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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549

 

FORM 8-K

 

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): January 11, 2026

 

 

Beam Therapeutics Inc.

(Exact name of Registrant as Specified in Its Charter)

 

 

Delaware

001-39208

81-5238376

(State or Other Jurisdiction
of Incorporation)

(Commission File Number)

(IRS Employer
Identification No.)

 

 

 

 

 

238 Main Street

 

Cambridge, Massachusetts

 

02142

(Address of Principal Executive Offices)

 

(Zip Code)

 

Registrant’s Telephone Number, Including Area Code: 857 327-8775

 

 

(Former Name or Former Address, if Changed Since Last Report)

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:


Title of each class

 

Trading
Symbol(s)

 


Name of each exchange on which registered

Common Stock, par value $0.01 per share

 

BEAM

 

Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

 


Item 2.02 Results of Operations and Financial Condition.

Although it has not finalized its full financial results for the fourth quarter and fiscal year ended December 31, 2025, Beam Therapeutics Inc. (the “Company”) announced in a press release on January 11, 2026 that it estimates that it had cash, cash equivalents and marketable securities of approximately $1.25 billion as of December 31, 2025.

The information contained in this Item 2.02 regarding the Company’s estimated cash balance as of December 31, 2025 is preliminary, unaudited and is subject to completion of the Company’s financial statement closing procedures. This estimate also does not present all information necessary for an understanding of the Company’s financial condition as of December 31, 2025 and its results of operations for the three months and year ended December 31, 2025. Accordingly, undue reliance should not be placed on this preliminary estimate.

The information in this Item 2.02 is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended (the “Securities Act”) or the Exchange Act, except as expressly set forth by specific reference in such a filing.

Item 7.01 Regulation FD Disclosure.

On January 11, 2026, the Company issued a press release announcing progress across its base editing portfolio and outlining key anticipated milestones. The full text of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference. In addition, on January 12, 2026, the Company updated its corporate presentation that it intends to use in connection with presentations at conferences and meetings, including an investor presentation at the 44th Annual J.P. Morgan Healthcare Conference on January 13, 2026. The slides from the Company’s corporate presentation are furnished as Exhibit 99.2 to this Current Report on Form 8-K and are incorporated herein by reference.

The information in this Item 7.01 (including Exhibits 99.1 and 99.2 attached hereto) is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Exchange Act or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference into any filing under the Securities Act or the Exchange Act, except as expressly set forth by specific reference in such filling.

Cautionary Note Regarding Forward-Looking Statements

Statements in this Current Report on Form 8-K (including the exhibits attached hereto) about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to: the Company's upcoming presentations at the 44th Annual J.P. Morgan Healthcare Conference; the therapeutic applications and potential of the Company's technology, including with respect to SCD, AATD and GSD1a; the Company's plans, and anticipated timing, to advance its programs and present data from ongoing clinical trials; the clinical trial designs and expectations for risto-cel, BEAM-103, BEAM-301 and BEAM-302; the Company's anticipated regulatory interactions and filings; the Company's estimated cash, cash equivalents and marketable securities as of December 31, 2025 and its expectations related thereto; the Company's potential receipt of additional cash consideration upon the release, if any, of certain escrows relating to the acquisition of Orbital Therapeutics by Bristol-Myers Squibb; the sufficiency of the Company's capital resources to fund operating expenses and capital expenditure requirements and the period in which such resources are expected to be available; and the Company's ability to develop life-long, curative, precision genetic medicines for patients through base editing. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: the Company's ability to develop, obtain regulatory approval for, and commercialize its product candidates, which may take longer or cost more than planned; the Company's ability to raise additional funding, which may not be available; the Company's ability to obtain, maintain and enforce patent and other intellectual property protection for its product candidates; the uncertainty that the Company's product candidates will receive regulatory approval necessary to initiate or continue human clinical trials; that preclinical testing of the Company's product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, the Company's clinical trials may take longer than expected; that the Company's product candidates, including the delivery modalities it relies on to administer them, may cause serious adverse events; that the Company's product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; whether the Company's actual audited results will be consistent with its estimated cash, cash equivalents and marketable securities as of December 31, 2024; and the other risks and uncertainties identified under the headings “Risk Factors Summary” and “Risk Factors” in the Company's Annual Report on Form 10-K for the year ended December 31, 2024, the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2025 and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this Current Report on Form 8-K.


