Beam Therapeutics used its presentation at the Wells Fargo 21st Annual Healthcare Conference on Tuesday, September 8, 2026, to outline progress across its base-editing pipeline. CEO John Evans joined Wells Fargo biotech analyst Yanan Zhu in a session that covered the company's alpha-1 antitrypsin deficiency, sickle cell disease, phenylketonuria and glycogen storage disease programs. Following the presentation, Beam shares traded at $26.63, with a market capitalization of $2.67 billion. The stock was up nearly 49% over the past year, and InvestingPro data showed a current ratio of 14.57, with the company holding more cash than debt.
In the alpha-1 antitrypsin deficiency program, BEAM-302, Evans said the company is getting patients into the mid-teens, with every patient at or above the 11 micromolar protective threshold. Baseline levels had been in the mid-single digits, while Cohort A and the 60 mg cohort reached mid-teens. Earlier reports cited 16.1 micromolar using LC-MS and 14.4 micromolar using turbidimetry. Evans said the effects have lasted 18 months or more after a single dose. Toxic Z protein fell by more than 80 percent, with specific reductions of 83 percent to 84 percent, and was reduced to under 10 percent. Functional M protein made up more than 90 percent of circulating protein, with one figure cited at 93 percent. Evans described the shift from an all-Z patient to a dominantly M patient as quite dramatic and said the profile supports a one-and-done approach because it restores a protected level while lowering Z and increasing M.
In Part B of the liver cohort, five patients were studied. AAT levels were in the mid-teens, around 13 micromolar, up from a baseline of about 4.7 micromolar, a roughly threefold increase. Safety events were mostly Grade 1 liver function test changes, alongside one patient with a Grade 3 AST and ALT rise that resolved within weeks. The company said there was no bilirubin elevation, no liver injury signal and no hospitalization. Cohort C, the pivotal Phase 2b cohort, dosed its first patient in July, and enrollment is taking place across 14 sites in six countries. Competitors mentioned included YolTech Therapeutics, Tessera, CRISPR Therapeutics and Prime Therapeutics; YolTech presented data at ERS with at least one patient in the mid-20s micromolar range and a mean around 20 micromolar.
For sickle cell disease, Beam discussed risto-cel, supported by a $500 million financing facility from Sixth Street Partners, with about $300 million expected to be drawn over time, roughly $100 million a year. The company targeted a biologics license application filing for year-end, a launch by the end of 2025 and revenue by the end of the decade. A total of 50 patients had been treated, including 30 patients matching the CASGEVY label required for filing; the 30th patient was dosed in July 2023. The VLC 12 endpoint was measured 15 months after dose in October 2024, and median vein-to-vein time was 4.5 months. Launch spending is expected to target a small number of treatment centers, specifically about 50 to 70 centers. The company referenced Vertex and CRISPR Therapeutics' CASGEVY and bluebird bio's LYFGENIA, which reached profitability in 2024, and described an in vivo program aimed at expanding access to as many as 100,000 sickle cell patients in the United States.
In phenylketonuria, BEAM-304 received IND clearance in mid-2024, with dosing expected to begin between year-end 2024 and early 2025. The first two editors are expected to cover about 45 percent of patients, with initial targets including the R408W allele and one additional allele. The clinical design includes dose escalation followed by a registration-enabling expansion cohort of about 50 patients. In glycogen storage disease type 1a, BEAM-301 targets the R83C mutation, the most common mutation, carried by about 300 of roughly 1,000 GSD1A patients. Dose-escalation cohorts with data are expected in 2026. The company mentioned Ultragenyx's approved gene therapy, which showed a 44 percent reduction in cornstarch use.
The presentation covered base editing across multiple rare diseases, with AAT data showing durability and the sickle cell program moving toward regulatory filing and commercial launch.
