Nes Ziona, Israel, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (NASDAQ: ENLV), a clinical-stage macrophage reprogramming immunotherapy company targeting diseased macrophages in patients with sepsis, COVID-19 and solid tumors, today announced the closing of its previously announced offering of 2,296,107 ordinary shares, par value NIS 0.40 per share, of the Company at a price to the public of $20.00 per ordinary share, less underwriting discounts and commissions.

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Enlivex Announces Closing of Previously Announced Bought Deal Offering of Approximately $46.0 Million Ordinary Shares

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FDA Approves G1 Therapeutics’ COSELA™ (trilaciclib): The First and Only Myeloprotection Therapy to Decrease the Incidence of Chemotherapy-Induced...

HOUSTON, Feb. 13, 2021 (GLOBE NEWSWIRE) -- Bio-Path Holdings, Inc., (Nasdaq:BPTH), a biotechnology company leveraging its proprietary DNAbilize® liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, today announced that it intends to offer and sell, subject to market and other conditions, shares of its common stock in a public offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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Bio-Path Holdings, Inc. Announces Proposed Public Offering of Common Stock

Prime Minister, Boris Johnson visits Newcastle-based QuantuMDx

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Prime Minister, Boris Johnson visits Newcastle-based QuantuMDx

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Neck Hammock Reviews – Does this gadget really help? – Product Review by Mike Vaughn

Machine learning for medicine

Small-molecule screens aimed at identifying therapeutic candidates traditionally search for molecules that affect one to several outputs at most, limiting discovery of true disease-modifying drugs. Theodoris et al. developed a machine-learning approach to identify small molecules that broadly correct gene networks dysregulated in a human induced pluripotent stem cell disease model of a common form of heart disease involving the aortic valve. Gene network correction by the most efficacious therapeutic candidate generalized to primary aortic valve cells derived from more than 20 patients with sporadic aortic valve disease and prevented aortic valve disease in vivo in a mouse model.

Science, this issue p. eabd0724

Determining the gene-regulatory networks that drive human disease allows the design of therapies that target the core disease mechanism rather than merely managing symptoms. However, small molecules used as therapeutic agents are traditionally screened for their effects on only one to several outputs at most, from which their predicted efficacy on the disease as a whole is extrapolated. In silico correlation of disease network dysregulation with pathways affected by molecules in surrogate cell types is limited by the relevance of the cell types used and by not directly testing compounds in patient cells.

In principle, mapping the architecture of the dysregulated network in disease-relevant cells differentiated from patient-derived induced pluripotent stem cells (iPSCs) and subsequent screening for small molecules that broadly correct the abnormal gene network could overcome this obstacle. Specifically, targeting normalization of the core regulatory elements that drive the disease process, rather than correction of peripheral downstream effectors that may not be disease modifying, would have the greatest likelihood of therapeutic success. We previously demonstrated that haploinsufficiency of NOTCH1 can cause calcific aortic valve disease (CAVD), the third most common form of heart disease, and that the underlying mechanism involves derepression of osteoblast-like gene networks in cardiac valve cells. There is no medical therapy for CAVD, and in the United States alone, >100,000 surgical valve replacements are performed annually to relieve obstruction of blood flow from the heart. Many of these occur in the setting of a congenital aortic valve anomaly present in 1 to 2% of the population in which the aortic valve has two leaflets (bicuspid) rather than the normal three leaflets (tricuspid). Bicuspid valves in humans can also be caused by NOTCH1 mutations and predispose to early and more aggressive calcification in adulthood. Given that valve calcification progresses with age, a medical therapy that could slow or even arrest progression would have tremendous impact.

We developed a machine-learning approach to identify small molecules that sufficiently corrected gene network dysregulation in NOTCH1-haploinsufficient human iPSC-derived endothelial cells (ECs) such that they classified similar to NOTCH1+/+ ECs derived from gene-corrected isogenic iPSCs. We screened 1595 small molecules for their effect on a signature of 119 genes representative of key regulatory nodes and peripheral genes from varied regions of the inferred NOTCH1-dependent network, assayed by targeted RNA sequencing (RNA-seq). Overall, eight molecules were validated to sufficiently correct the network signature such that NOTCH1+/ ECs classified as NOTCH1+/+ by the trained machine-learning algorithm. Of these, XCT790, an inverse agonist of estrogen-related receptor (ERR), had the strongest restorative effect on the key regulatory nodes SOX7 and TCF4 and on the network as a whole, as shown by full transcriptome RNA-seq.

Gene network correction by XCT790 generalized to human primary aortic valve ECs derived from explanted valves from >20 patients with nonfamilial CAVD. XCT790 was effective in broadly restoring dysregulated genes toward the normal state in both calcified tricuspid and bicuspid valves, including the key regulatory nodes SOX7 and TCF4.

