Neurogene Announces EMA Grants Orphan Drug Designation to CLN5 Batten Disease Gene Therapy
Retrieved on:
Tuesday, June 29, 2021
Biotechnology, FDA, Health, Genetics, Pharmaceutical, Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Gene therapy, Emerging technologies, Medical genetics, Adeno-associated virus, Batten disease, Therapy, Gene therapy of the human retina, Orphan Drug Designation, CLN5, a subtype of Batten disease, Genetic Testing, Neurogene Inc., ORPHAN DRUG DESIGNATION, CLN5, A SUBTYPE OF BATTEN DISEASE, GENETIC TESTING, NEUROGENE INC.
Neurogene is collaborating with Batten disease experts, regulatory authorities, and caregivers to provide a safe and effective gene therapy for this disease as quickly as possible.
Key Points:
- Neurogene is collaborating with Batten disease experts, regulatory authorities, and caregivers to provide a safe and effective gene therapy for this disease as quickly as possible.
- Applications for orphan designation are examined by the EMAs Committee for Orphan Medicinal Products (COMP), using the Committees network of experts.
- Our lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene.
- Neurogene is also developing novel gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully address.