Atidarsagene autotemcel

Atidarsagene autotemcel (also known as ATI-50002) is a gene therapy product developed by Audentes Therapeutics, a subsidiary of Astellas Pharma. It is designed for the treatment of X-linked Dystonia-Parkinsonism (XDP), a rare and severe neurodegenerative disorder.

Overview

Atidarsagene autotemcel is an investigational adeno-associated virus (AAV) vector-based gene therapy. It works by delivering a healthy copy of the TAF1 gene to cells in the body. The TAF1 gene is responsible for producing a protein that is essential for normal cell function. Mutations in this gene are the cause of X-linked Dystonia-Parkinsonism.

Development

The development of Atidarsagene autotemcel began with the identification of the TAF1 gene mutation in patients with XDP. This discovery led to the development of a gene therapy approach to treat the disorder. The therapy involves the use of an AAV vector to deliver a healthy copy of the TAF1 gene to cells in the body. The goal is to restore normal protein production and improve symptoms of the disorder.

Clinical Trials

Atidarsagene autotemcel is currently in clinical trials to evaluate its safety and efficacy in patients with XDP. The trials are being conducted by Audentes Therapeutics and are in the early stages of development.

Potential Impact

If successful, Atidarsagene autotemcel could provide a new treatment option for patients with XDP, a disorder for which there are currently no effective treatments. The therapy could potentially slow or halt the progression of the disease, improving quality of life for patients and their families.

See Also

• Gene therapy
• X-linked Dystonia-Parkinsonism
• Audentes Therapeutics
• Astellas Pharma