Gene therapy faces complexities in delivering treatments due to persistent safety concerns and daunting immune responses, but Next Generation Gene Therapeutics Inc. has found a way around this issue using dual-functional vectors to simultaneously remove harmful, mutated genes and replace them with normal, healthy genes to restore cellular function.
Belief Biomed Inc.’s gene therapy drug BBM-D101 has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD).
Retinitis pigmentosa (RP) is the most common hereditary degenerative eye disease that leads to progressive vision loss, primarily caused by retinal degeneration.
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.
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.
Ring Therapeutics Inc. has joined forces with Singapore’s Agency for Science, Technology, and Research and with the Singapore Eye Research Institute to advance the first new class of viral vectors in more than 50 years, Ring CEO Tuyen Ong told BioWorld.
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.
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