Dr. Cherqui Update from UCSD Stem Cell and Gene Therapy Clinical Trial!

CRF has a positive update from the UCSD Stem Cell and Gene Therapy clinical trial! This trial is possible because of the dedication and commitment of CRF families and donors who supported the early research and continue to support this trial today. We are so thankful to the first three patients who volunteered for the trial. They have paved the way for the entire community. And thank you, Stéphanie Cherqui , PhD, for your brilliant work. We are changing lives together.

Dr. Cherqui, reports: “The three patients dosed in the ongoing stem cell gene therapy clinical trial show strong data across multiple measures and have remained off oral cysteamine and cysteamine eye drops since gene therapy dosing.

At one-year post-gene therapy, the vector copy number (VCN) of Patient #1 was at 0.9, and his leukocyte cystine level was 1.7 nmol half cystine/mg protein.  His level of cystine crystals in the skin and rectal tissue substantially decreased.  Additionally, imaging shows a marked reduction in crystal density in his corneas, as well as a two-grade clinically meaningful improvement (from grade 3 to grade 1) in his photophobia.

At 6-month post-gene therapy, the VCN for Patient #2 was 1.7, and his leukocyte cystine level was 1.3 nmol half cystine/mg protein.  Patient #3 visit at UC San Diego is coming soon.”

The next three patients should be transplanted this year. We will continue to keep you updated as the data is released.

Read the AVROBIO Press Release with highlights on Cystinosis Phase 1 -2 clinical trial.

Cystinosis: Continued positive data across multiple measures; all three patients remain cysteamine-independent post-gene therapy