Beacon Therapeutics to develop gene therapies for retinal disease


Beacon Therapeutics Holdings Limited has been launched to develop gene therapies to treat rare and common retinal diseases that result in blindness.

Syncona 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 the development candidate Beacon Therapeutics through the value inflection point.

Beacon Therapeutics’ key clinical asset is AGTC-501, a gene therapy program currently in phase II clinical trials for the treatment of X-linked retinitis pigmentosa (XLRP) acquired as part of Syncona’s acquisition of AGTC in November 2022.

XLRP is mostly caused by mutations in the retinitis pigmentosa GTPase regulatory (RPGR) gene. Unlike other approaches in space, AGTC-501 correctly expresses the full-length RPGR protein, overcoming the full gamut of XLRP-induced photoreceptor damage, including loss of rods and cones.

About Beacon Therapeutics’ pre-clinical assets

Beacon Therapeutics’ first pre-clinical asset is a new intravitreal delivered (IVT) AAV-based program for dry age-related macular degeneration (dry AMD). Dry AMD is the leading cause of irreversible vision loss in people over 60 years old, if left untreated. About 20 million people in the US have AMD.

A second pre-clinical asset targets cone-rod dystrophy (CRD) caused by a null mutation in the Cadherin Related Family Member 1 (CDHR1) gene. This program has been licensed from the laboratory of Robert MacLaren, professor of ophthalmology at the University of Oxford. MacLaren will become the company’s principal scientific adviser and will join the board of directors.

To strengthen its channel going forward, Beacon Therapeutics also has access to a target-generation technology platform that will identify, screen, and search for secreted proteins in the ophthalmology suite.

Beacon Therapeutics will be led by David Fellows, former CEO of Nightstar Therapeutics. He will join Nadia Waheed as chief medical officer, formerly of Gyroscope Therapeutics. Abraham Scaria also joins the team from AGTC as chief scientific officer.

Fellows said: “Beacon Therapeutics combines extensive development pathways, deep scientific foundations, strong clinical networks, and a highly experienced management team to drive a unique late-stage clinical and pre-clinical pathway. With 12 months of data from our SKYLINE phase II trial for AGTC-501 expected soon and two highly innovative and differentiated pipeline assets for common and rare diseases of blindness, we are excited to establish a new leader in the ophthalmic gene therapy space.”

MacLaren added: “I have dedicated many years to translating pioneering gene therapies from the lab to the clinical trial phase with the support of my team at the Nuffield Laboratory of Ophthalmology and the NIHR Oxford Biomedical Research Center (BRC). Beacon Therapeutics is quite unique because it is a company that at its launch has been supported by excellent clinical trial data. The company has very innovative retinal gene therapy programs and the means to target them to the leading causes of blindness in both young and old.”

Extensive path of ophthalmic development

The company’s premier clinical gene therapy program AGTC-501 has strong clinical evidence targeting XLRP, a rare inherited form of retinitis pigmentosa that causes progressive vision loss in boys and young men. AGTC-501 demonstrated a significant efficacy and favorable safety profile in the recent HORIZON phase I/II trials and is expected to publish 12 months of data from the SKYLINE phase II trial in the second half of 2023. In addition, Beacon Therapeutics is currently awaiting feedback from the US Food and Drug Administration (FDA) regarding the design of the upcoming VISTA clinical trial study, a phase II/III study to assess the effect of AGTC-501 on symptomatic retinitis pigmentosa in additional patients with XLRP.

First preclinical program featuring intravitreal delivered (IVT) gene therapy for dry AMD. IVT delivery provides access to a much larger number of patients than sub-retinal delivery because it can be delivered in an outpatient clinic rather than requiring surgery.

The second preclinical program, CRD, targets mutations in the CDHR1 gene, which can cause central vision loss similar to dry AMD in the elderly and severe vision loss in younger patients.

About gene therapy for retinal disease

Since 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 want to dive deeper into the topic of gene therapy for blindness, we recommend reading the following article.


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