Factors or events that could cause the Company's actual results to differ may emerge from time to time, and it is not possible for the Company to predict all of them. The Company undertakes no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

Exhibit

Description

99.1

Press Release dated January 11, 2026

99.2

Beam Therapeutics Inc. Corporate Presentation

104

Cover Page Interactive Data File (embedded within the Inline XBRL document)

 


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

 

 

BEAM THERAPEUTICS INC.

 

 

 

 

Date:

January 12, 2026

By:

/s/ John Evans

 

 

 

John Evans
Chief Executive Officer

 


EX-99.1 2 beam-ex99_1.htm EX-99.1 EX-99.1

 

 

 

Beam Therapeutics Sets Strategic Priorities for its Genetic Disease and Hematology Franchises to Drive Execution of Late-Stage Clinical Programs and Extends its Operating Runway through Commercial Transition

 

Alignment Reached with U.S. FDA on Potential Accelerated Approval Pathway for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) Based on Biomarker Endpoints

 

U.S. Biologics Licensing Application (BLA) Submission for risto-cel (Previously Known as BEAM-101) Expected as Early as Year-End 2026

 

Expansion of Liver-targeted Genetic Disease Franchise Underway with New Program to be Announced in First Half of 2026

Ended 2025 with Estimated $1.25 Billion in Cash, Cash Equivalents and Marketable Securities; Projected Operating Runway Extension into 2029 Supports Anticipated risto-cel Launch and Execution of BEAM-302 Pivotal Development Plan

 

Cambridge, Mass., January 11, 2026 – Beam Therapeutics Inc. (Nasdaq: BEAM) today announced continued progress toward its mission to build a sustainable, predictable model for the advancement of precision genetic medicines, highlighting recent updates for its liver-targeted genetic disease and hematology franchises and strategic priorities in 2026, supported by an extended anticipated operating runway.

 

“Over the past year, we have continued to demonstrate the power and consistency of our base editing platform as we work to redefine what is possible in genetic medicine,” said John Evans, chief executive officer of Beam Therapeutics. “Our approach is rooted in precision and predictability – designing one-time treatments to reverse genetic disease, executing against our portfolio priorities with discipline, and generating differentiated clinical data that compound across programs. As our science and company matures, each milestone strengthens the foundation for the next, enabling us to advance a growing pipeline of programs with increasing confidence and speed. We are now further extending this platform-driven rigor into regulatory execution, including alignment with the FDA on a potential path to accelerated approval for BEAM-302 and a planned BLA submission for risto-cel as early as the end of 2026. Backed by a strong balance sheet and expected cash runway into 2029, we believe we are uniquely positioned to translate scientific innovation into meaningful, lasting benefit for patients with serious diseases.”

 

Recent Pipeline Updates and 2026 Anticipated Milestones

 

Liver-targeted Genetic Disease Franchise

 

Beam is building a platform approach for single-course, precision gene editing therapies for liver-targeted genetic diseases by delivering base editors through intravenous infusion of lipid-nanoparticles (LNPs), a clinically validated technology for delivery of nucleic acid payloads to the liver.

 


 

 

 

BEAM-302: Beam’s lead genetic disease program is designed to be a best-in-class and first-in-class liver-targeting therapy for alpha-1 antitrypsin deficiency (AATD) that addresses the underlying pathophysiology of both liver and lung disease. In an open-label Phase 1/2 clinical trial, treatment with BEAM-302 demonstrated the first-ever clinical in vivo genetic correction of a disease-causing mutation and established clinical proof of concept in AATD.

To date, more than 25 AATD patients with lung and/or liver disease have been treated in the dose-exploration portions of the trial.
Beam has reached alignment with the U.S. Food and Drug Administration (FDA) on a potential accelerated approval pathway for BEAM-302 based on AAT biomarkers evaluated over 12 months. To support a future BLA submission, the company anticipates enrolling approximately 50 additional patients to be treated with the selected optimal biologic dose of BEAM-302 in an expansion of the ongoing Phase 1/2 study.
BEAM-302 has also been accepted into the FDA’s Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program aimed at facilitating CMC development of products with expedited clinical development timeframes.
Beam expects to report updated data from the Phase 1/2 trial and next steps for pivotal development by the end of the first quarter of 2026.

