Jun 12, 2023

Beacon Therapeutics to Advance XLRP, Cone-Rod Dystrophy, and Dry AMD Gene Therapies

Eye On the Cure Research News

An emerging XLRP gene therapy acquired from AGTC is the company’s lead clinical program

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Beacon Therapeutics, a company focused on development of gene therapies for retinal diseases, has been launched with £96 million ($120 million) in investments from Syncona Limited, Oxford Sciences Enterprises (OSE), and additional partners. Initially, the new company will focus its development efforts on three emerging gene therapies:

  • The X-linked retinitis pigmentosa (RPGR) gene therapy program acquired from Applied Genetics Technology Corporation (AGTC) currently in the Phase 2 SKYLINE clinical trial. AGTC previously reported vision improvements for some patients in SKYLINE.
  • A preclinical cone-rod dystrophy (CDHR1) program licensed from the laboratory of Robert MacLaren, MB ChB, PhD, professor of Ophthalmology at the University of Oxford.
  • An intravitreally delivered gene therapy in preclinical development for the dry form of age-related macular degeneration (AMD).

Beacon will also complete data collection for the achromatopsia clinical trials (CNGA3, CNGB3) previously sponsored by AGTC, though it will not continue the development of either achromatopsia product candidates.

Beacon will be led by David Fellows, the former chief executive officer of Nightstar Therapeutics. Nadia Waheed, MD, formerly of Gyroscope Therapeutics, will be Beacon’s chief medical officer.  Abraham Scaria, PhD, formerly of AGTC,  will serve as Beacon’s chief scientific officer.

Dr. MacLaren is a co-founder of Beacon and will serve as a scientific advisor.