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Families of SMA Releases Newsletter Describing the Scientific Strategy Behind Antisense Drugs for Spinal Muscular Atrophy

Elk Grove Village, IL (PRWEB) December 09, 2013

Families of SMA has released a new edition of the Compass Newsletter, a publication dedicated to research updates. This newsletter was produced in a collaboration between Families of SMA, Biogen Idec, and Isis Pharmaceuticals. It describes the scientific strategy behind Antisense Drugs. It also provides updates on the ongoing research and clinical trials for this class of SMA drugs.

This edition of Compass, includes information about:

-The ABC's of Antisense Drugs

-ISIS-SMNRX Program Update on Ongoing and Future Clinical Trials

-FSMA Grant Award to Dr. Adrian Krainer at CSHL to Assess Influence of Backbone Chemistry on Antisense Drugs

-FSMA Grant Award to Drs. Arthur Burghes and Christian Lorson for Novel Antisense Therapies

-Report on New Data from the Completed Phase I Study Evaluating the Safety of Isis-SMNRX

Antisense drugs are small snippets of synthetic genetic material that bind to RNA (short for ribonucleic acid). The main factor driving where the antisense drug binds to the RNA is the nucleic acid sequence used. Nucleic acids consist of a chain of linked units called nucleotides. Antisense drugs are typically 12-21 nucleotides that are chemically modified to bring about the appropriate drug response.

How does the antisense approach work for SMA?
SMA is caused by a loss of, or defect in, the survival motor neuron 1 (SMN1) gene. The SMN1 gene produces most of the SMN protein, which is critical to the health and survival of the nerve cells in the spinal cord responsible for muscle function. The severity of SMA is correlated with the amount of SMN protein in the cell. A second closely related back-up gene called SMN2 exists that normally produces a truncated, low-functioning form of SMN protein. Antisense therapy could be used to change the functioning of the SMN2 gene by utilizing alternative RNA splicing. Alternative splicing is a normal mechanism that the cell uses to produce different, but closely related proteins from a single gene.

About Families of SMA
Families of SMA has awarded four new drug discovery projects since the start of 2013. The research progress provides hope that one day we will live in a world without SMA. There are now 15 new SMA drug programs in development, including 4 in clinical trials. This pipeline has expanded from just 4 programs 5 years ago. Families of SMA has funded over half of all the ongoing drug programs for SMA. The FSMA research approach funds programs at early stages, and then partners with companies to take them through clinical trials. Supporting multiple programs gives different approaches for a SMA therapy, which increases the chances of success and accelerates the timeline to a treatment and cure.

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