Beacon Therapeutics

Beacon Therapeutics is proud to partner with rare disease patients and their families in recognizing #RareDiseaseDay, 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

We are pleased to welcome Martin Quinn to the Beacon team. Welcome Martin, who joins us as VP of Commercialization!

🚀 Beacon Therapeutics’ Laru-Zova Advances with FDA RMAT Approval Beacon Therapeutics’ Laru-Zova has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, marking a significant milestone in the fight against X-linked retinitis pigmentosa (XLRP). This designation accelerates development and review, bringing hope to patients with this progressive blinding disease. Backed by promising data from the Phase 2 DAWN and SKYLINE trials, Laru-Zova is designed to restore RPGR protein function, targeting both rod and cone photoreceptor degeneration. With additional Fast Track (FDA), PRIME (EMA), and ILAP (UK) designations, its potential impact on vision loss is undeniable. Read more here: https://lnkd.in/dtCKYw5Q #GeneTherapy #VisionLoss #XLRP #FDA #Innovation #ClinicalTrials #Ophthalmology #EyeHealth

We're #hiring a new Director Biostatistics - REMOTE in Cambridge, Massachusetts. Apply today or share this post with your network.

Beacon Therapeutics is pleased to announce the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (AGTC-501), an investigational gene therapy for patients with X-linked retinitis pigmentosa (XLRP). Learn more: https://lnkd.in/e5vFHVd3 #genetherapy #XLRP

Thanks to Oxford University Innovation for including our CEO, Lance Baldo, in this great event!

We are excited to be kicking off 2025 at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco next week. Our CEO, Lance Baldo, MD will be presenting on Tuesday, January 14. CFO, Tom Biancardi and Head of Portfolio Strategy, Amy Adelman House will also be attending. We look forward to sharing an update on the work we are doing at Beacon to bring pioneering ocular gene therapies to patients. #JPM25 #JPM2025 #genetherapy

𝐁𝐞𝐚𝐜𝐨𝐧 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬 𝐡𝐚𝐬 𝐚𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐝 𝐞𝐧𝐜𝐨𝐮𝐫𝐚𝐠𝐢𝐧𝐠 𝟑-𝐦𝐨𝐧𝐭𝐡 𝐢𝐧𝐭𝐞𝐫𝐢𝐦 𝐫𝐞𝐬𝐮𝐥𝐭𝐬 𝐟𝐫𝐨𝐦 𝐢𝐭𝐬 𝐏𝐡𝐚𝐬𝐞 𝟐 𝐃𝐀𝐖𝐍 𝐭𝐫𝐢𝐚𝐥 𝐞𝐯𝐚𝐥𝐮𝐚𝐭𝐢𝐧𝐠 𝐥𝐚𝐫𝐮-𝐳𝐨𝐯𝐚 (𝐀𝐆𝐓𝐂-𝟓𝟎𝟏) Beacon Therapeutics Beacon Therapeutics has announced encouraging 3-month interim results from its Phase 2 DAWN trial evaluating laru-zova (AGTC-501) in patients with X-linked retinitis pigmentosa (XLRP). The gene therapy was well-tolerated, with no treatment-related adverse events, including ocular inflammation. Notably, early improvements were observed in low luminance visual acuity (LLVA), a critical measure of visual function. These findings support the continued development of laru-zova as a potential treatment for XLRP, a severe inherited retinal disease that often leads to blindness by middle age and currently lacks approved therapies. Beacon Therapeutics is also enrolling patients in the pivotal Phase 2/3 VISTA trial to further assess laru-zova's efficacy and safety. #GeneTherapy #Ophthalmology #XLRP #ClinicalTrials #BeaconTherapeutics

According to data presented at #FLORetina: Laru-zova demonstrated positive safety and early efficacy in improving low luminance #visualacuity in patients with X-linked #retinitispigmentosa. Lead author Prof. Paulo Eduardo Stanga, MD, spoke with Healio🔗 #OcularSurgeryNews #Ophthalmology https://lnkd.in/eqjdvsGJ

Syncona portfolio company, Beacon Therapeutics, today announced the presentation of three-month data from the Phase II DAWN trial of its lead asset, laru-zova (AGTC-501). The data support the ongoing development of laru-zova in XLRP, a severe, aggressive, inherited retinal disease. With no treatment options available, XLRP impacts boys and young men and often leads to blindness by middle age. Read the full release here: https://lnkd.in/egZrDRsN #lifesciences #biotech #ophthalmology