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 addition to founding the California Institute for Biomedical Research where the Families of SMA funded project will take place. Dr. Schultz also was Institute Director at the Genomics Institute of the Novartis Research Foundation (GNF) from 1999 to 2010. Dr. Schultz has founded multiple biotech companies, including Affymax Research Institute, Syrrx, Kalypsys, Phenomix, Symyx Technologies, Ilypsa, Ambrx and Wildcat Technologies. Dr. Schultz has published over 500 scientific publications, and received numerous awards for his work including: the Waterman Award of the National Science Foundation, the 1994 Wolf Prize in Chemistry, the 2003 Paul Ehrlich Prize, and the 2005 Arthur C. Cope Award of the American Chemical Society. He is a member of the National Academy of Sciences and the Institute of Medicine.

“Dr. Schultz is greatly respected in the research community, both as an academic scientist and in the biotech industry. He has successfully led many drug programs that continued on to clinical development”, said Jill Jarecki, Ph.D., FSMA Research Director. “FSMA is very pleased to have a chemist and drug developer of Dr. Schultz’s experience spearheading a program for Spinal Muscular Atrophy. We are also excited that Spinal Muscular Atrophy is one of the initial drug programs at the newly formed California Institute for Biomedical Research.”

This new institute will focus on innovative new approaches to unmet medical needs, including neglected and rare diseases. Their expertise will be in translating basic research discoveries from bench to bedside. Therefore, this group will be ideally poised to move newly identified SMN enhancing compounds through all the preclinical steps of drug development towards first-in-human clinical trials.

"I am pleased to be collaborating with Families of SMA on a new small molecule drug discovery project for SMA.  CALIBR will provide a unique opportunity to move therapies for rare diseases towards the clinic, due to our pre-clinical drug discovery infrastructure, coupled with our focused ability to move projects rapidly through early pre-clinical stages." Dr. Peter Schultz, Institute Director, CALIBR.

About Families of Spinal Muscular Atrophy

Families of SMA funds and directs the leading SMA research programs to develop a treatment and cure for the disease.  The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease.  The charity has invested over $50 million in research and been involved in funding half of all the ongoing novel drug programs for SMA.  Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 85,000 members and supporters throughout the United States, and 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 healthcare professionals and the public about SMA; enlisting government support for SMA; embracing all touched by SMA in a caring community.  For more information: www.curesma.org. Please contact Kenneth Hobby at Kenneth@fsma.org or call 1-800-886-1762 if you have any questions.

About California Institute for Biomedical Research

In early March 2012, Merck announced that it will invest $90 million the California Institute for Biomedical Research (CALIBR, www.calibr.org), a private, nonprofit center to be led by Peter Schultz, a renowned chemist at The Scripps Research Institute. The goal of the new institute is to bridge the gap to promote translational research and early drug development. The roughly 25,000-square-foot research center will be equipped for programs in immunology, autoimmune and metabolic disorders, cardiovascular disease, regenerative medicine, cancer biology and neurodegenerative disease.