A new West Coast biotech has emerged into the busy in vivo cell therapy space, this time with the backing of Nobel Prize-winning CRISPR pioneer Jennifer Doudna, Ph.D. Azalea Therapeutics has bloomed ...
The FDA is said to be making a U-turn on uniQure’s one-time gene therapy candidate for Huntington’s disease despite the ...
Pivotal results from uniQure’s gene therapy for Huntington’s disease have brought new light to patients who have known only ...
Researchers at Mount Sinai have identified the Cyclin A2 gene, which reactivates the human heart's natural healing ability.
Medication has long been the cornerstone of treatment for people with epilepsy, but it doesn't stop seizures for everyone and ...
The agency is establishing a network of care centers to expand access to regenerative medicine beyond regions served by major ...
A top US regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare ...
Shortly after taking reins at BioMarin, CEO Alexander Hardy laid out three paths the company could take for its struggling ...
Mother Daphne Graskewicz-Prado of Southern Illinois told Newsweek: “I wasn’t going to accept the terminal diagnosis.” ...
Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...
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