GENETIC DISEASE, INBORN
Clinical trials for GENETIC DISEASE, INBORN explained in plain language.
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One-shot gene therapy aims to stop rare nerve disease in its tracks
Disease control Recruiting nowThis study tests a single dose of a gene therapy called NTLA-2001 in 60 people with a rare inherited nerve disease (ATTRv-PN). The goal is to see if it can safely reduce harmful protein buildup and slow nerve damage compared to a placebo. Participants must have a confirmed diagno…
Matched conditions: GENETIC DISEASE, INBORN
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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Blood cell donations fuel future immune disorder treatments
Knowledge-focused Recruiting nowThis study collects blood stem cells and immune cells from healthy volunteers and patients with immune or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advance treatments, not to…
Matched conditions: GENETIC DISEASE, INBORN
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated May 26, 2026 03:23 UTC