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Families of SMA Awards New Funding to Advance a CNS Delivered Gene Therapy for Spinal Muscular Atrophy
May 2, 2012.
Families
of SMA is pleased to announce the award of up to $750,000 for an
important new grant to Dr. Brian Kaspar at Nationwide Children’s
Hospital. This award will support preclinical development of a
CNS-delivered Gene Therapy for Spinal Muscular Atrophy. With funding
from FSMA, Dr. Kaspar’s team will initiate the studies needed for an
Investigational New Drug (IND) application for this therapy to the Food
and Drug Administration (FDA).
“Families of SMA is excited
to be awarding new goal-directed drug discovery funding for this gene
therapy program. This work follows up on a 2010 grant from FSMA to test
the age-dependence in primates of this gene therapy. The new funding
will allow us to accomplish several key goals simultaneously”, says Jill
Jarecki, PhD, FSMA Research Director. “First, it will allow us to
advance this very promising new therapy for SMA towards human clinical
trials. Second, it will allow FSMA to fund multiple SMA drug programs
concurrently, which have different approaches. Doing this will increase
our community’s chances of successfully finding a treatment for SMA.”
“
This
is extremely important funding from FSMA to allow us to collect
additional pre-clinical data for a CNS delivered AAV gene delivery into
the cerebrospinal fluid, which will be important information to present
to the FDA. It also jump starts research prior to obtaining government
and commercial involvement which we are actively pursuing. We are quite
hopeful for a positive funding decision on a recent NIH proposal for
co-funding of this project with FSMA.” Brian Kaspar, PhD, Associate
Professor, Principal Investigator The Research Institute at Nationwide
Children’s Hospital, The Ohio State University.
The overall project goals are:
1) to optimize the dosing regimen for CNS-delivered SMA gene therapy;
2) to conduct the GLP toxicology, immune response, and bio-distribution experiments required by the FDA;
3) to prepare and hold a pre-IND meeting with the FDA;
4) to submit an IND to the FDA to begin human clinical trials; and
5) to produce clinical grade material for human studies.
The overall timeline for this work is expected to be three years.
This
Program was chosen for funding by the FSMA Translational Advisory
Committee (TAC), after reviewing multiple potential new drug programs.
Every drug program carries risk of encountering hurdles at each of the
stages described above. Therefore, a project specific Steering Committee
has been put in place, which is comprised of experts in both gene
therapy and in SMA biology, with representatives from academia and
industry. This committee will help manage the project, ensuring it
progresses in an efficient and well-run manner. In addition, project
funding will be awarded upon meeting predetermined milestones, decided
on by the Steering Committee.
“I am incredibly excited by FSMA’s
decision to support Dr. Kaspar and his team in this very important
project. As a pharmaceutical scientist who works every day in drug
discovery and development, I am encouraged by the quality of the science
and the fact that it aims to address SMA treatment from a different
vantage point from other programs in the SMA drug pipeline. This is only
the first step, but it’s a critically important step toward assessing
whether gene therapy is a viable approach in SMA. Time will tell but I,
for one, am incredibly hopeful and look forward to working with FSMA to
facilitate the efforts of Dr. Kaspar and his team. I should add that as a
parent of an SMA child, I am always looking for a medical breakthrough
that could the transform the lives of SMA patients.” Timothy P. Reilly,
PhD, DABT Director, Drug Safety Evaluation, Bristol-Myers Squibb. TAC
Member.
fsma.org
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