Families of Spinal Muscular Atrophy (FSMA) and Paratek Pharmaceuticals, Inc. today announced they have extended their joint R&D collaboration to develop a drug candidate for the treatment of Spinal Muscular Atrophy (SMA), the leading genetically inherited cause of death of children under the age of two years. The collaboration is focused on optimizing and advancing into the clinic a novel small molecule within Paratek’s library derived from the tetracycline class of compounds.

Spinal Muscular Atrophy is an often-fatal genetic disorder resulting from the loss of both copies of the Survival Motor Neuron (SMN1) gene. This causes a chronic deficiency in the production of the SMN protein, which is essential to the proper functioning of the motor neurons in the spinal cord and to the control of muscles in the limbs, neck and chest.

“We are excited by the progress in our program with Paratek, which in the past year has demonstrated positive results in early cell models of this disease,” said Kenneth Hobby, Executive Director of FSMA. “The continuation of this collaboration also represents FSMA’s broader commitment to expand our relationships with life sciences companies and build a pipeline of drug candidates. As reported recently in the Wall Street Journal, we believe these kinds of interactions and early stage support can provide a critical impetus for companies to innovate new therapies for serious diseases such as SMA that may have been otherwise overlooked.”

Dr. Ken Tanaka, Paratek Pharmaceuticals’ Vice President of Research and Development, stated, “It is an honor to work with the committed and resourceful team at FSMA. Together with FSMA and with the team of scientists led by Dr. Adrian Krainer at Cold Spring Harbor Laboratory and Dr. Arthur Burghes at Ohio State University , we have come to a greater understanding of the underlying biology of SMA, especially the processes that regulate SMN splicing. Our lead candidate has been shown in cells in culture and recently in cells in animals to enhance the expression of the SMN2 gene. Our goal is to find a compound that can restore normal levels of SMN protein in nerve tissue and prepare it for human studies to test the ability of this drug to slow or even reverse the disease process.”
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