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Families of Spinal Muscular Atrophy Announces Latest Developments on Three SMA Drug Discovery Programs
Date:6/18/2009

LIBERTYVILLE, Ill., June 18 /PRNewswire-USNewswire/ -- Families of Spinal Muscular Atrophy has just released details of the latest developments for the Families of SMA funded therapeutic pipeline.

Families of SMA is currently funding three distinct drug discovery programs: 1) Quinazolines to boost gene expression; 2) Tetracyclines at Paratek Pharmaceuticals to correct gene splicing; and 3) Motor neuron replacement program at California Stem Cell and UCI. These three programs represent an investment of over $16 Million so far to build a SMA drug pipeline.

Quinazoline Program:

In June 2009, the IND-enabling safety studies were completed for the FSMA-directed Quinazoline program. This is the program previously worked on by Vertex Pharmaceuticals and deCODE genetics, and fully funded by Families of SMA.

The next step for this program is to request a pre-IND meeting with the FDA and to prepare the required data package. Families of SMA will be working over the next several months with toxicology and regulatory experts to prepare for this meeting. According to Jill Jarecki, Ph.D., Research Director FSMA, "This meeting will be an exciting milestone for the project, and an essential step in the path towards human clinical trials of the drug candidate."

Motor Neuron Replacement Program:

California Stem Cell (CSC) is preparing for a final FDA pre-IND meeting to take place in mid 2009. This is a critical step on the track to submitting a formal Investigational New Drug (IND) application to the FDA to begin clinical trials in SMA Type I. It is the intent of CSC to gain approval to begin FDA-approved clinical trials for the use of these cells in development of a cell replacement therapy for SMA Type I.

Numerous pre-clinical efficacy studies have been completed, demonstrating that the cells work. The pivotal animal safety study, required to support an application to be
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SOURCE Families of Spinal Muscular Atrophy
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