FDA OKs Trial of NGN-101 Gene Therapy in CLN5 Patients

FDA OKs Trial of NGN-101 Gene Therapy in CLN5 Patients

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FDA OKs Trial of NGN-101 Gene Therapy in CLN5 Patients

The U.S. Food and Drug Administration (FDA) has cleared Neurogene’s request to conduct a Phase 1/2 clinical trial to evaluate the safety, tolerability, and efficacy of NGN-101, an investigational gene therapy to treat CLN5, a form of late-infantile Batten disease. The therapy uses a harmless adeno-associated virus (AAV) to deliver a functional copy of the CLN5 gene, which is faulty in people with CLN5 Batten. Although the exact function of CLN5 is unclear, mutations in…

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