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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 initiation, timing, prog

Key Takeaway: Beam Therapeutics recently discussed its forward-looking statements and progress within their pipeline of gene editing therapies, including the BEACON and BEAM-201 trials. The company is focusing on developing precision genetic medicines that could offer curative solutions for various diseases. However, Beam acknowledged several risks, such as potential delays in regulatory approvals and manufacturing challenges, which could impact their projects. Despite these challenges, the advancements in their portfolio hold promise for addressing unmet patient needs through innovative therapies.

Market Sentiment Analysis

POSITIVE FACTORS

  • Advancement of multiple clinical trials, including BEACON and BEAM-201.
  • Development of potentially curative, precision genetic medicines through base editing.
  • Strong pipeline with diverse applications, including CAR-T and therapies for rare diseases.

CONCERNS & RISKS

  • Risks associated with obtaining regulatory approval for product candidates might take longer than expected.
  • Potential manufacturing or supply interruptions for product candidates.
  • The impact of the COVID-19 pandemic may affect development timelines.

Full Press Release Details

PRECISION GENETIC MEDICINES THROUGH
BASE EDITING Beam Therapeutics NASDAQ: BEAM Exhibit 99.1
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 initiation, timing, progress and results of preclinical studies and research and
development programs, including the initiation and progress of clinical trials, including our BEACON trial and our BEAM-201 trial; the advancement of our pipeline, including the advancement of BEAM-101, BEAM-201, BEAM-301, BEAM-302, additional CAR-T
and liver programs, and Stargardt disease program in multiple preclinical studies; our current expectations and anticipated results of operations, including our expected use of capital; the potential activities and benefits under license and
collaboration agreements and the formation of new collaborations; and the therapeutic applications and potential of our technology, including our potential to develop life-long, 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," "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; the potential impact of the COVID-19 pandemic; 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 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, 2021, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2022, our Quarterly Report on
Form 10-Q for the quarter ended June 30, 2022, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, 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. Cautionary note regarding forward-looking statements .
Potential for one-time, curative
therapies Gene editing for rare and common diseases Platform for rapidly-programmable precision medicines Our vision is to provide life-long cures for patients suffering from serious diseases
Base editing is a differentiated,
potentially best-in-class gene editing technology Precise targeting? Yes (guide RNA or ZF/TALE) Yes (guide RNA) Durability of edit? Permanent Permanent Double strand breaks? Yes No Applications? Primarily knockout Correct, modify, activate,
multiplex Editing predictability Random insertions and deletions 100s of uncharacterized edits Single base edits All edits fully characterized Efficiency of precise edit? Low - dividing cells only High - any cell type Nuclease CRISPR,
ZFN, TALENs Base editing
A precise gene editing technology with
highly versatile applications CRISPR Protein Deaminase Guide RNA Correct proteins (eg, BEAM-301, BEAM-302) 3 Modify surface proteins (eg, ESCAPE) 4 Activate expression (eg, BEAM-101) X Knock out proteins (eg, BEAM-201) 2 Multiplex simultaneous edits
(eg, four gRNAs in BEAM-201) X + - 1 5 And many other applications possible
We are establishing a leading platform
for precision genetic medicine Payload Manufacturing Delivery Suite of gene editing technologies Base editing ABE: A-to-G (or T-to-C) editors CBE: C-to-T (or G-to-A) editors Additional kinds of base editors Nuclease editing RNA editing Prime editing
