gene

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

July 11, 2025 16:30 ET  | Source: Ultragenyx Pharmaceutical Inc. Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls…

10 months ago

Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS

NEW YORK, June 30, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. (NASDAQ: KLTO) today announced that it is moving forward with…

10 months ago

Klotho Neurosciences, Inc. and the Okinawa Research Center for Longevity Science, Leading Experts on the Okinawa “Blue Zone”, Announce a Plan to Study Tissue Levels of the Human Klotho Gene and Protein in the World’s Longest-Lived Population

NEW YORK, June 24, 2025 /PRNewswire/ -- Klotho Neuroscience, Inc. (NASDAQ: KLTO), a US-based biogenetics company developing cell and gene-…

10 months ago

[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys gene therapy for Duchenne muscular dystrophy in non-ambulatory patients

After a thorough clinical review, the benefit-risk for the use of Elevidys in non-ambulatory patients with Duchenne has been re-assessed,…

10 months ago
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