A critical mission of our center is to bring novel therapies to the affected patients. We structured our center to facilitate translational research into clinical trials with speed and caution. We have collaborated on and assisted with developing multiple ocular gene therapy INDs, documents required by the FDA before initiating a clinical trial. In particular, our collaborative work on Leber’s Congenital Amaurosis type 2 (LCA caused by RPE65 mutations) with the Children’s Hospital of Philadelphia (CHOP) has resulted in the only Phase 3 gene therapy study currently underway in the world.

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Geno/Phenotype

Geno/Phenotype

CAROT will advise investigators on steps that can be taken to obtain genetic diagnoses for various ophthalmic conditions on either a research basis or as a clinically certified test. Additional discussions will relate to establishing connections between genotype and phenotype.