Beacon Therapeutics raises $170M in series B to advance gene therapies for eye disease

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The fundraising will accelerate the clinical development of Beacon Therapeutics’ lead gene therapy candidate, which is currently in phase 2/3 trials.

Beacon Therapeutics has closed a $170 million series B round led by Forbion, and joined by Syncona, Oxford Science Enterprises, Oxford University, TCGX and Advent Life Sciences. The investment will support the development of Beacon’s lead asset, AGTC-501, as well as other programs in the company’s pipeline.

The mission of Beacon Therapeutics is to save and restore the vision of patients with retinal diseases that cause blindness. The company’s lead program targets X-linked retinitis pigmentosa (XLRP), a rare genetic condition for which there are no treatments approved.

XLRP is caused by a mutation in the gene that encodes the RPGR protein, which is essential for the development of photoreceptor cells in the retina. The loss of RPGR causes photoreceptor cells to die over time, resulting in the progressive loss of sight.

AGTC-501 is currently being evaluated as a treatment for XLRP in a pivotal phase 2/3 clinical trial. The gene therapy is designed to replace the missing RPGR protein in XLRP patients in order to halt vision loss.

“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, Chief Executive Officer of Beacon Therapeutics.

“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."

Part of the proceeds from the fundraising will go towards an upcoming phase 1/2 clinical trial focusing on dry age-related macular degeneration (dAMD), a retinal disease that causes progressive loss of vision. There is currently no medication that can reverse or stop the progression of the disease. 

Beacon Therapeutics was founded in June 2023 as a spin out of the University of Oxford. Its launch involved a £96 million fundraising, the biggest for any companies spun out of Oxford so far. The closing of the series B round comes just a year after Beacon’s foundation, bringing the total funds raised to date up to approximately $290 million.

The company is following the footsteps of Nightstar Therapeutics, another spinout of Oxford University that, like Beacon, was based on the work of Robert MacLaren, professor of ophthalmology at the University of Oxford and a world leader in ophthalmology. Founded in 2014, Nightstar was acquired five years later by Biogen for $800 million, which marked a record deal for the university.