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COMPANY

Beacon Therapeutics

HQ: Cambridge, MA (US office) source · beacontx.com ↗

Clinical Phase 3

Our lead asset, laru-zova, is an investigational gene therapy program for the treatment of XLRP. From company website Source · Oct 13, 2026

Keywords: gene therapy xlrp X-Linked Retinitis Pigmentosa Achromatopsia X-linked Retinoschisis Alpha-1 Antitrypsin Deficiency Leber Congenital Amaurosis rAAV2tYF-GRK1-RPGR Adeno-associated virus vector expressing a human RPGR gene rAAV2tYF-GRK1-hRPGRco rAAV2tYF-CB-hRS1 AGTC-402 rAAV2tYF-PR1.7-hCNGB3 rAAV1-CB-hAAT rAAV2-CB-hRPE65

Location: Cambridge, MA (US office) · source · Oct 12, 2026

Profile checked Oct 10, 2026

$75.0MRaised (2 yrs)13Trials (company is sponsor)

Raises and deals from press releases

Raises announced in these releases: $245M disclosed.

From the company’s own releases; this list is partial. All raises and deals

Timeline

  1. Raise Private equity round — Series C: $75M (total, as published), announced Jan 8, 2026. Source: company release on GlobeNewswire. source ↗
  2. Raise Series C · more than $75M source ↗
  3. Trial start 2: A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa source ↗
  4. Raise Series B · $170M source ↗
  5. Trial start Phase 2/Phase 3: A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP source ↗
  6. Trial start Phase 2: A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN) source ↗
  7. Trial start Phase 2: A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) source ↗
  8. Trial start Phase 1/Phase 2: Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations source ↗
  9. Trial start —: Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP) source ↗
  10. Trial start Phase 1/Phase 2: Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial) source ↗
  11. Trial start Phase 1/Phase 2: Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial) source ↗
  12. Trial start 1: Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis source ↗
  13. Trial start Phase 1/Phase 2: Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS) source ↗
  14. Trial start —: Clinical and Genetic Characterization of Individuals With Achromatopsia source ↗
  15. Trial start —: Clinical Evaluation of Patients With X-linked Retinoschisis source ↗
  16. Trial start Phase 2: Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency source ↗

Upcoming milestones

Estimated primary completion per ClinicalTrials.gov. Dates move.

Funding

Raised: $75.0M. Last 2 years.

Clinical trials (lead sponsor) 13

RecordTitlePhaseStatusStartPrimary completion
NCT07174726A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa2Active, not recruitingSep 10, 2025Dec 31, 2029 (estimated)
NCT04850118A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRPPhase 2/Phase 3Active, not recruitingMar 14, 2024Jun 30, 2026 (estimated)
NCT06275620A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)Phase 2Enrolling by invitationNov 14, 20232025-11 (estimated)
NCT06333249A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)Phase 2Active, not recruitingApr 13, 2021Apr 11, 2023 (actual)
NCT03316560Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR MutationsPhase 1/Phase 2Active, not recruitingApr 16, 2018Nov 9, 2023 (actual)
NCT02416622Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)Phase 1/Phase 2Completed2015-052019-04 (actual)
NCT03314207Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)Not listedCompletedDec 1, 2017Feb 10, 2022 (actual)
NCT02935517Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)Phase 1/Phase 2Unknown status (ClinicalTrials.gov)Aug 3, 20172022-08 (estimated)
NCT02599922Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)Phase 1/Phase 2Unknown status (ClinicalTrials.gov)Apr 11, 20162022-07 (estimated)
NCT01054339Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin DeficiencyPhase 2Completed2010-062011-10 (actual)
NCT00749957Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis1CompletedJun 17, 2009Sep 23, 2014 (actual)
NCT02331173Clinical Evaluation of Patients With X-linked RetinoschisisNot listedCompleted2012-112016-10 (actual)
NCT01846052Clinical and Genetic Characterization of Individuals With AchromatopsiaNot listedCompleted2013-062017-04 (actual)
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