RARE Daily

Beacon Launches with $120 Million to Develop Gene Therapies for Retinal Diseases

June 12, 2023

Rare Daily Staff

Beacon Therapeutics launched with $120 million (£96 million) in funding to develop a new generation of gene therapies to treat a range of rare and prevalent retinal diseases that result in blindness.

Syncona, along with additional investors including Oxford Science Enterprises, provided the funding to acquire AGTC, a biotech with a late-stage gene therapy candidate for X-linked retinitis pigmentosa (XLRP) that had struggled for funding, and take each of Beacon Therapeutics development candidates through to value inflection points.

Beacon’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, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men, is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. 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.

AGTC-501 has a strong body of clinical evidence targeting XLRP, demonstrating 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 US Food and Drug Administration 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.

“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,” said David Fellows, CEO of Beacon Therapeutics.

The company’s preclinical assets include an intravitreally (IVT) delivered novel AAV based program for dry age-related macular degeneration (dry AMD) and 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. MacLaren, a retinal expert and an experienced biotech company founder is a co-founder of Beacon Therapeutics and will become a key scientific advisor to the company and will join the board of directors.

To bolster its pipeline in the future, Beacon 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 will lead Beacon, and will be joined by retinal expert Nadia Waheed as chief medical officer, formerly from Gyroscope Therapeutics. Abraham Scaria 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.

Photo: David Fellows, CEO of Beacon Therapeutics

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