Global biopharmaceutical company PTC Therapeutics and Boston-based Massachusetts General Hospital have agreed to partner on research for the treatment of rare genetic disorders resulting from pre-mRNA splicing defects.
Here are four things to know about the collaboration.
1. Under the agreement, PTC will receive an exclusive worldwide license to compounds that modulate alternative splicing of the IKBKAP pre-mRNA — the predominant cause of familial dysautonomia, also known as Riley-Day syndrome, a rare developmental disorder of the sensory and autonomic nervous system.
2. PTC will pay an upfront license fee, make payments upon successful completion of certain development and commercialization milestones, as well as royalties on worldwide product sales.
3. PTC will also pay for collaborative research and licensing activities, and be responsible for the development and commercialization of products arising from the collaboration.
4. Susan Slaugenhaupt, PhD, scientific director of the Mass General Research Institute, professor of neurology (genetics) at Harvard Medical School, and the Elizabeth G. Riley and Dan E. Smith Jr. MGH Research Scholar in the MGH Center for Human Genetic Research, expressed excitement about the collaboration. "PTC's leadership in developing treatments for rare diseases and expertise in RNA biology, combined with our innovative research capabilities, makes this a powerful collaboration to carry forward our research program to develop therapies for splicing-related genetic conditions," she said in a news release.
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