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