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Forward Looking Statement 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" o

Key Takeaway: Vor Biopharma Inc. provided an update on its forward-looking statements regarding the development of telitacicept, a BAFF/APRIL inhibitor for autoimmune diseases. The company highlights the potential of telitacicept in treating generalized myasthenia gravis and primary Sjögren's disease, anticipating it to be a best-in-class treatment. Vor Biopharma currently holds a strong cash position that extends its runway for critical milestones into early 2027. However, the company acknowledged various risks including uncertainties in clinical trials and regulatory approvals.

Market Sentiment Analysis

POSITIVE FACTORS

  • Vor Biopharma is optimistic about the potential of telitacicept in autoimmune diseases.
  • The company has a strong cash position of $190 million, providing a runway into 1Q27.
  • Clinical trial data shows favorable safety profiles with no burdensome vaccination requirements.

CONCERNS & RISKS

  • There are uncertainties regarding the initiation and completion of clinical trials.
  • Regulatory approvals for trials or marketing products have risks attached.
  • Reliance on third parties may pose operational challenges.

Full Press Release Details

Global Science. One Purpose. Corporate
Presentation September 2025 Exhibit 99.1
Forward Looking Statement 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 Vor Bio's plans for development and
commercialization of telitacicept, the potential of telitacicept in various indications including generalized myasthenia gravis (gMG) and primary Sj gren's disease, the potential of telitacicept to be a best- and first-in-class BAFF/APRIL
inhibitor globally in gMG, the potential best-in-disease profile of telitacicept in primary Sj gren's disease, the availability of data from clinical trials including those conducted by third parties, the expected safety profile of
telitacicept, the market opportunities for telitacicept, the addressable patient populations in the indications Vor Bio intends to treat, telitacicept's therapeutic potential, Vor Bio's cash runway and other statements that are not historical fact.
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; 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; Vor Bio's reliance on third parties over which it may not always have full control; and the availability of funding sufficient for Vor Bio's foreseeable and unforeseeable operating expenses and capital expenditure requirements. 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 the Company believes these third-party sources to be reliable as of the date of this Presentation, the Company has not independently verified, and makes 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 the Company believes its own
internal research is reliable, such research has not been verified by any independent source. All brand names or trademarks appearing in this Presentation are the property of their respective owners.
China Is Now the Epicenter of Drug
Innovation 50%+ of new global molecules originate in China Out-licensing deals are surging ($58B+ in 2025) ICH, International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use; Source: Bloomberg and Bernstein
analysis; 1. Bloomberg, SMMT:NASDAQ 2. BioNTech and BMS release The Next Breakthrough Wave Is Coming From China Validation in China is Globally Credible Strong data across biotech sector Faster, larger trials with strong signals NMPA aligned with
ICH standards Western Deals Unlock Massive Value Summit/Akeso1: $17B+ market cap gain Biotheus - BioNTech - BMS2: $1B to $11B+ Proven model: China assets scale globally
01 The Medicine Meets the Moment 02 03
04 Telitacicept Dual BAFF/APRIL Inhibitor Optimal approach for B cell driven autoimmune diseases Addresses upstream and downstream signaling Myasthenia Gravis The Beachhead Indication Best-in-disease foundation from China Phase 3 clinical study
Market shifting from symptom control to disease modification Significant Expansion Opportunities Blockbuster potential in multiple autoimmune indications Sj gren's Disease: The next multi-billion-dollar opportunity World Class Management
