- Andelyn Biosciences has been selected by the Drake Rayden Foundation to manufacture clinical-grade AAV using its AAV Curator® Platform for a gene therapy targeting Nonketotic Hyperglycinemia (NKH).
- The collaboration aims to support the development of a treatment for NKH, a rare genetic disorder caused by mutations in the GLDC gene.
Andelyn Biosciences, Inc. has been selected by the Drake Rayden Foundation to manufacture clinical-grade adeno-associated virus (AAV) using its AAV Curator® Platform for the treatment of Nonketotic Hyperglycinemia (NKH), a rare inherited metabolic disorder caused by mutations in the GLDC gene.
The program focuses on developing a gene therapy for NKH, a condition that disrupts the glycine cleavage system and leads to toxic accumulation of glycine, particularly in the brain. The disorder is associated with severe neurological symptoms, including seizures, lethargy, breathing complications, and profound intellectual disability. According to the company, approximately 85% of diagnosed children have a severe form of NKH, with many surviving only days or weeks after birth.
Under the agreement, Andelyn will work with the Drake Rayden Foundation and scientists from Dr. Steven Gray’s laboratory at the University of Texas Southwestern to establish the manufacturing process using its AAV Curator® Platform. The platform is designed to support reliable speed and quality in AAV manufacturing. The work will involve production of clinical-grade material to support advancement of the gene therapy program.
“We are elated that our established and rapidly growing work on clinical and commercial gene therapy programs for the biotech industry allows us to also support family foundations seeking treatments for loved ones-often children with rare diseases.”
Matt Niloff, Chief Commercial Officer at Andelyn Biosciences
Andelyn operates as a cell and gene therapy CDMO, providing process development and clinical and commercial contract manufacturing services. The company stated that the NKH collaboration reflects its approach of supporting biotech companies, non-profit organizations, and family foundations working to advance gene therapies for patients with rare genetic diseases.
