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JASPER THERAPEUTICS, INC. Management’s Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) – Marketscreener.com

By daniellenierenberg

You should read the following discussion and analysis of our financial conditionand results of operations together with the condensed consolidated financialstatements and related notes included in Part I, Item 1 of this Quarterly Reporton Form 10-Q (this "Quarterly Report") and with the audited financial statementsand the related notes included in our Annual Report on Form 10-K for the fiscalyear ended December 31, 2021 filed with the Securities and Exchange Commissionon March 18, 2022. Certain of the information contained in this discussion andanalysis or set forth elsewhere in this Quarterly Report, including informationwith respect to plans and strategy for our business, includesforward-looking statements that involve risks and uncertainties. As a result ofmany factors, including those factors set forth in the section entitled "RiskFactors", in Part II, Item 1A of this Quarterly Report, our actual results coulddiffer materially from the results described in or implied by theforward-looking statements contained in the following discussion and analysis.You should carefully read the section entitled "Risk Factors" to gain anunderstanding of the important factors that could cause actual results to differmaterially from our forward-looking statements. Please also see the section ofthis Quarterly Report entitled "Cautionary Note RegardingForward-Looking Statements." The events and circumstances reflected in ourforward-looking statements may not be achieved or may not occur, and actualresults could differ materially from those described in or implied by theforward-looking statements contained in the following discussion and analysis.As a result of these risks, you should not place undue reliance on theseforward-looking statements. We assume no obligation to revise or update anyforward-looking statements for any reason, except as required by law.OverviewWe are a clinical-stage biotechnology company dedicated to enabling curesthrough hematopoietic stem cell therapy. We are focused on the development andcommercialization of safer and more effective conditioning agents and mRNA-basedstem cell engineering to allow for expanded use of stem cell transplantation andex vivo gene therapy, a technique in which genetic manipulation of cells isperformed outside of the body prior to transplantation. We are also developingnovel therapeutics directed at diseased hematopoietic stem cells.Our drug development pipeline includes multiple product candidates designed toimprove hematopoietic stem cell therapy. Our lead product candidate, JSP191, isin clinical development as a novel conditioning antibody that clearshematopoietic stem cells from bone marrow in patients prior to undergoingallogeneic stem cell therapy or stem cell gene therapy. We plan to initiate aregistrational clinical study in acute myeloid leukemia ("AML") patientsundergoing stem cell transplantation by the end of the first quarter of 2023.Based on the single agent depletion observed in our Phase 1 study ofmyelodysplastic syndrome ("MDS") patients undergoing stem cell transplant, weare also initiating a pilot study of JSP191 as a therapeutic in lower-risk MDS,which we expect to commence in the second half of this year. Beyond JSP191, weare developing stem cell grafts transiently reprogrammed using mRNA that have acompetitive advantage over endogenous hematopoietic stem cells ("HSCs"),enabling higher levels of engraftment designed to remove the need for highlytoxic conditioning of the patient and lower the risk of other seriouscomplications that limit current stem cell transplants. We plan to continue toexpand our pipeline to include other novel stem cell therapies based on immunemodulation, graft engineering and cell or gene therapies. Our goal is to expandthe use of curative stem cell transplant and gene therapies for all patients,including children and the elderly.Stem cell transplantation is among the most widely practiced forms of cellulartherapy and has the potential to cure a wide variety of diseases, includingcancers, genetic disorders, and autoimmune diseases. Yet currently, patientsmust receive highly toxic and potentially life-threatening conditioning agentsto prepare their bone marrow for transplantation with either donor stem cells ortheir own gene-edited stem cells. Younger, fitter patients capable of survivingthese toxic side effects are typically given myeloablative, or high-intensity,conditioning whereas older or less fit patients are typically given reducedintensity, but still toxic, conditioning which leads to less effectivetransplants. These toxicities include a range of acute and chronic effects tothe gastrointestinal tract, kidneys, liver, lung, endocrine, and neurologictissues. Depending upon the conditioning regimen, fitness of the patient, andcompatibility between the donor and recipient, the risk of transplant-relatedmortality ranges from 10% to more than 50% in older patients. Less toxic ways tocondition patients have been developed to enable transplant for older patientsor those with major comorbidities, but these regimens risk less potent diseaseelimination and higher rates of disease relapse. Even though stem cell therapycan be one of the most powerful forms of disease cure, these limitations ofnon-targeted conditioning regimens have seen little innovation over the pastdecade. 20Our lead product candidate, JSP191, is a monoclonal antibody designed to blockthe specific signal on stem cells required for survival. It is currently indevelopment as a highly targeted conditioning agent prior to stem cell therapyas well as a therapeutics in lower-risk MDS patients, which we expect tocommence in the second half of 2022. We are also sponsoring two clinical studiesof JSP191 as a conditioning agent prior to stem cell transplant. The firstclinical study is an open label Phase 1/2 trial in two cohorts of severecombined immunodeficiency ("SCID") patients: patients with a history of a priorallogeneic transplant for SCID but with poor graft outcomes and newly diagnosedSCID patients. The primary endpoint in this study is to evaluate the safety andtolerability of JSP191. The secondary goal of this study is to evaluate theefficacy of JSP191 as a conditioning agent in conjunction with a stem celltransplant. Based on preliminary results from our ongoing Phase 1/2 clinicaltrial, we believe JSP191 has demonstrated the ability as a single agent toenable engraftment of donor HSCs as determined by donor chimerism, or thepercentage of bone marrow cells in the patient that are of donor origin aftertransplant. Engraftment was observed in seven out of ten T-B-NK+ SCID patientswith prior allogeneic transplant, as evidenced by CD15+ donor chimerism of morethan 5% averaged from 12-24 weeks post-transplant. Increased nave donor T cellproduction was observed in the majority of T-B-NK+ subjects, as well as clinicalimprovement. No JSP191 treatment-related serious adverse events ("SAEs") havebeen reported to date and pharmacokinetics have been consistent with earlierstudies in healthy volunteers. We expect to complete enrollment in this Phase1/2 clinical trial by mid-2023.

The FDA has granted rare pediatric disease designation to JSP191 as aconditioning treatment for patients with SCID. In addition, the FDA grantedorphan drug designation to JSP191 for conditioning treatment prior tohematopoietic stem cell transplantation.

We expect our expenses will increase substantially in connection with ourongoing and planned activities, as we:

? advance product candidates through preclinical studies and clinical trials;

? procure the manufacture of supplies for our preclinical studies and clinical

? attract, hire and retain additional personnel;

? operate as a public company;

? implement operational, financial and management systems;

? pursue regulatory approval for any product candidates that successfully

? establish a sales, marketing, and distribution infrastructure to commercialize

any product candidate for which we may obtain marketing approval and related

commercial manufacturing build-out; and

? obtain, maintain, expand, and protect our portfolio of intellectual property

Business Impact of the COVID-19 Pandemic

Stanford License Agreement

Other collaboration and clinical trial agreements

Collaboration with Stanford University

Components of Results of Operations

External research and development costs include:

? costs incurred under agreements with third-party CROs, CMOs and other third

parties that conduct preclinical and clinical activities on our behalf and

manufacture our product candidates;

? costs associated with acquiring technology and intellectual property licenses

that have no alternative future uses;

? consulting fees associated with our research and development activities; and

? other costs associated with our research and development programs, including

Internal research and development costs include:

? employee-related costs, including salaries, benefits and

stock-based compensation expense for our research and development personnel;

? other expenses and allocated overheads incurred in connection with our research

Our future research and development costs may vary significantly based onfactors, such as:

? the scope, rate of progress, expense and results of our discovery and

preclinical development activities;

? the costs and timing of our chemistry, manufacturing and controls activities,

including fulfilling cGMP-related standards and compliance, and identifying and

? per patient clinical trial costs;

? the number of trials required for approval;

? the number of sites included in our clinical trials;

? the countries in which the trials are conducted;

? delays in adding a sufficient number of trial sites and recruiting suitable

patients to participate in our clinical trials;

? the number of patients that participate in the trials;

? the number of doses that patients receive;

? patient drop-out or discontinuation rates;

? the duration of patient participation in the trials and follow up;

? the cost and timing of manufacturing our product candidates;

? the phase of development of our product candidates;

? the efficacy and safety profile of our product candidates;

? the timing, receipt, and terms of any approvals from applicable regulatory

authorities, including the FDA and non-U.S. regulators;

? maintaining a continued acceptable safety profile of our product candidates

following approval, if any, of our product candidates;

? changes in the standard of care on which a clinical development plan was based,

which may require new or additional trials;

? the extent to which we establish additional strategic collaborations or other

? the impact of any business interruptions to our operations or to those of the

Other Income (Expense), Net

Three Months Ended March 31, 2022 and 2021

The following table summarizes our results of operations for the three monthsended March 31, 2022 and 2021 (in thousands):

Research and Development Expenses

The following table summarizes our research and development expenses for thethree months ended March 31, 2022 and 2021 (in thousands):

Our external costs by program for the three months ended March 31, 2022 and 2021were as follows (in thousands):

General and Administrative Expenses

Liquidity and Capital Resources

Future Funding Requirements - Going Concern

Contractual Obligations and Commitments

We have contractual obligations and commitments as described in Note 9,Commitments and Contingencies, within our condensed consolidated financialstatements included in Part I, Item 1 of this Quarterly Report.

Our future financing requirements will depend on many factors, including:

? the timing, scope, progress, results and costs of research and development,

preclinical and non-clinical studies and clinical trials for our current and

? the number, scope and duration of clinical trials required for regulatory

approval of our current and future product candidates;

? the outcome, timing and costs of seeking and obtaining regulatory approvals

from the FDA and comparable foreign regulatory authorities for our product

candidates, including any requirement to conduct additional studies or generate

additional data beyond that which we currently expect would be required to

support a marketing application;

? the costs of manufacturing clinical and commercial supplies of our current and

future product candidates;

? the costs and timing of future commercialization activities, including product

manufacturing, marketing, sales and distribution, for any of our product

candidates for which we receive marketing approval;

? any product liability or other lawsuits related to our product candidates;

? the revenue, if any, received from commercial sales of any product candidates

for which we may receive marketing approval;

? our ability to establish a commercially viable pricing structure and obtain

approval for coverage and adequate reimbursement from third-party and

? the costs to establish, maintain, expand, enforce and defend the scope of our

intellectual property portfolio, including the amount and timing of any

payments we may be required to make, or that we may receive, in connection with

licensing, preparing, filing, prosecuting, defending and enforcing our patents

or other intellectual property rights;

? expenses incurred to attract, hire and retain skilled personnel;

? the costs of operating as a public company; and

? the impact of the COVID-19 pandemic, which may exacerbate the magnitude of the

10,752

Cash Flows Used in Operating Activities

Net cash used in operating activities was $14.2 million and $6.2 million for thethree months ended March 2022 and 2021, respectively.

Cash Flows Used in Investing Activities

Cash Flows from Financing Activities

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JASPER THERAPEUTICS, INC. Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - Marketscreener.com

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Aileron Therapeutics Announces Late-Breaking Oral Presentation of Non-Clinical Data Demonstrating ALRN-6924 Protected Human Hair Follicles and Their…

By daniellenierenberg

Aileron Therapeutics, Inc.

