Expression Therapeutics LLC

ATLANTA, GA, USA I December 11, 2024 I Expression Therapeutics, Inc., has presented the results of a Phase 1 clinical trial for its novel lentiviral vector-based gene therapy — ET3 — for hemophilia A at the Annual Meeting of the American Society of Hematology (ASH) in San Diego, California. The results of the study, “Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A” (Srivastava et al ), were published by the New England Journal of Medicine , timed for release with the ASH presentation. Hemophilia A results from a deficiency in blood coagulation Factor VIII (FVIII). It is the most common severe congenital bleeding disorder, with an incidence of approximately 1 in 5,000 live male births. Without treatment, severe hemophilia A is crippling and fatal by late adolescence to early adulthood. The average annual cost per patient of managing hemophilia A in the United States ranges from over $200,000 to $850,000, making it one of the most expensive treatments over a lifetime for any genetic disorder. As a result, a curative gene therapy for hemophilia A can lead to significant long-term cost savings for healthcare systems compared to current prophylaxis treatments, in addition to improved quality of life for patients. New gene therapy offers hope of a permanent cure for hemophilia A patients. The clinical trial, conducted at the Christian Medical College, Vellore, India, recruited five participants 22 to 41 years of age with severe hemophilia A. Autologous hematopoietic stem cells (HSCs) were transduced with CD68-ET3-LV — a lentiviral vector including a novel FVIII transgene (ET3) with a myeloid-directed CD68 promoter. Before gene therapy, all participants reported annualized bleeding rates of at least 20 events. No spontaneous bleeding events occurred in any participant on study. These results were consistent with measured FVIII levels, which in all five participants increased from undetectable to levels predicted to produce therapeutic efficacy. The median follow-up was 14 months (range, 9 to 27 months). Except for neutropenia and thrombocytopenia (which were expected), no adverse events greater than grade 2 occurred in any participant. Inhibitors to FVIII did not develop in any participant after drug-product infusion. Integration-site analysis performed at 4 to 22 months after drug-product infusion showed no safety concerns. The results of this Phase 1 clinical trial validate a new approach to gene therapy for hemophilia A that can provide lifelong durability of expression. The candidate CD68-ET3-LV-CD34+ gene therapy product overcomes the limitations of the AAV gene therapy approaches to hemophilia A, including lack of durability of expression, ineligibility of patients on the basis of age, and preexisting anti-AAV antibody related conditions. Expression Therapeutics lentiviral gene therapy is currently the only approach that offers the possibility of a permanent cure for hemophilia A and provides an opportunity to reach both pediatric and adult populations. “This Phase I clinical trial produced no unexpected safety issues and remarkable clinical efficacy. Lentiviral vector hematopoietic stem cell therapy offers the hope of permanent cure of hemophilia A patients that ultimately may be extended to the pediatric population,” said Pete Lollar, MD, Chairman of the Board of Expression Therapeutics and Hemophilia of Georgia Professor at Emory University School of Medicine. “We are grateful to Dr. Alok Srivastava, Christian Medical College/Centre for Stem Cell Research/inSTEM, and, most importantly, the participants and their families for collaborating on this unique therapeutic development program with curative potential. We have demonstrated safety and efficacy in our Phase 1 trial and look forward to continued development of a lentiviral mediated hematopoietic stem cell gene therapy for the benefit of hemophilia A patients worldwide,” said Mohan Rao, Ph.D., the Chief Executive Officer of Expression Therapeutics. About Expression Therapeutics, Inc.: Expression Therapeutics, Inc., headquartered in Atlanta, Georgia, with a state-of-the-art GMP manufacturing facility in Cincinnati, Ohio, is a clinical-stage gene and cell therapy company advancing curative therapies for hemophilia A and developing innovative oncology solutions targeting neuroendocrine tumors and neuroblastoma. SOURCE: Expression Therapeutics

