Families of SMA and Nationwide Children’s Hospital Announce Multi-Million Dollar Award from NINDS

Nationwide Children’s Hospital and Families of Spinal Muscular Atrophy announce the award of a multi-million dollar cooperative agreement from the NINDS to advance a gene therapy development program for Spinal Muscular Atrophy.

Elk Grove Village (I-Newswire) March 18, 2013 - This three-year multi-million dollar cooperative agreement to Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital in the amount of $3,752,462, funds pre-clinical drug development up to the filing of an Investigational New Drug Application (IND) to the Food and Drug Administration (FDA). This agreement represents an innovative collaboration between Government, Advocacy and Academic groups to advance a promising new therapy for SMA.

"At Families of SMA we are extremely pleased that our initial investment at an early stage of this program has provided the preliminary data to leverage larger funding from the NIH. We feel this grant award is positive validation of the Families of SMA research funding and partnering strategy, as well as for this approach for gene therapy in SMA,” said Jill Jarecki PhD, Research Director at Families of SMA. “The Families of SMA funding strategy for preclinical drug development is to invest seed funds to begin early-stage programs for SMA. As programs advance, we look for funding to transition from non-profit to government and commercial sources.”

“My research team at Nationwide Children’s Hospital is excited to advance this promising cerebrospinal fluid delivery approach of AAV9-SMN to the clinic for SMA patients and we are extremely grateful to FSMA and NINDS for the support of this important work,” said Dr. Kaspar, also a faculty member at The Ohio State University College of Medicine. “We stand committed to bring SMA experimental therapeutics to the clinic in the most rapid and safe manner.”

“Development of therapies requires collaboration of academics, advocacy, industry, and government—no single party has the resources to do this alone. The collaboration between Dr. Brian Kaspar, Families of SMA, and the NIH is an exciting model in leveraging resources and expertise in the hope of accelerating therapy development for SMA,” said Dr. John Porter, PhD, Program Director at the National Institute of Neurological Disorders and Stroke