PTC Therapeutics and CHDI Foundation Announce a Collaboration on a Small-Molecule Therapeutic for Huntington’s Disease
SOUTH PLAINFIELD, N.J., April 4, 2018 – PTC Therapeutics, Inc. (NASDAQ: PTCT) and the CHDI Foundation, Inc. (CHDI) today announced 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 has the expanded human huntingtin transgene. Read more…