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.

 •  4/18/2025 Letter Continued (Full PDF)
 •  4/21/2023 BEAM Stockholder Letter
 •  4/19/2024 BEAM Stockholder Letter
 •  More "Biotechnology" Category Stockholder Letters
 •  BEAM Benford's Law Stock Score = 69

BEAM Shareholder/Stockholder Letter Transcript:

Dear Fellow Shareholders,
At Beam, our vision is to develop lifelong cures for patients suffering from serious diseases. This
has never felt more tangible than it does today, having achieved clinical proof-of-concept across
both of our core franchises in hematology and liver genetic diseases, further establishing our
leadership in base editing and our commitment to transforming genetic medicine.
We believe base editing can enable one-time curative therapies, fundamentally reshaping how
diseases are treated while significantly reducing the burdens of today   s chronic disease
management. With a pioneering platform, world-class capabilities, and a highly skilled team,
Beam is positioned to drive innovation and lead the next generation of genetic medicine.
Fully Integrated, Clinically Validated Base Editing Platform
    Delivery Excellence: We have successfully delivered therapies ex vivo, using CD34 and T
cells, and in vivo, using our lipid nanoparticle (LNP) technology for liver-directed
treatments.
    Manufacturing Strength: Our North Carolina facility has completed over 100 GMP
batches, reinforcing our robust production and scaling capabilities.
    Regulatory and Clinical Execution: With seven investigational new drug (IND) or
clinical trial authorization (CTA) approvals across five countries and 30+ active clinical
trial sites, our programs continue to advance rapidly.
High-Value Franchises with Best-in-Class Profiles
Hematology
    Clinical Proof-of-Concept with BEAM-101: Our lead Wave 1 base editor, BEAM-101, is
an investigational autologous cell therapy designed to efficiently and uniformly increase
fetal hemoglobin without using double-stranded DNA breaks. BEAM-101 delivers a
genetically modified cell product through stem cell transplant, enabled by chemotherapy
conditioning, for patients with severe sickle cell disease (SCD). In our ongoing BEACON
Phase 1/2 clinical trial of BEAM-101, initial data demonstrated a robust increase in fetal
hemoglobin of >60%, a decrease in hemoglobin S to <40%, resolution of anemia in all
patients, a low number of cell collection cycles, and a rapid time to engraftment. These
data underscore the potential for BEAM-101 to offer a differentiated, best-in-class
treatment for patients with SCD.
    Proof-of-Concept with ESCAPE in Non-Human Primates (NHPs): Our Wave 2
innovation, ESCAPE (Engineered Stem Cell Antibody Evasion), is developing a nongenotoxic alternative to traditional myeloablative conditioning, which is associated with
challenging side effects and risks. As a result, ESCAPE could open up the potential to
bring the benefits of curative therapy to the vast majority of SCD patients who are
ineligible for or unwilling to undergo a chemotherapy-based transplant. Proof-of-concept
for ESCAPE in NHPs demonstrated successful engraftment of base-edited cells using
only antibody conditioning and no chemotherapy. These data represent a potential
paradigm shift in transplant medicine, providing a strong foundation for advancing
ESCAPE into the clinic this year.


In Vivo Editing to Expand the Reach of Our Medicines: Our Wave 3 innovation aims to
deliver base editors intravenously via LNPs targeted to hematopoietic stem cells,
eliminating the need for transplantation altogether. In so doing, our in vivo editing
technology has further potential to simplify delivery and expand the application of base
editing to reach many more patients.
Liver Genetic Diseases
    Clinical Proof-of-Concept for BEAM-302 and Correction of a Disease-Causing
Mutation: BEAM-302 is designed to correct the PiZ allele, the most common gene
variant linked to severe alpha-1 antitrypsin deficiency (AATD). BEAM-302 has the
potential to simultaneously reduce the aggregation of mutant, misfolded AAT protein that
causes toxicity to the liver and increase circulating levels of corrected and functional AAT
protein, addressing the underlying pathophysiology of both the liver and lung disease. In
our ongoing Phase 1/2 clinical trial of BEAM-302, initial data demonstrated a favorable
safety profile, durable, dose-dependent correction of the PiZ mutation, and a clinically
meaningful increase in total AAT in patients. To our knowledge, these are the first data to
demonstrate a genetic correction of a disease-causing mutation in DNA, representing a
potential breakthrough treatment for patients.
    Advancing BEAM-301 into Clinical Development: BEAM-301 is designed to correct the
R83C mutation, the most common disease-causing mutation that results in the most
severe form of glycogen storage disease type 1a (GSD1a), a severe metabolic disorder
with potentially life-threatening and long-term complications. BEAM-301 aims to
normalize blood glucose without continuous supplementation to improve metabolic
parameters. Preclinical data have shown that a single administration of BEAM-301
directly and durably corrected the R83C mutation in vivo, with an ongoing significant
survival benefit at one-year post-treatment.
Executing with Speed and Precision
    BEAM-101: In our BEACON trial, we have achieved our target enrollment of adult
patients, with more than 40 enrolled to date, and begun enrolling adolescent patients. We
expect to have dosed 30 patients by mid-year, with more mature clinical data updates
planned in 2025. Given this progress, we believe we are on track for a future Biologics
License Application (BLA) submission for the potential regulatory approval of BEAM101.
    ESCAPE: We have initiated Phase 1-enabling toxicology studies for our ESCAPE
programs, BEAM-103 and BEAM-104. Preparations are underway to begin a Phase 1
trial of BEAM-103, an anti-CD117 monoclonal antibody, in healthy volunteers by yearend 2025.
BEAM-302: We are continuing the dose-escalation portion of Part A of our ongoing
Phase 1/2 trial and expect to report additional clinical data in the second half of 2025. In
addition, we plan to begin dosing in Part B, which will include AATD patients with mild
to moderate liver disease, in the second half of 2025.
    BEAM-301: Our first in vivo IND, for BEAM-301, has been cleared by the U.S. FDA.
We have activated our first clinical trial site in the U.S. and expect patient dosing to begin
in early 2025.

Well-Positioned for Long Term Growth, Value, and Patient Impact
With three proof-of-concept datasets delivered across our pipeline in 2024 and early 2025, Beam
is entering a new era of genetic medicine leadership. Our hematology pipeline continues to
expand, with BEAM-101   s initial data providing a differentiated profile for SCD and ESCAPE
exploring new transplant alternatives, expanding potential therapeutic opportunities for patients
with SCD, beta-thalassemia, and beyond. The proof-of-concept data for BEAM-302 mark a
historic milestone, not only modulating gene expression but directly rewriting and repairing the
genetic code. This breakthrough also validates our in vivo LNP delivery capabilities, reinforcing
our potential to drive innovation in liver-targeted therapies. Combined with our financial strength
and focus, we are well-positioned for sustained progress.
As we move forward, the key elements of Beam   s long-term success are firmly in place: a
clinically validated, fully integrated base editing platform; a portfolio of high-value,
commercially meaningful programs with best-in-class potential; a strong foundation for
continued execution across our research, development, and business operations; and a robust
balance sheet expected to fund the company   s late stage development and commercialization
activities into 2028.
We are deeply grateful for the continued support of our shareholders. Your confidence allows us
to push the boundaries of what   s possible in genetic medicine. Thank you for being part of our
journey     we look forward to shaping the future of genetic medicine together.
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, 2024, 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.



 4/18/2025 Letter Continued (Full PDF)