 

BEAM-301: BEAM-301 aims to correct the most common disease-causing mutation, R83C, in patients with glycogen storage disease type Ia (GSDIa). BEAM-301 has the potential to normalize blood glucose in these patients without continuous supplementation and improve key metabolic parameters.

BEAM-301 is currently being evaluated in an open-label Phase 1/2 dose-exploration trial in patients with GSDIa. Dosing is complete in the first cohort and enrollment has been initiated in the second cohort.
Beam expects to report initial clinical data in 2026.

 

Pipeline Expansion: Beam expects to disclose the next clinical program for its liver-targeted genetic disease franchise in the first half of 2026.

 

Hematology Franchise

 

Beam is pursuing a long-term, multi-wave development strategy for sickle cell disease (SCD), intended to progressively expand the reach of the company’s base editing approach to broader subsets of patients.

 

Risto-cel: Ristoglogene autogetemcel (risto-cel, formerly known as BEAM-101) is an investigational autologous cell therapy with a potential best-in-class profile for the treatment of SCD. The most recent data from the ongoing BEACON Phase 1/2 clinical trial presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition continue to show evidence of risto-cel’s differentiated treatment profile. Risto-cel has demonstrated a deeper resolution of SCD markers and reduced time in the hospital driven by a median of one cell collection cycle, rapid engraftment, and low number of neutropenic days.

 


 

 

Importantly, risto-cel’s predictable, robust manufacturing process led to fewer mobilizations and rapid delivery from cell collection to dose, potentially improving patient experience as well as treatment center capacity.

The adult and adolescent cohorts of the BEACON trial were fully enrolled in mid-2025.
Manufacturing of all doses was completed as of December 2025.
In addition, Beam completed interactions with the FDA on the anticipated BLA package for risto-cel, which is expected to align with regulatory precedent set by previously approved SCD gene therapies.
Risto-cel has also been accepted into the FDA’s CDRP program.
Beam expects to submit a BLA for risto-cel as early as year-end 2026.

 

Next-generation Programs in Sickle Cell Disease and Hematology: Beam continues to make significant investments in developing targeted LNPs for delivery of genetic payloads outside of the liver. Based on recent advances using this technique to deliver gene editing to hematopoietic stem cells (HSCs), Beam is now prioritizing in vivo delivery for its next wave approach in SCD, complementing risto-cel as a potential best-in-class ex vivo therapy. Beam’s long-term goal is to provide accessible, transformative genetic therapies for all patients with SCD. Beam is also continuing development of its proprietary ESCAPE platform, a powerful technology to potentially enable non-genotoxic treatment strategies that can be delivered either ex vivo or in vivo, including as part of any future in vivo program for SCD.

The ongoing Phase 1 healthy volunteer clinical trial of BEAM-103, an anti-CD117 monoclonal antibody (mAb) that enables ESCAPE, is expected to complete dosing in the first half of 2026.
Multiple targeted LNPs for HSC delivery have been identified and are in lead optimization.

 

Cash Position and Updated Operating Runway

Beam estimates that it had $1.25 billion in cash, cash equivalents and marketable securities as of December 31, 2025. This estimate is inclusive of the $255.1 million in closing cash consideration received from the acquisition of Orbital Therapeutics by Bristol-Myers Squibb. In addition, Beam has the right to receive up to approximately $26.3 million in additional cash consideration upon the release, if any, of certain escrows from the transaction. This estimate of cash on hand is preliminary, unaudited and subject to completion of Beam’s financial statement closing procedures. This estimate does not present all information necessary for an understanding of Beam’s financial condition as of December 31, 2025, and its results of operations for the three months and year ended December 31, 2025. Accordingly, undue reliance should not be placed on this preliminary estimate.

 

Beam now expects that its estimated cash, cash equivalents and marketable securities as of December 31, 2025, will enable the company to cover its anticipated operating expenses and capital expenditure requirements into 2029, funding the company through the anticipated launch of risto-cel in SCD and execution of the BEAM-302 pivotal development plan in AATD.

 

J.P. Morgan Healthcare Conference

 


 

 

Management will present and discuss Beam’s pipeline and business updates during a presentation at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026, at 5:15 p.m. PT. A live webcast will be available in the investor section of the company’s website at www.beamtx.com and will be archived for 60 days following the presentation.

