WebFeb 24, 2024 · Graphite Bio is discontinuing development of its experiment sickle cell disease gene-edited stem cell therapy; Intellia Therapeutics said its partner Novartis … WebApr 11, 2024 · Fulcrum Therapeutics’ sickle cell disease therapy has been placed on a clinical hold by the FDA, capping off a tough week for drug development for the condition that saw three separate treatments discontinued. ... Graphite Bio ended development of nulabeglogene autogedtemcel (nula-cel) after the first patient dosed experienced an …
Biotech, Medical Device & Pharma Layoffs BioSpace
WebNula-cel is being studied in the CEDAR trial, a Phase 1/2 multicenter, open-label clinical trial designed to evaluate the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics in patients with severe SCD. Learn more about the Phase 1/2 CEDAR clinical trial ... WebFeb 23, 2024 · Astellas presents update on recently cleared Pompe gene therapy trial. Published: Feb 23, 2024 . By Heather McKenzie. BioSpace. After overcoming a clinical hold, Astellas presented preliminary safety and efficacy data from the Phase I/II FORTIS trial of AT845 in late-onset Pompe disease (LOPD) at the 19th Annual … tax tips instacart
FDA Places Hold on Gilead HIV Trials Over Vial Quality Concerns
WebDec 14, 2024 · The US Food and Drug Administration (FDA) has granted Investigational New Drug (IND) permission enabling Graphite Bio to test the investigational, potentially revolutionary gene editing therapy GPH101 developed under the supervision of Matthew Porteus, MD, PhD, in a clinical trial for people with sickle cell disease (SCD). WebDec 22, 2024 · Published: Dec 22, 2024. By Mark Terry. BioSpace. The U.S. Food and Drug Administration (FDA) placed a clinical hold on Gilead Sciences ’ use of injectable lenacapavir in borosilicate vials in all ongoing clinical studies for HIV and HIV pre-exposure prophylaxis (PrEP). There are emerging worries about the compatibility of vials … WebOct 12, 2024 · Graphite Bio is evaluating GPH101 in the CEDAR trial, an open-label, multi-center Phase 1/2 clinical trial designed to assess the safety, engraftment success, gene correction rates, total ... taxtips ontario 2022