Taxanes, such as paclitaxel and docetaxel, cause severe and often permanent chemotherapy-induced hair loss (alopecia)

New non-clinical data demonstrate proof of principle that ALRN-6924 can temporarily arrest the cell cycle in human scalp hair follicles and their stem cells

ALRN-6924-induced cell cycle arrest protected hair follicles from paclitaxel-induced toxicity and irreversible stem cell damage

Ailerons precision medicine-based approach is designed to selectively protect normal, healthy cells from chemotherapy while ensuring chemotherapy cannot protect cancer cells

Ailerons ongoing non-small cell lung cancer (NSCLC) clinical trial and upcoming breast cancer clinical trial will evaluate ALRN-6924s protection against chemotherapy-induced bone marrow toxicities and other side effects, including alopecia

BOSTON, May 10, 2022 (GLOBE NEWSWIRE) -- Aileron Therapeutics (Nasdaq: ALRN), a chemoprotection oncology company that aspires to make chemotherapy safer and thereby more effective to save more patients lives, today announced a late-breaking oral presentation at the upcoming Society for Investigative Dermatology (SID) Annual Meeting, which will be held May 18 21, 2022 in Portland, Oregon. The presentation will highlight new non-clinical data developed in collaboration with Professor Ralf Paus, M.D., DSc, FRSB and his colleagues at the Dr. Phillip Frost Department of Dermatology & Cutaneous Surgery at the University of Miami Miller School of Medicine. This collaboration has generated promising ex vivo data demonstrating that ALRN-6924 protected human hair follicles and their stem cells from chemotherapy-induced acute and permanent damage. Details of the presentation are as follows:

Title:

ALRN-6924, a dual inhibitor of MDMX and MDM2, protects human scalp hair follicles and their epithelial stem cells from paclitaxel-induced toxicity (LB1018)

Presenter:

Jennifer Gherardini, Ph.D.; Paus Laboratory, University of Miami Miller School of Medicine

Date:

Thursday, May 19th

Time:

8:45 AM 11:15 AM PT

Session:

Late-Breaking Abstract Concurrent Session

Chemotherapy-induced toxicities range from severe and life-threatening to those that impact and diminish patients quality of life, sometimes long after chemotherapy has been completed. These toxicities occur because chemotherapy destroys normal, healthy cells while simultaneously destroying cancer cells, said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer at Aileron. Previously, we showed chemoprotection against severe bone marrow toxicities in small cell lung cancer patients receiving topotecan and demonstrated in healthy volunteers the mechanism of action cell cycle arrest underlying this chemoprotection benefit. We are excited to now present new data that may suggest ALRN-6924s ability to also protect against chemotherapy-induced hair loss, another devastating chemotherapy-induced side effect for millions of cancer patients.

Dr. Paus commented, These results got us quite excited as they directly follow in the footsteps of our prior work that showed arresting the cell cycle can have a strong protective effect against taxane-induced hair follicle damage. Until our research with ALRN-6924, we had not come across a cell cycle arrest-inducing drug that is in clinical testing for protection of normal cells without protecting cancer cells. Thus, ALRN-6924 invites a very promising and completely novel selective protection approach. In addition, we found that ALRN-6924 may exert some additional benefits that could reduce the risk of long-term damage of human hair follicle stem cells by taxanes.

Story continues

Aileron is currently developing ALRN-6924, a first-in-class MDM2/MDMX dual inhibitor, to selectively protect healthy cells in patients with cancers that harbor p53 mutations to reduce or eliminate chemotherapy-induced side effects while preserving chemotherapys attack on cancer cells. ALRN-6924 is designed to activate p53 in normal cells, which in turn upregulates p21, which pauses cell cycle in normal cells but not in p53-mutated cancer cells. The companys vision is to bring chemoprotection to all patients with p53-mutated cancer regardless of the type of cancer or chemotherapy.

About the Findings

Taxanes, such as paclitaxel and docetaxel, are known to cause severe and often permanent chemotherapy-induced alopecia. Over 90% of patients treated with this chemotherapy class experience alopecia, and approximately 10% (paclitaxel) to 25% (docetaxel) of patients experience permanent alopecia. Dr. Paus and his team previously demonstrated that paclitaxel damages human scalp hair follicles by inducing massive mitotic defects and apoptosis in hair matrix keratinocytes as well as bulge stem cell DNA damage, and that pharmacological induction of transient cell cycle arrest can protect hair follicles and stem cells (Purba et al. EMBO Molecular Medicine 2019). Aileron previously conducted in vitro studies showing that ALRN-6924 protected human fibroblasts in cell culture from multiple chemotherapies, but not p53-mutant breast cancer cells.

In the new non-clinical findings to be presented at the SID meeting, when organ-cultured anagen (i.e., active growth phase) scalp hair follicles from two human donors were pre-treated with ALRN-6924 or vehicle (i.e., placebo), followed by paclitaxel or vehicle, ALRN-6924 significantly increased the number of p21-positive hair matrix keratinocytes and bulge stem cells compared to vehicle or paclitaxel alone, confirming cell cycle arrest ex vivo. Further, pretreatment of paclitaxel-treated human hair follicles with ALRN-6924, led to a reduction in the number of melanin clumps, a marker of hair follicle cytotoxicity and dystrophy, as well as a reduction in apoptosis, pathological mitosis, and DNA damage. Aileron believes that these findings support clinical investigation of ALRN-6924 to prevent both acute and permanent chemotherapy-induced alopecia, in addition to its ongoing evaluation of ALRN-6924 to protect against chemotherapy-induced bone marrow and other toxicities.

About Ailerons Clinical Trials of ALRN-6924

Aileron is on track to initiate a Phase 1b randomized, controlled trial of ALRN-6924 in patients with p53-mutated ER+/HER2- neoadjuvant breast cancer in 2Q 2022. The planned breast cancer trial will evaluate ALRN-6924s protection against chemotherapy-induced bone marrow toxicities, as well as other toxicities, including alopecia, in patients with p53-mutated ER+/HER2- breast cancer treated with a doxorubicin plus cyclophosphamide and docetaxel chemotherapy regimen.

The company is currently enrolling patients in a Phase 1b randomized, double-blind, placebo-controlled trial evaluating ALRN-6924s protection against chemotherapy-induced bone marrow and other toxicities in patients with advanced p53-mutated non-small cell lung cancer undergoing treatment with first-line carboplatin plus pemetrexed with or without immunotherapy. While patients in this trial are monitored for alopecia, historically, only a small percentage of patients treated with carboplatin plus pemetrexed experience acute alopecia. Aileron is on track to report interim results on the first 20 patients enrolled in the NSCLC trial in June 2022 and topline results on 60 patients in 4Q 2022.

About Aileron Therapeutics

Aileron is a clinical stage chemoprotection oncology company that aspires to make chemotherapy safer and thereby more effective to save more patients lives. ALRN-6924, our first-in-class MDM2/MDMX dual inhibitor, is designed to activate p53, which in turn upregulates p21, a known inhibitor of the cell replication cycle. ALRN-6924 is the only reported chemoprotective agent in clinical development to employ a biomarker strategy, in which we exclusively focus on treating patients with p53-mutated cancers. Our targeted strategy is designed to selectively protect multiple healthy cell types throughout the body from chemotherapy without protecting cancer cells. As a result, healthy cells are spared from chemotherapeutic destruction while chemotherapy continues to kill cancer cells. By reducing or eliminating multiple chemotherapy-induced side effects, ALRN-6924 may improve patients quality of life and help them better tolerate chemotherapy. Enhanced tolerability may result in fewer dose reductions or delays of chemotherapy and the potential for improved efficacy.

Our vision is to bring chemoprotection to all patients with p53-mutated cancers, which represent approximately 50% of cancer patients, regardless of type of cancer or chemotherapy. Visit us at aileronrx.com to learn more.

Forward-Looking Statements

Statements in this press release about Ailerons future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements about the potential of ALRN-6924 as a chemoprotective agent, including its ability to prevent both acute and permanent chemotherapy-induced alopecia, and the Companys strategy and clinical development plans. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether Ailerons cash resources will be sufficient to fund its continuing operations for the periods anticipated or with respect to the matters anticipated; whether initial results of clinical trials will be indicative of final results of those trials or results obtained in future clinical trials, including trials in different indications; whether ALRN-6924 will advance through the clinical trial process on a timely basis, or at all; whether the results of such trials will be accepted by and warrant submission for approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies; whether ALRN-6924 will receive approval from regulatory agencies on a timely basis or at all or in which territories or indications ALRN-6924 may receive approval; whether, if ALRN-6924 obtains approval, it will be successfully distributed and marketed; what impact the coronavirus pandemic may have on the timing of our clinical development, clinical supply and our operations; and other factors discussed in the Risk Factors section of Ailerons annual report on Form 10-K for the year ended December 31, 2021, filed on March 28, 2022, and risks described in other filings that Aileron may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Aileron specifically disclaims any obligation to update any forward-looking statement, whether because of new information, future events or otherwise.

Investor Contact:Stern Investor RelationsAlexander Loboalex.lobo@sternir.com

Media Contact:Liz Melone617-256-6622

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Aileron Therapeutics Announces Late-Breaking Oral Presentation of Non-Clinical Data Demonstrating ALRN-6924 Protected Human Hair Follicles and Their...

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Gracell Biotechnologies to Present Clinical Data on BCMA/CD19 Dual-targeting CAR-T GC012F in RRMM and B-NHL and CD19/CD7 Dual-directed Allogeneic…

By daniellenierenberg

SAN DIEGO, Calif., SUZHOU and SHANGHAI, China , May 12, 2022 /PRNewswire/ -- Gracell Biotechnologies Inc. ("Gracell" or the "Company",NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, today announced the details of three abstracts that it will present at the European Hematology Association 2022 Hybrid Congress (EHA2022 Congress), being held from June 9 June 12 in Vienna, Austria. The abstracts highlight the clinical data from ongoing investigator-initiated trials (IITs) of BCMA/CD19 dual-targeting FasTCAR candidate GC012F in two indications of B-cell non-hodgkin's lymphoma (B-NHL) and relapsed/refractory multiple myeloma (RRMM), and allogeneic TruUCAR candidate GC502 in B-cell acute lymphoblastic leukemia (B-ALL).

"We are very excited to share our data for both our FasTCAR candidate GC012F in two indications of RRMM and B-NHL, and allogeneic TruUCAR candidate GC502 in B-ALL at the EHA2022 Congress," said Dr. Martina Sersch, Chief Medical Officer of Gracell. "The new data, including the expanded indication of GC012F into B-NHL, demonstrates the potential of our platforms and provides further validation. The clinical data of BCMA/CD19 dual-targeting GC012F in the treatment of B-NHL shows promising early results, along with benefits of the next-day manufacturing enabled by the FasTCAR platform. The CD19/CD7 dual-directed CAR-T therapy GC502 is our second allogeneic candidate on our TruUCAR platform, demonstrating the potential wide applicability of the TruUCAR design."

BCMA/CD19 Dual-Targeting FasTCAR-T GC012F for the Treatment of B-NHL

GC012F is an autologous CAR-T therapeutic candidate dual-targeting B cell maturation antigen (BCMA) and CD19. It is developed using Gracell's proprietary FasTCAR platform which enables next-day manufacturing, and is currently being evaluated in IITs in China including in RRMM and B-NHL. GC012F is the first BCMA/CD19 dual-targeting CAR-T in human trials for B-NHL.

Gracell will present the early results of the first-in-human phase 1 IIT in China evaluating the safety and tolerability of GC012F in B-NHL patients. Three patients who had received a median of two prior lines of therapy were enrolled, all of which presented with bulky disease. As of the February 22, 2022 data cutoff date, the enrolled patients had received one single infusion of GC012F at three different doses of 3.7x104 cells/kg and 2-3x105 cells/kg.

All three patients had achieved a complete response (CR) confirmed by PET- CT at day 28 after GC012F infusion. At 3-month follow-up, both of the two assessable patients had ongoing response. No dose-limiting toxicities were observed and no immune effector cell-associated neurotoxicity syndrome (ICANS) were observed. CRS presented as Grade 1 in two patients and Grade 3 in one patient (duration of two days) with no Grade 4 or 5 events.