Furthermore, XCT790 was sufficient to prevent as well as treat already established aortic valve disease in vivo in a mouse model of Notch1 haploinsufficiency on a telomere-shortened background. XCT790 significantly reduced aortic valve thickness, the extent of calcification, and echocardiographic signs of valve stenosis in vivo. XCT790 also reduced the percentage of aortic valve cells expressing the osteoblast transcriptional regulator RUNX2, indicating a reduction in the osteogenic cell fate switch underlying CAVD. Whole-transcriptome RNA-seq in treated aortic valves showed that XCT790 broadly corrected the genes dysregulated in Notch1-haploinsufficient mice with shortened telomeres, and that treatment of diseased aortic valves promoted clustering of the transcriptome with that of healthy aortic valves.

Network-based screening that leverages iPSC and machine-learning technologies is an effective strategy to discover molecules with broadly restorative effects on gene networks dysregulated in human disease that can be validated in vivo. XCT790 represents an entry point for developing a much-needed medical therapy for calcification of the aortic valve, which may also affect the highly related and associated calcification of blood vessels. Given the efficacy of XCT790 in limiting valve thickening, the potential for XCT790 to alter the progression of childhood, and perhaps even fetal, valve stenosis also warrants further study. Application of this strategy to other human models of disease may increase the likelihood of identifying disease-modifying candidate therapies that are successful in vivo.

A gene networkbased screening approach leveraging human disease-specific iPSCs and machine learning identified a therapeutic candidate, XCT790, which corrected the network dysregulation in genetically defined iPSC-derived endothelial cells and primary aortic valve endothelial cells from >20 patients with sporadic aortic valve disease. XCT790 was also effective in preventing and treating a mouse model of aortic valve disease.

Mapping the gene-regulatory networks dysregulated in human disease would allow the design of network-correcting therapies that treat the core disease mechanism. However, small molecules are traditionally screened for their effects on one to several outputs at most, biasing discovery and limiting the likelihood of true disease-modifying drug candidates. Here, we developed a machine-learning approach to identify small molecules that broadly correct gene networks dysregulated in a human induced pluripotent stem cell (iPSC) disease model of a common form of heart disease involving the aortic valve (AV). Gene network correction by the most efficacious therapeutic candidate, XCT790, generalized to patient-derived primary AV cells and was sufficient to prevent and treat AV disease in vivo in a mouse model. This strategy, made feasible by human iPSC technology, network analysis, and machine learning, may represent an effective path for drug discovery.

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Network-based screen in iPSC-derived cells reveals therapeutic candidate for heart valve disease - Science

Drinking certain energy drinks may cause significant damage to the heart, according to new findings published in Food and Chemical Toxicology.

Because the consumption of these beverages is not regulated and they are widely accessible over the counter to all age groups, the potential for adverse health effects of these products is a subject of concern and needed research, lead researcher Ivan Rusyn, MD, PhD, a professor at Texas A&M University in College Station, said in a prepared statement.

Rusyn et al. assessed a total of 17 popular energy drinks, studying their chemical profiles and looking for any associations with potential cardiac complications. Energy drinks sold by Adrenaline, Shoc, Bang Star, C4, CELSIUS, HEAT, EBOOST, Game Fuel, GURU, Kill Cliff, Kickstart, Monster Energy, Red Bull, Reign, Rockstar, RUNA, UPTIME, Venom Energy and Xyience Energy were all part of the teams analysis.

Overall, the authors found that stem cell-derived cardiomyocyteshuman heart cells grown in a laboratoryshowed signs of an increased beat rate after being exposed to some energy drinks. Also, theophylline, adenine and azelate were all ingredients the team associated with potentially contributing to QT prolongation in cardiomyocytes.

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Energy drinks may damage the heart, researchers warnshould the FDA get involved? - Cardiovascular Business

Apamistamab (Iomab-B) conditioning treatment with targeted radioimmunotherapy to the bone marrow resulted in high rates of successful allogeneic hematopoietic stem cell transplants in patients with active, relapsed, or refractory acute myeloid leukemia (AML), according to interim results from the phase 3 SIERRA trial, which were presented virtually at the 2021 Transplant and Cellular Therapies Meetings.1

In these patients with relapsed or refractory AML, we observed high rates of allogeneic stem cell transplant with curative intent [in] 88% of patients on the Iomab-B arm, 18% of patients who were randomized to the conventional care arm achieved complete remission and received standard of care allo-transplant, and an overall rate of 79% of allo-transplant in all enrolled patients, Boglarka Gyurkocza, MD, said in a virtual presentation.

Investigators sought to prove with this study that targeted radiation to the marrow with apamistamab, a radioactive iodine (131I)labeled anti-CD45 antibody, could enable the successful engraftment of patients despite active disease in the marrow. Safety and robust efficacy had previously been demonstrated with the agent in 271 patients treated in 9 different phase 1 and 2 clinical trials.