 

About Beam Therapeutics

Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.

 

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: our upcoming presentations at the 44th Annual J.P. Morgan Healthcare Conference; the therapeutic applications and potential of our technology, including with respect to SCD, AATD and GSDIa; our plans, and anticipated timing, to advance our programs and present data from ongoing clinical trials; the clinical trial designs and expectations for risto-cel, BEAM-103, BEAM-301 and BEAM-302; our anticipated regulatory interactions and filings; our estimated cash, cash equivalents and marketable securities as of December 31, 2025 and our expectations related thereto; our potential receipt of additional cash consideration upon the release, if any, of certain escrows relating to the acquisition of Orbital Therapeutics by Bristol-Myers Squibb; the sufficiency of our capital resources to fund operating expenses and capital expenditure requirements and the period in which such resources are expected to be available; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the uncertainty that our product candidates will receive regulatory approval necessary to initiate or continue human clinical trials; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates, including the delivery modalities we rely on to administer them, may cause serious adverse events; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; whether our actual audited results will be consistent with our estimated cash, cash equivalents and marketable securities as of December 31, 2025; and the other risks and uncertainties identified under the headings “Risk Factors Summary” and “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2024, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, and in any subsequent filings with the Securities and Exchange Commission.

 


 

 

These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

 

Contacts:

Investors:

Holly Manning

Beam Therapeutics

hmanning@beamtx.com

 

Media:

Josie Butler

1AB

josie@1abmedia.com

 


EX-99.2 3 beam-ex99_2.htm EX-99.2

Slide 1

The Power of Predictability JANUARY 2026 NASDAQ: BEAM


Slide 2

Cautionary note regarding forward-looking statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding: the therapeutic applications and potential of our technology, including with respect to SCD, AATD, GSDIa and beta-thalassemia; our plans, and anticipated timing, to advance our programs, including the clinical trial designs and expectations for risto-cel, BEAM-103, BEAM-301 and BEAM-302; our plans and anticipated timing to present data from ongoing clinical trials; our anticipated regulatory interactions and filings; our current expectations and anticipated results of operations, including our expected use of capital; the sufficiency of our capital resources to fund operating expenses and capital expenditure requirements and the period in which such resources are expected to be available; and the therapeutic applications and potential of our technology, including our potential to develop lifelong, curative, precision genetic medicines for patients through base editing, including potential safety advantages, all of which are subject to known and unknown important risks, uncertainties and other factors that may cause our actual results, performance or achievements, market trends, or industry results to differ materially from those expressed or implied by such forward-looking statements. Therefore, any statements contained herein that are not statements of historical fact may be forward-looking statements and should be evaluated as such. Without limiting the foregoing, the words "anticipate," "expect," "suggest," "plan," "vision," “strategy,” “possibility,” “promise,” "believe," "intend," "project," "forecast," "estimates," "targets," "projections," "potential," "should," "could," "would," "may," "might," "will," and the negative thereof and similar words and expressions are intended to identify forward-looking statements. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of our clinical trials may take longer than expected; that our product candidates or the delivery modalities we rely on to administer them may cause serious adverse events; the uncertainty that our product candidates will receive regulatory approval necessary to initiate or continue human clinical trials, that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the headings "Risk Factors Summary" and "Risk Factors" and elsewhere in our annual report on Form 10-K for the year ended December 31, 2024, our quarterly reports on Form 10-Q, and in any subsequent filings with the Securities and Exchange Commission (the "SEC") which are available on the SEC's website at www.sec.gov. Additional information will be made available by our annual and quarterly reports and other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date of this presentation. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law. .


Slide 3

Our vision is to provide lifelong cures for patients suffering from serious diseases POTENTIAL FOR one-time, curative therapies PLATFORM FOR rapidly programmable precision medicines GENE EDITING FOR rare and common diseases


Slide 4

LESS GENOTOXICITY than traditional gene editing DURABLE correction for one-time cures CONSISTENT gene sequence outcomes Predictable, ReproducibleOutcomes for Patients Beam was founded based on a simple concept with profound implications BASE EDITINGTECHNOLOGY


