Built from biotech parts, Beacon launches with $120M to develop eye gene therapies
12 Jun 2023
AcquisitionGene TherapyClinical Result
Backed by millions of dollars of funding and a team of drug industry veterans, a new biotechnology company aims to create genetic medicines for eye diseases that lead to blindness.
Beacon Therapeutics was founded earlier this year and has raised 96 million British pounds, or roughly $120 million, to support its mission. Its lead investor, Syncona Limited, is a London-based firm that focuses on the life sciences and has shown a particular interest in gene therapies for the eye. Syncona was a founding investor of two other startups dedicated to this research, Nightstar Therapeutics and Gyroscope Therapeutics, and last fall agreed to acquire another company in the space, Applied Genetic Technologies Corp.
Beacon now acts as a blend of those companies. Its most advanced gene therapy program comes from AGTC, and is in human testing as a potential treatment for a rare disorder called X-linked retinitis pigmentosa.
The disorder mainly affects males and is characterized by the destruction and loss of light-detecting cells in the eye, which ultimately causes blindness. A small trial evaluating AGTC’s therapy reported positive data last year, and Beacon hopes to confirm those results with a larger, Phase 2/3 study.
In addition to the AGTC program, Beacon says it’s working on two preclinical assets. The first targets “dry AMD,” a leading cause of vision loss in people older than 60. The therapy is delivered via an injection into the eye and relies on adeno-associated viruses, a type of benign virus commonly used to shuttle genetic medicine to cells in the body.
The second asset is meant to treat a group of conditions called cone-rod dystrophy. It was licensed from the laboratory of Robert MacLaren, an ophthalmology professor at the University of Oxford who is also a Beacon co-founder.
In a statement, Beacon also noted how it plans to further grow its slate of programs by using technology that will “identify, screen, and search secreted proteins in the ophthalmology space.”
“By acquiring AGTC and combining its late-stage program with highly complementary and innovative science from the University of Oxford and elsewhere, we have created an exciting new gene therapy company in an area where we have a great breadth and depth of knowledge and expertise,” said Chris Hollowood, CEO of Syncona.
Other investors, including Oxford Science Enterprises, have also funded Beacon.
Among gene therapy developers, the eyes have become an attractive research target. They are small and compact compared to other organs, and so may require less potent or smaller amounts of therapy. Over the last year or so, Regeneron Pharmaceuticals and the Swiss pharmaceutical giant Roche each inked deals with biotechs focused on ocular gene therapies.
Johnson & Johnson and partner MeiraGTx are also developing their own gene therapy for X-linked retinitis pigmentosa. The companies disclosed positive results from an early-stage study last June, and are currently dosing patients in a late-stage study set to produce data next year.
But alongside the investment, there have been setbacks. Multiple assets from Nightstar failed key clinical trials, and Biogen recently said that it has exited eye disease research while “refocusing” investments into gene therapy.
In April, Vedere Bio II, a small company specializing in eye gene therapies, shut down after just two years of operation because preclinical testing generated disappointing data. Another player in the space, Adverum Biotechnologies, turned to job cuts and a restructuring after clinical testing raised safety concerns about one of its most advanced therapies.
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