Details of the presentation are as follows:

BCMA/CD19 Dual-Targeting FasTCAR-T GC012F for the Treatment of RRMM

Gracell will also present as an oral abstract presentation the updated results from the first-in-human IIT evaluating GC012F for the treatment of RRMM patients. This data is currently under embargo and will be published on the EHA2022 Hybrid Congress website on Thursday, May 26 concurrently with ASCO.

Details of the presentation are as follows:

CD19/CD7 Dual-directed Allogeneic TruUCAR-T GC502 for the Treatment of B-ALL

GC502 leverages the novel dual-directed CAR design of Gracell's proprietary TruUCAR platform, designed to generate high-quality allogeneic CAR-T cell therapies that can be administered "off-the-shelf" at lower cost and with faster patient's access. TruUCAR-enabled GC502 utilizes the dual-directed CAR design with one CAR targeting CD19 on malignant cells and a second CAR targeting CD7 to suppress host-versus-graft rejection. An enhancer molecule is embedded in the basic construct of TruUCAR to enhance proliferation of TruUCAR T cells.

Between September 2021 and January 2022, four r/r B-ALL patients were enrolled and treated in an open-label, non-randomized, prospective IIT in China in two different dose levels and with two different formulations. Patients were heavily pretreated, and all had previously received either autologous or donor derived CD19 or CD19/CD22 targeted CAR-T therapy. As of the January 28, 2022 data cutoff date, all four patients had received a single dose of GC502, including one patient at dose level 1 (DL1) 1.0x107 cells/kg and three patients at dose level 2 (DL2) 1.5x107 cells/kg. Patients received a Flu/Cy based lymphodepletion regimen prior to treatment with GC502.

Three of four patients achieved minimal residual disease negative complete response or complete response with incomplete count recovery (MRD- CR/CRi), and one patient achieved a partial response at month one and subsequently received allogeneic hematopoietic stem-cell transplantation (allo-HSCT) on day 39.

Cytokine release syndrome (CRS) presented as Grade 2 and Grade 3 with no Grade 4 or 5 events. No immune effector cell-associated neurotoxicity syndrome (ICANS) or acute graft-versus-host disease (aGvHD) were observed.

Details of the presentation are as follows:

For more information about the EHA2022 Hybrid Congress, visit http://www.ehaweb.org.

About GC012F

GC012F is a FasTCAR-enabled dual-targeting CAR-T product candidate that is currently being evaluated in IIT studies in China for the treatment of multiple myeloma and B-cell non-Hodgkin's lymphoma. GC012F simultaneously targets CD19 and BCMA to drive fast, deep and durable responses, which can potentially improve efficacy and reduce relapse in multiple myeloma and B-NHL patients.

About B-NHL

Non-Hodgkin's lymphoma (NHL) is a group of blood cancers that developed from lymphocytes, most commonly derived from B cells (B-NHL). Globally, approximately 510,000 patients are diagnosed with NHL every year with about 80,470 patients expected to be diagnosed with NHL in the United States in 2022[1]. B-NHL accounts for approximately 85% of NHL diagnoses.

[1] Data source: American Cancer Society

About GC502

GC502 is a TruUCAR-enabled CD19/CD7 dual-directed, off-the-shelf allogeneic CAR-T product candidate that is being studied in an ongoing Phase 1 IIT in China for the treatment of B-cell malignancies. GC502 is manufactured using T cells from non-human leukocyte antigen (HLA) matched healthy donors. An enhancer molecule is embedded in the basic construct of TruUCAR to enhance proliferation of TruUCAR T cells. Optimized for CD19/CD7 dual-CAR functionality and in vivo durability, GC502 has demonstrated robust anti-tumor effects with potential to suppress host versus graft (HvG) rejection in preclinical models.

About B-ALL

Acute lymphoblastic leukemia (ALL) is a type of blood cancer characterized by proliferation of immature lymphocytes in the bone marrow, which can involve either T lymphocytes (T-ALL), or B lymphocytes (B-ALL). Globally, approximately 64,000 patients are diagnosed with ALL every year with an estimated 6,660 new cases to be diagnosed in the United States in 2022[2]. B-ALL accounts for 75% of ALL diagnoses in adults.

[2] Data source: American Cancer Society

About FasTCAR

CAR-T cells manufactured on Gracell's proprietary FasTCAR platform appear younger, less exhausted and show enhanced proliferation, persistence, bone marrow migration and tumor cell clearance activities as demonstrated in preclinical studies. With next day manufacturing, FasTCAR is able to significantly improve cell production efficiency which may result in meaningful cost savings, and, together with fast turnaround time, enables enhanced accessibility of cell therapies for cancer patients.

About TruUCAR

TruUCAR is Gracell's proprietary technology platform and is designed to generate CAR-T cell therapies from high quality allogeneic T cells that can be administered "off-the-shelf" at lower cost and with improved accessibility of cell therapies for cancer patients. With differentiated design enabled by gene editing, TruUCAR is designed to control HvG as well as GvHD without the need for being co-administered with additional strong immunosuppressant after conventional lymphodepletion. The novel dual-CAR design allows tumor antigen-CAR moiety to target malignant cells, while the CD7 CAR moiety is designed to suppress rejection (HvG response) of allogeneic CAR-T cells by host T and NK cells (HvG).

About Gracell

Gracell Biotechnologies Inc.("Gracell") is a global clinical-stage biopharmaceutical company dedicated to discovering and developing breakthrough cell therapies. Leveraging its pioneering FasTCAR and TruUCAR technology platforms and SMART CARTMtechnology module, Gracell is developing a rich clinical-stage pipeline of multiple autologous and allogeneic product candidates with the potential to overcome major industry challenges that persist with conventional CAR-T therapies, including lengthy manufacturing time, suboptimal cell quality, high therapy cost, and lack of effective CAR-T therapies for solid tumors. For more information on Gracell, please visit http://www.gracellbio.com.Follow @GracellBio on LinkedIn.

Cautionary Noted Regarding Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the expected trading commencement and closing date of the offering. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including factors discussed in the section entitled "Risk Factors" in Gracell's most recent annual report on Form 20-F as well as discussions of potential risks, uncertainties, and other important factors in Gracell's subsequent filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Gracell specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Readers should not rely upon the information on this page as current or accurate after its publication date.

Media contacts

Marvin Tang[emailprotected]

Kyle Evans[emailprotected]

Investor contacts

Gracie Tong[emailprotected]

Stephanie Carringtonsteph[emailprotected]

SOURCE Gracell Biotechnologies Inc.

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Gracell Biotechnologies to Present Clinical Data on BCMA/CD19 Dual-targeting CAR-T GC012F in RRMM and B-NHL and CD19/CD7 Dual-directed Allogeneic...

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Researchers share insights about the mechanisms of human embryo and create method to develop transcriptionally similar cells in tissue culture -…

By daniellenierenberg

Paper Title: Identification of a retinoic acid-dependent hemogenic endothelial progenitor from human pluripotent stem cells

Journal: Nature Cell Biology

Authors:Christopher Sturgeon, PhD, Associate Professor of Cell, Developmental & Regenerative Biology and Medicine, Hematology & Medical Oncology in the Black Family Stem Cell Institute at the Icahn School of Medicine at Mount Sinai, and other coauthors.

Bottom Line:Blood-forming stem cells found in bone marrow are the life-saving component used in bone marrow transplants. However, suitable donors often cannot be found in many cases. This study reveals how the human embryo develops the precursor to blood forming stem cells, which researchers say can be used in the novel method they developed to generate blood-forming stem cells from cells in tissue culture.

The studyled by researchers from Mount Sinai and the San Raffaele Telethon Institute for Gene Therapy in Milan Italyconfirms many aspects of cell development, including origins and regulation, which are known to occur within both the mouse and human embryo. In the mammalian embryo, blood-forming stem cells emerge from a specialized cell type called hemogenic endothelium. These cells develop in response to a critical signal pathway known as retinoic acid, which is essential for growth. Their analysis found that stem cell populations derived from human pluripotent stem cells were transcriptionally similar to cells in the early human embryo.

Results: For years, researchers in the field of regenerative medicine have been able to obtain hemogenic endothelium from embryonic stem cells, but these cells do not produce blood-forming stem cells. In the embryo, blood-forming stem cell development requires signaling by retinoic acid.But, current state-of-the-art methods for deriving blood progenitors from human pluripotent stem cells do so in the absence of retinoic acid. In this latest study, researchers examined the dependence on retinoic acid in early cell types derived from human pluripotent stem cells. They performed single cell RNA sequencing of stem cells in vitro to better understand patterns of mesodermal cell types during early development. The research team identified a new strategy to obtain cells that are transcriptionally similar to those hemogenic endothelial cells found in the human embryo by stimulating a very discrete original population with retinoic acid.

Why the Research Is Interesting:This new method brings researchers and scientists closer to developing blood-forming stem cells in tissue culture, but also provides a pathway to establishing specialized blood cell types for transfusions and other treatments for cancer since the new method makings it possible to obtain the same original cells in adult blood that are found in a developing embryo.

Said Mount Sinai's Dr. Christopher Sturgeon of the research:We have made a major breakthrough in our ability to direct the development of stem cells in a tissue culture dish into cells that have the same gene expression signature as the immediate progenitor of a blood-forming stem cell found in the developing embryo. With this, now we can focus our efforts at understanding how to capture embryonic blood-forming stem cells, with the goal of using them as a substitute for bone marrow.

Researchers from the Washington University School of Medicine in St. Louis, MO contributed to this study.

###

To request a full copy of the paper or to schedule an interview with the researcher, please contact the Mount Sinai Press Office at stacy.anderson@mountsinai.org or 347-346-3390.

Nature Cell Biology

28-Apr-2022

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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La Conner native raising funds to cure blood cancer – La Conner Weekly News

By daniellenierenberg

There are few things more grueling than running a marathon.

One of those is battling cancer.

A La Conner native knows all about the former, having previously run the Denver Colfax Marathon. Now shes helping bring greater awareness to the latter by raising funds for the Leukemia & Lymphoma Society as she trains for the Chicago Marathon in October.

Morgan Harlan, a 2020 Baylor University grad now teaching kindergarten in Denver, is hoping to raise $4,000 for LLS by running the urban Chicago course with two friends this fall. The Chicago Marathon is typically viewed by more than a million spectators.

I hope to raise as much money as I possibly can for such a worthy cause that is so important to my family, she told the Weekly News on Friday.

Her family has seen first-hand the life-saving potential of bone marrow and blood stem cell transplants, and is committed to helping find cures and ensure access to treatments for all blood cancer patients.

Harlans grandfather, longtime La Conner resident and Dunlap Towing retiree Mit Harlan, waged a successful battle against leukemia over a decade ago.

My grandfather, said Harlan, is alive because of a stem cell transplant.

While a student at Baylor, where she was a journalism/public relations major and played club soccer, Harlan signed up for Be the Match, which connects patients with transplant donors.

As a college student with a family member who had experienced cancer, said Harlan, I thought I was doing my due diligence by signing up for the registry.

Last December, four years after joining Be the Match, Harlan flew to Seattle to donate her stem cells.

Her patient was a 65-year-old male with leukemia the same age her grandfather was when he received his transplant.

When Be the Match called to inform me that I was the match and asked me if I would be willing to donate my stem cells, Harlan added, my response was: Absolutely. I hope I can give another little girl or boy more time with their grandpa like I was given.

Harlan has not stopped there. She has taken on fundraising for the cause, doing so in a way that shows she is in it for the long run.

