Families of Spinal Muscular Atrophy Licenses Potential Treatment for SMA to Repligen Corporation.
October 22, 2009.
SMA, the leading genetic killer of children under the age of two, is typically marked by the degeneration of muscle movement including the muscles that control crawling, walking, swallowing or breathing. There are no approved therapies for the treatment of SMA, which affects one in every 6,000 babies. One in every 40 people carries the gene that causes SMA, indicating approximately 7.5 million carriers in the United States.
Families of SMA was started by a small group of parents in 1984 who wanted to raise funds for SMA research and support all affected families. FSMA has funded $50 million for SMA research, raised from individual donations and through fundraising events held by volunteer families and Chapters.
FSMA made investments of $13 million during the last decade to bring this specific program to the cusp of clinical development. Through FSMA’s leadership, the research has resulted in a drug candidate that treats the underlying cause of SMA. In preclinical studies, the drug has been shown to efficiently cross the blood brain barrier - a critical feature for a neurological drug - and prolong survival significantly in two different mouse models of SMA.
Often an “Orphan Disease,” such as SMA, will not be "adopted" by the pharmaceutical industry because there is limited financial incentive to make new medications to treat smaller patient populations. This license agreement marks a significant milestone for the SMA community by securing the commitment of an industrial partner to develop potential treatments through the highly expensive human clinical trial phases.
“At this point in the program, joining forces with a corporate partner to advance into clinical studies is the best way to meet our objective of accelerating drug development for SMA,” said Kenneth Hobby, President of Families of SMA. “Repligen is an ideal partner for this program with the necessary resources and expertise to invest in and focus on successfully developing this new treatment for SMA. We are very excited to partner with Repligen and look forward to making our combined efforts successful in delivering an effective treatment to our patients.”
“Families of SMA has made remarkable progress in defining a series of highly potent compounds which may be clinical candidates for SMA,” stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. “We look forward to working with FSMA and their collaborators in the development of what we hope will be an important new treatment for SMA.”
Through the agreement with Repligen, Families of SMA has the potential to recover its investments through a series of milestone payments if the program successfully progresses through clinical stages and eventually reaches market approval.
About Families of SMA
Families of Spinal Muscular Atrophy (FSMA) is dedicated to creating a treatment and cure by: funding and advancing a comprehensive research program; supporting SMA families through networking, information and services; improving care for all SMA patients; educating health professionals and the public about SMA; enlisting government support for SMA; embracing all touched by SMA in a caring community. FSMA’s vision is a world where Spinal Muscular Atrophy is treatable and curable.
Families of SMA funds and directs the leading SMA research programs. Successful results and progress from basic research to drug discovery programs to clinical trials provide real hope for families and patients. Families of SMA is a non-profit, 501(c)3 tax exempt organization with 26 Chapters throughout the United States and over 65,000 members and supporters. Additional information about FSMA can be found at www.curesma.org.
About Repligen Corporation
Repligen Corporation is a biopharmaceutical company focused on the development of novel therapeutics for diseases that affect the central nervous system. In addition, we are the world’s leading supplier of recombinant Protein A, the sales of which partially fund the advancement of our development pipeline while supporting our financial stability. Repligen's corporate headquarters are located at 41 Seyon Street, Building #1, Suite 100, Waltham, MA 02453. Additional information may be requested from www.repligen.com.
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