Gene Therapy
Clinical Trials
Currently, two clinical trials are open:
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
Gene Therapy lead
Alpha Stem Cell Clinic Information
