On this page of StockholderLetter.com we present the latest annual shareholder letter from Beam Therapeutics Inc. — ticker symbol BEAM. Reading current and past BEAM letters to shareholders can bring important insights into the investment thesis.
Dear Fellow Shareholders, At Beam, our vision is to provide lifelong cures for patients suffering from serious diseases. In 2025, that vision moved decisively from promise to proof. Over the past year, we demonstrated that base editing can reliably deliver predictable, durable genetic correction across multiple diseases and delivery modalities in patients. We have also confirmed regulatory pathways to potential approval for our lead programs, which are positioned to become first ever ex vivo and in vivo base editing medicines. These achievements, just a few years after entering clinical development, validate not only our science but also our strategy to build a scalable, high-value genetic medicine company grounded in execution and long-term impact. The Power of Predictability Beam was founded on a simple idea with profound implications: if we can precisely rewrite a single letter in DNA without creating double-stranded breaks, we can achieve consistent gene sequence outcomes, durable correction, and potentially lower genotoxicity than traditional gene editing. In 2025, that idea was validated in the clinic across our hematology and liver genetic disease programs, reinforcing our belief that predictability is the defining advantage of base editing. Predictability is not an abstract concept; it is a driver of progress. It enables streamlined R&D cycles, reduces development risk, supports regulatory acceleration, builds physician confidence, improves patient experience, and aligns outcomes with payer expectations. As this flywheel turns faster, it creates a repeatable model for advancing genetic medicine one that benefits patients, healthcare systems, and shareholders alike. A Transformational Year of Execution in 2025 2025 marked a pivotal inflection point for Beam. We achieved several firsts and milestones that collectively established clinical proof of concept for our platform and set the foundation for sustainable value creation. We delivered the first-ever human proof of concept for in vivo gene correction with BEAM-302, presented differentiated and potentially best-in-class clinical data for risto-cel in sickle cell disease, exceeded clinical enrollment and timeline targets across both programs, and gained FDA regulatory alignment on accelerated pathways for our lead assets. We also significantly strengthened our financial position in the last 12 months through an equity raise and a non-dilutive facility that collectively provide Beam with up to $1 billion. Together, these funds have enabled a significant extension of our anticipated operating runway into mid2029. This financial discipline allows us to advance our wholly owned pipeline through key value inflection points, including potential commercialization, while continuing to invest thoughtfully in next-wave innovation.
Two High-Value Franchises Anchoring Long-Term Growth Our strategy is focused on building multiple, growing franchises powered by a fully integrated base editing platform, reusable delivery technologies, scalable internal manufacturing, and flexible regulatory pathways. Today, two core franchises anchor our value proposition: livertargeted genetic diseases and hematology. Liver-Targeted Genetic Disease Franchise Our liver franchise exemplifies the power of in vivo base editing. BEAM-302, our lead program in alpha-1 antitrypsin deficiency (AATD), is designed to directly correct the underlying diseasecausing mutation of AATD, addressing both liver and lung manifestations with a single-course treatment. In 2025, we established clinical proof of concept for BEAM-302 showing durable, dose-dependent correction of the PiZ mutation, significant reduction of mutant Z-AAT, and increases in total AAT above the protective threshold. A year later, we reported a robust, compelling clinical data update from 29 patients treated with BEAM-302 that supports this potentially first- and best-in-class profile. Importantly, these data support the advancement of BEAM-302 into pivotal development in pursuit of a potential accelerated approval pathway. Beyond BEAM-302, we advanced BEAM-301 in glycogen storage disease type Ia (GSDIa) into a Phase 1/2 clinical trial and expect to report initial data from the program this year. In early 2026, we expanded our liver franchise with a new program, BEAM-304, for the treatment of phenylketonuria (PKU), a disease with significant unmet need that affects approximately 20,000 individuals in the U.S. We are advancing BEAM-304 using an innovative development approach in which multiple mutation-specific base editors are developed efficiently within a single clinical program, providing us with the potential to create transformative, onetime therapies for the vast majority of patients with PKU. Hematology Franchise In hematology, our multi-wave strategy focuses on near-term commercial opportunity through ex vivo therapy, followed by in vivo delivery to maximize scalability and reach. In 2025, we made significant operational and clinical progress for risto-cel, our lead ex vivo candidate, completing enrollment in the BEACON study and presenting an impressive, differentiated dataset from 31 patients with SCD with severe vaso-occlusive crises. The data, first presented at the ASH Annual Meeting then published in the New England Journal of Medicine, demonstrate deep resolution of red cell dysfunction and reduced time in hospital, driven by low cell collection cycles and rapid engraftment. These important differentiating factors are driven in part by the elegant mechanism of base editing, and in part by our efficient and highly automated internal manufacturing process, which has the potential to address key bottlenecks that currently limit adoption of gene therapies in SCD and improve the patient and treatment center experience. Looking ahead, we re squarely focused on advancing risto-cel toward a planned BLA submission as early as year-end 2026 and bringing this therapy to patients as quickly as possible.
Well Positioned for Durable Value Creation As we enter the next phase of Beam s evolution, we have established the foundational elements for long-term success: a clinically validated and fully integrated base editing platform; two highvalue franchises with clear paths to approval and commercialization; a growing pipeline supported by reusable delivery and manufacturing capabilities; and financial strength to execute with focus and discipline. We believe the years ahead will continue to be defined by predictable execution, expansion, and impact for patients and for shareholders. We are deeply grateful to the patients, families and physicians who have partnered with us in our clinical trials, and to our shareholders for your continued confidence and support. Together, we are shaping the future of genetic medicine. Sincerely, John Evans Chief Executive Officer Cautionary Note Regarding Forward-Looking Statements This letter contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to, among other things, future operating performance, product development, market position and business strategy. The reader is cautioned not to rely on these statements, which are based on current expectations of future events. For important information about these statements, including the important risks, uncertainties and other factors that could cause actual results to differ materially from the assumptions, expectations and projections expressed in any forward-looking statements, the reader should review the enclosed Annual Report on Form 10-K for the fiscal year ended December 31, 2025, including the sections captioned Risk Factors Summary and Item 1A. Risk Factors. Beam Therapeutics Inc. does not undertake to update any forward-looking statement as a result of new information, future events or developments or otherwise, except as may be required by applicable law.
 • shareholder letter icon 4/17/2026 Letter Continued (Full PDF)
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 • stockholder letter icon 4/18/2025 BEAM Stockholder Letter
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 • Benford's Law Stocks icon BEAM Benford's Law Stock Score = 64


