Milestones
- First patient dosed in Phase 1/22024Complete.
- First data: first fix of a disease typo in patients10 Mar 2025Complete.
- Key trial group (~50 patients) first dosedJul 2026Complete.
- Updated data at ERS Congress 20268 Sep 2026Complete.
- Finish key group for a fast-track FDA filingNowCurrent milestone.
Most important updates
- 8 Sep 2026
- Jul 2026
- 7 May 2026
- 10 Mar 2025
Current obstacles
- Proving it helps patientsFast-track approval rests on protein levels over 12 months; proving lung and liver benefit takes far longer.
Physics limits
- 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.
- Fat-bubble delivery mostly reaches the liverIn the blood, lipid nanoparticles pick up a protein (ApoE) that liver cells absorb. That makes the liver easy to edit, but most other organs (brain, muscle, lungs) remain hard to reach with enough dose.
- Edits in the body are permanent and can't be recalledOnce liver cells are edited, the change lasts for life and is copied as cells renew. Any off-target cut or unexpected long-term effect can't be undone, so safety must be proven over years.
How it works
Deliver
Fat bubbles to the liver
Lipid nanoparticles carry mRNA for a base editor and a guide RNA into liver cells, where AAT protein is made.
Fix
Rewrite one letter
The base editor chemically changes the single wrong DNA letter (the 'Z' mutation in SERPINA1) back to normal, without cutting both DNA strands.
Effect
Protect lungs and liver
Corrected cells make working AAT that shields the lungs from enzyme damage, and stop making the misfolded form that clogs the liver.
Update log
Tue 8 Sep
- Minor: PressResearch
Jul 2026
- Minor: PressTest
Thu 7 May
- Minor: PressTest
Mon 10 Mar 2025
- Major: PressTest
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