Beam Therapeutics Inc. · NASDAQ:BEAM
Beam Therapeutics — Investor Presentation (January 2026)
27 slides from Beam Therapeutics's investor presentation, Jan 12, 2026
Slide 1: The Power of Predictability
The Power of Predictability JANUARY 2026 NASDAQ: BEAM
Slide 2: Cautionary note regarding forward-looking statements
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 techn
Slide 3: Our vision is to provide lifelong cures for patients suffering from serious diseases
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: Predictable, Reproducible Outcomes for Patients: base editing technology
LESS GENOTOXICITY than traditional gene editing DURABLE correction for one-time cures CONSISTENT gene sequence outcomes Predictable, Reproducible Outcomes for Patients Beam was founded based on a simple concept with profound implications BASE EDITING TECHNOLOGY
Slide 5: Predictability as a driver of progress across the healthcare ecosystem
Predictability as a driver of progress: The potential to ripple through the broader healthcare ecosystem Predictable, Reproducible Outcomes for Patients Streamlined R&D Cycles Reduced development risk Regulatory Acceleration Efficient regulatory pathways Physician Confidence Predictable safety and d
Slide 6: Precision genetic medicines platform
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 partne
Slide 7: Establishing a foundation of financial strength for sustainable growth
Establishing a foundation of financial strength for sustainable growth Wholly owned pipeline Significant addressable markets Sustainable, platform-enabled pipeline expansion and growth $1.25 billion in cash as of Dec. 31, 2025*, inclusive of $255.1 million proceeds from Orbital acquisition Expected
Slide 8: Significant progress in 2025 against all key value drivers
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 clinica
Slide 9: Liver-targeted Genetic Disease Franchise
Liver-targeted Genetic Disease Franchise
Slide 10: Rapidly advancing a portfolio of liver-targeted in vivo programs for genetic diseases
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 Glyc
Slide 11: Severe AATD impacts >100,000 individuals in the U.S. with limited treatment options
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
Slide 12: BEAM-302 potential first single-course treatment for full spectrum of AATD
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 th
Slide 13: BEAM-302 directly corrects the mutation causing AATD
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
Slide 14: Rapid execution of BEAM-302 Phase 1/2 trial
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
Slide 15: Beam intends to pursue accelerated approval pathway for BEAM-302
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 approximate
Slide 16: BEAM-301 Phase 1/2 trial in GSDIa patients with R83C mutation
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 mutatio
Slide 17: Hematology Franchise
Hematology Franchise 17
Slide 18: Multi-wave strategy in SCD: ex vivo commercial opportunity, then in vivo delivery
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
Slide 19: BEACON results show potential differentiation of base editing and risto-cel
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 Time in Hospital HbF levels >60% and HbS levels <40%, comparable to sickle trait Resolutio
Slide 20: Bottlenecks for SCD gene therapy adoption: manufacturing capacity and process efficiency
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+ tr
Slide 21: End-to-end, efficient risto-cel process
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
Slide 22: Preparing for BLA submission and a potential best-in-class launch
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 package as early as YE 2026 Initiate efficient commercial build to
Slide 23: In vivo HSC editing: next wave in SCD
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, o
Slide 24: Capabilities assembled to enable in vivo delivery of SCD gene editing
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 Precis
Slide 25: Partnerships have resulted in more than $900M non-dilutive funding
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 va
Slide 26: Beam is well positioned to realize the power of predictability in 2026
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 Repo
Slide 27: Thank you
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
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