BEAM Shareholder/Stockholder Letter Transcript:

Dear Fellow Shareholders,
At Beam, our vision is to provide lifelong cures for patients suffering from serious diseases. In
2025, that vision moved decisively from promise to proof. Over the past year, we demonstrated
that base editing can reliably deliver predictable, durable genetic correction across multiple
diseases and delivery modalities in patients. We have also confirmed regulatory pathways to
potential approval for our lead programs, which are positioned to become first ever ex vivo and
in vivo base editing medicines. These achievements, just a few years after entering clinical
development, validate not only our science but also our strategy to build a scalable, high-value
genetic medicine company grounded in execution and long-term impact.
The Power of Predictability
Beam was founded on a simple idea with profound implications: if we can precisely rewrite a
single letter in DNA without creating double-stranded breaks, we can achieve consistent gene
sequence outcomes, durable correction, and potentially lower genotoxicity than traditional gene
editing. In 2025, that idea was validated in the clinic across our hematology and liver genetic
disease programs, reinforcing our belief that predictability is the defining advantage of base
editing.
Predictability is not an abstract concept; it is a driver of progress. It enables streamlined R&D
cycles, reduces development risk, supports regulatory acceleration, builds physician confidence,
improves patient experience, and aligns outcomes with payer expectations. As this flywheel turns
faster, it creates a repeatable model for advancing genetic medicine     one that benefits patients,
healthcare systems, and shareholders alike.
A Transformational Year of Execution in 2025
2025 marked a pivotal inflection point for Beam. We achieved several firsts and milestones that
collectively established clinical proof of concept for our platform and set the foundation for
sustainable value creation. We delivered the first-ever human proof of concept for in vivo gene
correction with BEAM-302, presented differentiated and potentially best-in-class clinical data
for risto-cel in sickle cell disease, exceeded clinical enrollment and timeline targets across both
programs, and gained FDA regulatory alignment on accelerated pathways for our lead assets.
We also significantly strengthened our financial position in the last 12 months through an equity
raise and a non-dilutive facility that collectively provide Beam with up to $1 billion. Together,
these funds have enabled a significant extension of our anticipated operating runway into mid2029. This financial discipline allows us to advance our wholly owned pipeline through key
value inflection points, including potential commercialization, while continuing to invest
thoughtfully in next-wave innovation.