Slide 5

Predictability as a driver of progress: The potential to ripple through the broader healthcare ecosystem Predictable, ReproducibleOutcomes for Patients Streamlined R&D Cycles Reduced development risk Regulatory Acceleration Efficient regulatory pathways Physician Confidence Predictable safety and durability Clarity in treatment decisions Patient Experience Improvement Reliable therapeutic outcomes Enhanced quality of daily living Payer & System Impact Sustainable, outcomes-aligned payer models Reduced lifetime healthcare utilization


Slide 6

PRECISION GENETIC MEDICINES PLATFORM Underpinned by a unified culture that operates with integrity and transparency The power of predictability: Beam is building a reliable model for advancing genetic medicine Multiple growing, high-value franchises Active pipeline expansion Creative platform partnerships REUSABLE delivery technology CONSISTENT preclinical and clinical outcomes HIGHLY SCALABLE internal manufacturing FLEXIBLE emerging regulatory framework Easily ADAPTABLE to new targets


Slide 7

Establishing a foundation of financial strength for sustainable growth Wholly owned pipeline Significant addressable markets Sustainable,platform-enabled pipeline expansionand growth $1.25 billion in cash as of Dec. 31, 2025*, inclusive of $255.1 million proceeds from Orbital acquisition Expected operating runway into 2029 Anticipate funding through risto-cel launch and execution of BEAM-302 pivotal development plan Financial Strength Focused Investment Clear Path to Value and Sustainable Growth Optimized spend and expense management strategy in place Efficient commercial build for potential risto-cel launch Accelerated pathway for BEAM-302 development *Inclusive of cash, cash equivalents, and marketable securities. This estimate is preliminary, unaudited and is subject to completion of Beam’s financial statement closing procedures.


Slide 8

Significant progress in 2025 against all key value drivers Completed $500M financing and runway extension into 2029 Advanced next wave programs Exceeded clinical enrollment and timeline targets Gained FDA regulatory alignment across late-stage programs Presented differentiated, best-in-class clinical data for risto-cel in SCD Achieved first human proof of concept for in vivo gene correction with BEAM-302 2025


Slide 9

Liver-targeted Genetic Disease Franchise


Slide 10

Rapidly advancing and growing a portfolio of liver-targeted in vivo programs for genetic diseases PROGRAM DISEASE DELIVERY EDITING APPROACH RESEARCH LEAD OPTIMIZATION IND ENABLING PHASE I/II PIVOTAL BEAM-302 Alpha-1 antitrypsin deficiency (AATD) In vivo LNP Correction of E342K mutation BEAM-301 Glycogen storage disease type Ia (GSDIa) In vivo LNP Correction of R83C mutation Undisclosed Undisclosed In vivo LNP Undisclosed Industry-leading LNP capabilities Platform synergies well positioned for novel regulatory pathways Potential best-in-class and first-in-class AATD program Near-term pipeline expansion LNP well tolerated clinically LNP = lipid nanoparticle


Slide 11

Severe AATD (PiZZ genotype) impacts >100,000 individuals in the U.S. with limited treatment options Single G to A point mutation in the SERPINA1 gene(PiZ or “Z” mutation) Routine COPD care IV augmentation therapy given weekly is only approved option   Supportive care and liver transplant for advanced disease No approved treatments for liver disease Progressive liver disease with fibrosis and cirrhosis due to: Aggregation and accumulation of mutant Z-AAT Progressive lung disease due to: Low and poorly functioning systemic Z-AAT levels Circulating Z-AAT aggregates, causes inflammation


Slide 12

BEAM-302 has the potential to be first, single-course treatment to address full spectrum of disease manifestations of AATD Durable, single-course treatment Address both lung and liver manifestations Liver produces M-AAT for the first time Significantly reduce Z-AAT Total AAT above 11µM protective threshold Increased total AATis functional AAT increases with inflammatory response Correction at root cause of disease Restore physiologic control of AAT GOALS OF BEAM-302 TREATMENT


Slide 13

BEAM-302 directly corrects the mutation causing AATD, with initial clinical data delivering potential best-in-class profile Total AAT in circulation at Day 28, mean 12.4µM % change in Z-AAT at Day 28, mean -78% Corrected M-AAT reached >90% of total circulating AAT at Day 28 Significant reduction in Z-AAT in circulation M-AAT comprises majority of total AAT in circulation Total AAT above protective threshold of 11µM Data from Feb. 28, 2025 datacut: 60 mg dose of BEAM-302, n=3


