Gene Therapy

Clinical Trials

Two clinical trials currently are open:

  1. CRISPR-SCD
  2. Gene Therapy to Reduce All Sickle Pain 

Funded through a four-year, $8 million grant from the California Institute for Regenerative Medicine, UCSF Alpha Stem Cell Clinic aims to accelerate the development and delivery of stem cell therapies to patients through high-quality clinical trials.

FDA-approved gene therapy/gene editing therapies

LYFGENIA - the longest-studied approved gene therapy for sickle cell disease 

CASGEVY - a gene therapy that is made by editing the BCL11A gene

Learn more about our Gene Therapy team.

Alpha Stem Cell Clinic Information