The SIERRA trial is looking to enroll 150 patients, and the trial is already over 75% enrolled. Recently, an independent data monitoring committee recommended that the trial continue to the planned full enrollment based on a positive pre-planned ad-hoc analysis.2

In the study, patients with active, relapsed, refractory AML are randomized 1:1 to receive either apamistamab conditioning therapy and allogeneic HCT or conventional care. In the control arm, patients who do not achieve a complete remission (CR) by day 42 are allowed to cross over to receive Iomab-B, and those who do have a CR undergo HCT or receive standard-of-care therapy of the physicians choice.

Durable CR (dCR) rate is the primary end point of the study, characterized as complete response at 6 months after initial CR, and the secondary end point is overall survival (OS) rate at 1 year.

Patients are eligible for enrollment if they have marrow blast count 5% or the presence of peripheral blasts, age 55 years, a Karnofsky score 70, and related/unrelated donor matching at human leukocyte antigen (HLA)-A, HLA-B, HLA-C, and DRB-1. Active, relapsed, or refractory AML was defined for the sake of the trial as primary induction failure after 2 cycles of therapy including chemotherapy or 2 cycles of venetoclax (Venclexta) with a hypomethylating agent or low-dose cytarabine, first early relapse after first CR of less than 6 months, relapse refractory to salvage chemotherapy regimen, or second or subsequent relapse. Secondary or treatment-related AML was also allowed.

In the SIERRA trial, patient-specific dosimetry was used to generate an individualized therapeutic dose to target marrow and spare non-hematopoietic organs. Patients in the investigational arm received a dosimetric dose of apamistamab ( 20 mCi) approximately 19 days prior to HCT followed by a therapeutic dose of apamistamab, which is individually calculated for each patient based on an upper limit of 24 Gy to the liver. After, patients remain on radiation isolation for several days before receiving fludarabine conditioning therapy (30 mg/m2/day for 3 days) and finally low-dose total body irradiation (200 cGy) prior to HCT.

Among the first 75% of enrolled patients (n = 113), patients in the apamistamab arm (n = 56) had a median age of 63 years (range, 55-77), 35% had intermediate risk and 61% had adverse risk, the median

percent of marrow blasts at baseline was 29% (range, 4%-95%), and had received a median of 3 prior treatment regimens (range, 1-7). At randomization, 56% were in primary induction failure, 16% were in first early relapse, 15% had relapsed or refractory disease, and 13% were in their second or later relapse.

In the conventional care arm, the median age was 65 years (range, 55-77), 32% had intermediate risk and 63% had adverse risk, median marrow blasts was 20% (range, 5%-97%), and had received a median of 3 prior regimens (range, 1-6). At randomization, 49% were in primary induction failure, 21% were in first early relapse, 21% had relapsed or refractory disease, and 8.8% were in their second or later relapse. Patients who crossed over to receive apamistamab (n = 30) had similar baseline characteristics.

Forty-nine patients in the apamistamab-randomized arm were able to go on and undergo allogeneic HCT compared with 10 patients in the conventional care arm. In the investigational arm, a median of 646 mCi (range, 3541027) of apamistamab was infused at a dose of 14.7 Gy (range, 4.6-32) to the marrow. The median infused CD34-positive cell count was 5.6 x 106/Kg (range, 1.8-208). Forty-five patients received peripheral blood stem cells (PBSCs), 3 received marrow grafts, 17 had related donors, and 31 had unrelated.

Individualized therapy of Iomab-B provided myeloablative doses of radiation to the marrow, Gyurkocza, a medical oncologist at Memorial Sloan Kettering Cancer Center, commented.

These patients had a median of 30 days (range, 23-60) to HCT after randomization and 14 days (range, 9-22) to neutrophil engraftment, with no graft failure reported. Patients also had 18 days (range, 4-39) until platelet engraftment.

We also observed 100% neutrophil and platelet engraftment in patients who received Iomab-B conditioning, despite a heavy leukemia burden, Gyurkocza said.

In patients in the conventional arm who went on to HCT, conditioning regimens for HCT consisted of fludarabine/melphalan in 2, fludarabine/melphalan/total body irradiation in 1, busulfan/fludarabine in 1, cyclophosphamide/fludarabine/total body irradiation in 2, and 4 had no data on conditioning regimens available. Eight of these patients had PBSCs, 2 had marrow, 3 had related donors, 6 had unrelated, and 1 was unreported.

Median days to HCT was 67 (range, 52-104) with 17 days (range, 13-83) to neutrophil engraftment and 22 days (range, 8-35) to platelet engraftment. There was 1 graft failure.