Quality System Expert Joins Hemogenyx Pharmaceuticals as Director of Quality LONDON, UK / ACCESSWIRE / August 22, 2022 / Hemogenyx Pharmaceuticals plc (LSE:HEMO), the biopharmaceutical group developing new therapies and treatments for blood diseases, announces the appointment of Mr Stuart Tinch as Director of Quality. Stuart brings over seven years of Good Manufacturing Practice ("GMP") expertise to Hemogenyx Pharmaceuticals. He will be instrumental in creating a culture and system of quality to ensure that the Company's therapies, such as HEMO-CAR-T, which is currently being prepared for clinical trials, are held to the standards of current GMP regulations. Stuart's appointment takes the Company forward toward its first clinical clinical trials. Stuart joins Hemogenyx Pharmaceuticals from the manufacturing division of Expression Therapeutics INC ("Expression") where he was the Senior Manager of Viral Vector Manufacturing. There he worked to set up Expression's GMP facility and to assist in the design and implementation of its quality systems. Stuart previously worked at the Vector Production Facility ("VPF") at Cincinnati Children's Hospital Medical Center where he progressed to be Senior GxP Specialist and Supervisor of GMP manufacturing of viral vectors. At the VPF he was involved in and supervised over 70 GMP manufacturing cycles both for academics and pharmaceutical companies. Stuart received his BS in Biochemistry from Miami University. Dr Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented: "We are excited to welcome Stuart as Director of Quality. His rich experience and deep insight into the cutting-edge GMP manufacturing of viral vectors and cell therapies as well as his expertise in establishing and maintaining quality systems will undoubtedly help to accelerate Hemogenyx Pharmaceuticals' product candidates. Stuart will help us to establish manufacturing of our lead product candidate, HEMO-CAR-T, as well as potentially other cell therapies, according to the highest standards of quality to facilitate their transition into the clinic." Mr Stuart Tinch, Director of Quality of Hemogenyx Pharmaceuticals, commented: "I have been privileged to join Hemogenyx Pharmaceuticals to establish its quality system. It is great to be able to facilitate the transition of brilliant science into the clinic for the benefit of patients." Enquiries: Hemogenyx Pharmaceuticals plc Dr Vladislav Sandler, Chief Executive Officer & Co-Founder headquarters@hemogenyx.com Peter Redmond, Director peter.redmond@hemogenyx.com SP Angel Corporate Finance LLP Tel: +44 (0)20 3470 0470 Matthew Johnson, Vadim Alexandre, Adam Cowl Peterhouse Capital Limited Tel: +44 (0)20 7469 0930 Lucy Williams, Duncan Vasey, Charles Goodfellow About Hemogenyx Pharmaceuticals plc Hemogenyx Pharmaceuticals is a publicly traded company (LSE: HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility. The Company is a pre-clinical stage biopharmaceutical group developing new medicines and treatments to treat blood and autoimmune disease and to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as platform technologies that it uses as engines for novel product development. For more than 50 years, bone marrow transplantation has been used to save the lives of patients suffering from blood diseases. The risks of toxicity and death that are associated with bone marrow transplantation, however, have meant that the procedure is restricted to use only as a last resort. The Company's technology has the potential to enable many more patients suffering from devastating blood diseases such as leukemia and lymphoma, as well as severe autoimmune diseases such as multiple sclerosis, aplastic anemia and systemic lupus erythematosus (Lupus), to benefit from bone marrow transplantation. This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit . SOURCE: Hemogenyx Pharmaceuticals PLC View source version on accesswire.com:

