Date: 2014-09-22
Type of information: Collaboration agreement
Compound: small molecule candidate therapies for CMT1A
Company: Genzyme (USA - MA), a Sanofi company (France) Charcot-Marie-Tooth Association (CMTA) (USA - IL)
Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases
Type agreement: collaboration - research
Action mechanism:
Disease: Charcot-Marie-Tooth disease type 1A (CMT1A)
Details:
- • On September 22, 2014, the Charcot-Marie-Tooth Association (CMTA) announced the formation of an alliance with Genzyme, a Sanofi company, to discover therapies for CMT1A. CMT1A is the most common Charcot-Marie-Tooth disorder, comprising at least 60 percent of all patients with CMT Type 1. This hereditary neuropathy is caused by a duplication of the gene for Peripheral Myelin Protein 22 (PMP22), located on Chromosome 17, and is inherited in an autosomal dominant fashion.
CMT1A usually presents with progressive symptoms including muscle weakness and atrophy, loss of balance, and sensory loss. There are currently no available therapies to treat either the initiation or progression of the disease, and patients often resort to bracing for ankle support.
Under the agreement, a close collaboration will be formed between the CMTA STAR network of investigators and Genzyme that will leverage the Sanofi U.S. high-throughput screening facility in Tucson, Arizona. Assays developed at the University of Wisconsin and the NIH will be used to screen compound libraries that include more than 2 million small molecules. Additional investigative STAR participants include laboratories at the University of Iowa and University of Buffalo, which will participate via the sharing of assays specific to the regulation of PMP22 in peripheral nerves. Preclinical investigation of the in vivo activity of select molecules will be pursued in rat and mouse models of CMT1A through the CMTA\'s service relationship with Psychogenics. The aim of this consortium effort driven by the CMTA will be to identify small molecule candidate therapies for CMT1A, which can be further advanced into clinical testing in a patient trials network supported by the NIH and the CMTA.
Financial terms:
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