She has enlisted a coach, La Conner alum Carlee Daub, to help her train for Chicago. Daub is an owner of Wahoo Running, an online platform that provides coaching to runners throughout the nation.

My first marathon, Harlan recalled, I was focused on completion. I wanted to prove to myself that I had the physical and mental grit to get through 26.2 miles. The Chicago Marathon will be focused more on speed and race strategy.

As Harlan has lowered her running times, her fundraising numbers have increased.

My fundraising has gone really well because of the wonderful people around me, she said. I am very thankful to have generous family members, friends, and community members.

My original goal was to raise $2,000, Harlan explained, which I was able to raise in the first week. I have since raised my goal to $4,000.

Committing to run the Chicago Marathon on behalf of LLS is a big step for Harlan. After graduating from Burlington-Edison High School, having competed in soccer and track there, Harlan chose to go out of state for college.

I wanted to travel outside of Washington for my four years of college and live somewhere new, she said. Baylor had a great mix of academic strength, athletics success and extracurriculars.

While on the Waco, Texas campus, Harlan regularly wrote for the student newspaper, the Baylor Lariat.

Now, as she preps for the Chicago Marathon and generates support for LLS, Harlan is making rather than reporting the news.

For her, its a story whose headliner is her grandfather.

Hes one of the best humans I know, said Harlan. Growing up, he never missed a soccer match (of mine), including a tournament in Spain. Hes very giving with his time and money, especially towards charities like LLS.

Harlan, daughter of Mike and Jennifer Harlan, of Landing Road, southeast of La Conner, said the best ways to donate are through either her donation page: (https://pages.lls.org.tnt/rm.chicago22/MHarlan) or Facebook.

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I would not be here were it not for the blood: Duval residents describe impact of blood donation – Yahoo News

By daniellenierenberg

Community members are speaking out about the role blood donation has played in their personal lives.

This comes as Action News Jax and our Family Focus partners have teamed up for our annual Spring Into Action Blood Drive.

Jacksonville resident Penelope McGowan told Action News Jax reporter Kennedy Dendy that having the opportunity to give blood is an honor. My father needed a life-saving procedure, so it became more important to me to start giving blood, McGowan said.

She then became a regular giver, knowing the impact donation truly has.

That allowed him the time to spend time with his grandchildren, McGowan said. He walked his granddaughter down the aisle and got to see some of his great-grandchildren.

McGowan said blood donation made that moment possible.

Now that hes passed away, I want to give that gift of time to other families, McGowan said. So, its so important to me to give blood.

RELATED: OneBlood, Action News Jax team up for the Spring into Action Blood Drive

Action News Jax also spoke with John Dean, who is a patient at the Mayo Clinic. Hes from South Carolina but has been living in Jacksonville since January.

I got the bone marrow transplant, which is basically a stem cell infusion on January 17th, Dean said. I have been dealing with myelodysplastic syndrome.

Dean said its a form of bone marrow cancer hes been battling since 2017.

During that time, I had become increasingly dependent upon blood because the syndrome destroys my bodys ability to make red blood cells, Dean said. So when the blood numbers drop, I get very very sick.

He said the transplant was designed to cut down on his need to get the blood, but that hasnt happened yet.

Ive been more blood dependent since January than I had been before I came down here, Dean said.

Dean spoke with me just moments after he received a blood transfusion at the hospital -- but he wanted one message out there.

Youre transmitting a miracle, Dean said. Im a living example of that. I would not be here were it not for the blood.

Story continues

OneBlood said to donate youll need an ID, and you must be 16 years and older.

Randy Varner donated double red blood cells at Tuesdays drive.

My wife has had to have two heart valves replaced, so shes had to have blood before at the hospital -- so I try to help out when I can, Varner said.

Varner shared that if youre able to -- you should give.

Theres nothing to it, Varner said. You go in there. You answer a few questions. You lay down. You can take a little nap if you have to.

Nicole Payne is the Senior Program and Membership Director with the Brooks Family YMCA, one of the many sites for the drive.

Theres always a lack of blood available for people that come into any traumatic situation, Payne said. We want to make sure that we can hopefully combine some of the best parts of Jacksonville -- and thats through OneBlood being here to help people have access to donate.

The Spring into Action Blood Drive kicked off Tuesday and runs through Friday.

When you donate you will receive a free t-shirt, a $20 e-gift card, and an additional gift depending on the location where you donate.

CLICK HERE to find out when and where you can donate.

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Interim Data Targeting CD117 Show Promising MRD Results and Safety in MDS/AML – Targeted Oncology

By daniellenierenberg

Early outcomes with the combination of JSP191, fludarabine, and low-dose total body radiation (TBI) demonstrated facilitation of full donor myeloid chimerism, clearing of minimal residual disease (MRD), and a well-tolerated safety profile in older patients with myelodysplastic syndrome/acute myeloid leukemia (MDS/AML) receiving non-myeloablative (NMA) allogenic hematopoietic cell transplantation (AHCT).

Results from the phase 1 trial (NCT04429191) presented at the 2022 Transplantation & Cellular Therapy Meetings, showed there were no infusion toxicities or serious adverse events with JSP191, and no instances of primary graft failure in first 24 patients enrolled on the trial; only 1 patient had secondary graft failure and went on to have successful retransplant. Additionally, MRD clearance was observed in 12 patients, and JSP191 pharmacokinetics were shown to be predictable.

AHCT is the only curative treatment for many patients with MDS/AML, even though there have been advancements in therapy for these patients in recent years. While transplant has proven feasible for adults well into their 70s, the optimal conditioning regimen for older adults remains unknown as more intensive regimens tend to be associated with transplant-related mortality, while less intensive nonmyeloablative regimens have resulted historically in higher rates of disease relapse and progression, Lori Muffly, MD, MS, said in her presentation.

Therefore, a conditioning regimen that results in minimal toxicity but has enhanced disease control is needed in order to improve transplantation outcomes in this population, Muffly, associate professor of medicine (blood and marrow transplantation and cellular therapy) at Stanford Healthcare, continued.

JSP191 is a humanized monoclonal antibody meant to block stem cell factor binding site on CD117, which is necessary for hematopoietic stem cell (HSC) survival and HSC interactions in the bone marrow niche. After the bone marrow niche is emptied because of JSP191 binding to CD117, healthy donor cells are able to engraft. Preclinical models showed synergy between anti-CD117 monoclonal antibodies and low-dose TBI to help deplete HSC and facilitate donor cell engraftment.

For the first 24 patients with MDS (n = 13) or AML (n = 11), primary end points evaluated were safety, tolerability, and pharmacokinetics of the combination. Secondary end points included engraftment and donor chimerism, MRD clearance, relapse-free survival, graft-vs-host disease (GVHD), non-relapse mortality, and overall survival. Patients received AHCT, then 200 to 300 cGy of TBI, 30 mg/m2 of fludarabine for 3 days, and 0.6 mg/kg of intravenous JSP191.

To determine the starting date of fludarabine, real-time pharmacokinetic measurements and modeling were used after JSP191 was administered. For the first 7 patients, TBI was increased from 200 to 300 cGy to aid lymphoablation. Tacrolimus, sirolimus, and mycohphenolate motefil were used as GVHD prophylaxis.

Consistent pharmacokinetics and predictable clearance were observed with JSP191 over the 2 weeks after administration. All patients were able to receive donor cell infusion between 9 and 15 days following administration of the antibody. Interestingly, we did see in some patients very low levels of the antibody present on the day of donor cell infusion, and this did not appear to impact donor cell engraftment, Muffly said.

Bone marrow aspirations taken at screening and between administration of the antibody and fludarabine/TBI showed JSP191 depletes hemopoietic stem and progenitor cells (HSPC). In the CD34-positive, CD45RA-negative population, there was a 66% mean depletion of HSPC. The investigators do not believe this reflects the nadir of HSPC depletion, Muffly explained, and that the depletion continues until donor stem cell infusion.

All patients experienced neutropenia followed by neutrophil engraftment between TD+15 and TD+26. Primary engraftment was seen in all patients, with only 1 patient losing myeloid chimerism early, which was associated with disease progression. T cell chimerism improved when patients went up from 200 to 300 cGy.

Using flow cytometry, cytogenetics, and next-generation sequencing, investigators were able to track MRD in patients with de novo AML (n = 8) and AML from MDS (n = 3). Of the 9 patients with AML who were MRD positive at the time of enrollment, 6 were MRD negative at the time of follow-up. Eleven of 13 patients with MDS were MRD positive at enrollment, and 8 were MRD negative at the last follow-up.

After 6 months median follow-up (range, 2-12 months), there were no reports of classical grade II-IV acute GVHD. One case of late onset grade III-IV acute gastrointestinal GVHD was reported as of the latest follow-up, but this patient had non-relapse mortality. Any instances of chronic GVHD has yet to be reported due to insufficient median follow-up time. Morphologic relapse occurred in 4 patients, 3 with AML and 1 with MDS.

The median age for these patients was 70 years (range, 62-79), with a requirement of 60 years of age or older or an AHCT-comorbidity index of 3 or more to enroll in the trial. They could not have prior AHCT and needed a human leukocyte antigenmatched related or unrelated donor. Over half of patients received only a hypomethylating agent-containing regimens.

JSP191 in combination with fludarabine and low-dose TBI is a novel conditioning platform that appears safe, well tolerated, has demonstrated on-target effects of HSPC depletion, permits full donor myeloid chimerism, and results in promising early MRD clearance, Muffly concluded.

Reference:

Muffly L, Lee CJ, Gandhi A, et al. Preliminary data from a phase 1 study of JSP191, an anti-CD117 monoclonal antibody, in combination with low dose irradiation and fludarabine conditioning is well-tolerated, facilitates chimerism and clearance of minimal residual disease in older adults with MDS/AML undergoing allogeneic HCT. Presented at: 2022 Transplantation & Cellular Therapy Meetings; Salt Lake City, UT; April 23-26, 2022. Abstract LBA4. https://bit.ly/3xRTwee

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Bone Therapeutics announces 2021 full year results – GlobeNewswire

By daniellenierenberg

REGULATED INFORMATION

Strategic focus revised and fully focused on achieving topline results of the ALLOB Phase IIb study in tibial fractures.

Discussions for ALLOB global partnership still ongoing.

Board of Directors and Management exploring all strategic options to protect shareholder value.

Strengthening financial position with EIB agreement and private placement in 2021 and a new bond issuance foreseen in May 2022

Management to host conference call today at 3pm CEST / 9am EST - details provided below

Mont-Saint-Guibert, Belgium, 29April 2022, 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today announces its business update and full year financial results for the year ending 31 December 2021, prepared in accordance with IFRS as adopted by the European Union.

Incomplete fracture healing remains a seriously unmet medical need affecting hundreds of thousands of patients worldwide. Despite the pandemic and subsequent seriously geopolitical and economic global events, Bone Therapeutics still remains on target for delivery of topline results for its Phase IIb study of its allogeneic cell therapy product, ALLOB, in patients with difficult tibial fractures, said Miguel Forte, MD, PhD, CEO of Bone Therapeutics. We believe ALLOB could provide difficult tibial fracture patients a convenient treatment option with a potentially superior outcome. Having successfully completed two clinical studies showing promising safety profile and efficacy signals in more than 60 patients, we firmly believe that ALLOB has the highest potential of near-term value creation and is focused on completing the current Phase IIb study. In addition, Bone Therapeutics has made a serious contribution for the future into the use of Induced Pluripotent Stem Cell (iPSC) derived, genetically engineered MSCs. Bone Therapeutics is continuing its efforts to establish value adding business collaborations and to strengthen its financial position.