Among the patients who crossed over to receive apamistamab before HCT, the median infused dose was 592 mCi (range, 313-1013) with 15.5 Gy (range, 6.3-42) to the marrow. The median infused CD34-positive cell count was 5.1 x 106/Kg (range, 1.8-16.1). Twenty-eight patients had PBSCs, 2 had marrow, 10 had related donors, and 20 had unrelated.

Patients had a median of 62 days (range, 36-100) to HCT, 14 days (range, 10-37) to neutrophil engraftment, and 19 days (range, 1-38) to platelet engraftment. No graft failure was reported in this group.

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Apamistamab Conditioning Treatment Induces High Rates of HCT Success in AML - OncLive

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, today announced the results of a Phase 3 clinical study of omidubicel presented in an oral session at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR), or the TCT Meetings. Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant solution for patients with hematologic malignancies.

This clinical data set was from the international, multi-center, randomized Phase 3 study of omidubicel that was designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. This is the first presentation of these data in a peer-reviewed conference. The full presentation is available on the Gamida Cell website.

The results of this global Phase 3 study of omidubicel in patients with hematologic malignancies show that omidubicel resulted in faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital, all of which are meaningful results and represent potentially important advancements in care when considering the patient experience following transplant, said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. The comparator, a transplant with umbilical cord blood, has been historically shown to result in low incidence of graft versus host disease (GvHD) in relation to other graft sources, and in this study, omidubicel demonstrated a GvHD profile similar to the comparator. Moreover, previous studies have shown that engraftment with omidubicel is durable, with some patients in the Phase 1/2 study receiving their transplant more than 10 years ago. The data presented at this meeting indicate that omidubicel has the potential to be considered a new standard of care for patients who are in need of stem cell transplantation but do not have access to a matched donor.

Details of Phase 3 Efficacy and Safety Results Shared at the TCT Meetings

Patient demographics including racial and ethnic diversity and baseline characteristics were well-balanced across the two study groups. The studys intent-to-treat analysis included 125 patients aged 1365 years with a median age of 41. Diseases included acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma. Patients were enrolled at more than 30 clinical centers in the United States, Europe, Asia, and Latin America.

Gamida Cell previously reported in May 2020 that the study achieved its primary endpoint, showing that omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment, a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and a key milestone in a patients recovery from a bone marrow transplant. The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001).

All three secondary endpoints demonstrated a statistically significant improvement among patients who were randomized to omidubicel in relation to patients randomized to the comparator group (intent-to-treat). Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 35 percent for the comparator (p = 0.028). The rate of infection was significantly reduced for patients randomized to omidubicel, with the cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.027). Hospitalization in the first 100 days after transplant was also reduced in patients randomized to omidubicel, with a median number of days alive and out of hospital for patients randomized to omidubicel of 60.5 days, compared to 48.0 days for the comparator (p = 0.005). The details of these data were first reported in December 2020.

Previously unpublished data from the study relating to exploratory endpoints also support the clinical benefit demonstrated by the studys primary and secondary endpoints. There was no statistically significant difference between the two patient groups related to grade 3/4 acute GvHD (14 percent for omidubicel, 21 percent for the comparator) or all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Non-relapse mortality was shown to be 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09).

These clinical data results will form the basis of a Biologics License Application (BLA) that Gamida Cell expects to submit to the U.S. Food and Drug Administration (FDA) in the second half of 2021.

We believe that omidubicel has the potential to transform the field of hematopoietic bone marrow transplant by expanding access to this potentially curative cell therapy treatment for thousands of patients who are in need of a transplant but lack access to a matched related donor, said Julian Adams, Ph.D., chief executive officer of Gamida Cell. Sharing the results of the Phase 3 study of omidubicel with the transplant community is a major moment for Gamida Cell, and we are forever grateful to the patients who participated in this study, their caregivers, and the work of the investigators and their teams.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About Gamida Cell

Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of anticipated regulatory submissions, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the progress and expansion of Gamida Cells manufacturing capabilities and other commercialization efforts and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on February 26, 2020, its Report on Form 6-K filed with the SEC on August 12, 2020, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

1 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374.

2 Gamida Cell press release, Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies, issued May 12, 2020. Last accessed August 31, 2020.

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Gamida Cell Presents Efficacy and Safety Results of Phase 3 Study of Omidubicel in Patients with Hematologic Malignancies at the 2021 TCT Meetings of...

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced positive preliminary findings from its ongoing multicenter Phase 1 clinical trial of JSP191, a first-in-class anti-CD117 (stem cell factor receptor) monoclonal antibody, as a conditioning agent in older patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) undergoing hematopoietic (blood) cell transplantation.

