New Hope Research Foundation and Forge Biologics Announce cGMP Manufacturing Partnership to Accelerate Gene Therapy for Patients with Tay-Sachs Disease - Forge Biologics

New Hope Research Foundation and Forge Biologics Announce cGMP Manufacturing Partnership to Accelerate Gene Therapy for Patients with Tay-Sachs Disease


COLUMBUS, Ohio – June 28, 2023 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, announced today a development and cGMP manufacturing partnership to advance the Foundation’s novel gene therapy, NHR01, into Phase I/II clinical trials for patients with GM2 gangliosidosis.

“We are proud to be the CDMO partner for New Hope Research Foundation, an organization that is significantly advancing a gene therapy approach to provide hope for patients suffering from Tay-Sachs disease,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge Biologics. “At Forge, we are ready to partner with companies, nonprofit organizations, and gene therapy developers around the world, working towards the mission we share to bring potentially life-saving gene therapies to patients.”

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.

About 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.

About New Hope Research Foundation
Founded in 2006, New Hope Research Foundation is a 501(c)(3) non-profit operating foundation with a mission of developing genetic cures for GM2 gangliosidosis and other lysosomal storage diseases that affect the central nervous system. The Foundation is led by a seasoned management, science, and engineering team with extensive experience in developing innovative and life saving medical technology and products. 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. To learn more, visit

About Forge Biologics
Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life-changing gene therapies and help bring them from idea to reality. Forge’s 200,000 square foot facility utilizes 20 cGMP suites in Columbus, Ohio, the Hearth, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV manufacturing and hosts scalable, end-to-end manufacturing services. Offerings include process and analytical development, plasmid DNA manufacturing, viral vector manufacturing, final fill, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most. To learn more, visit

Media Inquiries
Simone LaBonté – New Hope Research Foundation
Communications Director

Marina Corleto – Forge Biologics
Associate Director, Marketing and Communications   

Client Development
Taleen Barsoumian – Forge Biologics
Vice President, Client Development


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