Beacon signals intent with $170m to advance gene therapies for retinal diseases

Significant Series B funding will enable clinical trials of dry AMD gene therapy delivered by intravitreal injection.

British-US biotech Beacon Therapeutics has successfully raised $170 million in a Series B funding round to advance its ophthalmic gene therapies aimed at treating retinal diseases. The funding will be used to advance its pipeline of therapies, including moving its dry age-related macular degeneration program into Phase 1/2 clinical trials.

With operations in London and Cambridge, MA, Beacon is developing gene therapy treatments designed to save and restore vision for patients suffering from retinal diseases that lead to blindness. The clinical stage company has completed multiple clinical trials that it says demonstrate the “precision, effectiveness and safety” of its gene therapy approach.

While a substantial portion of the new funds will support the clinical progression of Beacon’s lead program targeting the rare orphan disease X-Linked Retinitis Pigmentosa (XLRP), longevity watchers will be more interested in the company’s work in dry AMD, a leading cause of irreversible vision loss among people over the age of 60. The condition describes the gradual deterioration of the macula’s cells over many years, leading to significant visual impairment. Patients with dry AMD, particularly in its advanced stages, experience severe reductions in central vision, night vision difficulties, increased blurriness, distortion of straight lines and text, and a faded perception of colors.

Beacon’s dry AMD program employs a novel AAV-based gene therapy delivered via an intravitreal injection. The company claims this method of delivery is less invasive compared with traditional surgical approaches, requires less specialized training for clinicians, and can be administered in a clinical setting rather than an operating room, thus potentially making it more accessible to a broader patient population.

The financing round, which brings the total raised by Beacon to approximately $290 million, was led by Forbion, with participation from existing investors Syncona Limited, Oxford Science Enterprises, and the University of Oxford, as well as new investments from TCGX and Advent Life Sciences. The company also expanded its Board of Directors, appointing Dmitrij Hristodorov and Wouter Joustra from Forbion, and Cariad Chester from TCGX, while Dominic Schmidt from Advent Life Sciences joins as a Board Observer.

“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,” said David Fellows, CEO of Beacon. “I am confident that along with the addition of Dmitrij Hristodorov, Wouter Joustra and Cariad Chester to the Beacon Board, these funds will support the ongoing development of our late-stage and pre-clinical pipeline and enable acceleration of the development of AGTC-501 as we progress through the clinic and toward commercialization.”

Photograph: javi_indy/Envato