Suite of delivery technologies Autologous cell therapy Allogeneic cell therapy mRNA LNP vectors Viral vectors Internal manufacturing capability 100,000 square foot cGMP clinical/commercial facility in NC, phased build, anticipated to be operational
Advancing a diversified pipeline into
the clinic LNP = Lipid Nanoparticle; AAV = Adeno Associated Virus; HSC = Hematopoietic Stem Cell; T-ALL / T-LL = T-Cell Acute Lymphoblastic Leukemia / T-Cell Lymphoblastic Lymphoma; AML = Acute Myeloid Leukemia; ESCAPE:
Engineered Stem Cell Antibody Paired Evasion DELIVERY PROGRAM / DISEASE EDITING APPROACH RESEARCH LEAD OPTIMIZATION IND ENABLING PHASE I/II PIVOTAL Ex vivo HSCs BEAM-101 Sickle Cell Disease Beta Thalassemia Activation of
fetal hemoglobin BEAM-102 Sickle Cell Disease Correction of HbS sickle mutation ESCAPE Sickle Cell Disease Beta Thalassemia Multiplex CD117 edit-antibody pair Ex vivo T cells BEAM-201 T-ALL / T-LL CD7+ AML Multiplex silenced CD7 CAR-T In vivo LNP
BEAM-301 Glycogen Storage Disease Ia Correction of R83C mutation In vivo LNP BEAM-302 Alpha-1 Antitrypsin Deficiency Correction of E342K mutation In vivo LNP Glycogen Storage Disease Ia Correction of Q347X mutation In vivo LNP Hepatitis B Virus
Multiplex silencing In vivo LNP Complement Pathway (Apellis) Undisclosed 3 undisclosed targets (Pfizer) Undisclosed AAV Stargardt Disease Correction of G1961E mutation Refocusing on ESCAPE or in vivo delivery
Lead Programs: Potentially de-risk
technology (higher probability of technical success, faster path), generate revenue, and benefit patients with high unmet need Future platforms: Expand addressable patient populations to create highly valuable, differentiated franchises through
further innovation in editing and delivery HEMATOLOGY BEAM-101 ESCAPE for conditioning In vivo delivery GENETIC DISEASES BEAM-301, BEAM-302 Multiple new liver targets Barcoded LNP beyond liver Near term: Future platforms: IMMUNOLOGY- ONCOLOGY
BEAM-201 Next-generation allogeneic platform (4-6+ edits) Beam is developing medicines across three franchises, each with near- and long-term potential ESCAPE: Engineered Stem Cell Antibody Paired Evasion
Key progress and anticipated milestones
2022 Achievements Upcoming Milestones Data presentation on multiple patients from BEACON in 2024 Regulatory filing for BEAM-301 by late 2023 / early 2024 Complete sentinel cohort enrollment and initiate
expansion cohort of BEACON in 2023 Dose first BEAM-201 patient by mid 2023 Regulatory filing for BEAM-302 in early 2024 Refocused on new technology: ESCAPE & LNP Initiate IND-enabling studies for BEAM-301 Nominated BEAM-302 development candidate
First subject enrolled for BEAM-101 Submit IND for BEAM-201 and respond to hold Refocused on next gen allogeneic strategies Strategic platform partnerships (Pfizer, Orbital) Hematology Immunology - Oncology Genetic disease Platform
Designed for best-in-class profile:
One-time therapy with potential for highest fetal hemoglobin (HbF) induction Direct editing of HbF genes to turn them on Potential for greatest reduction of disease-causing HbS due to hemoglobin switching Non-viral: No detectable random insertion
Non-cutting: Lower risk for genotoxic stress and chromosomal abnormalities Investment in patient delivery to differentiate: Wholly owned manufacturing: control over quality and connection to patient services Investment in patient services:
optimizing patient experience BEAM-101: Designed to treat sickle cell disease with a potentially one-time, direct, non-cutting activation of HbF Sickle Cell Disease: 100,000 patients in the US; severe pain crises, multi-organ damage, early mortality
HPFH = Hereditary Persistence of Fetal Hemoglobin HBB HBG1 HBG2 A single base editor + gRNA edits regulatory element of both fetal hemoglobin genes, without cutting DNA Sickle hemoglobin (HbS) gene Duplicated fetal hemoglobin (HbF) genes
Potentially best-in-class attributes
of BEAM-101 product Base editing at HBG1/2 promoters1 HbF protein levels2 Preclinical data presented at ASGCT 2020; Edited human HSPCs analyzed 16 weeks after infusion in NBSGW mice (Mean SEM, n=4-6); 1. Sorted human Lineage-CD34+ bulk bone
marrow; 2. Sorted erythroid cells (GlyA+) HbS protein levels2 Edited human CD34+ cells followed by 16 week engraftment in mice >90% 65% <40% Potential for highest HbF induction and lowest residual HbS levels versus other approaches in the