Clinical and operational highlights (including post-period events)

In January 2021, Bone Therapeutics initiated the treatment of patients in the Phase IIb study of its allogeneic cell therapy product, ALLOB, in patients with difficult tibial fractures. Bone Therapeutics anticipates finalizing patient recruitment of this study in 2022. This recruitment finalization is subject, as across the industry, to evolution of the ongoing COVID-19 pandemic and the associated containment measures. Although early recruitment rates were very promising, the recruitment rates have temporarily slowed in subsequent months due to pandemic-related factors, such as reduced site activities due to staff availability and the number of available patients due to less occurrence of accidents. Bone Therapeutics has implemented several mitigating measures in collaboration with the involved clinical research organization to improve and facilitate recruitment. These measures include site expansion, training, information, best practices sharing and close monitoring of progress. As a result of these measures and the improving recruitment rate, Bone Therapeutics continues to currently expect the release of topline data by Q1 2023.

In January 2021, Bone Therapeutics signed an initial agreement for a process development partnership with the mesenchymal stromal cell (MSC) specialist, Rigenerand. This collaboration focused on further developing and enhancing Bone Therapeutics bone-forming platform.

In June 2021, Bone Therapeutics published the positive results of its Phase I/IIa clinical trial with ALLOB in patients with delayed union fractures. The results were published in Stem Cell Research & Therapy, the international peer-reviewed journal focusing on translational research in stem cell therapies. ALLOB was generally well-tolerated and that all patients met the primary endpoint.

In August 2021, Bone Therapeutics announced topline results from the Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004, its legacy non-MSC product. JTA-004 had a favorable safety profile. However, the study did not meet the primary and key secondary endpoints. No statistically significant difference in pain reduction could be observed between the treatment, placebo and comparator groups, with all treatment arms showing similar efficacy.

In September 2021, Bone Therapeutics signed a research evaluation agreement with Implant Therapeutics, the developer of hypoimmunogenic and safe harbor engineered IPSC derived cells. The agreement enables Bone Therapeutics to access, evaluate and materially transfer Implant Therapeutics Induced Pluripotent Stem Cell (iPSC) derived, genetically engineered MSCs, including lines, media, differentiation protocols and expertise.

In November 2021, Bone Therapeutics signed a non-binding term sheet for the global rights for ALLOB, Bone Therapeutics allogeneic osteoblastic cell therapy product, with one of its current Chinese partners, Link Health Pharma Co., Ltd (Link Health). The negotiations for the global rights agreement are still ongoing but take longer than expected. The envisaged completion of a final binding agreement has been delayed and is now contemplated over the course of Q2 2022.

Corporate highlights (including post-period events)

In March, 2021, Bone Therapeutics appointed the stem cell therapy industry veteran, Anthony Ting, PhD, as Chief Scientific Officer. Dr. Ting is responsible for Bone Therapeutics research activities.

In July 2021, Bone Therapeutics appointed Dr. Anne Leselbaum as Chief Medical Officer. Dr. Leselbaum brings three decades of experience in strategic international clinical development, clinical operations and medical affairs. As CMO, she takes responsibility for the leadership of all clinical development and medical affairs strategies and activities across the entire Bone Therapeutics pipeline and will oversee the regulatory interactions.

In September 2021, Bone Therapeutics appointed Lieve Creten, as interim Chief Financial Officer (CFO), succeeding Jean-Luc Vandebroek. Lieves extensive financial experience ensures the continued optimal financial control, oversight and compliance.

In October 2021, Bone Therapeutics appointed key experts to its Scientific Advisory Board (SAB). The members of the SAB consist of world-recognized scientists and clinicians in the cell and gene therapy field.

In March 2022, Bone Therapeutics announced it was redefining its strategic priorities to concentrate specifically on the development of its most advanced clinical asset, ALLOB. As a result, Bone Therapeutics will focus its R&D activities to support the clinical development of ALLOB and all activities related to the development of the pre-clinical iMSCg platform as well as all other non ALLOB related activities, were stopped. In this context, some members of Bone Therapeutics' management team will depart Bone Therapeutics in the following months in alignment with the refocus in activity. This includes Miguel Forte (CEO), Tony Ting (CSO), Stefanos Theoharis (CBO) and Lieve Creten (CFO). During the transition, CEO, Miguel Forte, will remain in function. The Scientific Advisory Board was also dissolved.

Financial highlights (including post-period events)

In July 2021, Bone Therapeutics secured a loan agreement of up to 16.0 million with the European Investment Bank (EIB). The EIB loan financing will be disbursed in two tranches of 8.0 million each, subject to conditions precedent. Following the approval of the issuance of associated warrants by Bone Therapeutics General Meetings at the end of August 2021, Bone Therapeutics received a payment from the EIB for the first tranche of 8.0 million and the EIB was granted 800,000 warrants approved by the Extraordinary General Meeting.

In August 2021, Bone Therapeutics also renegotiated 800 convertible bonds issued on May 7, 2020 (for an amount of 2 million) to Patronale Life into a loan subject to the same repayment terms as the agreement with the EIB, with the issuance of 200,000 additional warrants approved by the Extraordinary General Meeting.

In December 2021, Bone Therapeutics raised additional 3.3 million funding through a private placement with current and new institutional investors to advance its lead orthopedic asset, ALLOB, through mid-stage clinical development.

The total revenues and operating income for 2021 amounted to 2.7 million compared to 3.7 million in 2020. As a result of the reduced clinical activities following the completion of the Phase III JTA-004 study, and the slower pace of patient enrollment for the ALLOB TF2 Phase IIb study due to the COVID-19 pandemic, operating loss for the period decreased to 12.0 million from 15.0 million for the full year 2020. Consequently, cash used for operating activities amounted to 12.8 million for the full year 2021. Year-end cash position amount to 9.5 million compared to 14.7 million year-end 2020.

In April 2022, Bone Therapeutics signed a binding term sheet for a 5 million convertible bonds (CBs) facility arranged by ABO Securities. The proceeds of the financing will be used to advance the clinical development of Bone Therapeutics lead asset, the allogeneic bone cell therapy, ALLOB. ABO Securities, on behalf of the CB investor, commits to subscribe to up to 5 million in CBs. Subject to the fulfillment of condition precedents, Bone Therapeutics and ABO Securities aim to agree on and execute the final subscription agreement for the CBs and to issue the first tranche of CBs by the beginning of May 2022.

Outlook for the remainder of 2022

In the ongoing Phase IIb ALLOB clinical study in difficult tibial fractures, Bone Therapeutics clinical team, in partnership with its clinical research organization, is continuing to institute measures to mitigate the impact of the pandemic and will closely monitor the recruitment progress. As a result of the initial mitigation actions and the improving recruitment rate due to the gradual lifting of COVID-19 related measures in Europe, Bone Therapeutics expects to report topline results as scheduled by the first quarter of 2023. However, a delay cannot be excluded. Should the pandemic continue to have impact on patient availability, Bone Therapeutics may have to re-evaluate this timeline and, in that eventuality, will communicate again to the market.

The negotiations for ALLOB, with one of Bone Therapeutics current Chinese partners, for the global rights agreement are still ongoing but are taking longer than originally anticipated. The potential completion of a final binding agreement has been delayed into Q2 2022.

Subsequent to some preliminary contacts, the board of directors of Bone Therapeutics is currently examining various opportunities to combine certain activities within Bone Therapeutics, taking into account the interests of its shareholders and other stakeholders. Further announcements will be made in due course, if and when circumstances so allow or require.

Following the restructuring of the management team announced on 12 April 2022, Bone Therapeutics has initiated the search for a new CEO and CFO.

Disciplined cost and cash management will remain a key priority. The operating cash burn for the full year 2022 is expected to be in the range of 8-10 million, assuming normal operations as the effect of the ongoing COVID-19 epidemic cannot be excluded. The situation will be actively and closely monitored. The company anticipates having sufficient cash to carry out its business objectives into Q1 2023, assuming, amongst other, full issuance of the new convertible bond facility. Bone Therapeutics refers to the going concern statement in the Annual Report 2021 for all key assumptions taken.

Conference call

Miguel Forte, MD, PhD, Chief Executive Officer will host a webcast with conference call today at 3:00 pm CEST / 9:00am EST. To participate in webcast or the conference call, please use the following link:

https://us06web.zoom.us/j/81633950602

Or select your dial-in number from the list below quoting the conference ID 816 3395 0602#:

Belgium: +32 2 290 9360France: +33 1 7095 0103United Kingdom: +44 208 080 6592United States: +1 646 876 9923

The presentation will be made available on the Investors section - Presentations of the Bone Therapeutics website shortly prior to the call.

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. Currently Bone Therapeutics is concentrating specifically on the development of its most advanced clinical asset, the allogeneic cell therapy platform, ALLOB.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell and gene therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Its leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the Louvain-la-Neuve Science Park in Mont-Saint-Guibert, Belgium. Further information is available at http://www.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerLieve Creten, Chief Financial Officer ad interimTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicBert BouserieTel: +32 (0)488 40 44 77bert.bouserie@bepublicgroup.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Hairy cell leukemia: Outlook, treatment, and what to expect – Medical News Today

By daniellenierenberg

Hairy cell leukemia is a rare type of blood cancer that can affect adults. In people who receive treatment, the long-term outlook for hairy cell leukemia is good.

Hairy cell leukemia (HCL) occurs when bone marrow produces too many white blood cells called lymphocytes.

The disease gets its name from the hairlike projections on its cells. HCL cells can affect the bone marrow, spleen, liver, and lymph nodes.

According to the National Organization for Rare Disorders, HCL is more common in males over the age of 50 years.

HCL affects roughly 6,000 people in the United States, with around 600800 new diagnoses each year. Around 12% of all adult leukemias are HCL.

In many cases, the long-term outlook for HCL is good, with people often continuing to live good-quality lives for years with medical care.

In this article, we look at the outlook and survival rates for HCL, the risk of secondary cancers, and treatment options.

Learn about the symptoms of HCL here.

HCL is a chronic disease, and although there is no cure for it, the condition is treatable. Treatment is usually highly effective and can help people continue to live normal lives.

According to the National Cancer Institute, HCL progresses slowly or does not worsen at all.

The Leukemia and Lymphoma Society reports that the 5-year event-free survival rate for HCL is 90% in people who received initial treatment with the chemotherapy drug cladribine. This means 90% of people will still be alive 5 years after diagnosis.

Treatment with cladribine has led to roughly 85% complete remission and around 10% partial response in people with HCL.

A 2020 study looked at survival rates in 279 people diagnosed with HCL between 1980 and 2011. The median age of the participants was 59 years old. In 208 of the participants, the first-line treatments were the drugs cladribine or pentostatin.

A 10-year follow-up found that the median survival rate was 27 years overall, with 11 years of relapse-free survival. There was a relapse rate of 39%. The study concluded that people with HCL have a good long-term outlook.

Research suggests that there may be racial disparities in HCL outcomes. A 2015 study included participants of the following racial groups:

The study found that the 10-year survival rate was worse in African American participants than in those of other racial groups.

Half of African American participants were alive at the 10-year follow-up, whereas more than two-thirds of those in other racial groups were alive at the follow-up.

The researchers concluded that the biological, socioeconomic, and health system factors contributing to this disparity need further investigation.

According to a 2020 study, people with HCL have an increased risk of secondary cancer.

Among 279 participants, 59 people developed at least one secondary cancer. The most common secondary cancers were prostate cancer, nonmelanoma skin cancer, and blood cancers.

The study did not find that treatment with purine analogs, such as cladribine or pentostatin, was a risk factor for secondary cancers.

However, according to the National Cancer Institute, cladribine and pentostatin may increase the risk of Hodgkin lymphoma and non-Hodgkin lymphoma.

