EVRY, France — Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular dystrophy in patients treated at therapeutic doses in the first phase of an international multicenter clinical trial...
treatment News
GNT0004 product confirms its clinical efficacy with stabilization of motor functions in patients treated at the effective dose for up to 2 years. Sustained significant reduction in levels of creatine phosphokinase (CPK), a biomarker of muscle damage, with an average decrease of over 75% at 18 months in the 3 patients treated...
PARIS, France — Genethon, a worldwide pioneer and leader in research and development in gene therapy for rare genetic diseases, has received approvals from regulatory authorities, MHRA and EMA*, to begin pivotal Phase 3 clinical trials in France and the UK of its gene therapy, GNT0004, for Duchenne muscular dystrophy...
PARIS, France – Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today its research and clinical trial data will be featured in eight oral presentations and 13 posters at the American Society of Gene & Cell Therapy Annual Meeting May 11-15...
VANCOUVER, BC – Genetrack Biolabs, working in partnership with Canada’s Michael Smith Genome Sciences Centre as part of a landmark research–industry collaboration, has announced the development of a revolutionary cancer testing platform called GenTraceDx™. Instead of providing a single snapshot of someone’s cancer, this innovative technology delivers ongoing genomic updates...
SARASOTA, Fla. and NOVATO, Calif. – GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced that they have received clearance from Health Canada to begin enrolling the Phase 1/2 study of GTX-102 in...
Collaboration supports manufacture of the OPTF01 product to Drive Next-Generation Chimeric Antigen Receptor T Cell (CAR-T) Development INDIANAPOLIS and EHIME, Japan — Genezen, a leading viral vector Contract Development and Manufacturing Organization (CDMO), and Optieum Biotechnologies, Inc. (Optieum), a preclinical stage company dedicated to the discovery and development of innovative CAR-T cell therapies,...
Lille, France, Cambridge, Massachusetts and Zurich, Switzerland – GENFIT (Euronext: GNFT), a biopharmaceutical company dedicated to improving the lives of patients with rare and life-threatening liver diseases, today reports encouraging preliminary Phase 1b data from its CCA clinical trial evaluating GNS561 in combination. Clinical trial context and objective CCA is a rare...
SHANGHAI, China — GenFleet Therapeutics, a clinical-stage biotechnology company developing cutting-edge therapies in oncology and immunology, announced that U.S. Food and Drug Administration (FDA) has recently granted another two designations for SLS009 (GFH009). GFH009 (highly selective CDK9 inhibitor) so far has received both fast track and orphan drug designations from the...
Results from the EPCORE® NHL-2 trial show investigational treatment with epcoritamab in combination with rituximab, ifosfamide, carboplatin, and etoposide (R-ICE) led to an overall response rate (ORR) of 87 percent and a complete response (CR) rate of 65 percent in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL)...