GENETIC DISEASE, INBORN
Clinical trials for GENETIC DISEASE, INBORN explained in plain language.
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Gene-Editing shot aims to halt rare nerve disease in phase 3 trial
Disease control Recruiting nowThis study tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a genetic disease that damages nerves. The goal is to see if the treatment can stop or slow the disease by lowering a harmfu…
Matched conditions: GENETIC DISEASE, INBORN
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 06, 2026 16:02 UTC
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Blood cell donors needed to advance 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 create better treatments f…
Matched conditions: GENETIC DISEASE, INBORN
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated May 15, 2026 11:57 UTC