Slide 14

Rapid execution of BEAM-302 Phase 1/2 trial sets up opportunity for accelerated pivotal development path Most advanced genetic medicine clinical program for AATD >25 patients dosed across cohorts to date First U.S. sites open Significant patient and physician enthusiasm Part A: AATD-associated Lung Disease Part B: AATD-associated Liver Disease with or without Lung Disease Assess early safety and efficacy and identify optimal dose for pivotal study Dose Exploration Dose Exploration 30 mg, 60 mg Dose Exploration 15 mg, 30 mg, 60 mg, 75 mg, 2x-60 mg Updated clinical data across all cohorts expected by end of first quarter 2026


Slide 15

Beam intends to pursue accelerated approval pathway for BEAM-302 based on FDA feedback to date Alignment reached with FDA on a potential accelerated approval pathway for BEAM-302 in AATD Primary endpoint expected to be based on AAT biomarkers evaluated over 12 months Anticipate enrolling approximately 50 additional patients in expansion of open-label Phase 1/2 trial Accepted to FDA CMC Development and Readiness Pilot (CDRP) program – for programs with expedited clinical development timelines Clinical proof of concept U.S. IND clearanceINTEREST RMAT Designation Orphan Drug Designation FDA alignment Q2 2025 Q1 2025 Q4 2025 IND = investigational new drug; RMAT Designation = Regenerative Medicine Advanced Therapy Designation


Slide 16

KEY ENDPOINTS Safety and tolerability Time to hypoglycemia during fasting Changes from baseline in starch supplementation Phase 1/2 Open-label, single-ascending dose trial Cohort 1 20 mg N = 3 Cohort 2 40 mg N = 3-6 Add’l Cohort(s) N = 3-6 BEAM-301 Phase 1/2 trial in GSDIa patients with R83C mutation designed to achieve early clinical proof of concept Orphan disease Inability to convert glycogen back to glucose to sustain blood sugar while fasting Constant risk of hypoglycemia that can result in seizures, coma or death Standard of care is cornstarch taken every 2-4 hours, even overnight Correct liver G6PC mutation to restore enzyme activity and enable normal glucose metabolism Animal studies suggest ~11% enzyme activity sufficient for restoring metabolic profile Same LNP as BEAM-302 Glycogen Storage Disease Type Ia BEAM-301 Potential Plan to report initial clinical data in 2026


Slide 17

Hematology Franchise


Slide 18

Multi-wave strategy in SCD focuses on near-term ex vivo commercial opportunity, followed by in vivo delivery for maximum scalability ESCAPE technology has potential broad utility Severe SCD gene therapy market (~10K patients) poised for growth with significant demand in U.S. Clinical differentiation of risto-cel from base editing and manufacturing process   In vivo HSC editing progress, prioritized for next wave program PROGRAM DISEASE DELIVERY EDITING APPROACH RESEARCH LEAD OPTIMIZATION IND ENABLING PHASE I/II PIVOTAL Risto-cel Sickle cell disease (SCD) Ex vivo HSC Activation of fetal hemoglobin (HbF) BEAM-103 SCD Beta-thalassemia Ex vivo or in vivo HSC CD117 edit- antibody (ESCAPE) In vivo HSC editing SCD Beta-thalassemia In vivo LNP Activation of HbF


Slide 19

BEACON results provide evidence of potential differentiation and best-in-class profile of base editing and risto-cel for severe SCD Rapid neutrophil and platelet engraftment Low number of neutropenic days Less Timein Hospital HbF levels >60% and HbS levels <40%, comparable to sickle trait Resolution of anemia Markers of hemolysis and oxygen delivery normalized or improved Deeper Resolution of SCD Median of 1 cell collection cycle Consistently high yields and viability Median 2.9 months from start of mobilization to drug product release Beam NC facility allows flexible scheduling for patients and treatment centers Predictable Manufacturing, Fast Patient Delivery Data from BEACON trial presented at ASH 2025


Slide 20

Major bottlenecks for SCD gene therapy commercial adoption are limited manufacturing capacity and low process efficiency We have patients knocking at the door every day. Current manufacturers cannot handle the volume of patients. – Southeast U.S. KOL “ “ Patient waiting lists at major centers 70+ treatment centers active Patient outcomes have been positive Pricing support and no payment rejections to date Inefficient, unpredictable process Multiple cell collection cycles Manufacturing slots fully booked PATIENTINTEREST TREATMENTCENTERS PATIENT OUTCOMES CELL COLLECTION & MANUFACTURING REIMBURSEMENT Source: Beam market research, competitive intelligence, sell side research and ASH 2025 data