Los Angeles, USA, May 05, 2021 (GLOBE NEWSWIRE) -- T-cell Leukemia Pipeline: Comprehensive Analysis of 15+ Key Players and 15+ Key Therapies Out of all T-cell Leukemia pipeline therapies, HBI-8000 is set to capture the maximum patient pool owing to its immunomodulatory function. DelveInsight’s ‘T-cell Leukemia Pipeline Insights’ report provides a holistic view of the pipeline therapies that are under development in pre-clinical as well as clinical stages of development, and growth prospects across the T-cell Leukemia domain. Some of the key highlights of the T-cell Leukemia Pipeline report: Got queries? Want to know more? Request for Sample @ T-cell Leukemia Emerging Therapies and Forecast The report lays down a complete coverage of the therapeutics by development stage, product type, and route of administration, molecule type, and MOA type for T-cell Leukemia across the complete product development cycle, including all clinical and non-clinical stages. The T-cell Leukemia pipeline report puts on the table business opportunities, threats, prospective collaborations, strong competitors, growth strategies, and failed as well as discontinued drugs. T-cell Leukemia Pipeline Drugs Request for Sample to know more @ T-cell Leukemia Pipeline Analysis, Key Companies and Futuristic Trends T-cell Leukemia Therapeutic Assessment The T-cell Leukemia Pipeline report proffers exhaustive insights into active pipeline assets segmented by Stage, Product Type, Route of Administration, Molecule Type, Target, and Indications of various drugs. By Product Type By Stage By Molecule Type By Route of Administration By Mechanism of Action By Targets Get in touch with our Business executive for Rich and Deep Market Assessment and Consulting Solutions Scope of the Report Coverage: GlobalKey Players: Daiichi Sankyo; Bioniz; Otsuka Pharmaceutical; Seagen /Takeda; HUYA Bioscience; iCell Gene Therapeutics; Gracell Biotechnology; Ascentage Pharma Group; Celgene Corporation; Shorla Pharma; AVM Biotechnology; Expression Therapeutics; miRagen Therapeutics, and others.Key T-cell Leukemia Pipeline Therapies: Valemetostat; BNZ-1; ASTX 660; Brentuximab vedotin; HBI-8000; CD4CAR; CD7 UCAR-T cells; APG-115; Romidepsin; SH-111; Cobomarsen; AVM-0703, and others. Reach out @ T-cell Leukemia Pipeline: Novel therapies and emerging technologies Table of Contents Visit to know more of what’s covered @ T-cell Leukemia Emerging Therapies Browse through Related Reports CAR T-Cell Therapy for Acute Lymphoblastic LeukemiaDelveInsight’s “CAR T-Cell Therapy for Acute Lymphoblastic Leukemia (ALL)- Market Insights and Market Forecast- 2030” report. CAR T-Cell Therapy for Acute Lymphoblastic Leukemia Pipeline DelveInsight’s, “CAR T-Cell Therapy for Acute Lymphoblastic Leukemia - Pipeline Insight, 2021,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in CAR T-Cell Therapy for Acute Lymphoblastic Leukemia pipeline landscape. Adult T Cell Leukemia Lymphoma MarketDelveInsight's "Adult T-Cell Leukemia-Lymphoma - Market Insights, Epidemiology, and Market Forecast-2030" report. Acute Myeloid Leukemia MarketDelveInsight's "Acute Myeloid Leukemia (AML) Market Insights, Epidemiology, and Market Forecast-2030" report. B-Cell Chronic Lymphocytic Leukemia MarketDelveInsight's "B-Cell Chronic Lymphocytic Leukemia - Market Insights, Epidemiology, and Market Forecast-2030" report. Hairy Cell Leukemia Market Insight Epidemiology and Market ForecastDelveInsight's 'Hairy Cell Leukemia (HCL)- Market Insights, Epidemiology and Market Forecast-2028' report. Bowens Disease MarketDelveInsight's "Bowen's Disease - Market Insights, Epidemiology, and Market Forecast-2030" report. Buergers Disease MarketDelveInsight's "Buerger’s Disease - Market Insights, Epidemiology, and Market Forecast-2030" report. Brucellosis MarketDelveInsight's "Brucellosis - Market Insights, Epidemiology, and Market Forecast-2030" report. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end-to-end comprehensive solutions to improve their performance.  Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. For more insights, visit Pharma, Healthcare, and Biotech News