The brief
- Mar 2025: BEAM-302 made the first-ever fix of a disease-causing DNA typo in patients, raising working AAT protein.
- A ~50-patient key trial group of BEAM-302 began in Jul 2026, aiming at fast-track approval.
- An FDA filing for risto-cel in sickle cell is planned as early as end of 2026.
Programs
Beam Therapeutics, two programs
Updated 8 Sep 2026BEAM-302 (AATD)Fat bubbles carry a base editor to the liver, where it fixes the one-letter typo behind alpha-1 antitrypsin deficiency, so the liver makes normal protein again.PilotNextFinish key group for a fast-track FDA filingUpdated 8 Sep 2026View program →Risto-cel (BEAM-101)A patient's own stem cells are base-edited to keep making fetal hemoglobin, like people born with a harmless natural variant, without cutting the DNA.PilotNextFDA filing · Q4 2026Updated 12 May 2026View program →
Research · Proto · Pilot · Deploy · Scaling
Research & demos
- Nov 2017paperProgrammable base editing of A-T to G-C in genomic DNA without DNA cleavage (Nature) (opens doi.org)
Gaudelli et al. introduced A-to-G base editors, the basis of BEAM-302.
- May 2016
Komor et al. introduced the first base editors (C to T).
Peers
Company & funding
- Listed
- BEAM (opens google.com)NASDAQ
- HQ
- US
- Founded
- 2017