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APPROVAL TRANSPLANT COLLECTION MANUFACTURING 17 days (median) to neutrophil engraftment* Internal manufacturing with optimized process and product release Predictable scheduling and consistent process outcomes Faster process can also expand market: higher throughput per center End-to-end, efficient risto-cel process designed to reduce burden and optimizes experience for patients and treatment centers With a more predictable and shorter process overall, we could treat more patients per year. -Northeast KOL “ “ Conditioning & GTx Infusion DrugProduct Manufacturing Release Testing Mobilization, Apheresis & Shipment Prior Auth/Payer Approval Release from Hospital 2.9 months (median) Start of mobilization to drug release* 4.5 months (median) Start of mobilization to dosing* 1 cycle per patient (median)* 19 days (median)to platelet engraftment* TOTAL: 4-6 months * BEACON trial data


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Preparing for BLA submission and a potential best-in-class launch in a growing market Engaged with regulators in 2025 to provide clarity on BLA package Completed manufacturing of all doses in BEACON trial Plan to submit risto-cel BLA packageas early as YE 2026 Initiate efficient commercial build to support potential risto-cel launch


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In vivo HSC Editing:Next wave in SCD Recent progress optimizing delivery to HSCs enabled by multiple breakthroughs in LNP technology Advancing in vivo approach to DC Potential to enhance in vivo editing with ESCAPE technology Healthy volunteer study of BEAM-103, an anti-CD117 monoclonal antibody, ongoing Maintain optionality to pursue ex vivo applications in SCD and other hematologic diseases Up to 100% ~100,000 10% ~10,000 Expanding curative therapy for all patients with SCD will be enabled by improvements in delivery Potential Eligible SCD Patient Population (U.S.) Ex vivo HSC Transplant:Potential best-in-class profile with risto-cel Source: Market research, internal Beam estimates


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Beam has assembled all capabilities needed to enable in vivo delivery of SCD gene editing LNP Potent, wholly-owned, highly biodegradable ionizable lipid and LNP process Extra-hepatic Targeting LNP modifications to de-target liver Targeting binders enable cell-specific delivery in vivo Payload Precise and efficient gene editing payloads ESCAPE Selectively suppresses diseased cells, enabling non-genotoxic therapy both ex vivo and in vivo In vivo LNP delivery to HSCs In vivo LNP delivery to T cells NHP proof of concept Progressing to clinical studies Acquisition by BMS for $1.5B Multiple HSC-targeting LNPs identified Development scale-up ready Lead optimization ongoing


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Partnerships have resulted in more than $900M non-dilutive funding to date and more than $1B in potential milestones Gained rights to innovative and complementary technologies Advanced Beam science toward patients in non-core areas Beam’s innovative partnership strategy has consistently delivered value creation and therapeutic impact Acquired by Lilly Opted-in to FcRn program Acquired by BMS


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Beam is well positioned to realize the power of predictability in 2026 Report updated Phase 1/2 data for BEAM-302 and provide next steps for pivotal development by the end of Q1 2026 Plan to submit risto-cel BLA as early as YE 2026 Disclose next liver-targeted genetic disease program in 1H 2026 Report initial BEAM-301 data by YE 2026 Complete BEAM-103 healthy volunteer study in 1H 2026 Advance in vivo HSC editing program $1.25 billion in cash as of Dec. 31, 2025* Expected runway into 2029 through anticipated risto-cel launch and execution of BEAM-302 pivotal development plan Pursue Path to Approval for Lead Programs Maintain Financial Strength Advance and Expand Pipeline *Inclusive of cash, cash equivalents, and marketable securities. This estimate is preliminary, unaudited and is subject to completion of Beam’s financial statement closing procedures.


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THANK YOU Kyle LIVING WITH SICKLE CELL DISEASE Dan LIVING WITH ALPHA-1 ANTITRYPSIN DEFICIENCY Alyssa and Gayle LIVING WITH GLYCOGEN STORAGE DISEASE TYPE IA