October 2026 - North Bethesda, MD

NovaGen Research Fund Receives Defense Medical Research and Development Program (DMRDP) Grant from Defense Health Agency (DHA) to Advance Novel Gene Therapy for Amyotrophic Lateral Sclerosis (ALS)

This multi-site program will develop and test an AAV-based therapeutic targeting a retroviral driver of ALS, with support from the Defense Health Agency's Defense Medical Research and Development Program 

NORTH BETHESDA, MD — NovaGen Research Fund (NGRF) announced it will manage a grant from the Defense Health Agency (DHA) through the Defense Medical Research and Development Program (DMRDP) to support a multi-year research initiative aimed at advancing a novel gene therapy candidate for amyotrophic lateral sclerosis (ALS). The award, administered under the Amyotrophic Lateral Sclerosis Research Program, will fund a coordinated effort spanning research and manufacturing sites, with NGRF providing scientific operational management across the collaboration. The program will execute the titled project “shRNA to a Human Endogenous Retrovirus as a Therapeutic Approach to ALS” with our partners. 

"ALS remains one of the most devastating diagnoses a person can receive, and the need for therapies that address its underlying biology — not just its symptoms — is paramount," said Joseph Tasto, M.D., CEO of NGRF. "This award allows our partners to move a scientifically compelling approach through critical manufacturing, preclinical testing, and biomarker development to research alternatives for those in need." 

About the Collaboration 

The collaboration brings together a multidisciplinary team of organizations, each contributing specialized expertise to advance the program. The National Institute of Neurological Disorders and Stroke (NINDS) will lead product development, animal model testing, and biomarker research under the leadership of Principal Investigator Avindra Nath, M.D. The company SK Pharmteco will provide plasmid and viral vector manufacturing capabilities, while Twilight Biosciences will develop the biomarker assays used to evaluate treatment outcomes. NovaGen Research Fund (NGRF) will provide scientific program and project management support and serve as the central coordinator across the organizations, facilitating communication, integration of activities, and overall program execution. "Bringing together vector manufacturing expertise, an experienced research team, and biomarker science under a well-coordinated program allows this research to move forward in an effective manner for patients," said Reema Railkar, Ph.D., who will serve as NGRF's point of contact for the program.  

About NovaGen Research Fund (NGRF) 

NGRF is a 501(c)(3) nonprofit collaborative research hub dedicated to accelerating scientific research and facilitating multi-institutional collaborations to move science forward. NGRF provides data science and AI analysis and tools, grants management, program coordination, and scientific administration to assist research teams in translating promising discoveries into real-world therapies and advances.