Two High-Value Franchises Anchoring Long-Term Growth
Our strategy is focused on building multiple, growing franchises powered by a fully integrated
base editing platform, reusable delivery technologies, scalable internal manufacturing, and
flexible regulatory pathways. Today, two core franchises anchor our value proposition: livertargeted genetic diseases and hematology.
Liver-Targeted Genetic Disease Franchise
Our liver franchise exemplifies the power of in vivo base editing. BEAM-302, our lead program
in alpha-1 antitrypsin deficiency (AATD), is designed to directly correct the underlying diseasecausing mutation of AATD, addressing both liver and lung manifestations with a single-course
treatment. In 2025, we established clinical proof of concept for BEAM-302 showing durable,
dose-dependent correction of the PiZ mutation, significant reduction of mutant Z-AAT, and
increases in total AAT above the protective threshold. A year later, we reported a robust,
compelling clinical data update from 29 patients treated with BEAM-302 that supports this
potentially first- and best-in-class profile. Importantly, these data support the advancement of
BEAM-302 into pivotal development in pursuit of a potential accelerated approval pathway.
Beyond BEAM-302, we advanced BEAM-301 in glycogen storage disease type Ia (GSDIa) into
a Phase 1/2 clinical trial and expect to report initial data from the program this year.
In early 2026, we expanded our liver franchise with a new program, BEAM-304, for the
treatment of phenylketonuria (PKU), a disease with significant unmet need that affects
approximately 20,000 individuals in the U.S. We are advancing BEAM-304 using an innovative
development approach in which multiple mutation-specific base editors are developed efficiently
within a single clinical program, providing us with the potential to create transformative, onetime therapies for the vast majority of patients with PKU.
Hematology Franchise
In hematology, our multi-wave strategy focuses on near-term commercial opportunity through ex
vivo therapy, followed by in vivo delivery to maximize scalability and reach. In 2025, we made
significant operational and clinical progress for risto-cel, our lead ex vivo candidate, completing
enrollment in the BEACON study and presenting an impressive, differentiated dataset from 31
patients with SCD with severe vaso-occlusive crises. The data, first presented at the ASH Annual
Meeting then published in the New England Journal of Medicine, demonstrate deep resolution of
red cell dysfunction and reduced time in hospital, driven by low cell collection cycles and rapid
engraftment. These important differentiating factors are driven in part by the elegant mechanism
of base editing, and in part by our efficient and highly automated internal manufacturing process,
which has the potential to address key bottlenecks that currently limit adoption of gene therapies
in SCD and improve the patient and treatment center experience. Looking ahead, we   re squarely
focused on advancing risto-cel toward a planned BLA submission as early as year-end 2026 and
bringing this therapy to patients as quickly as possible.

Well Positioned for Durable Value Creation
As we enter the next phase of Beam   s evolution, we have established the foundational elements
for long-term success: a clinically validated and fully integrated base editing platform; two highvalue franchises with clear paths to approval and commercialization; a growing pipeline
supported by reusable delivery and manufacturing capabilities; and financial strength to execute
with focus and discipline. We believe the years ahead will continue to be defined by predictable
execution, expansion, and impact for patients and for shareholders.
We are deeply grateful to the patients, families and physicians who have partnered with us in our
clinical trials, and to our shareholders for your continued confidence and support. Together, we
are shaping the future of genetic medicine.
Sincerely,
John Evans
Chief Executive Officer
Cautionary Note Regarding Forward-Looking Statements
This letter contains forward-looking statements within the meaning of the Private Securities
Litigation Reform Act of 1995 relating to, among other things, future operating performance,
product development, market position and business strategy. The reader is cautioned not to rely
on these statements, which are based on current expectations of future events. For important
information about these statements, including the important risks, uncertainties and other factors
that could cause actual results to differ materially from the assumptions, expectations and
projections expressed in any forward-looking statements, the reader should review the enclosed
Annual Report on Form 10-K for the fiscal year ended December 31, 2025, including the
sections captioned    Risk Factors Summary    and    Item 1A. Risk Factors.    Beam Therapeutics
Inc. does not undertake to update any forward-looking statement as a result of new information,
future events or developments or otherwise, except as may be required by applicable law.


shareholder letter icon 4/17/2026 Letter Continued (Full PDF)
 

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