Data from the first six patients who received a single dose of JSP191 prior to transplantation showed successful engraftment in all six patients. Complete donor myeloid chimerism (equal or greater than 95%) was observed in five of six evaluable patients at 28 days, and all three evaluable patients had total donor chimerism equal or greater than 95% observed at day 90. In addition, at 28 days, three of five evaluable patients showed complete eradication of measurable residual disease (MRD) as measured by next-generation sequencing. Two of the five evaluable patients showed substantial reductions in MRD. No treatment-related serious adverse events were reported.

The findings were presented by lead investigator Lori Muffly, M.D., M.S., Assistant Professor of Medicine (Blood and Bone Marrow Transplantation) at Stanford Medicine, as a late-breaking abstract at the 2021 Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).

These early clinical results are the first to demonstrate that JSP191 administered in combination with a standard non-myeloablative regimen of low-dose radiation and fludarabine is well tolerated and can clear measurable residual disease in older adults with MDS or AML undergoing hematopoietic cell transplantation a patient population with historically few options, said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. These patients could be cured by hematopoietic cell transplantation, but the standard-of-care myeloablative conditioning regimens used today are highly toxic and associated with high rates of morbidity and mortality particularly in older adults. Traditional lower intensity transplant conditioning regimens are better tolerated in older adults, but are associated with higher rates of relapse in MDS/AML patients with measurable residual disease. JSP191, a well-tolerated biologic conditioning agent that targets and depletes both normal hematopoietic stem cells and those that initiate MDS and AML, has the potential to be a curative option for these patients.

The open-label, multicenter Phase 1 study (JSP-CP-003) is evaluating the safety, tolerability and efficacy of adding JSP191 to the standard conditioning regimen of low-dose radiation and fludarabine among patients age 65 to 74 years with MDS or AML undergoing hematopoietic cell transplantation. Patients were ineligible for full myeloablative conditioning. The primary outcome measure of the study is the safety and tolerability of JSP191 as a conditioning regimen up to one year following a donor cell transplant.

We designed JSP191 to be given as outpatient conditioning and to have both the efficacy and safety profile required for use in newborn patients and older patients for successful outcomes, said Wendy Pang, M.D., Ph.D. Executive Director, Research and Translational Medicine, of Jasper Therapeutics. We are enthusiastic about the reduction of measurable residual disease seen in these patients, especially given that it is associated with improved relapse-free survival. We are excited to continue our research in MDS/AML, with plans for an expanded study. We are evaluating JSP191, the only antibody of its kind, in two ongoing clinical studies and are encouraged by the positive clinical data seen to date.

About MDS and AML

Myelodysplastic syndromes (MDS) are a group of disorders in which immature blood-forming cells in the bone marrow become abnormal and do not make new blood cells or make defective blood cells, leading to low numbers of normal blood cells, especially red blood cells.1 In about one in three patients, MDS can progress to acute myeloid leukemia (AML), a rapidly progressing cancer of the bone marrow cells.1 Both are diseases of the elderly with high mortality. Each year, about 5,000 patients with MDS and 8,000 people with AML in the G7 countries receive hematopoietic cell transplants. These transplants are curative but are underused due to the toxicity of the current high-intensity conditioning regimen, which includes the chemotherapy agents busulfan and fludarabine.

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate clinical studies in hematopoietic cell transplantation. A Phase 1/2 dose-escalation and expansion trial is evaluating JSP191 as a sole conditioning agent to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplantation for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. Data presented at the 62nd American Society of Hematology (ASH) Annual Meeting showed that a single dose of JSP191 administered prior to stem cell transplantation in a 6-month-old infant was effective in establishing sustained donor chimerism followed by development of B, T and NK immune cells. No treatment-related adverse events were reported. A Phase 1 clinical study is evaluating JSP191 in combination with another low-intensity conditioning regimen in patients with MDS or AML undergoing hematopoietic cell transplantation. For more information about the design of these two ongoing clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

1 https://www.cancer.org/cancer/myelodysplastic-syndrome/about/what-is-mds.html

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Jasper Therapeutics Announces Positive Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Patients with...

Gene therapy companies have been under pressure lately, but Orchard Therapeutics got a lift yesterday from promising early data with its mucopolysaccharidosis type I candidateOTL-203.

The company is seeking to supersede the current standard of care, enzyme-replacement therapy or bone marrow transplant. But other gene therapy contenders are not too far behind, notablyRegenxbio, which in December started a proof-of-concept study of its rival project, RGX-111.

Good IDUA

Both projects seek to deliver the -l-iduronidase (IDUA) gene, which is mutated in MPS-I, leading to a deficiency of the IDUA enzyme. This enzyme usually breaks down glycosaminoglycans (GAGs), so in MPS-I patients these build up, causing tissue and organ damage. Symptoms of MSP-I, also known as Hurler syndrome, include cognitive impairment and skeletal deformity; if left untreated, patients rarely survive beyond the age of 10.

And both OTL-203 and RGX-111 are designed as one-time therapies, whereas the current enzyme replacement, Biomarin/Sanofis Aldurazyme, is given intravenously once a week.