field Building capabilities for potential best-in-class patient delivery including internal manufacturing HEMATOLOGY
BEAM-101 is the first clinical base
editing program in the U.S., accelerating path to patients and the market BEACON-101 Phase 1/2 Study Design Manufacturing Conditioning & Transplant Engraftment Follow-up Patient 1 Patient 2 Patient 3 Patients 4-45 Transfusion & Mobilization
6 months (+ / -) Select safety endpoints Proportion of patients with successful neutrophil engraftment by day 42 Safety and tolerability assessments Select inclusion criteria Patients with sickle cell disease (SCD) with severe veno-occlusive crises
despite hydroxyurea or other supportive measures Age 18 to 35 years for initial cohort Select efficacy endpoints Severe vaso-occlusive crises Transfusion requirements Hemoglobin F levels Quality of life and ability to function Markers
of red blood cell function and organ damage HEMATOLOGY
Well positioned to deliver
potentially best-in-class regimens for SCD patients, now and in the future Precise gene editing (non-cutting, non-viral) Busulfan conditioning BEAM-101 (HbF) Less toxic conditioning selects for edited cells - potential to expand to younger and
broader patient population ESCAPE-1 (HbF+CD117) ESCAPE-2 (Makassar+CD117) In vivo editing after infusion of HSC-targeted LNPs (no transplant) HbF + Makassar available Wave 1 Base Editing + HSC Transplant Wave 2 Improved Conditioning Wave 3 In vivo
Delivery HEMATOLOGY * ESCAPE: Engineered Stem Cell Antibody Paired Evasion
Stem cell factor (SCF) signaling via
CD117 is required for HSC survival and proliferation A single base edit changes an epitope on the CD117 receptor and is designed not to impact HSC biology Customized conditioning antibody depletes diseased unedited cells, but enables CD117-edited
cells to "ESCAPE" and grow normally ESCAPE* designed for selective depletion of diseased cells, which may enable non-genotoxic conditioning Enrichment of edited cells in presence of antibody Paired CD117 antibody (ng/mL) HSC eHSC Cell
Dies Cell Survives * ESCAPE: Engineered Stem Cell Antibody Paired Evasion HEMATOLOGY
GSD1a unmet need: Low G6PC activity
can result in severe drop in blood glucose levels within 1-3 hrs Hypoglycemia may result in seizures or can be lethal Multiple organ dysfunction (e.g. renal and liver) BEAM-301 potential: Near-normal serum metabolites, G6PC activity, hepatic
morphology, increased survival in mice Animal studies suggest 11% editing sufficient for restoring fasting glucose1 Key points: Beam's first in vivo DC First DC in industry with in vivo direct correction gene editing2 Regulatory filing
expected by late 2023 / early 2024 Chou & Mansfield. 2007. Curr. Gen. Ther. Based on publicly announced development candidates Wildtype G6PC gene G6PC R83C mutation BEAM-301 program aims to restore impaired glycogen metabolism which otherwise
causes significant morbidity Liver Low G6P conversion High G6P conversion Glycogen Storage Disease Ia: 900 US R83C patients; severe hypoglycemia, liver & kidney dysfunction GENETIC DISEASE
Preclinical data presented at ESGCT
2021; 1. Homozygous huG6PC-R83C mice untreated or treated with LNP via temporal vein shortly after birth, and untreated mice survived less than 3 days with glucose therapy BEAM-301 program aims to restore impaired glycogen metabolism which otherwise
causes significant morbidity ABE correction of GSDIa R83C mutation associated with improved survival of R83C mice1 Near-normal serum metabolites, G6PC activity, hepatic morphology and lipid deposition GENETIC DISEASE
Potential one time treatment to
create permanent correction of E342K and enable normal A1 secretion and gene regulation Designed to address disease pathology in both the liver and lung In preclinical studies, lead candidate delivered up to 27% correction editing, that resulted in
>3X increase in A1AT protein (> 11 uM protective threshold) at clinically-relevant dose of 0.75mpk In a minority of cells, correction resulted in wildtype allele plus D341G allele (bystander) that was observed to function normally BEAM-302
nominated for development; regulatory filing expected in early 2024 BEAM-302: Development candidate nominated for potential one-time treatment of AATD Corrected SERPINA1 (wildtype) E342K (PiZ) mutation SERPINA1 with D341G bystander edit GENETIC
