Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) set up its second overseas regulatory office in Washington, four months after the drug and med-tech regulator opened its first Asia base in Bangkok, Thailand, in July 2024.
Gene editing strategies, from epigenetic engineering to cell reprogramming and genetic vaccines, are accelerating the development of new therapies that awaken the immune system to treat cancer, as presented last month in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Some of these advances are taking advantage of the conditions of the tumor microenvironment, where cancer cells coexist with immune cells, microorganisms and blood vessels.
The PLN-R14del mutation is often tied to dilated or arrhythmogenic cardiomyopathy, which can progress to end-stage heart failure. Cardiac phospholamban (PLN) plays a crucial role in calcium modulation in cardiomyocytes, which is disrupted by the PLN-R14del mutation.
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York.
The prospect of a gene therapy for Alzheimer’s disease has kept Wall Street steadily interested in Lexeo Therapeutics Inc., and another increment of intrigue was added when the New York-based firm offered positive interim results from the 52-week, 15-subject phase I/II study of LX-1001 for the treatment of the condition when APOE4-associated.
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York. The agency is trying to set up cell and gene companies for success and that’s a very different agency than what it was years ago, said Paul Bresge, CEO of Ray Therapeutics Inc.
Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.
Although more and more gene therapies are getting the FDA stamp of approval, concerns persist about their potential long-term risks. U.S. lawmakers have proposed several pieces of legislation over the past few years to address some of the uncertainties. Now the Congressional Research Service (CRS) is suggesting other requirements Congress may want to consider to improve the regulatory landscape for gene therapies, especially those intended to treat blood disorders.
The Muscular Dystrophy Association (MDA)’s Kickstart program has announced receipt of both U.S. orphan drug and rare pediatric disease designations in support of work for congenital myasthenic syndrome caused by choline acetyltransferase (CHAT) gene deficiency. The FDA awarded the orphan drug designation to AVCHAT-01X (AAV serotype 9 human choline acetyltransferase).
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