However, the gene therapy candidates go about restoring IDUA enzyme activity in different ways. OTL-203 uses hematopoietic stem cells taken from the patient, then genetically modified using a lentiviral vector to express the IDUA gene, before being reinfused.

RGX-111, meanwhile, uses an adeno-associated viral vector to deliver the gene directly to the brain, getting around a central problem with Aldurazyme, which cannot cross the blood-brain barrier.

Getting into the brain should not be a problem for OTL-203 either, Orchards head of medical affairs, Leslie Meltzer, told Evaluate Vantage. She explained that hematopoietic stem cells naturally cross the blood-brain barrier and, once in the CNS, differentiate into a microglial-like cell.

This claim appears to be supported by the latest data, which admittedly come in just a handful of subjects. The eight-patient phase I/II trial, presented at the World Symposium yesterday, found increases in the IDUA enzyme in patients blood and cerebrospinal fluid. There was also a decrease in GAGs in the CSF and urine.

Encouragingly, this activity appears to have translated into a clinical benefit: all eight patients showed stable cognitive scores and stable motor function versus baseline, as well as growth in the normal range for patients age.

Its a progressive disease, so youd expect these things to worsen over time, but the fact they continued to be stable is very promising, Ms Meltzer said.She admitted that the data were early, with only around a year of follow-up on most of the clinical endpoints.

Orchard plans to start a registrational study by the end of this year.Ms Meltzer would not give any details ondesign, saying this would be finalised after feedback from regulators.

Regenxbios proof-of-concept study of RGX-111 is due to complete in November, putting the project about a year behind OTL-203.

One candidate that will go no further is Sangamos SB-318. The company reported disappointing data with the in vivo zinc finger nuclease genome-editing project two years ago, and has since said it would focus on second-generation zinc finger projects.

Still, even two gene therapies might be too many for an ultra-rare disease like MPS-I, which affects just one in 100,000 people. Asked whether this market could support more than one gene therapy, Ms Meltzer said newborn screening recently implemented in countries including the UScould lead to a revision of that estimate.

But, as in other rare disorders that have attracted several gene therapy players, a battle over a limited patient pool could be shaping up.

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World Symposium Orchard leads the crop of Hurler syndrome hopefuls - Vantage

Global Hematopoietic Stem Cell Transplantation (HSCT) Report offers market size, share, overview, segmentation by types, application, countries, key manufactures, cost analysis, industrial chain, sourcing strategy, downstream buyers, marketing strategy analysis, distributors/traders, factors affecting market, forecast and other important information for key insight.

The research report on Hematopoietic Stem Cell Transplantation (HSCT) market is an in-depth analysis of pivotal drivers, challenges, and growth prospects prevailing in the business space and their impact on the expansion graph over the ensuing years.

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According to the report, Hematopoietic Stem Cell Transplantation (HSCT) market is anticipated to record a y-o-y growth rate of XX% over the analysis duration (2020-2026) and is poised to amass substantial revenues by the end of study term.

Various disruptions are being observed on account of lockdowns imposed to control COVID-19 spread, leading to uncertainties. While all industry verticals are facing revenues troubles presently, some sectors will continue to fight these challenges even as economy emerges from pandemic blowback.

As a result, all the businesses are revising their budgets to formulate new profit trajectory for the forthcoming years. Our thorough analysis of this industry space will enable you to come up with contingency plans and prepare you to manage market qualms.

The research document scrutinizes different segmentations to offer comprehensive insights about the growth opportunities in the market.

Major points summarized in Hematopoietic Stem Cell Transplantation (HSCT) market report:

Elucidating Hematopoietic Stem Cell Transplantation (HSCT) market segmentations:

Regional terrain:

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Product scope:

Application landscape:

Competitive hierarchy:

Important Highlights of the Report:

Industry Size & Forecast: Estimations on the global Hematopoietic Stem Cell Transplantation (HSCT) industry size on the basis of value and volume are provided in this part of the report

Segmental Analysis: The report has examined the high-growth segments including product type, application, and end-users

Future Prospects: Future opportunities are estimated to emerge in the industry

Geography-Wise Analysis: The authors of the report have studied the regions having growth potential to help companies plan their future investments

Study on Competitive Landscape: The industry experts have offered thorough information about the strategic tactics adopted by the industry participants.

Key questions answered in the report:

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Growth Factors of Hematopoietic Stem Cell Transplantation (HSCT) Market with Emerging Trends and Revenue Estimation By 2026 - AlgosOnline

UCART19 produced a manageable safety profile in 2 separate phase 1 studies examining heavily pretreated pediatric and adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), according to data published in The Lancet.

For the first time, these studies support the feasibility of UCART19 and other genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor (CAR) T-cells to treat this group of patients with aggressive forms of ALL.

