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Beacon Therapeutics Begins Registrational Gene Therapy Trial in Rare Retinal Disorder

NEW YORK – Beacon Therapeutics on Wednesday said the first patient with X-linked retinitis pigmentosa has received its experimental gene therapy AGTC-501 within a registrational trial.

AGTC-501, also known as laruparetigene zovaparvovec, is the company's lead asset under development for treating X-linked retinitis pigmentosa, a rare and inherited retinal disease that primarily affects men.

In the randomized-controlled registrational trial, known as VISTA, investigators will assess the safety and efficacy of AGTC-501 on vision and other symptoms. AGTC-501 is designed to express the full length of the RPGR protein, which plays an important role in vision. Mutations in the RPGR gene account for most cases of X-linked retinitis pigmentosa.

Patients who receive AGTC-501 within the VISTA trial will get one of two dose levels and be compared to an untreated control group.

Investigators are initially enrolling patients in the US but have plans to open sites in other countries.

Beacon, which launched last year and is headquartered in Alachua, Florida, said it will submit data from the VISTA trial in regulatory applications seeking AGTC-501's approval in the US and EU.