Siren Biotechnology Inc. has unveiled its lead asset, SRN-101, for the treatment of high-grade gliomas. The FDA has granted orphan drug and rare pediatric disease designations to SRN-101 for high-grade gliomas and pediatric-type diffuse high-grade gliomas, respectively.
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.
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.
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).
Some rare skin diseases not only reduce the quality of life of patients, but also can be devastating conditions, leading to amputations or death. At the 31st annual congress of the European Society of Gene and Cell Therapy (ESGCT), held last week in Rome, different laboratories showcased their approaches to editing mutations related to this group of diseases.
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