Beacon Therapeutics Launches with Goal of Developing Gene Therapies for Retinal Diseases
Beacon Therapeutics has launched and will develop a new generation of gene therapies to treat a range of rare and prevalent retinal diseases that result in blindness.
Syncona Limited, with additional investors including OSE, has provided £96 million ($120 million) to fund the acquisition of AGTC and provide capital.
Beacon Therapeutics’s lead clinical asset is AGTC-501, a gene therapy program currently in phase 2 clinical trials for the treatment of XLRP that was acquired as part of Syncona’s acquisition of AGTC in November 2022. XLRP 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, thereby addressing the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss, according to a company news release.
Beacon Therapeutics’s first preclinical asset is an intravitreally (IVT) delivered novel AAV based program for dry age-related macular degeneration (AMD).
The second preclinical 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 Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford. Prof. MacLaren, a retinal expert, experienced biotech company founder, and a Co-Founder of Beacon Therapeutics, will become a key scientific advisor to the company and will join the board of directors.
To bolster its pipeline, Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.
Beacon Therapeutics will be led by David Fellows, the former CEO of Nightstar Therapeutics with over 40 years’ experience in the ophthalmology field. He will be joined by Dr. Nadia Waheed, MD, as Chief Medical Officer, formerly from Gyroscope Therapeutics. Abraham Scaria, PhD, also joins the team from AGTC as Chief Scientific Officer bringing 25 years of experience in gene therapy, ranging from discovery research to early-stage clinical trials.
“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," Mr. Fellows said in a company news release. "With the 12-month data from our phase 2 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.”
Ophthalmic Development Pipeline
The 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 1/2 HORIZON trial and expects to be publishing 12-month data from its phase 2 SKYLINE trial in the second half of 2023. In addition, Beacon Therapeutics is awaiting feedback from the FDA regarding the study design of its upcoming VISTA clinical trial, a phase 2/3 study to assess the effect of AGTC-501 on the symptoms of retinitis pigmentosa in additional patients with XLRP.
The 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.
The 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.
The company has a new location in London, complementing existing facilities in Boston and Alachua, Florida.