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Afraxis Enters Collaboration with NIH as Part of Therapeutics for Rare and Neglected Disease Program
Date:9/26/2011

SAN DIEGO, Sept. 26, 2011 /PRNewswire/ -- Afraxis, a San Diego-based biotechnology company developing drugs to treat rare genetic diseases through the modulation of p21-activated kinase (PAK), announced today that it has been selected to participate in the National Institutes of Health's (NIH) Therapeutics for Rare and Neglected Disease (TRND) Program. As part of the program, Afraxis will partner with the NIH to pursue the ongoing development of the company's disease-modifying treatments for Fragile X syndrome. Fragile X syndrome is a rare genetic disorder that is the most common inherited form of mental retardation.

"TRND collaborations help companies focusing on rare and neglected diseases overcome one of the biggest obstacles facing all drug development companies: advancing programs through that 'valley of death' between identification of a promising compound, and clinical stage development when a program becomes more attractive to a big pharma partner," said Jay Lichter, Ph.D., president and CEO of Afraxis. "This is a tremendous opportunity for Afraxis as the NIH is providing access to state-of-the-art laboratory facilities, the expertise of collaborating NIH scientists, and the financial resources to continue our research and development efforts through the initiation of human clinical trials."

Afraxis is developing disease-modifying treatments for Fragile X syndrome and other central nervous system disorders based on a leadership position in the understanding of PAK biology. Fragile X symptoms can range from learning disabilities to more severe cognitive or intellectual disabilities, such as mental retardation. Fragile X is the most common known cause of autism or "autistic-like" behaviors. The disease is caused by a mutation in the FMR gene on t
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