essence, as delays in producing therapies can be the difference between life and death, but the approval process takes time. The process of working out all kinks in manufacturing also remains a challenge. Rapid production is difficult, too, because of the necessary customization of doses and the need to ensure a safe and effective transfer of cells from the patient to the manufacturing center and back into the patient.7

Other factors that can slow down launches include insurance coverage, site certification, staff training, reimbursement, and patient identification. The question of how to reimburse has not been definitively answered; at this point, insurers are being asked to issue 6- or even 7-figure payments for treatments and therapies that may not work.8

CAR T, I think, will become part of the standard of care, Gottschalk said. The question is how to best get that accomplished. To address the tribulations of some autologous products, a lot of groups are working with off -the-shelf products to get around some of the manufacturing bottlenecks. I believe those issues will be solved in the long run.

References

1. Statement from FDA Commissioner Scott Gottlieb, MD, and Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. News release. FDA website. January 15, 2019. https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics. Accessed January 13, 2021.

2. Bristol Myers Squibb provides regulatory update on lisocabtagene maraleucel (liso-cel). News release. Bristol Myers Squibb; November 16, 2020. Accessed January 11, 2021. https://news.bms.com/news/details/2020/Bristol-Myers-Squibb-Provides-Regulatory-Update-on-Lisocabtagene-Maraleucel-liso-cel/default.aspx

3. Wei J, Guo Y, Wang Y. et al. Clinical development of CAR T cell therapy in China: 2020 update. Cell Mol Immunol. Published online September 30, 2020. doi:10.1038/s41423-020-00555-x

4. Stein R. CRISPR for sickle cell diseases shows promise in early test. Public Radio East. November 19, 2019. Accessed January 11, 2021. https://www.publicradioeast.org/post/crisprsickle-cell-disease-shows-promise-early-test

5. Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-Cas9 gene editing for sickle cell disease and -Thalassemia. N Engl J Med. Published online December 5, 2020. DOI: 10.1056/NEJMoa2031054

6. Esrick EB, Lehmann LE, Biffi A, et al. Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease. N Engl J Med. Published online December 5, 2020. doi:10.1056/NEJMoa2029392

7. Yednak C. The gene therapy race. PwC. February 5, 2020. Accessed January 11, 2021. https://www.pwc.com/us/en/industries/healthindustries/library/gene-therapy-race.html

8. Gene therapies require advanced capabilities to succeed after approval. PwC website. Accessed January 11, 2021. https://www.pwc.com/us/en/industries/health-industries/library/commercializing-gene-therapies.html

Read more here:
The Untapped Potential of Cell and Gene Therapy - AJMC.com Managed Markets Network

To Read More: The Untapped Potential of Cell and Gene Therapy – AJMC.com Managed Markets Network
categoriaCardiac Stem Cells commentoComments Off on The Untapped Potential of Cell and Gene Therapy – AJMC.com Managed Markets Network | dataFebruary 19th, 2021
Read All

Page 20«..10..19202122..3040..»