Ambition: Curing Blood Cancers through cell and genome engineering

March 2024

Disclaimer

This presentation (the "Presentation") contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 about Vor Biopharma Inc. ("Vor," "Vor Bio" or the "Company"). The words "aim," "anticipate," "believe," "can," "could," "design," "enable" "estimate," "expect," "intend," "may," "ongoing," "plan," "potential," "project," "should," "target," "towards," "will," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this Presentation include those regarding the feasibility of a trem-cel (formerly VOR33) transplant to be successfully manufactured, to engraft normally, to maintain blood counts following treatment with Mylotarg following allogeneic hematopoietic cell transplant and to be well tolerated, the potential of VCAR33ALLO in

combination with trem-cel as a Treatment System, the potential of trem-cel to enable targeted therapies in the post-transplant setting including Mylotarg and CD33-targetedCAR-Ts, the potential of Vor Bio's platform, Vor Bio's plans, strategies, expectations and anticipated milestones for its preclinical and clinical programs, including the availability and timing of

results from preclinical studies and clinical trials, the timing of regulatory filings, and the timing of dosing patients, Vor Bio's expectations regarding expedited regulatory review of its product candidates as a result of Fast Tracks designation or otherwise, the expected safety profile of Vor Bio's product candidates, cash runway and expected capital requirements, Vor Bio's expectations regarding addressable patient population and reimbursement rates for its product candidates, if approved, and its plans and expectations related to the Company's manufacturing and facilities. Vor Bio may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward- looking statements as a result of various factors, including: uncertainties inherent in the initiation, completion of, and availability and timing of results from, preclinical studies and

clinical trials and clinical development of Vor Bio's product candidates; whether preclinical data or interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; the uncertainty of regulatory approvals to conduct trials or to market products; the success of Vor Bio's in-house manufacturing capabilities and efforts; and

availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements. The interim data for trem-cel presented in this Presentation is based on eight patients and future results for these patients or additional patients may not produce the same or consistent results. These and other risks are described in greater detail under the caption "Risk Factors" included in Vor Bio's most recent annual or quarterly report and in other reports it has filed or may file with the Securities and Exchange Commission. Any forward-looking statements contained in this Presentation speak only as of the date of this Presentation, and Vor Bio expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law.

Certain information contained in this Presentation relates to or is based on studies, publications, surveys and other data obtained from third party sources and Vor Bio's own internal

estimates and research. While we believe these third-party sources to be reliable as of the date of this Presentation, we have not independently verified, and make no representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third party sources. In addition, there can be no guarantee as to the accuracy or reliability of

any assumptions or limitations that may be included in such third-party information. While we believe our own internal research is reliable, such research has not been verified by any independent source.

2

Our Vision: Cure Blood Cancers Through Cell and Genome Engineering

Unique Approach

Shielded stem cell transplants enabling targeted therapy

VCAR33ALLO

Trem-cel

CD33-directed

Positive POC demonstrated

novel transplant donor

in AML with shielded

CAR-T in clinic with initial

CD33-deleted transplant

data in 2H 2024

Additional data in 2H 2024

$137M as of Dec. 31, 2023 Cash runway into 2H 2025

In-house GMP

Clinical Manufacturing

Reliable process with rapid

release time

3

AML is a Common Leukemia with High Unmet Need

20,000 new cases/year (US)2

(10% of new blood cancer cases)2

Standard of Care is Transplantation

Replace Diseased Bone Marrow with Healthy Donor Cells

Relapse

10,000 deaths/year (US)2

(20% of blood cancer deaths)2

Relapse Despite Transplantation

is still common in AML patients

0.8Post-transplant Incidence of Relapse1

Measurable Residual Disease (MRD) positive

3

3

1

2

3

4

Conditioning

Harvest

Transplant

Watchful Waiting

Incidence of

0.6

0.4

Active disease

MRD negative

Toxic therapies

Mobilize and collect

Infuse stem cells

Monitor for relapse;

to kill existing

stem cells from

into patient to

any follow-up

bone marrow

matched healthy

restore the blood

treatment will damage

donor

system

the transplant

Cumulative

0.2

0.0

0

1

2

Years Since HCT

HCT, Hematopoietic Cell Transplant.

  • 1 Araki et al, JCO 2016. 2 LLS 2023

Pipeline Generating Multiple Clinical Readouts over Next 12 Months

Description

Preclinical

Clinical

Anticipated Milestones

Program / Trial

Modality

Indication

Discovery/

IND-

Phase 1/2

Phase

Validation

Enabling

2/3

VCAR33ALLO

AML

Initial data expected in the

(healthy transplant donor

CD33-directed transplant donor CAR-T

Post-

second half of 2024

CAR-T) / VBP301

transplant

AML

Clinical update in second half of

Trem-cel + Mylotarg /

Shielded CD33-deleted transplant +

2024

VBP101

CD33-directed ADC

MDS

Trem-cel + VCAR33

Shielded CD33-deleted transplant +

AML

IND filing following initial

Treatment System

CD33-directed transplant donor CAR-T

trem-cel and VCAR33ALLO data

CD33-CLL1 Treatment

Multi-specificCAR-T

AML

System

Multiplex-edited shielded transplant

AML

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Cell Therapies Designed to Synergize to Potentially Cure AML

VCAR33ALLO

Healthy transplant donor CD33-directed

CAR-T

Trem-cel

Shielded CD33-deleted

stem cell transplant

Vor Bio Treatment

System

A shielded healthy marrow

co-existing with a

potent, persistent CAR-T

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VCAR33ALLO: Healthy Transplant Donor CAR-T

VCAR33ALLO

Healthy transplant donor

CD33-directed

CAR-T

7

A New Way of Generating CAR-T Therapy

Traditional Approaches

Autologous cells

Allogeneic cells

(derived from patient)

(off-the-shelf)

Exhausted, depleted T cells

Poor expansion and persistence

High manufacturing failure

Poorer clinical durability

Vor Bio Approach

Transplant Donor Cells

T cells exactly matched to patient's new immune system, more likely to persist

Stem-like,CAR-T cells more likely to expand and less prone to exhaustion

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Vor Bio's T Cell Manufacturing Process Preserves Stemness

T Cell Phenotype from VCAR33ALLO Process

100

90

Effector (TEM, TEMRA)

80

70

Central Memory (TCM)

60

Stem-like,

50

long-lived

40

Stem Memory (TSCM)

cells capable

30

of self-

20

renewal

10

0

CD4+

CD8+

Rapid ~7-day process to preserve stemness

Transplant donor cells

VCAR33ALLO

9

VBP301: VCAR33ALLO Phase 1/2 Clinical Trial

Patient Journey

Enroll

VCAR33ALLO Infusion

MRD+ or relapsed AML

Arm A: Blasts ≥ 5%

following SOC transplant or

Lymphodepletion

trem-cel transplant

Arm B: MRD+

3x3 dose escalation starting at 1 x 106 CAR+ cells/kg

Prior TransplantDonor

Consent

Collect

VCAR33ALLO

Manufacturing

~7-day process

First patient treated in Jan 2024 with initial data expected in 2H 2024

Day 28 Follow-up

2nd transplant

if required

Key Endpoints

01 Safety

02 Expansion, persistence

03 Disease control/response

10 Most clinical trial sites overlap with VBP101 trem-cel study

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Disclaimer

Vor Biopharma Inc. published this content on 20 March 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 20 March 2024 20:31:00 UTC.