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Beacon Therapeutics Debuts With $120M to Develop Gene Therapies for Eye Diseases

NEW YORK – Beacon Therapeutics launched on Monday with £96 million ($120 million) in funding, which the firm will use to develop gene therapies for retinal diseases.

Syncona, a British investment trust focused on the life sciences space, is Beacon's lead investor; additional investors include Oxford Science Enterprises, which funds companies spun out from the University of Oxford. London-based Beacon, which has assets acquired by Syncona and preclinical research licensed from the University of Oxford, is focused on improving vision for patients with various retinal diseases that cause blindness.

Beacon's lead clinical asset is AGTC-501, a gene therapy in a Phase II trial, designed to treat X-linked retinitis pigmentosa (XLRP), a rare, inherited disease that's frequently caused by mutations in the RPGR gene. AGTC-501, which Syncona purchased as part of its Applied Genetic Technologies acquisition last year, aims to treat XLRP by expressing full-length RPGR protein.

Beacon said it expects to have a 12-month data readout from the AGTC-501 Phase II trial in the second half of this year and is awaiting feedback from the US Food and Drug Administration about the design of a planned Phase II/III trial of the gene therapy.

One of the company's preclinical assets, licensed from the University of Oxford, is for cone-rod dystrophy caused by a null mutation in the CDHR1 gene. Beacon also has access to a target generation technology platform that it will use to home in on secreted proteins of interest in the ophthalmology space. 

The preclinical asset originated in the lab of Robert MacLaren, a professor of ophthalmology at the University of Oxford who cofounded Beacon and who will serve as an adviser and a member of the firm's board of directors. The company will be led by David Fellows, former chief executive of Nightstar Therapeutics and an expert in the ophthalmology field.