Phase 1 trials in paediatric and adult patients with late-stage relapsed or refractory B-cell acute lymphoblastic leukaemia have shown the feasibility, safety, and activity of UCART19, an off-the-shelf CAR T-cell product, wrote the investigative team. The results of these trials represent a substantial step forward in the development of CAR T cells and could herald a new, effective, and easily accessible cell therapy for patients with B-cell acute lymphoblastic leukaemia.

The results determined that the most common adverse event between both phase 1 studies was cytokine release syndrome (CRS), observed in 19 patients (91%). Three patients (14%) experienced grade 3/4 CRS.

More, 8 patients (38%) experienced grades 1/2 neurotoxicity, 2 (10%) experienced grade 1 acute skin graft-versus-host disease, and 6 (32%) had grade 4 prolonged cytopenia.

The research team recorded 2 treatment-related deaths between the 2 studies. The first was caused by neutropenic sepsis in a patient with concurrent CRS and the other was from pulmonary hemorrhage in a patient with persistent cytopenia.

Overall, 14 of 21 patients (67%) experienced a complete response or complete response with incomplete hematological recovery at 28 days following infusion. Median duration of response was recorded at 4.1 months, with 10 of 14 adult patients (71%) progressing to subsequent allogeneic stem cell transplant. The progression-free survival rate at 6 months was 27%, with an overall survival rate of 55%.

The adverse effects observed with UCART19 to date seem similar to those reported for autologous anti-CD19 CAR T cells, wrote the investigators. Cytokine release syndrome was encountered in the majority of patients in whom UCART19 expansion was detected and appeared no more severe than with approved autologous products.

The 2 ongoing, multicenter, clinical trials (NCT02808442 and NCT02746952) enrolled 7 pediatric and 14 adult patients from June 3, 2016, through October 23, 2018, to examine the safety profile and antileukemic activity of UCART19.

The dose-escalation studies began with patients undergoing lymphodepletion with fludarabine and cyclophosphamide, with or without alemtuzumab (Lemtrada), followed by different doses of UCART19 for adults and children. The primary end point of the data was adverse events.

The small sample size for the investigation is the leading limitation for the research, but the research team also mentioned the differing trial designs, lymphodepletion regimens, and UCART19 cell doses to be among limitations of both trials.

The results [of these studies] are an encouraging step forward for the field of allogeneic CAR T cells, and UCART19 offers the opportunity to treat patients with rapidly progressive disease and where autologous CAR T-cell therapy is unavailable, wrote the investigators.

Reference:

Benjamin R, Graham C, Yallop D, et al. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5

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Manageable Safety Profile Observed in Phase 1 Studies Examining UCART19 for Pediatric and Adult Patients with B-Cell ALL - Cancer Network

You might associate platelet rich plasma therapy (PRP) with the renowned vampire facial and youd be right. However, theres so much more to this ground-breaking treatment than what youve seen in mainstream media. Founder of Anti-Aging Art Medical Aesthetics, Dr Reza Mia, shares more on this cutting-edge procedure.

PRP, or platelet rich plasma therapy, as its technically known, is a non-invasive treatment performed to accelerate healing, minimise the signs of ageing, accentuate parts of the body and to relieve pain. Incredibly versatile, this therapy is used among athletes to accelerate the healing of injuries, it is used to treat arthritis and tendonitis and is also popularly used as an anti-ageing facial treatment. PRP is considered a long-lasting solution to the bodys natural healing process, with results ranging from instant, to appearing after a couple of weeks, and lasting up to several years.

The science behind it

Plasma is the liquid part of the blood, consisting mainly of water and protein. It allows red and white blood cells, as well as platelets (a type of blood cell responsible for making blood clot, as well as for facilitating healing) to move through the bloodstream. Platelets are rich in connective tissue growth and healing factors; they initiate repair in the body and attract stem cells to injuries. This amazing healing ability is what makes them so effective in platelet rich plasma therapy.

To collect plasma, we draw blood from the body and then inserts it into a machine called a centrifuge, which spins the blood at high speeds, separating platelet-rich plasma from the rest of the blood. The red blood cells are discarded, and one is left with a concentration of platelets above normal values. This concentration can then be injected into various areas of the body to treat injuries or concerns.

Sports-related injuries

PRP has become a popular therapy among athletes. It is used to treat injured tendons, ligament sprains and tears, damaged ligaments and joints. Not only does it stimulate the healing of cartilage but it also helps reduce pain. Soft tissue injuries are most responsive to PRP treatment. Depending on the type of injury and the severity of it, some athletes who would have been side-lined for months have seen major results after around 6 weeks.

