Beacon Therapeutics

26/09/2025

We are excited to share that our CEO Lance Baldo was recently featured on the Eye on the Cure Podcast from the Foundation Fighting Blindness Thanks to host Ben Shaberman for a great discussion on the tremendous progress being made in ocular gene therapy. We’re proud that Beacon can help create the roadmap for how to build successful gene therapies and bring transformative treatments to patients.



Listen to the full podcast here: https://www.fightingblindness.org/podcasts/eyecurepodcast-39

In this week’s episode of the Eye on the Cure podcast, Lance Baldo, MD, CEO at Beacon Therapeutics, joins host Ben Shaberman to discuss the fully enrolled Phase 3 VISTA clinical trial for laru-zova, a gene therapy for X-linked retinitis pigmentosa (RPGR mutations) that has shown promising early results. They also dive into Beacon’s plans for a clinical trial targeting dry age-related macular degeneration.

Tune in now and share with your community to help spread the word about the progress being made in gene therapy for blinding diseases. https://bit.ly/4mwZabe

Image Description: Dark blue background with Foundation Fighting Blindness logo in the top left and Eye on the Cure Podcast logo in the top right. Teal half circle at the top center with "Ep. 92" inside. Below, text reads, "Listen to the latest podcast episode out now. September 25, 2025." To the left is Ben Shaberman with "Ben Shaberman, Host" beneath. To the right is Lance Baldo, MD with "Lance Baldo, MD, Guest" below.

28/02/2025

Beacon Therapeutics is proud to partner with rare disease patients and their families in recognizing , to raise awareness about rare diseases and their impact on the lives of the 300 million people worldwide living with a rare disease. To learn more, please visit www.rarediseaseday.org

08/07/2024

We are thrilled to announce the successful closing of our $170 million Series B, led by Forbion with contributions from existing investors Syncona Limited Oxford Science Enterprises and University of Oxford, as well as initial investments from TCGX and Advent Life Sciences. This represents a significant milestone in our journey to bring potentially transformative treatments to patients with blinding retinal diseases. The funds will be used to support the continued clinical development of AGTC-501 for X-Linked Retinitis Pigmentosa (XLRP) and our Dry Age-related Macular Degeneration (dAMD) program.

To read more about our Series B funding and pipeline momentum, click the link to the press release below.

https://www.beacontx.com/news-and-events/beacon-therapeutics-raises-170-million-in-series-b-funding-to-advance-development-of-ophthalmic-gene-therapies/

12/06/2024

Beacon Therapeutics is pleased to announce that the first patient has been treated in our Phase 2/3 VISTA clinical trial for our lead asset, AGTC-501. This is an important clinical milestone for AGTC-501 and another promising step toward a potential treatment for patients with X-Linked Retinitis Pigmentosa (XLRP), a blinding orphan disease for which there is no available treatment. VISTA is a randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 doses of AGTC-501 for the treatment of XLRP compared to an untreated control group.

To learn more about our ongoing VISTA trial, click the link to the announcement press release below.

Related posts: Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa Phase 2 Randomized, Controlled, Multicenter Clinical Trial (Skyline) 3-Month Results Beacon Therapeutics Announces Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis....

25/04/2024

We are excited to announce our Phase 2/3 Vista Clinical Trial is now open to enrollment. To learn more about our clinical trials and our science, visit beacontx.com.

29/02/2024

Today is Rare Disease Day! Beacon joins the rare disease patient community in recognizing to raise awareness amongst the general public and health care decision-makers about rare diseases and their impact on patients’ lives.

Beacon is proud to lead the fight against rare inherited retinal diseases. To learn more about , please visit: https://www.rarediseaseday.org.

08/02/2024

Beacon is excited to announce promising 12-month interim safety and efficacy results of the Phase 2 SKYLINE trial in patients with X-linked retinitis pigmentosa (XLRP). The 12-month data, presented by Mark Pennesi, M.D., Ph.D., FARVO, Director, Ophthalmic Genetics at the Retina Foundation at the 47th Annual Macula Society Meeting, represents another step toward a potential treatment for this devastating condition.

We look forward to announcing the initiation of our Phase 2/3 VISTA trial in the first half of 2024. To read more about the data, please visit our website: https://www.beacontx.com/news-and-events/

01/01/2024

Happy New Year! There is a lot to look forward to in 2024. Come check out what we are working on at

Beacon aids in restoring and improving the vision of patients with ophthalmic gene therapy. Find out more.

31/10/2023

We are excited to share that earlier this month, Professor Robert MacLaren presented interim safety data on the XLRP phase 1/2 gene therapy clinical trial. Visit www.beacontx.com to read his presentation

Our established scientific foundation combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP) with two innovative and differentiated preclinical programs for prevalent and rare blinding diseases.

25/10/2023

Beacon was a Proud sponsor of Foundation Fighting Blindness’s 16th Vision Walk! Thanks to the Foundation for another great year!

14/10/2023

We are proud to sponsor Black Ties & Blindfolds, an annual fundraising event hosted by MOMS for Sight, Inc.

MOMS for Sight is dedicated to raising awareness and funding sight saving research with Fighting Blindness!

Learn more at: https://www.momsforsight.org/2023-black-ties-and-blindfolds.html

13/10/2023

JOIN Beacon Therapeutics team members TOMORROW as we unite with tens of thousands of people who have taken important steps toward a cure by participating in the Foundation Fighting Blindness Fall VisionWalk. This event has raised over $60 million to fund sight-saving research.

Learn more on how to participate or donate here:
https://give.fightingblindness.org/event/boston-visionwalk-2023/e458266