RARE Daily

New Hope and Forge Enter cGMP Manufacturing Partnership to Accelerate Gene Therapy for Tay-Sachs Disease

June 30, 2023

Rare Daily Staff

The New Hope Research Foundation, a nonprofit organization dedicated to finding a genetic cure for GM2 gangliosidosis (including Tay-Sachs disease) and other lysosomal storage diseases, and Forge Biologics, a leading manufacturer of genetic medicines, entered into a development and cGMP manufacturing partnership to advance the Foundation’s novel gene therapy, NHR01, into phase 1/2 clinical trials for patients with GM2 gangliosidosis.

GM2 gangliosidosis, subclassified as Tay-Sachs disease or Sandhoff disease, is a hereditary neurogenerative disease which causes severe, progressive symptoms. As with other lysosomal storage diseases, GM2 gangliosidosis is caused by gene mutations leading to a deficiency of a specific enzyme. In the severest forms of the diseases, children succumb to the overwhelming neurodegeneration at an early age. With the late-onset form of this disease, symptoms do not become apparent until young adulthood. The disease in these individuals progresses slowly over many years resulting in major loss of balance, unintelligible speech, difficulty swallowing, muscle atrophy, and psychiatric issues. There are currently no FDA-approved treatments for the disease.

The Foundation has developed NHR01, a novel AAV gene vector for the treatment of GM2 gangliosidosis, and proprietary minimally invasive methods for targeting its delivery to the central nervous system. Preclinical evaluations of NHR01 conducted through collaborations with academic and hospital research centers have shown NHR01 to be highly effective in restoring the enzyme function that is deficient in both Tay-Sachs and Sandhoff diseases.

Through this partnership, Forge will provide adeno-associated virus (AAV) process development, analytical services, and cGMP manufacturing to New Hope Research Foundation. The Foundation will leverage Forge’s platform processes, including its proprietary HEK293 suspension Ignition Cells, to accelerate the initial production. All development and AAV manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy facility in Columbus, Ohio.

“Forge has proven to be a trusted partner to many gene therapy developers. We look forward to embarking on our manufacturing collaboration with their experienced team and tried and true platform process to help accelerate our therapy into clinical trials and deliver new hope for patients with GM2 gangliosidosis,” said Jack Keimel, co-founder and president of New Hope Research Foundation.

Photo: Jack Keimel, co-founder and president of New Hope Research Foundation

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