BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

Summary

This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD

Timeline

Start

2022-08-30

Primary completion

2028-02

Completion

2028-02

Publications

• Gupta AO, Sharma A, Frangoul H, Kanter J, Mapara MY, Dalal J, Alavi A, Jaroscak JJ, Ayala E, DiPersio JF, Ziga ED, Eapen M, Rifkin-Zenenberg S, Minella AC, Chen Y, Chesler S, Ambati S, Bowman TS, Habtemariam B, Joseney-Antoine M, Chockalingam PS, Lin L, Goyal S, Simon A, Thompson AA, Heeney MM; BEACON Investigators. Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease. N Engl J Med. 2026 May 7;394(18):1824-1835. doi: 10.1056/NEJMoa2504835. Epub 2026 Apr 1.
• Ligon JA, Cupit-Link MC, Yu C, Levine J, Foley T, Rotz S, Sharma A, Gomez-Lobo V, Shah NN. Pediatric Cancer Immunotherapy and Potential for Impact on Fertility: A Need for Evidence-Based Guidance. Transplant Cell Ther. 2024 Aug;30(8):737-749. doi: 10.1016/j.jtct.2024.06.006. Epub 2024 Jun 10.
• Sharma A, Young A, Carroll Y, Darji H, Li Y, Mandrell BN, Nelson MN, Owens CL, Irvine M, Caples M, Jerkins LP, Unguru Y, Hankins JS, Johnson LM. Gene therapy in sickle cell disease: Attitudes and informational needs of patients and caregivers. Pediatr Blood Cancer. 2023 Jun;70(6):e30319. doi: 10.1002/pbc.30319. Epub 2023 Mar 28.
• Persaud Y, Mandrell BN, Sharma A, Carroll Y, Irvine M, Olufadi Y, Kang G, Hijano DR, Rai P, Hankins JS, Johnson LM. Attitudes toward COVID-19 vaccine among pediatric patients with sickle cell disease and their caregivers. Pediatr Blood Cancer. 2023 May;70(5):e30274. doi: 10.1002/pbc.30274. Epub 2023 Mar 1.

Drugs

Indications