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Lysosomal Disorders

Rajni Agarwal-Hashmi, MD, on Safety Advantages of Gene Therapy in Fanconi Anemia Over SOC

The professor of pediatrics at Stanford Cancer Institute discussed recent data from clinical trials of the lentiviral gene therapy RP-L102.

Around the Helix: Cell and Gene Therapy Company Updates – January 25, 2023

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Rett Syndrome Gene Therapy Cleared for Clinical Trial

NGN-401 is Neurogene’s second investigational gene therapy to enter clinical trials.