FDA Grants Orphan Drug Designation to CUHK's Novel Peptide Drug Candidate
核心洞察
The FDA has granted Orphan Drug Designation to a peptide drug candidate for Huntington's disease (搜索) developed by CUHK and its spin-off Rare Power Limited (搜索).
The candidate is listed under the generic name 21-amino acid peptide targeting expanded CAG repeat RNA (搜索), a structurally abnormal RNA species.
The team is advancing preclinical work including manufacturing quality, safety and large-animal toxicology, aiming for first-in-human trials in one to two years.
A novel peptide drug candidate developed jointly by the School of Life Sciences at The Chinese University of Hong Kong and its biotechnology spin-off Rare Power Limited (搜索) has received Orphan Drug Designation from the U.S. Food and Drug Administration (搜索) for the treatment of Huntington's disease (搜索). The candidate appears in the FDA Orphan Drug Designations and Approvals database under the generic name "21-amino acid peptide targeting expanded CAG repeat RNA (搜索)."
Huntington's disease (搜索) is a rare, autosomal-dominant neurodegenerative disorder caused by an expanded CAG trinucleotide repeat in the huntingtin (搜索) (HTT) gene, producing progressive motor impairment including chorea, psychiatric symptoms and cognitive decline. Current treatments are primarily symptomatic, such as reducing chorea, and no approved medicine directly targets the cause of the disease or halts its progression. The CUHK and Rare Power programme treats the expanded CAG-repeat RNA itself as the pathogenic species, using a peptide designed to recognise the folded structure of that RNA and neutralise both the RNA and the toxicity that follows, rather than lowering mutant huntingtin protein or its messenger RNA through gene silencing.
Rare Power, founded in 2023, develops peptides against short tandem repeat rare neurodegenerative diseases including Huntington's disease (搜索), spinocerebellar ataxias (搜索) and C9ORF72 ALS/FTD (搜索). The company said the candidate showed promising activity against the RNA species in patient-derived neurons. The next stage is preclinical development covering manufacturing quality, safety and long-term toxicology in large animals, with a first-in-human study targeted in about one to two years. Orphan Drug Designation provides development incentives including regulatory guidance, certain fee waivers at later stages and potential eligibility for seven years of U.S. market exclusivity upon approval.
Source: CUHK press release