Gene Therapy Advances: Parkinson’s, Rare Diseases, FDA Approvals & More

Neurocrine returns two CNS gene therapy programs to Voyager, focusing Voyager’s pipeline on Parkinson’s and Gaucher diseases. Aspen Neuroscience’s Phase 1/2a Parkinson’s cell therapy shows promising patient improvements. Capsida and MavriX advance FDA-cleared gene therapies for epileptic encephalopathy and Angelman syndrome, respectively. MeiraGTx gains expedited review for Parkinson’s gene therapy. Sarepta’s Duchenne muscular dystrophy therapy wins approval in Japan. CRISPR and Vertex expand sickle cell treatment trials. Regenxbio’s MPS II therapy enters Priority Review. Genascence and Ensoma progress with therapies for osteoarthritis and X-CGD.

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