“This new drug development collaboration will focus on optimizing the drug features of a newly identified lead compound that directly influences SMN2 gene splicing,” said Dr. Jill Jarecki, FSMA Research Director. “FSMA-sponsored research has contributed to the identification of the SMN1 gene as well as a second disease-modifying copy of the gene called SMN2. Normally, the SMN2 gene produces reduced amounts of SMN protein due to a defect in mRNA splicing. This project aims to develop a SMA drug that safely and effectively restores the proper amount of SMN protein in the body in order to slow or reverse the disease process by correcting the splicing of SMN2 gene.”

“Paratek is excited about this collaboration for several reasons. First, we already have a compound with validated activity in hand,” said Dr. Ken Tanaka, Vice President of Research and Development for Paratek. “In addition, this project is of great scientific interest, as the compound has been shown to directly regulate SMN splicing and should lead to a greater biological understanding of the underlying disease process. With our chemistry expertise and the splicing assay capabilities at Cold Spring Harbor, we believe we have the right resources and team in place to lead us to a novel drug for treating SMA.”

The Krainer laboratory at Cold Spring Harbor will be assessing additional Paratek compounds from Paratek’s collection for effects on SMN2 splicing. In parallel, Paratek will be developing new proprietary compounds based on this initial lead. In related work, the FSMA scientific advisory board has recently awarded Dr. Michelle Hastings of Cold Spring Harbor Laboratory a two-year grant to investigate the molecular mechanism of this class of compounds.

“FSMA is excited to be funding this drug discovery effort,” said Audrey Lewis, Executive Director of FSMA. “We believe that this compound will work differently from the other SMN-increasing compounds that FSMA is developing, providing our community with an additional pathway to potentially treat this disease.”
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