The vampire facial or facelift gained worldwide fame when reality star Kim Kardashian famously posted about it on Instagram a few years ago. Today, it is a common anti-ageing treatment. PRP is injected into the face to reduce wrinkles and rejuvenate the skin. The treatment provides a gradual increase in volume by helping to stimulate the bodys natural collagen production. Other benefits of the treatment include skin tightening, lifting and smoothing, and a more even skin tone.

Also known as a vampire breast lift, PRP for breast enhancement is a non-surgical form of breast augmentation. Unlike a traditional breast lift or augmentation which requires incisions, this treatment is performed by utilising PRP injections to create a fuller and firmer bust. A vampire breast lift wont increase your cup size or change the shape of your breast. However, it will create a fuller and firmer appearance and minimise the appearance of wrinkles and stretch marks. The results have been likened to those achieved by wearing a great quality push-up bra.

PRP therapy can be used to improve sexual function in both men and women. Platelet rich plasma sexual rejuvenation, involves injecting your own activated blood into the vagina or penis. The v-shot can enhance clitoral orgasms; increase arousal and lubrication and vaginal tightness. The benefits of the p-shot are longer, firmer and more sustainable erections and proven penis enlargement. The question on everyones lips is, of course, whether or not the latter procedure is painful. No. It is completely painless!

Did you know that PRP therapy is also successful when used to enhance hair growth? The treatment has shown to effectively treat androgenic alopecia (also known as male pattern baldness). PRP injections trigger and maintain natural hair growth by stimulating blood flow to the hair follicles. This treatment may be combined with otherhair loss procedures or medications.

Undergoing PRP therapy is simple and painless. The entire procedure, from drawing the blood to solution preparation, takes around half an hour and is performed in your aesthetic practitioners office. The time spent on the treatment performed with the PRP afterwards, is dependent on the individual treatment. Generally speaking, PRP injections are not painful,. We make use of various pain management techniques to ensure the comfort of our patients during their procedures. Depending on your treatment, you may experience some swelling and bruising for a few days, but this clears up quickly.

While the vampire facial put PRP on the general publics map, theres so much more that can be achieved by making use of this incredible treatment. Whether youre looking to treat an injury, minimise your wrinkles, rejuvenate your sex life or increase your hair growth, Plasma rich platelet therapy is a safe and effective treatment option to consider.

Visit http://www.drreza.co.za or or follow Dr Mia on Instagram @drreza.sa and @antiagingart

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Platelet Rich Plasma Therapy: The answer to a rejuvenated body, skin, hair and sex life! - Longevity LIVE - Longevity LIVE

SAN DIEGO, Feb. 11, 2021 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company developing long-acting therapeutics designed to transform the standard of care for patients facing serious fungal or viral infections, today announced an oral presentation and four poster presentations at the 21st International Immunocompromised Host Society (ICHS) Symposium on Infections in the Immunocompromised Host, taking place virtually Feb. 17-19, 2021.

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Cidara Therapeutics to Present New Data for Rezafungin and Influenza AVCs at the 21st ICHS Symposium

NEW YORK, Feb. 11, 2021 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, will announce its fourth quarter and full year 2020 financial results prior to market open on Thursday, February 25, 2021. The announcement will be followed by a conference call and audio webcast hosted by Intercept management at 8:30 a.m. ET to discuss the results.

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Intercept to Announce Fourth Quarter and Full Year 2020 Financial Results on February 25, 2021

BOSTON, Mass., Feb. 11, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today announced that it will report its financial results for the fourth quarter and full year ended December 31, 2020, and provide an update on recent business highlights, on March 4, 2021.

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X4 Pharmaceuticals to Announce Fourth Quarter and Full Year 2020 Financial Results and Host a Conference Call and Webcast on March 4, 2021

PHILADELPHIA and OXFORDSHIRE, United Kingdom, Feb. 11, 2021 (GLOBE NEWSWIRE) -- Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, will report financial results and provide business updates for the fourth quarter and year ended December 31, 2020, before the US markets open on Thursday, February 25, 2021. Following the announcement, the Company will host a live teleconference and webcast at 8:00 a.m. EST (1:00 p.m. GMT) that same day (details below).

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Adaptimmune to Report Fourth Quarter/ Full Year 2020 Financial Results and Business Updates on Thursday, February 25, 2021

BRIDGEWATER, N.J., Feb. 11, 2021 (GLOBE NEWSWIRE) -- VYNE Therapeutics Inc. (Nasdaq: VYNE) (“VYNE” or the “Company”) today announced that its Board of Directors has approved a reverse stock split of the Company’s common stock at a ratio of 1-for-4. The reverse stock split was previously approved by stockholders at the Annual Meeting of Stockholders held on August 3, 2020.

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VYNE Therapeutics Announces Reverse Stock Split

Brings Track Record of Success to Expanded Role Brings Track Record of Success to Expanded Role

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BioDelivery Sciences CFO Terry Coelho Promoted to Executive Vice President