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Joint Development of Gene Editing Programs

IOB has established a research collaboration agreement with Beam Therapeutics, a Cambridge, Massachusetts-based biotechnology company developing precision genetic medicines through base editing.

Under the terms of the agreement, the partners will leverage IOB’s unique expertise in the field of ophthalmology along with Beam’s novel base editing technology to advance programs directed to the treatment of certain ocular diseases, including Stargardt disease.

Botond Roska, IOB Co-Director and Head of the Molecular Research Center and Bence György, Head of the IOB Translation Group in the Clinical Research Center will act as principal investigators for the program.

“The combination of IOB’s deep understanding of ophthalmic diseases with Beam’s precision approach to target single point mutations for disease, offers a promising avenue for the treatment of patients suffering from impaired vision,” said Botond Roska. “At IOB, we have developed proprietary human models to improve understanding of the retina, including how it is impaired in Stargardt disease. With this collaboration, we plan to explore the therapeutic opportunity of Beam’s novel base editing technology to precisely correct mutations that lead to Stargardt disease” explained Bence György.

Base editing represents an exciting new class of precision medicine, in which precise, single base changes in genes are made without cutting DNA, enabling a wide range of editing strategies, including correction, silencing, activation, modification and multiplexing. Through this collaboration, IOB will leverage insights from its research program to study the potential impact base editing could have on ocular diseases and translate this approach to the clinic.

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