Editas Medicine Shifts Focus: Inherited Retinal Disease Programs Seek New Partnerships

Editas Medicine Shifts Focus: Inherited Retinal Disease Programs Seek New Partnerships

For individuals and families impacted by inherited retinal diseases (IRDs), news from the biotech sector can bring both hope and uncertainty. A significant announcement made on January 9, 2023, by Editas Medicine, a clinical-stage genome editing company, outlined a strategic reprioritization of its portfolio. This decision means the company will no longer internally invest in its inherited retinal disease programs, including those for Leber Congenital Amaurosis 10 (LCA10) and rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP).

This shift is particularly relevant to the IRD community as Editas Medicine has been a notable player in gene editing research for these conditions. The company stated it would now prioritize resources towards its lead clinical program, EDIT-301, for severe sickle cell disease and transfusion-dependent beta thalassemia, and focus its research and development on hemoglobinopathies and in vivo gene editing.

Key facts from the announcement include:

• Discontinuation of Internal IRD Investment: Editas Medicine is discontinuing internal investments in its inherited retinal disease programs, specifically mentioning EDIT-101 for LCA10 and EDIT-103 for RHO-adRP.
• Seeking Partnerships: The company intends to seek partnerships for the further development of its IRD programs.
• Focus on Hemoglobinopathies: Resources will be prioritized for EDIT-301, a program targeting severe sickle cell disease and transfusion-dependent beta thalassemia.
• Workforce Reduction: As part of the strategic reprioritization, Editas Medicine reduced its workforce by approximately 20%, which is expected to extend its cash runway into 2025.

For patients and families navigating the complexities of IRDs, this news means that while Editas Medicine will no longer be driving the internal development of these specific retinal gene therapies, the programs themselves are not necessarily abandoned. The intent to seek partnerships suggests that these promising research avenues could continue under new collaborations. For example, the Phase 1/2 BRILLIANCE trial for EDIT-101, which targeted LCA10, had previously demonstrated clinical proof of concept with a favorable safety profile and preliminary efficacy signals for some patients. In fact, a recent study published in the New England Journal of Medicine, led by investigators at Mass General Brigham in collaboration with Editas Medicine, indicated that 11 of 14 patients with IRD in the BRILLIANCE trial achieved improvements in vision with no serious side effects. The company had paused further enrollment in the BRILLIANCE trial in November 2022, citing the small patient population for homozygous IVS26 mutation and the decision to seek a collaboration partner for continued development of EDIT-101.

Looking ahead, the inherited retinal disease community will be watching for potential new collaborations or licensing agreements that could advance these gene editing therapies. The hope remains that these programs, which have shown early promise, will find new pathways to continue their development and potentially reach patients in need.