A private biotech company, Beacon Therapeutics, based in London, UK (and Florida and Cambridge, USA), has received an investment of $170M from several funding partners, including Forbion, Syncona, Oxford Science Enterprises, TCGX and Advent Life Sciences. Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), is an ophthalmic gene therapy company “with a mission to save and restore the vision of patients with blinding retinal diseases”. The investment will be used to support the clinical development of “AGTC-501” (previously named as rAAV2tYF-GRK1-RPGR) – a gene therapy treatment for X-linked retinitis pigmentosa (XLRP) and generating a phase 1/2 clinical trial for a dry age-related macular degeneration (dAMD) program. The company was founded in 2023 to fund the acquisition of AGTC (Applied Genetic Technologies Corporation, Nasdaq: AGTC), a previous clinical-stage company focused on the commercialization of adeno-associated virus (AAV)-based gene therapies”.
XLRP, one of the most common forms of retinitis pigmentosa with mutations in one gene, the RP GTPase regulator gene (RPGR gene), is thought to account for approximately 75% of XLRP recorded cases. The gene encodes a ciliary protein that regulates trafficking of proteins to the outer segment of photoreceptors. Males are more severely affected by the X-linked pathology with night blindness generally occurring within the first decade of life, followed by restriction of the visual field and loss of visual acuity leading to legal blindness in most patients by the fourth to fifth decade of life. Unlike other approaches in the gene therapy space, AGTC-501 correctly expresses the full length RPGR protein, thereby addressing the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss.
Following the announcement, David Fellows, Chief Executive Officer of Beacon Therapeutics, said, “we are focused on progressing our pipeline of ophthalmic gene therapies to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness”. According to their press release, Beacon has raised approximately $290 million in funding to date. The funding was previously supported to dosing of the first patient in the “VISTA” registrational trial for AGTC-501, including the initiation of the phase II “DAWN” trial and the presentation of positive 12-month interim results of the phase 2 “SKYLINE” trial.
Beacon Therapeutics team is led by CEO David Fellows, previously at Nightstar Therapeutics Ltd., and Abraham Scaria, Chief Scientific Officer, previously at AGCT plc.
https://www.beacontx.com/about-us/