View Press Releases

Andelyn Biosciences to Manufacture First Gene Therapy for Infantile Neuroaxonal Dystrophy in the US

November 28, 2022

COLUMBUS, OH, November 29, 2022 - Andelyn Biosciences, Inc., a pioneering patient-focused gene therapy Contract Development and Manufacturing Organization, will help the INADcure Foundation bring the first-ever gene therapy for the treatment of Infantile Neuroaxonal Dystrophy (INAD) to clinical trials.

Infantile Neuroaxonal Dystrophy (INAD) is a rare inherited neurological disorder caused by mutations in a gene called PLA2G6 that encodes for an enzyme known as Phospholipase A2. The disease leads to neurodegeneration in the nervous system of patients accompanied by an inflammatory response and causes progressive loss of mental skills, muscular control, and vision.

The INADcure Foundation was formed in 2017 to support the development of treatments for INAD and other forms of PLA2G6-related neurodegeneration (PLAN). Due to the lack of FDA-approved therapies to prevent or slow the progression of the disease, the Foundation assembled a research team to develop a gene therapy treatment led by Dr. Neil Hackett, an expert in the clinical translation of AAV vectors.

Following an extensive review of the gene therapy manufacturing marketplace, Andelyn was selected as INADcure’s contract to manufacturer GMP (clinical grade) AAV vectors to be delivered in a ready-to-use format.

Kathrin Meyer, Ph.D., from the Center for Gene Therapy at Nationwide Children's Hospital and an advisor to the scientific team working on the program, commented, “Nationwide Children’s Hospital and Andelyn have collaborated on several research projects involving gene therapy manufacturing. Andelyn understands the high unmet need of these rare, devastating conditions."

Eric Blair, Chief Commercial Officer at Andelyn, said, “When we were first approached to support the work of the INADcure Foundation, we were determined to help them turn hope into reality. At Andelyn, we have the expertise to help make the journey to market easier for the Foundation and ensure the INAD therapy is safe for children. There are over 10,000 rare diseases that need a cure, and although they are rare, cumulatively, they’re not uncommon and impact over 50 million people worldwide. We are committed to manufacturing therapies to treat these diseases and providing hope to the children and adults impacted.”

Andelyn has recently expanded its facilities into a new GMP clinical and commercial manufacturing facility, the Andelyn Corporate Center (ACC), which completes a network of flexible and scalable sites that support gene therapy development and manufacturing.  Andelyn has the capabilities to start production of a therapy immediately, which is providing the Foundation, a not-for-profit organization, with a timely, but lower price point for services. With the ACC now available for manufacturing, Andelyn has successfully opened its new facilities and is actively producing end-to-end solutions to accelerate manufacturing speed. 

Leena Panwala, Founder & President of the INADcure Foundation, added: “When my daughter was first diagnosed with INAD, I quickly learned very little was known about the disease. My husband, Anil, and I established the INADcure Foundation with the goal of funding research that could lead to life-changing treatments, not only for our daughter, Ariya but for all children with INAD. Now, thanks to the support from INAD families around the world who are tirelessly raising awareness and funds, we can advance to this critical next phase.

“It was evident from the start that Andelyn was the perfect partner for us. Andelyn has the expertise and is passionate about helping organizations like ours that work against the clock to develop treatments for rare diseases. We are excited about our partnership with Andelyn which will bring a gene therapy for INAD to the clinic as quickly as possible."