Breaking News, Collaborations & Alliances

PTC, CHDI Enter Research Agreement

To advance PTC's Huntington's disease program

PTC Therapeutics and the CHDI Foundation have entered a research collaboration to advance PTC’s Huntington’s disease program.    The program is optimizing small-molecule compounds—identified using PTC’s splicing technology platform—that decrease the production of huntingtin protein. The current compounds have been shown to be orally bioavailable in animals, blood-brain barrier penetrant, and effective in decreasing the amount of huntingtin protein in a mouse model that ...

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