Syncona Limited has established Beacon Therapeutics Holdings Limited, a gene therapy company focused on restoring and improving vision in patients with prevalent and rare blinding retinal diseases including X-linked retinitis pigmentosa (XLRP).
Let’s start with Syncona.
Founded in 2012, London-based Syncona is a leading FTSE 250 healthcare company specializing in life science investments.
Note: FTSE (Financial Times Stock Exchange), commonly referred to as the “Footsie”, is the London Stock Exchange.
How does it tie into this new company?
Syncona, along with additional investors like Oxford Science Enterprises, raised $120 million (£96 million) to fund the acquisition of AGTC—a publicly-traded biotechnology company developing genetic therapies for rare inherited conditions—and provide capital for the newly-established Beacon Therapeutics.
And the purpose of Beacon Therapeutics?
The gene therapy company is focused on restoring and improving vision in patients with prevalent and rare retinal diseases that lead to blindness.
Gotcha. Now talk about its pipeline.
Beacon Therapeutics’ pipeline inventory includes AGTC-501, a late-stage and lead development candidate for XLRP (caused by mutations in the RPGR gene), as well as two Syncona-owned preclinical programs that target:
- Dry age-related macular degeneration (AMD)
- Cone-rod dystrophy (CRD), in-licensed from the University of Oxford.
According to the company, it also has access to a target generation technology platform that will be used to identify, screen, and search secreted proteins within the ophthalmology space.
Back up .. what do we know about those other two programs?
For dry AMD, the company is developing an intravitreally-delivered novel adeno-associated viruses (AAV)-based program that can be administered within an outpatient clinic rather than via surgery.
For CRD… the program targets mutations in the Cadherin Family Related Member 1 (CDHR1) gene—a potential cause for central vision loss (akin to dry AMD).
Additionally, Robert MacLaren, MB, ChB, DPhil, FRCOphth, FRCS, FACS, FMedSci, a professor of ophthalmology at the University of Oxford and co-founder of Beacon Therapeutics, is noted as an active advisor on the company’s CDHR1 program.
Now tell me about AGTC-501.
Originally developed by AGTC, AGTC-501 works by correctly expressing the full length RPGR gene protein and addressing the full complement of photoreceptor damage that XLRP causes (including both rod and cone loss).
Any clinical data on it?
Yup! According to the company, the phase 1/2 HORIZON trial reported that AGTC-501 demonstrated meaningful efficacy and a favorable safety profile.
Further, 12-month data from the completed phase 2 SKYLINE trial are expected to be published later in 2023.
What’s next?
The company is expecting to hear from the FDA regarding its upcoming phase 2/3 VISTA trial (NCT04850118), which will evaluate the efficacy of AGTC-501 in treating RP symptoms in additional patients with XLRP.