Treating LCA6 with an RPGRIP1 Gene Therapy

LCA6 Disease Overview

Leber congenital amaurosis (LCA) is a type of inherited eye disorder leading to vision loss at birth or in early childhood.

  • Accounts for 20% of blindness in school-age children
  • Vision-loss onset often seen at birth
  • Symptoms can include: profound vision loss, involuntary eye movements (nystagmus), crossed eyes (strabismus), and sensitivity to light (photophobia)

At least 27 different genes are associated with LCA. Mutations in both copies of the RPGRIP1 gene cause a spectrum of vision-related deficits most commonly diagnosed as LCA type 6 (LCA6). LCA6 patients are often completely blind from early childhood and have a rapid decline in function of the photoreceptor cells of the retina. The RPGRIP1 protein is needed for the normal function of the photoreceptors (rods and cones) of the retina, and loss of this protein is what causes the vision loss in patients. Vision loss caused by RPGRIP1 mutations is particularly devastating because of the rapid onset and progression.

Together we can prevent vision loss

Odylia is developing a gene therapy to treat vision loss caused by RPGRIP1 mutations, for which there is currently no treatment. This gene therapy uses the novel Anc80 vector technology developed by Odylia co-founder, Luk Vandenberghe, and builds upon proof-of-concept data generated at Massachusetts Eye and Ear in the labs of Eric Pierce and Luk Vandenberghe. Odylia is currently conducting late-stage preclinical experiments and is preparing for IND submission for use of the gene therapy in clinical trials. To move this program through IND, Odylia is seeking partnerships or philanthropic funding for the estimated $3.5 million of development costs required to advance the program through toxicology studies and fund clinical manufacturing costs.

Odylia is exploring hybrid models of funding which include donor support as well as private/commercial investment. We are committed to moving this gene therapy forward to ultimately prevent vision loss and are actively seeking partners and donors at this time. For more information on how you can support the Odylia Manufacturing Fund, click here. If your company may be interested in becoming a partner, please contact us here.

Clinical Advisory Group

Odylia has assembled a group of clinical thought leaders in the field of subretinally administered gene therapies.

Robert K. Koenekoop MD, MSc, PhD, FRCS(C), FARVO

Professor of Paediatric Surgery, Human Genetics and Adult Ophthalmology at McGill University
Director of the Laboratory for Retinal Genetics and Therapeutics
Chief Paediatric Ophthalmology

Montreal Children’s Hospital

Jiong Yan, MD

Associate Professor of Ophthalmology
Director, Vitreo-Retina Surgery Fellowship

Emory University School of Medicine

Tiansen Li, PhD

Senior Investigator, Retinal Cell Biology and Degeneration Section

National Eye Institute

Eric A. Pierce, MD, PhD

Director, Inherited Retinal Disorders Service, Massachusetts Eye and Ear
William F. Chatlos Professor of Ophthalmology, Harvard Medical School

Massachusetts Eye and Ear

Thaddeus (Ted) Dryja, MD

Professor of Ophthalmology, Harvard Medical School
Physician and Surgeon, Massachusetts Eye and Ear Infirmary

Massachusetts Eye and Ear Infirmary

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