Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.

The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease. 

Lead development candidate AGTC-501, is a gene therapy program in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises.

Find out more about us online at: www.beacontx.com. 



Syncona Partners LLP

Oxford Science Enterprises

Beacon Therapeutics

Beacon will be attending #BIO2024! Thank you to our team members representing Beacon - Amy Christenson Parker Davenport and Amy Adelman House. You can also see Amy House on the "Changing Clinical Trial Designs in the IRA Environment and Impacts on Stakeholders in Rare and Ultra-Rare Disease" panel on June 5.  
#genetherapy #raredisease #ophthalmology

We were excited to see Prof Robert MacLaren included in The Ophthalmologist Power List 2024. Robert is a scientific co-founder and board member at Beacon Therapeutics. Congratulations to Robert on this well-deserved honor!  https://lnkd.in/eqRjC89H

#ophthalmology

Beacon Therapeutics is attending the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Baltimore! Our team would be happy to discuss our mission to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. 

Thank you to Abraham Scaria Amy Adelman House Brendan M Zangari Bruno Doreste and Khalid Arhzaouy for representing Beacon at ASGCT 2024. 
#ASGCT2024 #genetherapy


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Beacon Therapeutics is an ophthalmic gene therapy company focused on saving and restoring vision in patients with various retinal diseases, including X-linked retinitis pigmentosa (XLRP), dry age-related macular degeneration (dAMD), and cone-rod dystrophy (CRD).

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Beacon Therapeutics