The VISTA Registrational Trial will assess the effect of AGTC-501 on the vision and other symptoms of X-Linked Retinitis Pigmentosa in additional patients with XLRP.

AGTC-501 is an investigational gene therapy expressing the full length RGPR protein, thus addressing the full complement of photoreceptor damage caused by XLRP.

VISTA will enroll patients in the United States, with additional sites expected to open in multiple countries.

Beacon Therapeutics Holdings Limited ('Beacon Therapeutics' or 'the Company'), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced the treatment of the first patient in its registrational VISTA clinical trial with its lead asset, AGTC-501 (laruparetigene zovaparvovec).

VISTA is a global randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 dose levels of AGTC-501 for the treatment of X-Linked Retinitis Pigmentosa (XLRP) compared to an untreated control group. Beacon Therapeutics plans to use the data generated from the VISTA trial, in combination with data from the Phase 1/2 HORIZON and Phase 2 SKYLINE studies, including all available long-term data, to support a BLA in the US and a MAA in Europe for the treatment of patients with XLRP. AGTC-501 has been awarded an Innovative Medicine Designation (ILAP) in the UK, Priority Medicine (PRIME) in the EU, and Fast Track in the US for the treatment of X-linked Retinitis Pigmentosa caused by mutations in the RPGR gene.

Darin Curtiss, PharmD, Global Vice President of Clinical Development at Beacon Therapeutics, said, "This is an important milestone for the continued clinical development of AGTC-501, which offers the potential to transform the prognosis of patients with XLRP by enabling them to see better or maintain their current vision. We look forward to progressing AGTC-501 in the clinic and moving toward our goal of bringing a treatment to market for this life-altering disease."

Dr. David Birch, Scientific Director at Retina Foundation of the Southwest, said, "We are delighted to start the final phase of this important trial of gene therapy for X-linked retinitis pigmentosa. It will bring hope to thousands of young men affected by this disease. The results so far have been very encouraging."

XLRP is an orphan disease affecting approximately 17,000 patients in the US and EU5. XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age with no treatment options available. The disease is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein and is therefore expected to rescue function of both rod and cone photoreceptors, making it uniquely well-suited as a potential treatment to improve the lives of patients with XLRP.

For more information about the VISTA study can be found at



About Beacon Therapeutics

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.

The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting cone-rod dystrophy (CRD), an inherited retinal disease.

Lead development candidate AGTC-501, is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises.

Find out more about Beacon Therapeutics at


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Syncona Ltd. published this content on 12 June 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 12 June 2024 14:28:08 UTC.