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Families of SMA and Nationwide Children’s Announce Multi-Million Dollar Award from NINDS to Advance CNS Gene Therapy for Spinal Muscular Atrophy

Elk Grove Village, IL. March 13, 2013--Nationwide Children’s Hospital (Columbus, OH) and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announce the award of a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS) to advance a gene therapy development program for Spinal Muscular Atrophy (SMA). 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,

Pfizer Licenses Families of Spinal Muscular Atrophy Quinazoline Drug Program from Repligen

Elk Grove Village, IL. January 3, 2013. Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen’s spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA). Families of SMA funded and directed the preclinical development of RG3039 with an investment of more than $13 million. This was the first drug discovery program ever conducted specifically for SMA.  Repligen licensed RG3039 in 2009 from Families of SMA. “We believe this collaboration with Pfizer, a leading

Families of Spinal Muscular Atrophy Committed $2.5 Million to Funding SMA Research in 2012

Elk Grove Village, IL. December 31, 2012--Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program, which includes: Basic Research to reveal the best ways of making SMA drugs; Drug Discovery to make practical new drugs, and; Clinical Trial resources to help test new drugs. Families of SMA committed new funding to the following SMA research projects during 2012:-$710,000 for Basic Research.-$1,450,000 for Drug Discovery.-$110,000 for Clinical Trial Resources.-$200,000 for the 16th SMA

Families of Spinal Muscular Atrophy Awards $710,000 for Basic Research to Identify New and More Effective Approaches for SMA Therapies

Elk Grove Village, IL. December 28, 2012--Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program.  Continued investment in basic research leads to greater understanding of the exact nature, causes, and consequences of SMA.  This knowledge is key to ensuring that the most effective SMA treatments can be identified and developed as quickly as possible. Basic research is vital to finding a treatment and cure for SMA. It provides fundamental information about what is going wrong in SMA by showing

Repligen Initiates Phase 1b Trial of RG3039 for Spinal Muscular Atrophy Licensed from Families of SMA

Elk Grove Village, IL. September 26, 2012. Repligen Corporation has initiated a Phase 1b clinical study of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA).  The primary objectives of the study are to further evaluate the safety and plasma pharmacokinetics (PK) of multiple doses of RG3039 in healthy volunteers. Repligen licensed RG3039 in 2009 from Families of Spinal Muscular Atrophy (FSMA).  FSMA funded and directed the preclinical development of RG3039 with an investment of more than $13 million.  This was the first drug

Families of SMA Announces $1.4 Million Planned for Next Round of Spinal Muscular Atrophy Research Funding.

Elk Grove Village, IL. June 7, 2012. Families of Spinal Muscular Atrophy (FSMA) is dedicated to creating a treatment and cure for Spinal Muscular Atrophy (SMA) by funding and advancing a comprehensive research program.  The organization is planning to award $1.4 Million in new research funding over the next 6 months.  This new round of research funding will be allocated into three areas: 1)Basic Research to understand the disease and provide ideas for drug making, 2)Drug Discovery to develop new SMA therapies, and 3)Clinical Research to help test new drugs effectively and to improve

Families of SMA Announces $700,000 Funding for Novel Drug Program at Newly Created California Institute for Biomedical Research.

Elk Grove Village, IL. May 10, 2012. This new program will focus on optimizing small molecule drug candidates which increase the SMA back-up gene in order to make them ready for human clinical trials. It is well demonstrated that increasing production from the back-up gene can lead to improvement in mouse models of SMA. Small molecule drug candidates have particular advantages: mainly ease of use by oral dosing and the ability to clear the drug quickly from the body. Dr. Peter Schultz, a renowned chemist and successful biotech leader, will be leading this new research effort. In

Families of SMA Awards New Funding to Advance a CNS Delivered Gene Therapy for Spinal Muscular Atrophy

Elk Grove Village, IL. 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.

Repligen Reports Positive Results From Phase 1 Clinical Trial for Spinal Muscular Atrophy (SMA).

Elk Grove Village, IL. April 25, 2012. Repligen Corporation (NASDAQ:RGEN) today announced positive results from a Phase 1 study to evaluate the pharmacokinetic (PK) and safety profile of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA). SMA is a inherited neurodegenerative disease in which symptoms of progressive damage to motor neurons including loss of muscle function typically appear very early in life and often progress to severe physical disability and early loss of life. The Phase 1 trial was a blinded, ascending, single

Update on the Spinal Muscular Atrophy Drug Development Pipeline from Families of SMA

Elk Grove Village, IL. January 19, 2012. Developing new therapies to treat and ultimately cure SMA is the driving force behind Families of SMA. To achieve this goal, Families of SMA aggressively invests in drug development research, funding the most successful and innovative scientists and organizations. Several key details demonstrate the great progress being made:1) Programs are advancing into the clinical trial stage of testing.There are now 3 novel programs actively being testing in clinical trials for SMA.2) There is a broad diversity of approaches in development.This

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