Milestones
- First patient dosed2023Complete.
- Dosing complete in adults and adolescents2026Complete.
- FDA filingTarget Q4 2026Not yet reached.
Most important updates
- 12 May 2026
Upcoming
- Q4 2026FDA filing (next)
- Q4 2026Planned US BLA submission for risto-cel (as early as end of 2026)
Current obstacles
- Same harsh chemo as CasgevyStill needs chemo to clear the marrow; Beam is working on a gentler antibody-based option.
Physics limits
- Edited stem cells need room: chemo clears the marrowBlood stem cells live in limited niches in the bone marrow. For edited cells to take over, the patient's own must be wiped out with chemotherapy like busulfan, which risks infections and infertility.
- Only so many stem cells can be collected and editedA patient gives a limited number of blood stem cells, and some are lost in editing and freezing. If too few survive, collection must be repeated, which stretches treatment to months.
- Base editors can only make certain letter swapsThey chemically turn C into T or A into G (and the matching pair): 4 of the 12 possible single-letter changes. They also act on nearby look-alike letters in a ~5-letter window.
How it works
Collect
Gather stem cells
Blood stem cells are collected from the patient's blood after a drug pushes them out of the bone marrow.
Edit
Mimic a harmless variant
A base editor changes letters in the switches of the fetal hemoglobin genes, copying natural variants that keep fetal hemoglobin on for life.
Return
Chemo, then infusion
Chemotherapy clears the marrow, and the edited cells are infused. They make red cells rich in fetal hemoglobin, which resists sickling.
Update log
Tue 12 May
- Minor: PressResearch
About Beam Therapeutics
Beam Therapeutics
Base editing, AATD, sickle cell
Base-editing company built on David Liu's lab work. Base editors change one DNA letter into another without cutting the DNA, fixing single-letter typos. Leads: BEAM-302 for alpha-1 antitrypsin deficiency and risto-cel for sickle cell.
- 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.
- Founded
- 20179 yrs
- Headquarters
- United States
- Status
- Public
- Listed
- BEAM (opens google.com)NASDAQ
- Valuation
- public
- Works in
- BiotechGene editing
- Coverage
- 2 programs · 6 updateslatest 8 Sep 2026checked 25 Sep
- People
- John Evans (opens beamtx.com)CEODavid Liu (opens en.wikipedia.org)Co-founder