Andelyn Biosciences Partners with Drake Rayden Foundation to Manufacture AAV Gene Therapy for Rare NKH Disease
核心洞察
Andelyn Biosciences (搜索) has been selected by Drake Rayden Foundation (搜索) to manufacture clinical-grade AAV gene therapy (搜索) for nonketotic hyperglycinemia (搜索) (NKH (搜索)), a rare inherited metabolic disorder.
NKH (搜索) affects the glycine cleavage system (搜索), causing toxic glycine buildup in the brain that leads to severe neurological problems and early death in 85% of severe cases.
The collaboration involves scientists from University of Texas Southwestern (搜索) and utilizes Andelyn's AAV Curator® Platform to develop manufacturing processes for this potential treatment.
Andelyn Biosciences (搜索), a leading cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been selected by the Drake Rayden Foundation (搜索) to manufacture clinical-grade adeno-associated virus (AAV) gene therapy for the treatment of nonketotic hyperglycinemia (搜索) (NKH (搜索)), a devastating rare genetic disorder.
Addressing a Critical Unmet Medical Need
NKH (搜索) is a debilitating inherited metabolic disorder caused by mutations in the GLDC gene (搜索) that disrupts the glycine cleavage system (搜索) (GCS). This disruption prevents the body from breaking down the amino acid glycine, leading to toxic accumulation particularly in the brain. The resulting neurological complications include seizures, lethargy, poor muscle tone, breathing difficulties, and profound intellectual disability.
The prognosis for NKH (搜索) patients is particularly grim. Children with the severe form of the disease, which comprises close to 85% of diagnosed cases, often survive only days or weeks after birth. Those who do survive face severe seizure disorders and a very poor long-term prognosis.
Foundation-Driven Research Initiative
The Drake Rayden Foundation (搜索), established in 2017, was formed with the specific mission to advance NKH (搜索) research toward viable treatments. The foundation was created by Eric and Tarah O'Sullivan after their son Drake Rayden was diagnosed with NKH at just two weeks old. Their daughter Vivian Faye was later also born severely affected by the disease.
"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," said Tarah O'Sullivan, founder and mother of Drake and Vivian. "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."
Manufacturing Partnership and Platform Technology
Andelyn will collaborate closely with the Drake Rayden Foundation (搜索) and scientists from Dr. Steven Gray's laboratory at the University of Texas Southwestern (搜索) (UTSW) to establish manufacturing processes using Andelyn's proprietary AAV Curator® Platform. This methodology is designed to offer reliable speed and quality in AAV production.
The partnership reflects Andelyn's broader commitment to accelerating gene therapies by working not only with biotech and pharmaceutical companies but also with individual families and non-profit organizations seeking treatments for rare diseases.
"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," said Matt Niloff, Chief Commercial Officer at Andelyn Biosciences (搜索). "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."
Company Capabilities and Experience
Andelyn Biosciences (搜索) brings significant expertise to this collaboration, with more than 20 years of experience in viral vector development and manufacturing. The company has produced cGMP material for more than 450 clinical batches and supported 75 global clinical trials. Operating from development and manufacturing facilities in Columbus, Ohio, Andelyn offers comprehensive services from concept through plasmid engineering, process development, and commercial manufacturing.
The company's manufacturing capabilities include cGMP production for suspension processes up to 2,000 liters and adherent processes, supported by a rigorous quality system, regulatory support, and supply chain vertical integration.