FDA grants orphan drug designation to CUHK peptide RP001v63 for Huntington's
The 21-amino-acid peptide binds expanded CAG-repeat RNA duplexes, the toxic species implicated in Huntington's and related repeat disorders.
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By Daniel Okafor · Regulatory Correspondent
Sep 23 08:03 ETSource: CUHK Communications and Public Relations Office
The wire
- 01The FDA awarded Orphan Drug Designation to a peptide candidate developed by The Chinese University of Hong Kong's School of Life Sciences and spin-off Rare Power Limited for Huntington's disease.
- 02Current Huntington's treatment is symptomatic, mainly for chorea, with no approved therapy targeting the cause; the team positions RP001v63 as a disease-modifying RNA-targeting peptide.
- 03The programme is still in preclinical development, with first-in-human trials planned within one to two years; no human efficacy or safety data have been reported.
From the source
Read at cpr.cuhk.edu.hkCUHK-developed novel peptide drug candidate granted FDA Orphan Drug Designation Offering new hope for Huntington’s disease patients
Co-founders of Rare Power Limited. From left: Dr Maggie Leong Lok-i, Mr Tony Wong, Professor Edwin Chan Ho-yin and Dr Aldrin Yim
