The first gene therapy to treat severe hemophilia A was among the drugs recommended for European approval by regulators from the EMA’s CHMP at its monthly meeting. Manufactured by Biomarin Pharmaceutical Inc., Roctavian (valoctocogene roxaparvovec) was recommended for conditional marketing authorization in the EU for severe hemophilia A in adults who do not have factor VIII inhibitors and no antibodies to adeno-associated virus serotype 5. Read More
Sarepta Therapeutics Inc. aims to resolve “very quickly” the clinical hold placed by the U.S. FDA on SRP-5051 (vesleteplirsen) for Duchenne muscular dystrophy (DMD), said Louise Rodino-Klapac, chief scientific officer. Read More
Tessa Therapeutics Ltd. has a closed a $126 million series A financing round to advance its ongoing clinical development, which includes the initiation of a pivotal trial of autologous CD30-CAR-T therapy (TT-11) and advancement of allogeneic CD30.CAR EBVST therapy (TT-11X) programs. Read More
Sanofi SA and GSK plc said new phase III data show their adjuvanted bivalent D614 and beta (B.1.351) COVID-19 vaccine candidate responded well against omicron despite it being designed to fight the original SARS-CoV-2 virus and the beta variant. Read More
The only way to avoid food allergies right now is to stay away from whatever triggers them and, at best, grab some rescue epinephrine in case there’s trouble. Peanut allergies have an FDA-approved treatment, but the remainder of the troublemakers don’t have any therapies. Hoping to change that, Alladapt Immunotherapeutics Inc. closed a $119 million financing to develop its food allergy treatments. The Menlo Park, Calif.-based company’s lead candidate is ADP-101, an oral immunotherapy that is in a phase I/II trial and an open-label extension study of children and adults. Read More
Although there has been huge progress in treatment of cystic fibrosis over the last decade, with Vertex Pharmaceuticals Inc. becoming the first to address the underlying cause of the disease with its Kalydeco (ivacaftor), approved in 2012, there are still many patients who aren’t eligible for treatment. Read More
Nonprofit deals with biopharma companies in 2022 indicate that 92% of the disclosed funds are going toward infectious disease therapies, with COVID-19 accounting for 79% of the total. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Albireo, Astrazeneca, Cognition, Dermbiont, Diffusion, Disc, Gilead, Novaremed, Scynexis, Timber, Uniqure, Vistagen, Vivoryon, Xenon. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Biogen, Can-Fite, Dr. Reddy’s, Eton, Galapagos, Greenfire, Mirum, Molecure, Redhill, Repertoire, Saniona, Siga, Soligenix, Turning Point, Versanis, Voltron, Xentria. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Abbvie, Alterity, Amryt, Astrazeneca, Biontech, CNS, Fusion, Menarini, Merck & Co., Novartis, Novavax, Radius, Realta, Rockwell, Valneva. Read More
