GENETIC DISEASES, INBORN

Clinical trials for GENETIC DISEASES, INBORN explained in plain language.

Never miss a new study

Get alerted when new GENETIC DISEASES, INBORN trials appear

Sign up with your email to follow new studies for GENETIC DISEASES, INBORN, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.

Our safety recommendation!

By submitting, you agree to our Terms of use

Trials to join now! 3 Not yet recruiting 1 Not yet finished but already full! 1 Completed 1

CRISPR gene therapy: a Long-Term look at safety for blood disease patients

Disease control ENROLLING_BY_INVITATION

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). Researchers will monitor participants for years to check for side effects, new cancers, and how well the thera…

Matched conditions: GENETIC DISEASES, INBORN

Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control

Last updated May 13, 2026 16:00 UTC

GENETIC DISEASES, INBORN

Get alerted when new GENETIC DISEASES, INBORN trials appear

CRISPR gene therapy: a Long-Term look at safety for blood disease patients