BEAM Outlines Key Strategic & Pipeline Goals for 2026, Stock Rises

Shares of Beam Therapeutics BEAM were up 22.3% on Monday after the company outlined strategic priorities and some expected milestones related to its gene therapy pipeline candidates for 2026.

Beam Therapeutics’ genetic disease pipeline includes BEAM-301 and BEAM-302, which are being developed for the treatment of glycogen storage disease type 1a (GSD1a) and alpha-1 antitrypsin deficiency (“AATD”), respectively, in separate studies.

BEAM’s Recent Updates on BEAM-302 & BEAM-301

Beam Therapeutics’ lead genetic disease pipeline candidate, BEAM-302, is being evaluated in an open-label phase I/II study for treating AATD, addressing the underlying pathophysiology of both liver and lung disease. More than 25 AATD patients with lung and/or liver disease have been treated with BEAM-302 in the dose-exploration portions of the study to date.

AATD is an inherited genetic disorder that can cause early onset of emphysema and liver disease. Currently, there are no approved curative treatments for the given indication.

Per the latest update, BEAM has reached alignment with the FDA on a potential accelerated approval pathway for BEAM-302 based on AAT biomarker data evaluated over 12 months. To support a future BLA filing for BEAM-302, the company plans to enroll around 50 additional patients at the optimal biologic dose of BEAM-302 in an expansion of the ongoing phase I/II study.

Updated data from the phase I/II study and next steps for pivotal development of BEAM-302 are expected to be announced by the end of the first quarter of 2026.

The FDA has also accepted BEAM-302 into its Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program, which supports faster chemistry, manufacturing, and controls development for products with accelerated clinical timelines.

BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the disease-causing PiZ mutation.

Meanwhile, BEAM-301 is currently being evaluated in an open-label phase I/II dose-exploration study for treating patients with GSDIa. Initial data from the study is expected later in 2026.

Beam Therapeutics also plans to unveil its next clinical program for the liver-targeted genetic disease franchise in the first half of 2026.

BEAM's Price Performance

Several investors and Wall Street analysts expressed optimism around Beam Therapeutics’ update related to BEAM-302 as the company reached alignment with the FDA on a potential accelerated approval pathway. Investors also seemed impressed with the company’s other pipeline goals, which resulted in the stock price increasing on Jan. 12.

In the past six months, shares of Beam Therapeutics have rallied 59.1% compared with the industry’s increase of 21%.

BEAM's Recent Updates With Risto-Cel

Beam Therapeutics is developing its hemoglobinopathy candidate, ristoglogene autogetemcel (risto-cel, formerly known as BEAM-101), an ex vivo therapy being evaluated in the phase I/II BEACON study for treating sickle cell disease (“SCD”).

Last month, BEAM reported updated data from this study at the 2025 ASH annual meeting, which continued to show evidence of risto-cel’s differentiated treatment profile in SCD patients.

Additionally, Beam Therapeutics has also completed discussions with the FDA on the planned biologics licensing application (BLA) package for risto-cel, which is expected to follow the regulatory precedent set by previously approved SCD gene therapies. The company plans to submit a BLA for risto-cel as early as year-end 2026.

The FDA has also accepted risto-cel in its CDRP program.

Beam Therapeutics recently initiated dosing in a phase I healthy volunteer study evaluating BEAM-103, an experimental anti-CD117 monoclonal antibody for the treatment of SCD.

Beam Therapeutics ended 2025 with approximately $1.25 billion in cash, cash equivalents and marketable securities, providing significant financial flexibility to fund ongoing clinical programs. Management expects this cash balance to fund the company’s anticipated operating expenses and capital expenditure requirements into 2029 while also aiding the launch of risto-cel in SCD and execution of the BEAM-302 pivotal development plan in AATD.

BEAM's Zacks Rank & Stocks to Consider

Beam Therapeutics currently carries a Zacks Rank #4 (Sell).

Some better-ranked stocks in the biotech sector are Keros Therapeutics KROS, Exact Sciences EXAS and Immunocore IMCR, each carrying a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, 2026 loss per share estimates for Keros Therapeutics have narrowed from $3.65 to $3.47. In the past six months, shares of KROS have risen 29.6%.

Keros Therapeutics’ earnings beat estimates in three of the trailing four quarters, while missing the mark on one occasion. On average, the company delivered a surprise of 9,098.63%.

In the past 60 days, estimates for Exact Sciences’ 2026 earnings per share have moved up from $1.35 to $1.40. EXAS stock has surged 88.2% in the past six months.

Exact Sciences’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 352.28%.

In the past 60 days, 2026 loss per share estimates for Immunocore have narrowed from $1.18 to 90 cents. In the past six months, shares of IMCR have lost 7.5%.

Immunocore’s earnings beat estimates in three of the trailing four quarters, while missing the mark on one occasion. On average, the company delivered a surprise of 53.96%.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

This article originally published on Zacks Investment Research (zacks.com).

Zacks Investment Research