Andelyn Biosciences to Manufacture AAV Gene Therapy for Rare NKH Disorder

Andelyn Biosciences, Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been selected by Drake Rayden Foundation to apply the AAV Curator® Platform to manufacture clinical grade AAV for the Treatment of Nonketotic Hyperglycinemia (NKH), a rare, inherited metabolic genetic disorder caused by a mutation in the GLDC gene.

NKH is a debilitating disease that causes a disruption of the glycine cleavage system (GCS), leading to the inability of the body to break down the amino acid glycine. Glycine builds up to toxic levels, particularly in the brain, which leads to severe neurological problems including seizures, lethargy, poor muscle tone, breathing issues, and profound intellectual disability. Children with the severe form of NKH, comprising close to 85% of diagnosed, often live only days or weeks after birth. Those that survive live with severe seizure disorders and a very poor prognosis.

Andelyn will work closely with the Drake Rayden Foundation and scientists from Dr. Steven Gray's laboratory at the University of Texas Southwestern (UTSW) to establish the manufacturing process in Andelyn's AAV Curator® Platform, a methodology that offers reliable speed and quality. This initiative reflects Andelyn's commitment to accelerate gene therapies to patients universally by working with not only biotech and pharma companies, but also individual families and non-profit organizations.

The Drake Rayden Foundation was formed in 2017 with the conviction to push research forward towards a treatment for NKH by raising funds and establishing key partnerships with research institutes and manufacturing organizations. The foundation founder stated, "We have clung to the verse in Galatians that brought such encouragement when we faced the exhaustion that comes from trying to create treatment in a rare disease. Chapter 6 verse 9 reminds us, 'Let us not become weary in doing good, for at the proper time we will reap a harvest if we do not give up.' This gene therapy is bringing such a season of hope for the future."

The Chief Commercial Officer at Andelyn Biosciences said, "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. The power of gene therapy continues to offer the much-needed hope for the many patients with genetic diseases, and we are honored to play a pivotal role in delivering these therapies."

Andelyn Biosciences has more than 20 years of experience and has produced cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of its development and manufacturing facilities in Columbus, Ohio, Andelyn supports clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Capabilities include cGMP manufacturing for suspension processes up to a 2,000-liter and adherent processes.