Some research suggests that HCL and its effects on the body may increase the risk of secondary cancer.

People with HCL must attend regular cancer screenings to detect any early signs of secondary cancer.

Blood cell changes in those with HCL may result in compromised immune systems, making people more susceptible to infection or autoimmune disease.

HCL responds very well to treatment, which aims to manage the cancer rather than cure it.

Unlike with many other types of cancer, doctors may choose to wait before starting treatment.

Doctors will monitor the condition and may only begin treatment if they believe it is necessary to control it. This can help avoid any unnecessary side effects of treatment.

The type of treatment will depend on each condition but may include the following:

Cladribine and pentostatin are purine analogs, which are the first-line treatment for HCL.

According to the Hairy Cell Leukemia Foundation, both medications are highly effective treatments and can result in long-term remission.

In 2018, the Food and Drug Administration (FDA) approved another drug, moxetumomab pasudotox, to treat HCL. Doctors may use this drug in people who have not responded to standard therapies.

Interferon is a drug that doctors may use to treat HCL. Interferon uses the bodys immune system to help fight off cancer. Interferon affects how cancer cells divide and helps slow tumor growth.

Doctors may also use a biologic drug called rituximab, known by the brand name Rituxan, if people with HCL have not responded to other treatments. Rituximab is an antibody that attaches to HCL cells. Doctors may also use rituximab in combination with chemotherapy as a first-line treatment.

Targeted therapies use medications or other substances to find and destroy cancer cells. Targeted therapies may cause less harm to healthy cells than other treatments, such as radiation therapy or chemotherapy.

One type of targeted therapy to treat HCL is monoclonal antibody therapy. A laboratory creates antibodies that attach to cancer cells and destroy them or prevent them from growing and spreading. The biologic drug rituximab is an example of a monoclonal antibody.

Splenectomy is a surgical procedure to remove the spleen. This may be necessary if HCL causes an enlarged spleen.

However, doctors rarely perform splenectomy for HCL because there are medications that can effectively reduce the size of the spleen.

Learn more about immunotherapy for leukemia here.

Treatments for HCL can have the following side effects:

Cancer treatments may also cause other side effects, such as fatigue, appetite loss, or nausea.

Before starting treatment, people can discuss any potential side effects and the risks and benefits of each treatment option with their healthcare team.

Learn more about side effects here.

HCL is a rare type of leukemia. Other types of leukemia include:

HCL is a rare type of adult leukemia. It is more common in males over the age of 50 years.

The overall outlook for people with HCL is good. Treatment with chemotherapy drugs, such as cladribine and pentostatin, is highly effective and may result in long-term remission.

Treatments for HCL may have side effects. People can discuss any treatments potential risks and benefits with their healthcare team.

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Hairy cell leukemia: Outlook, treatment, and what to expect - Medical News Today

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Molecular Imaging (PET and SPECT) for Children with Hypoxic-ischemic-encephalopathy and Cerebral Palsy before and after cell therapy – Newswise

By daniellenierenberg

Abstract: Glucose metabolism has been the focus of research in order to understand pathological conditions associated with diseases such as neonatal hypoxic-ischemic-encephalopathy (HIE), cerebral palsy (CP) and cerebral infarction.

Objective:To evaluate the use of molecular imaging (SPECT and PET) for children with HIE and CP before and after cell therapy, and to propose future perspectives on the use of those modalities for assessment of brain function in children with these conditions.

Methods:PubMed search for studies using PET or SPECT scans for HIE and CP in children.

Results:We identified 18 PET and 17 SPECT studies that have been performed in cases under age of 19 over the past three decades (19912021). Six papers on PET use consisted of one with human umbilical cord derived mesenchymal stromal cells, one mobilized peripheral blood mononuclear cells, three autologous bone marrow mononuclear cells and one allogeneic umbilical cord blood. 4/6 papers reported that PET-CT scan revealed increased glucose metabolism and 1/6 showed no significant change in glucose metabolism after cell therapy. One article on SPECT reported that 2/5 cases had improvement of cerebral perfusion in the thalamus after treatment.

Discussion:SPECT in the first few weeks of life is useful and more sensitive than MRI in predicting major neurological disability. SPECT is not appropriate for neonates because of the risk of radiation, improvement of other clinical test equipment. PET studies reported high glucose metabolism in the early neonatal periods in children with mild to moderate HIE, but not in the most severe cases, including those neonates that died.We suggested that PET could be more useful tool to estimate effectiveness of stem cell therapy than SPECT.

Conclusion:PET might be a good clinical modalities to clarify mechanism of stem cell therapy for CP. We need further clinical studies to clarify more precisely.

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Burden of arrhythmias and predictors of mortality among multiple myeloma patients with arrhythmias – Journal of Investigative Medicine

By daniellenierenberg

Abstract

Arrhythmias are a major cardiac complication reported among patients with multiple myeloma (MM), but these have not been further characterized in this population. We explored the prevalence of arrhythmias and examined the predictors of mortality among patients with MM with arrhythmias. The National Inpatient Sample data collected between 2016 and 2018 were used to conduct retrospective analyses. Multivariable logistic regression analyses were done to examine the predictors of mortality among patients with MM with arrhythmias. 16.9% of patients with MM reported a diagnosis of any arrhythmias and 70.7% of these were atrial fibrillation. Patients aged 70 years and above had 21% lower odds (adjusted OR (AOR): 0.79; 95% CI: 0.68 to 0.92) of inpatient mortality relative to younger patients. Those in the non-Hispanic black, Hispanic, and non-Hispanic other category were 1.38 (95% CI: 1.16 to 1.64), 1.53 (95% CI: 1.19 to 1.97), and 1.69 (95% CI: 1.29 to 2.21) times more likely to die during hospitalization compared with their counterparts who were non-Hispanic whites. Relative to patients with MM who were on Medicare, those on private (AOR: 1.28; 95% CI: 1.06 to 1.54) and other insurance types (AOR: 1.78; 95% CI: 1.23 to 2.58) had higher odds of mortality. Other predictors of inpatient mortality were elective admission (AOR: 0.67; 95% CI: 0.52 to 0.85) and Charlson comorbidity indices between 57 (AOR: 1.23; 95% CI: 1.07 to 1.41) and 8 (AOR: 1.45; 95% CI: 1.21 to 1.73) compared with comorbidity indices between 0 and 4. Our study adds to the body of knowledge on the need for proper diagnosis and management of cardiac arrhythmias in patients with MM. Research is needed to further assess the time of arrhythmia diagnosis and its impact on health outcomes among patients with MM.

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Versant-backed startup launches with plans to broaden cell therapy’s reach – BioPharma Dive

By daniellenierenberg

Stem cell transplants can effectively cure a wide range of diseases, from blood cancers to rare genetic disorders. They've been used for decades and are considered standard treatment for certain conditions.

But for a good number of patients, stem cell transplants are out of reach. Drug regimens used to prepare the body for a transplant are toxic and can cause serious side effects. The transplanted cells don't always "engraft," or take root in the bone marrow. Even when they do, patients' disease may linger or recur.

A biotech startup launching Wednesday with $50 million in funding hopes that, by combining cell, antibody and gene editing technologies, at least some of these problems can be overcome. Called Cimeio Therapeutics, the new company is led by a team of pharmaceutical industry veterans and an advisory board filled with scientific luminaries, including immunologist Jeffrey Bluestone and gene editing pioneer Fyodor Urnov.

Cimeio's approach involves "shielding" transplanted cells by genetically editing them in ways that allows paired immunotherapies to be safely used both before and after a transplant.

Thomas Fuchs

Courtesy of Cimeio Therapeutics

"We think that this can really unleash the power of hematopoietic stem cell transplant and make a lot more patients eligible for it," said Thomas Fuchs, Cimeio's CEO and a former Genentech executive.

The "shielding" technology used by Cimeio was developed in Switzerland at the laboratory of Lukas Jeker, a physician-scientist from Basel University Hospital who will join Cimeio as head of gene editing.

Jeker's lab discovered that protein receptors on the surface of cells could be genetically edited in such a way that prevented antibodies from binding to them, while leaving their function intact. In preclinical testing, these edits could cloak, or "shield," the cells from being depleted by antibody drugs and T cell therapies.

The work could have powerful implications for improving stem cell transplant and adoptive cell therapy, according to Fuchs.

Once a stem cell or T cell is shielded, a complementary immunotherapy could be used to either help ready patients for a transplant or to further treat disease afterwards, he said. "Maybe you could give a cycle or two of the paired immunotherapy, implant the shielded cells and then continue to administer the immunotherapy," he added.

If the shielding works as intended, Cimeio could develop treatments for conditioning that are more tolerable than the chemotherapy or radiation-based regimens currently in use. Shielding might also allow existing drugs that target cell proteins on healthy as well as diseased cells to be used more flexibly with transplants, such as to treat residual disease that lingers afterwards.

For example, Cimeio could engineer stem cells that are protected against binding via a protein called CD19 that's often the target for CAR-T therapies that treat lymphoma, but is also found on healthy B cells that help the immune system fight off threats.

"One benefit could be that you could prevent a lifetime of B cell depletion, which happens when you give a CAR-T," said Fuchs.

Alex Mayweg

Courtesy of Cimeio Therapeutics

Cimeio was built from Jeker's lab by Versant Ventures at the company's "Ridgeline" incubator in Basel, which has previously produced companies like Monte Rosa Therapeutics and Black Diamond Therapeutics. The initial $50 million Versant provided will fund Cimeio through next year, said Alex Mayweg, a managing director at the venture firm and a Cimeio board member. Additional investors will be brought on later this year or early next, Mayweg said.

Cimeio will need the money, as its research and development plans are expansive. The company has identified four drug candidates already and envisions a dozen more behind those, said Fuchs. Its research spans blood cancers, rare genetic diseases and autoimmune disorders.

In some cases, Cimeio will develop paired immunotherapies to go with the shielded cells. In others, it will use existing treatments. Three of the first four candidates involve protecting hematopoietic stem cells, while the fourth involves T cells. The company hopes to begin human testing next year.

Cimeio plans to choose gene editing technologies based on the type of alteration it needs to make to shield cells. "Rather than building up an internal editing capability," Mayweg said, "we wanted to stay as flexible as possible."

That might mean partnerships or alliances with other companies, some of which have reached out to Cimeio already, according to Mayweg.

Cimeio is aided by a group of scientific advisers notable for their work in areas the company is focusing on. Urnov, of the University of California, Berkeley, is well known for his research in gene editing using zinc finger nucleases and CRISPR. Bluestone previously led the Parker Institute for Cancer Immunotherapy and is CEO of the cell therapy-focused biotech Sonoma Biotherapeutics.

Suneet Agarwal, a co-program leader of the stem cell transplant center at Boston Children's Cancer and Blood Disorders Center, is also on the advisory board, while Cimeio has a research collaboration in place with Matthew Porteus, a gene editing specialist at Stanford University.

About 20 people currently work at Cimeio directly, a number Fuchs expects will grow as the company's research advances. Another 15 are currently supporting Cimeio from Versant's Ridgeline group.

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Molecular Characteristics of Resistance to CD19-Directed CAR T Cells Revealed in Pediatric ALL – Cancer Network

By daniellenierenberg

Investigators uncovered potential mechanisms of resistance to CD19-directed CAR T-cell therapy in patients with pediatric acute lymphoblastic leukemia.

