Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
Be among the first to receive Kisho Intelligence Briefings
Showing news related to
Showing 1–3 of 3 stories
The FDA has placed clinical holds on RegenXBio's investigational gene therapies RGX-111 and another for rare neurodevelopmental disorders, including mucopolysaccharidosis type I (Hurler syndrome). This decision follows the discovery of a neoplasm in a trial participant, impacting ongoing studies.
Read full storyRegenxbio's RGX-111, a gene therapy for Hurler syndrome, faces uncertainty after a cancer case was reported in its Phase 1/2 trial. This therapy aims to deliver a gene to central nervous system cells to produce the deficient enzyme, addressing the challenges of enzyme replacement therapy.
FDA has placed a clinical hold on Regenxbio's gene therapies RGX-111 and RGX-121 for severe mucopolysaccharidosis type I (MPS I) following an adverse event in a trial participant. This decision impacts the development of these therapies aimed at delivering the IDUA gene to improve patient outcomes.
Get the week's most important rare disease developments — pipeline moves, policy changes, and funding signals — delivered to your inbox every Monday.
Free weekly briefing. Unsubscribe anytime.