Beacon Therapeutics launches with goal to develop gene therapies for retinal diseases

12 Jun 2023
AcquisitionGene TherapyPhase 2
Beacon Therapeutics launched Monday with £96 million ($121 million) in funding, which was led by Syncona, to develop gene therapies for retinal diseases. The company's lead programme, AGTC-501 for late-stage X-linked retinitis pigmentosa (XLRP) programme, is currently in Phase II studies.
Syncona gained AGTC-501 as part of its acquisition of Applied Genetic Technologies Corporation (AGTC) last year, with Beacon's pipeline also including two preclinical programmes. "By acquiring AGTC and combining its late-stage programme with highly complementary and innovative science from the University of Oxford and elsewhere, we have created an exciting new gene therapy company," remarked Chris Hollowood, CEO of Syncona.
Along with Syncona, additional investors in Beacon include Oxford Science Enterprises, with the funding expected to provide enough capital to take each the development candidates through to value inflection points. One-year data from the Phase II SKYLINE study of AGTC-501 are anticipated in the second half, while Beacon is also awaiting feedback from the FDA regarding the design of the Phase II/III VISTA trial.
Meanwhile, Beacon's first preclinical asset is an intravitreally delivered AAV-based programme for dry age-related macular degeneration. The second preclinical asset - licensed from the University of Oxford - is targeting cone-rod dystrophy. The drugmaker will also utilise a target generation technology platform to identify, screen and search secreted proteins in the ophthalmology space to bolster its pipeline.
David Fellows, chief executive of Beacon, remarked "with the 12-month data from…SKYLINE…expected shortly and two highly innovative and differentiated pipeline assets for prevalent and rare blinding diseases, we are excited to be building a new leader in the ophthalmic gene therapy space." Fellows previously served as CEO of Nightstar Therapeutics.
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