Odylia Therapeutics Granted Orphan Drug and Rare Pediatric Disease Designations by the FDA for OT-004, an Investigational Gene Therapy for RPGRIP1- associated Retinal Dystrophies

Atlanta, GA — November 16th, 2021

Odylia Therapeutics, a nonprofit biotech focused on developing gene therapies for rare diseases, announces that the US Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designation for Odylia’s lead gene therapy, OT-004. OT-004 is a novel gene therapy to treat vision loss caused by mutations in the RPGRIP1 gene. The designations are granted for the “treatment of RPGRIP1 mutation-associated retinal dystrophies” which most commonly includes Leber congenital amaurosis 6 (LCA6), but is also associated with diagnoses of cone-rod dystrophy 13 (CORD13) and forms of early-onset retinitis pigmentosa (RP).

“Receiving orphan drug and rare pediatric disease designations represents an important milestone for Odylia and recognizes the potential of this gene therapy to deliver life changing results to LCA6 patients and RPGRIP1 associated retinal dystrophies,” said Ashley Winslow, PhD, Odylia’s CSO.

Orphan drug designation is granted to therapeutics intended for treatment, diagnosis, or prevention of diseases affecting fewer than 200,000 people in the US. Rare pediatric disease designation is granted to serious or life-threatening rare diseases that primarily affect individuals under 18 years old. Orphan drug and rare pediatric designations provide companies with benefits including access to research grants to support clinical studies, waiver of regulatory fees, 7-year marketing exclusivity, and eligibility for a priority review voucher.

About LCA6 and RPGRIP1-associated retinal dystrophies

RPGRIP1 mutation-associated retinal dystrophies including LCA6, CORD13 and juvenile RP collectively are rare and serious vision loss disorders leading to progressive blindness in children and young adults. Homozygous and compound heterozygous loss-of-function mutations in the RPGRIP1 gene are most commonly associated with a LCA6, characterized by early infantile onset with a lack of recordable cone ERG by 12 months of age. Mutations in RPGRIP1 are also associated with CORD13 and some forms of juvenile RP, both with a later age-at-onset usually in childhood or early adolescence. There are currently no treatments for RPGRIP1-associated retinal dystrophies.

About OT-004

OT-004 is an investigational AAV gene therapy that utilizes the Anc80 AAV vector for efficient gene transfer. Anc80 is an ancestral adeno-associated virus discovered in 2015 in the lab of Luk H. Vandenberghe, at Massachusetts Eye and Ear. Dr. Vandenberghe’s laboratory discovered Anc80 through their efforts to identify safer, more effective viral vector-based delivery systems. Anc80 is predicted to be the ancestor of the broadly utilized AAV1, AAV2, AAV8, and AAV9, and can effectively transduce the retina, central nervous system, muscle, liver, and cells of the inner ear to name a few.

About Odylia Therapeutics

Odylia is a nonprofit biotech focused on bringing life-altering and lifesaving treatments to those with rare diseases. Our vision is to change how drugs are developed for rare diseases by focusing on the science and patient needs, rather than the commercial potential. We partner with the patient community, researchers, clinicians, and financial donors to bring promising therapeutics to clinical trials. Our programs include preclinical programs in RPGRIP1-associated retinal dystrophies and Usher Syndrome. For additional information, visit odylia.org, and follow us on Twitter and LinkedIn.