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…

9 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…

9 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
Show previous Posts