DISEASE Alpha-1 Anti-trypsin Deficiency (AATD): 60,000 ZZ patients in US; severe progressive lung & liver disease
BEAM-302 program has the potential
to address both lung and liver pathology of AATD in one course treatment 4.9-fold increase in functional A1AT secretion Reduction in toxic liver aggregates Control Correction Group Preclinical data presented at ASGCT 2020; Editing in NSG-PiZ mice
with either control (PCSK9) or correction (E342K) provided above results Representative in vivo studies of PiZZ mouse with precursor base editors GENETIC DISEASE
Multiplex base editing: Unlike
nuclease editors, no detected chromosomal rearrangements, normal cell expansion, and no detected DNA damage response in preclinical studies Clinical-scale process: 96-99% editing, >90% quad edited1 BEAM-201 US IND cleared; first patient dosing
expected by mid-2023 BEAM-201: Base edited allogeneic cell therapy candidate with an opportunity to treat aggressive CD7+ leukemias T-Cell Acute Leukemia: 15% of ALL, not treated by B-cell CARTs, few options for relapsed/refractory patients
IMMUNOLOGY-ONCOLOGY Base editor mRNA TRAC gRNA: Prevent graft-vs-host disease CD52 gRNA: Enable allogeneic cell source PD1 gRNA: Prolong efficacy CD7 gRNA: Prevent fratricide from CD7 CAR C G C G C G C G Clinical-scale process yielded 96-99%
editing, >90% quad edited1 Unedited BEAM-201 CD7 CAR Preclinical data presented at SITC 2020; 1. Simultaneous base editing at four target loci using clinical-scale process as measured by NGS.
BEAM-201: Significant advantages of
multiplex base editing without double strand breaks Chromosomal rearrangements Impact on cell expansion 4 edits: TRAC, CD52, PD1, CD7 3 edits: TRAC, B2M, PD1 Base editing Nuclease Percent of cells with translocations1 Percent yield after editing2
Preclinical data presented at SITC 2020; 1. Base editing versus nuclease editing with the same four guide RNAs measured via G-banded karyotypes from 100 cells; updated analysis shows <0.1% translocations using first generation CBE (data
unpublished) 2. Extensive guide screen across three targets, with BE4 and spCas9 sgRNAs selected for high editing efficiency and expansion in single-plex test, final cell yields compared between 3 edits, normalized to electroporation only control
IMMUNOLOGY-ONCOLOGY <40% Multiplex editing more efficient with base editing which translates to better cell product Optimization of platform ongoing with focus on generating next generation "true allogeneic" products
BTX-ALO-001: Multiplex edited
BEAM-201 enables evaluation in aggressive T-cell cancers using optimized lymphodepletion (LD) Select safety endpoints Incidence and severity of treatment emergent adverse events (TEAE) and treatment-related AEs, including serious AEs (SAEs) and DLTs
Select inclusion criteria 18 to 50 yrs for dose exploration 1 yrs for peds after FDA review T-ALL or T-LL with one of following: Relapsed after 2nd CR Relapse after HSCT Primary refractory or R/R Eligible for allo HSCT (donor
available) Select efficacy endpoints Proportion of T-ALL pts with CR or CRi or T-LL pts with CR or PR any time post BEAM-201 Proportion of pts eligible for HSCT based on response to BEAM-201 Proportion achieving MRD-negative status Duration of
response, OS, etc Phase 1 dose exploration ( 36 pts) Phase 2 (~48 pts) Expansion Cohort 2 Expansion Cohort 1 Pediatric cohort (gated initiation of enrollment) LD Regimen 1 (Up to 3 CAR-T dose levels) LD Regimen 2 (Up to 3 CAR-T dose levels)

Frequently Asked Questions

What is Beam Therapeutics focused on developing?

Beam Therapeutics is focused on developing precision genetic medicines using base editing technology.

What are the key gene editing technologies used by Beam?

Beam utilizes base editing, CRISPR, ZFN, TALENs, and additional editing platforms.

What are key milestones for Beam in 2023?

Key milestones include dose administration for BEAM-201 and regulatory filings for BEAM-301 and BEAM-302.

What therapeutic applications does Beam target?

Beam targets rare and common diseases, including genetic disorders and hematological conditions.

How does Beam ensure precise gene editing?

Beam's base editing technology allows for high-precision, permanent gene edits without double-strand breaks.

Last updated: Jan 9, 2023