Andelyn Biosciences manufactures custom gene therapy for rare genetic disorder

Andelyn Biosciences has successfully developed and manufactured a custom gene therapy to treat a child with NEDAMSS, a rare and degenerative neurological disorder.

Using its AAV Curator Platform, the contract development and manufacturing organization (CDMO) completed the therapy within 10 months of starting development, just 14 months after the child, Elly Krueger, was diagnosed. The platform allows for the ability to support the exacting viral vector manufacturing needs of different types of patients, including for ultra-rare diseases, according to Andelyn.

NEDAMSS, caused by mutations in the IRF2BPL gene, is marked by developmental regression, seizures, and loss of motor and cognitive functions. Elly, diagnosed at eight months old, faced rapid deterioration.

Her family launched a foundation, Elly’s Team, to drive awareness and pursue treatment options. After evaluating multiple strategies, Andelyn was selected to produce the gene therapy. The company used its proprietary platform to accelerate development and manufacturing to meet the urgency of Elly’s case.

Elly received the gene therapy on April 3 at Weill Cornell in New York City, becoming the first patient treated for IRF2BPL deficiency. One month following the treatment, the therapy has been well tolerated.

While it is too early to determine long-term efficacy, Elly’s family and medical team are hopeful. The FDA has signaled support for exploring treatment expansion to other children with the same condition.

Andelyn’s AAV Curator Platform enables efficient gene therapy development through a streamlined, internally developed process with pre-reviewed FDA Drug Master Files. CEO Wade Macedone said the company is committed to programs of all sizes, including single-patient projects.

“As a company we are as proud of successfully completing single-patient programs as we are scaling a manufacturing process for 10,000 or more patients,” Macedone said in a statement. “We all know that with these projects there is someone, usually a young child, whose life depends on us.”