$160 million ‘moonshot’ backs Boston effort to revolutionize rare disease treatment
A new $160 million non-profit initiative in Boston aims to accelerate the development of genetic therapies for rare and ultra-rare diseases.
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A new $160 million non-profit initiative in Boston aims to accelerate the development of genetic therapies for rare and ultra-rare diseases.
MeiraGTx has secured a multi-million dollar funding package led by Oberland Capital to support the commercialization of two gene therapies.
New research links exercise to muscle rejuvenation—and a surprising potential cancer connection
Leadership transitions at the FDA coincide with a significant regulatory rejection for Unicycive Therapeutics.
A 23-year-old New Orleans man has become the first person in Louisiana to be functionally cured of sickle cell disease following a historic gene therapy.
Researchers have launched the first human clinical trial for an experimental gene therapy designed to reverse the biological aging of cells.
The FDA has reversed its position on Regenxbio's gene therapy for MPS II, allowing a new path for accelerated approval.
Gene therapy pioneer Sangamo Therapeutics files for bankruptcy, with Lilly and Astellas racing to acquire its assets