Bone marrow samples from a trial examining CD19-directed CAR T-cell therapy helped investigators identify epigenetic biomarkers predicting resistance to therapy in pediatric acute lymphoblastic leukemia (ALL), according to a presentation from the 2022 American Association for Cancer Research (AACR) Annual Meeting.1

Investigators observed 3 key features of leukemia cells that do not respond to CAR T-cell therapy: hypermethylation of DNA, a stem cell-like phenotype and inherent plasticity, and decreased antigen presentation. These are independent of CD19 status and leukemia subtype, indicating a new predictive biomarker.

Whats most important about this is we can detect it prior to therapy in patient samples, so this highlights its potential as a biomarker for response, Katherine E. Masih, BS, an NIH-Cambridge scholar in the Genetics Branch, Center for Cancer Research at the National Cancer Institute, said in a press conference. We hope that eventually this can improve patient selection and eligibility for CD19 CAR T-cell therapy.

CD19 is a common target of CAR T cells, and resistance to treatment can occur even if patients continue to show CD19 expression. The investigators explored primary non-response (PNR) to CAR T-cell therapy, which occurs in 10% to 20% of patients and whose causes are not fully understood. Known reasons for non-response to CAR T cells include collection of dysfunctional T cells and decreased death cell receptors on the cell surface.2,3

The investigators used bone marrow samples from 14 participants in the PLAT-02 trial (NCT02028455) of CD19-directed CAR T-cell therapy for relapsed/refractory pediatric ALL.1 These samples included those of 7 patients who had a complete response to therapy and 7 who had no response. Non-response was defined as not achieving and maintaining minimal residual disease negativity at 63 days.

A multiomic analysis of the bone marrow included whole-exome sequencing, RNA sequencing of the bulk cells, single-cell RNA sequencing, array-based methylation analysis, and ATAC-seq (Assay for Transposase-Accessible Chromatin using sequencing).

In patients who went on to have PNR, investigators discovered epigenetic markers including 238 regions of hypermethylated DNA, which is associated with inactivated genes (P = 8.15 10-25). These regions are known to be activated in stem cells.

The ATAC-seq analysis showed increased accessibility of chromatin at regions linked to stem cell proliferation (normalized enrichment score [NES] = 2.31; P < .0001) and cell cycling (NES = 2.27; P < .0001), indicating inherent plasticity that may allow leukemia cells to adapt to resist CAR T-cell therapy.

Investigators found that the epigenetic differences did not lead to differences in phenotype of B cells between primary sensitive and PNR patients. However, PNR cells did show an increase in regions associated with hematopoietic stem cells and myeloid and lymphoid progenitors. In addition, investigators observed decreased antigen presentation and processing that could lead to lack of response in cells that still express CD19 (P = .0001).

These factors characterized a potential novel biomarker associated with PNR that investigators named Stem-Cell Epigenome with Multi-Lineage Potential (SCE-MLP). Masih acknowledged that the sample size of 14 patients was small and more research on SCE-MLPs link to PNR is needed. We would love to see this validated in a larger cohort with more cases of PNR that exist around the country, she said.

Another potential use of SCE-MLP could be to find ways to overcome resistance to CAR T-cell therapy by combining it with targeted therapies that disrupt these epigenetic factors for resistance.

Currently, the investigators hope that this research will be used to shape patient selection for CAR T-cell therapy and alternative therapies.

If we can reliably identify responders, perhaps through screening for SCE-MLP, we can prioritize less toxic targeted therapies for our patients and overall improve outcomes for children with this devastating disease, Masih said.

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Columbus man to be one of first to receive transplant via new OhioHealth program – 10TV

By daniellenierenberg

Marc Howard was diagnosed with multiple myeloma. He will receive a transplant using his own cells later this year.

COLUMBUS, Ohio It all started with back pain that seemed to be progressively getting worse.

My back, the structure of my body, was like starting to deteriorate, and I could tell, he said. I'm tall, so when I started to lean over, and the pain and things of that nature, I'm like, yo, something's going on.

His longtime love Sonia Grant noticed, too. And she was right there to encourage him to get it checked.

When he did, doctors found holes in his spine where his bone had deteriorated. He had a vertebroplasty procedure to have those holes filled with bone cement. But that was not the end of his journey. In fact, it was really only the beginning.

After the surgery, he was okay for about a month, then I saw him (leaning) over again, and he couldnt get off the bed one day, Grant said. I said, uh uh, were going back up there (to the hospital). Theres something wrong.

And something was. Howard was diagnosed with multiple myeloma, a cancer that forms in the plasma cells.

I dont want to be the woe is me, Howard said. I want to be the success story for somebody, for the world to look at, like, that man went through a situation, and he made it.

And hes making it so far, with the help of Grant. Hes been doing weekly chemotherapy treatments and taking daily medication. Meanwhile, Grant is making sure hes eating his fruits and veggies and drinking plenty of water, too.

If youre not up to the challenge, I will help you get there, I will, Grant said. Because failure is just not a thing when it comes to fighting something like cancer. You gotta fight, you just gotta fight.

This fight will culminate with a major procedure later this fall via OhioHealths new Blood and Marrow Transplant Program. Howard will be one of the first patients to receive an autologous stem cell transplant, meaning the procedure will use his own cells.

Dr. Yvonne Efebera, the medical director for the program, explains this process is different than a procedure using donor cells.

BMT, blood and marrow transplant, is a process where, certain diseases require this, where non-functioning, deficient bone marrow or cancer cells are eliminated by giving high-dose chemotherapy, with or without radiation, and then replaced by new, healthy cells, Dr. Efebera said.

Shes been treating Howard throughout this process and points out that this is one of the benefits of the new program. Before, patients who needed transplants would have to be sent to other healthcare systems. Now, they can go from start to finish with the same clinical team.

Marc always wanted to be the first, she joked. Hes anxious to have his stem cells to be the first collected and the first admitted.

Both Howard and Grant are up to the challenge.

Its a battle, Grant said. Were halfway through the battle, and so, were going to get all the way to the end of the battle. Bruised, not broken. But were in the battle. But were going to get through it.

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Columbus man to be one of first to receive transplant via new OhioHealth program - 10TV

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Be wary of companies offering stem cell therapy for arthritis, joint pain, COVID, and more. Heres why – WATN – Local 24

By daniellenierenberg

ABC24 talked with Randy Hutchinson from the Better Business Bureau of the Mid-South about a lawsuit against one company and what consumers need to watch out for.

MEMPHIS, Tenn. The Federal Trade Commission and Georgia Attorney General have sued the founders of a company they claim has made unsubstantiated claims its stem cell therapy can treat arthritis, joint pain, and other orthopedic ailments.

The company is called Stem Cell Institute of America. It claimed its treatments are comparable to or better than surgery, steroid injections, and painkillers. The FTC said the company charged up to $5,000 per injection. It said a related company taught chiropractors and other healthcare professionals how to offer stem cell therapy.

ABC24 talked with Randy Hutchinson from the Better Business Bureau of the Mid-South about the claims and what consumers need to watch out for.

So what are the facts about stem cells?

They're sometimes called the body's "master cells" because they develop into blood, brain, bones, and other organs.

Stem cells from bone marrow or blood are used to treat certain kinds of cancer and disorders of the blood and immune system. But other uses have not been properly studied and approved.

The FDA cites these potential risks from unproven treatments:

There could be safety risks even using a persons own stem cells.

Other claims by some companies

The FTC has also looked into companies claiming their stem cell therapies can treat Parkinson's, multiple sclerosis, COVID, and a host of other ailments. They're sometimes referred to as "regenerative medicine."

So what do consumers need to do?

Take miracle health care claims with a grain of salt.

Check out a company and treatment online using terms like "complaints," "scam" and "reviews."

Consult your own health care provider before using any product or treatment.

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Be wary of companies offering stem cell therapy for arthritis, joint pain, COVID, and more. Heres why - WATN - Local 24

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microRNA-148a in Exosomes Derived from Bone Marrow Mesenchymal Stem Cells Alleviates Cardiomyocyte Apoptosis in Atrial Fibrillation by Inhibiting…

By daniellenierenberg

This article was originally published here

Mol Biotechnol. 2022 Apr 9. doi: 10.1007/s12033-022-00487-z. Online ahead of print.

ABSTRACT

Exosomes-related microRNAs (miRNAs) have been considered to be the significant biomarkers contributing to the development of atrial fibrillation (AF). We observed the implicit mechanism of exosomes-miR-148a derived from bone marrow mesenchymal stem cells (BMSCs) in AF. The AF cell and mice models were established firstly. QRT-PCR and Western blot analysis were applied to detect the expression of miR-148a, SPARC-associated modular calcium-binding protein 2 (SMOC2), Bcl-2, Bax, and caspase-3. BMSCs were separated from healthy mice and exosomes were obtained from BMSCs. BMSCs were transfected with mimics and inhibitor, and HL-1 cells were treated with mimics and pcDNA3.1. MTT assay were used to detect cell viability of cells. Flow cytometric analysis and TUNEL analysis were used for detecting cell apoptosis of cells. In our study, exosomes derived from BMSCs inhibited the development of AF, and miR-148a acted a vital role in this segment. SMOC2 was a target gene of miR-148a and promoted apoptosis of HL-1 cells. Additionally, miR-148a mimics decreased cellular apoptosis, eliminated SMOC2 expression, and elevated Bcl-2 expression in AF-treated cells. Collectively, miR-148a overexpressed in BMSC-exosomes restrained cardiomyocytes apoptosis by inhibiting SMOC2.

PMID:35397056 | DOI:10.1007/s12033-022-00487-z

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microRNA-148a in Exosomes Derived from Bone Marrow Mesenchymal Stem Cells Alleviates Cardiomyocyte Apoptosis in Atrial Fibrillation by Inhibiting...

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Penticton woman donated bone marrow to save stranger’s life, urges others to do the same – Penticton News – Castanet.net

By daniellenierenberg

Photo: Contributed

Salny Ehman, left, and Tom Ellison, the man whose life she saved through a bone marrow donation.

Penticton resident Salny Ehman saved a complete stranger's life, and hopes to inspire others to do the same.

Ehman, now 35, signed up to be a bone marrow donor at the age of 19, after watching her grandmother's sibling go through leukemia.

"She has a whole bunch of siblings, and one of them had blood cancer, and only she was a match. And the math of that blew me away, how likely is it to have a close relative that can help?" Ehman said.

"If it's that unlikely to have a match when you have that many siblings, then people need to sign up."

Ehman did just that. She registered with Canadian Blood Services, and a few months later, got a call that she was a match with an anonymous recipient signed up through an American service, fighting cancer and in need of bone marrow stem cells.

Despite not knowing who she was donating to, Ehman underwent the donation procedure, and then one year later, did the same thing again after learning the recipient's body had been rejecting the donated cells.

"One year after the second donation, we were both allowed to say yes, we would like to know the other person. And we both said yes, but I was living in Nova Scotia at the time," Ehman said, having learned her stem cell recipient, Tom Allison, lived in Seattle.

"The only information given to me about [my donor] was it was a 19 year old girl from Nova Scotia. And I thought, what's a 19 year old girl doing on a bone marrow registry for?" Allison said.

"Neither one of my boys were matches, and neither one of my brothers. So this seemed just a million to one chance for somebody out there with a match that's no relation to me."

Allison and Ehman ultimately got in touch and kept in touch for more than a decade.

"We would write each other letters over the years, and little postcards," Ehman said.

"And then I moved here [to Penticton] two years ago. So as soon as I moved here, I was like, 'Wow, I'm so close.' Just a quick drive, like three and a half hours away."

She and Allison, who is now in his 60s and healthy post-cancer, met up in person recently, and Ehman was thrilled to see him thriving thanks to her donation.

"Second to my daughters, [donating] was the best, best experience in my entire life. And to know that he was out there, and spending time with his family, his grandchildren, it really meant a lot. And it showed me what I was capable of," Ehman said.

