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New Nonprofit Biotech to Accelerate Therapies for Ultra-Rare Eye Diseases Launches on Rare Disease Awareness Day




(PRESS RLEASE) BOSTON, MA — Scientists and advocates announced the formation of Odylia Therapeutics, a nonprofit corporation dedicated to creating gene therapies for ultra-rare genetic conditions causing blindness. The company’s efforts will concentrate on developing laboratory findings into human clinical trials for select diseases, addressing an unmet need for drug development in an area where there is currently little commercial interest. Odylia Therapeutics is being launched with support from Massachusetts Eye and Ear and the Usher 2020 Foundation, a charitable organization focused on finding cures for rare eye diseases.

“With some genetic diseases affecting fewer than 1,000 patients worldwide per year, ultra-rare genetic blindness can be a risky investment for commercialization, leaving these patients with little hope for treatment in their lifetimes,” said Scott Dorfman, chief executive officer and co-founder of Odylia Therapeutics. “Given proof-of-concept evidence suggesting that successful gene therapies can safely restore or preserve vision, Odylia will focus on moving therapies for these conditions to clinical trial, giving hope to patients progressively going blind.”

Bringing Gene Therapy to Patients Losing Vision

Odylia Therapeutics’ initial focus will be on accelerating therapies for inherited retinal diseases, a group of conditions brought on by hereditary gene mutations that cause blindness due to degeneration of the retina, the light-sensing tissue at the back of the eye. More than 250 genetic forms of inherited retinal diseases have been identified.  A subgroup of these disorders is considered ultra-rare, and faces greater hurdles in the development of clinical treatments. Studies have shown pre-clinical proof-of-concept for correcting at least 30 of these gene mutations. While gene therapies comprise the company’s initial focus, Odylia may develop resources for other therapeutic approaches as opportunities arise.

In tackling these ultra-rare retinal diseases, the team hopes to fill a current void. Innovative treatments for many of these conditions have limited commercial appeal despite compelling studies that suggest a significant impact could be made on these patients’ vision and lives – leaving potential therapies behind in the lab.

Odylia Therapeutics develops strategies to move research programs from the lab to the clinic in a streamlined fashion by standardizing processes and documentation, centralizing regulatory guidance, vector manufacturing, pharmacology and safety testing, and clinical trial design and monitoring. In doing so, Odylia ultimately aims to expedite the regulatory process, work toward cost-savings in the challenging field of ultra-rare diseases, and to make these therapies more attractive for commercialization. Odylia’s innovative methods could be applied ultimately to a broader range of diseases.


“The progress in the field of retinal gene therapy has been astounding. New technologies, a deeper understanding of the science of retinal disorders and gene transfer, and clinical successes have created incredible momentum toward bringing possibly transformative medicine to patients,” says Luk H. Vandenberghe, Ph.D., co-founder of Odylia and Director of the Grousbeck Center for Gene Therapy at Mass. Eye and Ear and Harvard Medical School.  “We see that many rare ultra-retinal diseases unfortunately are not benefiting from this upsurge – in part understandably because of the limited commercial potential. Odylia is carefully designed to provide a path forward to reach clinical validation and eventually the patient.”

Led by Advocates and Experts on Genetic Eye Disease

Odylia Therapeutics is being launched with support from Massachusetts Eye and Ear, a specialty hospital of Harvard Medical School, and the Usher 2020 Foundation, a nonprofit dedicated to advancing research on Usher syndrome, a rare genetic condition causing blindness as well as hearing and balance impairment.

Odylia’s co-founders, who are deeply committed to its nonprofit mission, include world-leading scientists, clinicians and advocates for research in the area of genetic blindness.

  • Scott Dorfman, CEO and co-founder of Odylia Therapeutics
  • Luk H. Vandenberghe, Ph.D., Assistant Professor in Harvard’s Department of Ophthalmology and Director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear
  • Eric A. Pierce, M.D., Ph.D., Director of the Inherited Retinal Disorders Service at Massachusetts Eye and Ear, Director of the Harvard Ophthalmology Ocular Genomics Institute, and the Solman and Libe Friedman Professor of Ophthalmology at Harvard Medical School
  • Joan W. Miller, M.D., Chief of Ophthalmology at Massachusetts Eye and Ear and Massachusetts General Hospital, Chair of Ophthalmology and David Glendenning Cogan Professor of Ophthalmology at Harvard Medical School
  • John Fernandez, President of Massachusetts Eye and Ear

Joining Dorfman and Vandenberghe on Odylia Therapeutics’ Board of Directors are:

  • Alberto Auricchio, Ph.D., Associate Professor of Medical Genetics at Federico II University Napoli and Principal Investigator, TIGEM
  • Jean Bennett, M.D., Ph.D., Professor at the Perelman School of Medicine at University of Pennsylvania
  • Mathew Pletcher, Ph.D., Head of Rare Disease Discovery, Roche




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