SCRI worked with 29/36 (81%) of the
drugs approved for adult malignancies in
2023 prior to the original FDA approval
29 of 36
71%
FDA Approves First Gene-Editing Therapy
In December 2023, the FDA approved the CRISPR-Cas9 gene-editing therapy developed by Vertex Pharmaceuticals
and CRISPR Therapeutics to treat patients with Sickle Cell Disease who are 12 years and older with recurrent vaso-
occlusive crises. Victoria Gray, the first participant in the revolutionary clinical trial, was treated at SCRI under the
care of Haydar Frangoul, MD, MS. Victoria, now more than four years after her treatment, has said, "The life that I once
felt like I was only existing in I am now
thriving in. I stand here before you today
as proof that miracles still happen."
Just six weeks later, the FDA approved
this therapy for the treatment of patients
12 years and older with transfusion-
dependent beta thalassemia.
"The life that I once felt like I was
only existing in I am now thriving
in. I stand here before you today as
proof that miracles still happen."
2013 — 2022
10 Year Growth
Phase I Accruals
861 1,476
81%