She and Allison remain good friends, and she urges anyone who can help to either sign up to be a donor, or if they can't participate in that way, give financially to Canadian Blood Services.

"There's lots of opportunities to help that cause," she said.

Find out more about Canadian Blood Services and how you can help here, and learn how you can be the match that saves someone's life here.

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Donors needed to save young lives – Benoni City Times

By daniellenierenberg

Three children are fighting for their lives and require stem cell transplants to cure their blood cancers and disorders.

Rayaan (four months), Emily (five months) and Neo (7) all share one commonality: being diagnosed with life-threatening blood diseases. They are waiting on blood stem cell transplantations from unrelated donors.

Between 800 and 1 000 children in South Africa are diagnosed with cancer annually.

However, this number does not account for the almost 50% of cases of childhood cancer that are never diagnosed, due to a lack of knowledge regarding the disease and how it presents in children.

Because children still experience growth spurts within a short space of time, this may cause blood cancer and disorders to spread quicker and more aggressively. Therefore, diseases affecting young children are those most often occurring in the developing cells, such as bone marrow, blood, kidneys and nervous system tissues.

Rayaan, Emily and Neos families lives have been turned upside down by blood cancer and disorders.

Rayaan was diagnosed with life-threatening acute myeloid leukaemia when he was just eight weeks old. AML usually requires immediate treatment and for Rayaan, bone marrow or blood stem cell transplant is his only chance of a cure.

Without a successful transplant, Rayaan will have to endure continued chemotherapy and isolation, which will expose him to the terminal effects of infection. Rayaan is now in search of an unrelated matching donor, but the low representation of diverse stem cell donors across the country and in the global registry hampers the chances of saving this courageous baby.

Arlene said watching her son endure this pain is heartbreaking and there have been some very dark days. At one point, he had to be resuscitated after a spinal lumbar puncture, but their courageous little fighter battled on and still wakes up every day with a smile on his face.

Please help my baby to live. He is just too little to suffer like this. Dont delay, you could be his perfect match, Arlene added.

Meanwhile, five-month-old Emily from Johannesburg has been battling a blood disorder following her diagnosis in November at only three months. She was diagnosed with juvenile myelomonocytic leukaemia (JMML) and is undergoing treatment, hoping a stem cell transplant will be performed soon.

As in Rayaans case, Emilys best chance at survival is a blood stem cell transplant. Dr Theo Gerdener, a clinical haematologist at Albert Alberts Stem Cell Transplant Centre and medical director at DKMS Africa, said:

JMML, which is especially prevalent in young children, is a rare cancer of the blood and occurs when white blood cells, known as monocytes and myelocytes, mature abnormally. This cancer can occur spontaneously or, sometimes, is linked to other genetic disorders.

Leukaemia affects white blood cells and bone marrow, and alarmingly, childhood leukaemia accounts for around 25% of all cancer in children. With proper diagnosis and management, including stem cell transplantation, childhood leukaemia can be cured in 85% to 90% of patients.

According to Natalie, Emilys mother, her daughter has already endured multiple blood and platelet transfusions, frequent injections and other medication, lengthy hospital stays, including isolation and ICU admission, as well as surgery to insert a port in her chest for intravenous administration.

Her parents desperately hope to find a stem cell donor match through DKMS global stem cell registry and donor centre to provide them with this one in 100 000 chance.

Were keeping positive and are hoping a match will be found for Emily. We hope she grows up, has a normal childhood and becomes a beautiful, bright young lady, said Natalie.

Neo was diagnosed with Fanconi anaemia in April 2019 at only four years old. A couple of years earlier, his older sister, who was also diagnosed with the same blood disorder, received a stem cell donor transplant, giving her a second chance at life.

Their dad, Phoebus, recalls the late-night rushes to the hospital, overnight stays and time away from work, as both parents grappled with the unusual, but persistent symptoms in their child, as Neo endured uncontrollable nosebleeds, debilitating fatigue, prominent bruising and innumerable fevers and infections owing to his compromised immune system.

Neo is transfusion-dependent and receiving steroid treatment, among other things. He is also searching for his second chance at life through an unrelated donor match to provide him with a life-saving blood stem cell transplant.

A donor with the same ethnic background as a patient may be a better match than one who comes from an entirely different background.

Globally, there are a low number of registered donors among the black, Indian and mixed-ethnic populations, meaning the pool of prospective matches is significantly lower.

For patients like Neo, a substantial increase in the registration of black donors will directly impact his chances of a successful transplant from a matched donor.

Neos family has thrown its support behind DKMS Africa to champion the cause of education and awareness around blood diseases.

Also Read:Young couple says I do with the help of generous donors

People need to be aware of these medical conditions and empower themselves with the knowledge. Some people and organisations are there to help. People should not be afraid to reach out, said Phoebus.

If you are in good health and between the ages of 18 and 55 and considering joining the registry, visit http://www.dkms-africa.org or call 0800 12 10 82, weekdays between 08:30 and 16:30.

Once you have registered online, a swab kit is sent to you via courier and then collected when you have completed the process (at no cost to you). Take action, save a life!

Also Read:Become an organ donor and help save a life

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Donors needed to save young lives - Benoni City Times

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FDA Grants Direct Biologics Regenerative Medicine Advanced Therapy (RMAT) Designation for the use of ExoFlo in COVID-19 Related ARDS USA – English -…

By daniellenierenberg

AUSTIN, Texas, April 13, 2022 /PRNewswire/ -- Direct Biologics, an innovative biotechnology company with a groundbreaking extracellular vesicle (EV) platform drug technology, announced that the U.S. Food and Drug Administration (FDA) has awarded their EV drug product ExoFlo with a Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of Acute Respiratory Distress Syndrome (ARDS) associated with COVID-19. The RMAT program is designed to expedite the approval of promising regenerative medical products in the US that demonstrate clinical evidence indicating the ability to address an unmet medical need for a serious life-threatening disease or condition. Under the RMAT designation, the FDA provides intensive guidance on drug development and post-market requirements through early and frequent interactions. Additionally, an RMAT confers eligibility for accelerated approval and priority review of biologics licensing applications (BLA).

"After intensively reviewing our preclinical data, manufacturing processes, and clinical data from our Phase II multicenter, double blinded, placebo controlled randomized clinical trial, the FDA has recognized ExoFlo as a lifesaving treatment for patients suffering from Acute Respiratory Distress Syndrome (ARDS) due to severe or critical COVID-19," said Mark Adams, Chief Executive Officer. "The additional attention, resources, and regulatory benefits provided by an RMAT designation demonstrate that the FDA views ExoFlo as a product that can significantly enhance the standard of care for the thousands still dying from ARDS every week in the US," he said.

"We are very pleased that the FDA has recognized the lifesaving potential of our platform drug technology ExoFlo. The RMAT has provided a pathway to expedite our drug development to achieve a BLA in the shortest possible time," said Joe Schmidt, President. "I am very proud of our team. Everyone has been working around the clock for years in our mission to save human lives taken by a disease that lacks treatment options, both in the US and abroad. We are grateful for the opportunity to accelerate development of ExoFlo under the RMAT designation as it leads us closer to our goal of bringing our life saving drug to patients who desperately need it."

ExoFlo is an acellular human bone marrow mesenchymal stem cell (MSC) derived extracellular vesicle (EV) product. These nanosized EVs deliver thousands of signals in the form of regulatory proteins, microRNA, and messenger RNA to cells in the body, harnessing the anti-inflammatory and regenerative properties of bone marrow MSCs without the cost, complexity and limitations of scalability associated with MSC transplantation. ExoFlo is produced using a proprietary EV platform technology by Direct Biologics, LLC.

Physicians can learn more and may request information on becoming a study site at clinicaltrials.gov. For more information on Direct Biologics and regenerative medicine, visit: https://directbiologics.com.

About Direct BiologicsDirect Biologics, LLC, is headquartered in Austin, Texas, with an R&D facility located at the University of California, and an Operations and Order Fulfillment Center located in San Antonio, Texas. Direct Biologics is a market-leading innovator and cGMP manufacturer of regenerative medical products, including a robust EV platform technology. Direct Biologics' management team holds extensive collective experience in biologics research, development, and commercialization, making the Company a leader in the evolving segment of next generation regenerative biotherapeutics. Direct Biologics has obtained and is pursuing multiple additional clinical indications for ExoFlo through the FDA's investigational new drug (IND) process. For more information visit http://www.directbiologics.com.

Photo - https://mma.prnewswire.com/media/1781269/Direct_biologics_Logo.jpg

SOURCE Direct Biologics

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Speaker’s passing highlights the plight of cancer patients in Uganda – Monitor

By daniellenierenberg

Dr Jackson Orem, the Director of the Cancer Institute, gave a detailed patient history to highlight the cause of the departed former speaker Jacob Oulanyahs death at the State funeral in Kololo, Kampala. last Wednesday.

Eloquent he was in narrating that the patient had utilised the two lines of treatment available in Uganda and was only left with two experimental options including stem cell therapy.

Stem cell therapy involves extraction of bone marrow cells and implanting them in the body to rejuvenate its ability to produce white blood cells. In the case of the former Speaker, the Minister of Health Dr Ruth Aceng highlighted that the patient had lost his spleen decades ago compromising his immunity. She read from the post mortem report the patient suffered from a compound of various bacterial and viral infections and succumbed to multiple organ failure.

In all these, perhaps the only addition would have come from a pathologist to elaborate further and pinpoint the exact cause of death. Pathologists are the academic doctors who study forms of life in its innate forms, whether as tissue, examining dead bodies etc. In some analyses these are supported by microbiologists. All these people exist in close proximity on Mulago Hill and the new national testing center at Butabika. Listening to Dr Orem it is clear Ugandas problem is not the diagnostic part.

The diagnostic part has only failed in delivery to the general population due to its prohibitive cost. For most patients it is the treatment part that drives patients and their families to desperation.

The stories of the on-and off operations of the two cobalt machines in Mulago that administer radiotherapy is well documented. Radiotherapy burns the cancerous cells that are trying to outnumber the white cells that produce antibodies to defend the body from infections.

There are a few things Dr Orem may have inserted in the national conversation. First is the rising number of cancer cases. Last count says 30,000 cases but these are the diagnosed cases processed through the Cancer Institute. All non-communicable diseases are on the rise. Cancer is just behind cardio-vascular diseases. Others are chronic respiratory diseases, type 2 diabetes mellitus (DM), and chronic kidney disease.

A recent study in 2021 in BMC Journal shows that rural areas are seeing as much of an upswing as urban areas where conditions like cancer are associated with lifestyle. Non communicable diseases are a big profit center for big pharma as management costs are high. Its an area where name brands have overwhelmed generic drugs.

The other version of cancer triggers are stress and chemicals in the water supply. The introduction of contraceptives in the 1970s as women entered the workforce is blamed for a surge in a generation of mothers. GMO foods which have made cooking oil a staple in many homes are also a factor.

Processed foods are also a factor, including artificial colouring, hydrogenated fats and saturated oils. Very few Ugandans know that cooking oil can easily run a diesel engine.

During the Covid pandemic, NCD patients suffered more from the ravages of the disease, including debilitating strokes, heart attacks that played on the vulnerabilities NCD sufferers find themselves in.

Managing these diseases is only going to get more complicated as the big anchor in the healthcare supply system USAID is exiting.

The cancer center is already too small, patients waiting for hours in agony for relief. In the Covid interlude, Mulago (when patients dropped between January and April 2021), offered excellent care to in-patients.

This all round model can be replicated for cancer patients. If there is excess capacity at the new womens hospital it could be used. Cancer is a one way street, all the urgency that people are treated with dignity.

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