Beacon Therapeutics Holdings Limited has been launched to develop gene therapies to treat a range of rare and prevalent retinal diseases that result in blindness. \n\n\n\nSyncona Limited, with additional investors including Oxford Science Enterprises, has provided £96 million ($120 million) to fund the acquisition of AGTC and provide capital to take Beacon Therapeutics’ development candidates through to value inflection points. \n\n\n\nBeacon Therapeutics’s lead clinical asset is AGTC-501, a gene therapy program currently in phase II clinical trials for the treatment of X-linked retinitis pigmentosa (XLRP) that was acquired as part of Syncona’s acquisition of AGTC in November 2022. \n\n\n\nXLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Unlike other approaches in the space, AGTC-501 correctly expresses the full length RPGR protein, addressing the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss. \n\n\n\nAbout Beacon Therapeutics’s pre-clinical assets\n\n\n\nBeacon Therapeutics’s first pre-clinical asset is an intravitreally (IVT) delivered novel AAV based program for dry age-related macular degeneration (dry AMD). Dry AMD is a leading cause of irreversible vision loss in people over 60, if left untreated. Around 20 million people in the U.S. suffer from AMD.\n\n\n\nThe second pre-clinical asset is targeting cone-rod dystrophy (CRD) which is caused by a null mutation in the Cadherin Related Family Member 1 (CDHR1) gene. The program has been licensed from the laboratory of Robert MacLaren, professor of ophthalmology at the University of Oxford. MacLaren will become a key scientific advisor to the company and will join the board of directors.\n\n\n\nTo bolster its pipeline in the future, Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.\n\n\n\nBeacon Therapeutics will be led by David Fellows, the former chief executive of Nightstar Therapeutics. He will be joined by Nadia Waheed as chief medical officer, formerly from Gyroscope Therapeutics. Abraham Scaria also joins the team from AGTC as chief scientific officer. \n\n\n\nFellows said: "Beacon Therapeutics combines a broad development pipeline, a deep scientific foundation, a strong clinical network, and a highly experienced management team to drive forward a unique late-stage clinical and pre-clinical pipeline. With the 12-month data from our phase II SKYLINE trial for AGTC-501 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."\n\n\n\nMacLaren added: "I have dedicated many years to translating pioneering gene therapies from the lab through to clinical trial phase with the support of my team in the Nuffield Laboratory of Ophthalmology and NIHR Oxford Biomedical Research Centre (BRC). Beacon Therapeutics is quite unique in being a company that at launch is already underpinned by excellent clinical trial data. The company has both highly innovative retinal gene therapy programmes and the means to target them to the main causes of blindness in both the young and old." \n\n\n\nBroad ophthalmic development pipeline\n\n\n\nThe company’s lead clinical gene therapy program AGTC-501 has a strong body of clinical evidence targeting XLRP, an inherited rare form of retinitis pigmentosa that causes progressive vision loss in boys and young men. AGTC-501 demonstrated meaningful efficacy and a good safety profile in the recent phase I/II HORIZON trial and expects to be publishing 12-month data from its phase II SKYLINE trial in the second half of 2023. In addition, Beacon Therapeutics is awaiting feedback from the US Food and Drug Administration (FDA) regarding the study design of its upcoming VISTA clinical trial, a phase II/III study to assess the effect of AGTC-501 on the symptoms of retinitis pigmentosa in additional patients with XLRP. \n\n\n\nThe first preclinical program features intravitreally (IVT) delivered gene therapy for dry AMD. IVT delivery provides access to a significantly greater number of patients than sub-retinal delivery as it can be administered within an outpatient clinic rather than requiring surgery. \n\n\n\nThe second preclinical program, CRD, targets mutations in the CDHR1 gene, which may cause central vision loss similar to dry AMD in older people and severe sight loss in younger patients.\n\n\n\nAbout gene therapy for retinal diseases\n\n\n\nSince the initial approval of the first gene therapy for blindness, a wave of companies has emerged, actively developing gene therapy treatments with the potential to cure various forms of genetic blindness. If you would like to delve deeper into the topic of gene therapy for blindness, we recommend reading the following article.