Loren Eng is the President of the Spinal Muscular Atrophy Foundation, a nonprofit organization which develops treatments for SMA, the leading genetic cause of death in young children. Since its inception, the Foundation has funded over $150 million in research and therapeutics development. The Foundation collaborates with leading academic medical centers, the federal government (DoD, NIH, CDC, FDA), and nearly every pharmaceutical company in the world including: Roche, Biogen, and Novartis. When the SMA Foundation was established in 2003, there were no treatments for SMA. Due to the Foundation’s investments and drug discovery efforts, today there are three FDA-approved drugs.  

There are several others in advanced clinical development and in preclinical development. One of our first investments resulted in Spinraza which was the first drug approved for SMA. In 2017, Spinraza won the Prix Galien Award for Best Biotechnology Product of the year. In 2019, Dr. Frank Bennett (our collaborator at Ionis Pharmaceuticals) and Dr. Adrian Krainer (our collaborator at CSHL) received the Breakthrough Prize in Life Sciences (for the development of Spinraza). The SMA Foundation funded the basic science research and clinical trials network; and worked on the full scope of the therapeutics development which led to the drug Spinraza.

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