Team Domain experts in autoimmune diseases, clinical development, and commercialization
Telitacicept Leads the BAFF/APRIL
Field with Broad Approval Momentum Strong cash position of $190M* with runway into 1Q27 covers critical milestones Indications Preclinical Phase 1 Phase 2 Phase 3 Marketing Approval Milestones Systemic Lupus Erythematosus (SLE) Rheumatoid Arthritis
(RA) Myasthenia Gravis (MG) OLE 48-wk Data - AANEM (10.29.25) Topline Global Data 1H27 IgA Nephropathy (IgAN) Dataset at Upcoming Medical Meeting Primary Sj gren's Disease (pSD) Dataset at Upcoming Medical Meeting FDA Cleared Global
Phase 3 Neuromyelitis Optica Spectrum Disorder (NMSOD) Lupus Nephritis (LN) Membranous Nephritis (MN) and Other Indications China Marketed China Marketed China Marketed BLA Submitted BLA Submitted Phase 3 Phase 2 Phase 3 Phase 3 Ready Phase 2 Vor
- Global Trial RemeGen - China Trial Primary Endpoint Achieved *cash and cash equivalents as of June 30,2025
Disease Modification Through Upstream
and Downstream Control Ab, antibody Dual BAFF/APRIL blockade stops B cell survival and plasma cell antibody production Drives B cell development and survival Promotes proliferation of mature B cells BAFF Upstream BAFF APRIL Telitacicept TACI-Fc
fusion protein: dual inhibition of BAFF/APRIL B cell differentiation Isotype-switching B cell survival maturation Plasma cell survival Anti-inflammation B-cell function Sustains long-lived plasma cells in bone marrow Supports pathogenic Ab
production Drives Ig isotype class switching APRIL Upstream + Downstream Telitacicept Dual blockade of BAFF/APRIL Normalization of B cell function Cuts off plasma cell persistence + Ab production Restores immune balance Upstream + Downstream
doi.org/10.3390/cancers12041045
Commercial Approvals
Conditional Approval, Full Approval in 2023; *Accelerated Approval in China; Est., estimated Unprecedented Efficacy in China Across Autoimmune Diseases BLA Submissions Best-In-Disease in China 3 2 3 2021 - Systemic Lupus Erythematosus
(SLE) 2024 - Rheumatoid Arthritis (RA) 2025 - Myasthenia Gravis (MG) Est. 2026 - Primary Sj grens Disease (pSD) Est. 2026 - IgA Nephropathy (IgAN)* Validated Commercial Therapy in China Across Diverse Autoimmune Diseases Poised to
Further Expand Telitacicept Footprint in Large, Underserved Diseases in China Unique Dual BAFF/APRIL Inhibition Drives Superior Clinical Benefit Systemic Lupus Erythematosus Myasthenia Gravis Primary Sj grens Disease
*From pooled safety analysis across
all telitacicept trials. AEs, adverse events; SAEs, serious adverse events. Favorable Safety At Scale Favorable and Predictable Safety Profile Observed Among ~1,800* Patients Studied in Clinical Trials No Burdensome Vaccination Requirements No
Signature B Cell Depletion Associated SAEs Mild to Moderate AEs Placebo (n=527) Telitacicept (n=1211) Blood IgM decreased 2 6 Blood IgG decreased 1 5 Cough 3 5 Diarrhea 5 5 Urinary tract infection 9 10 Injection site reaction 2 17 Upper respiratory
tract infection 30 35 >70,000 Patients Treated Commercially in China Frequency (%) of safety events reported in clinical trials
A Pipeline of Autoantibody-Driven
Diseases & Blockbuster Opportunities ANCA-AAV, ANCA-associated Vasculitis; ITP, Immune Thrombocytopenic Purpura; CIDP, Chronic Inflammatory Demyelinating Polyradiculoneuropathy; NMOSD, Neuromyelitis Optica Spectrum Disorder; IgG4-RD,
IgG4-related disease. Telitacicept can potentially address more than 1 million patients1 in the US alone CIDP 30,000 Sj gren's Disease 290,000 ANCA-AAV 140,000 SLE 240,000 Myasthenia Gravis 90,000 NMOSD 25,000 ITP 65,000 Bullous
Pemphigoid 40,000 Membraneous Nephropathy 70,000 IgG4-RD 20,000 Lupus Nephritis 105,000 1. Vor metanalysis and estimates
Myasthenia Gravis Moving Beyond IgG
A Large and Growing Global
Opportunity in Myasthenia Gravis ~260,000 diagnosed MG patients across key markets1 Significant Burden A large, diagnosed patient population across key markets establishes a substantial initial opportunity Favorable Growth Drivers Prevalence is
growing due to increased awareness, improved diagnostics, and an aging population ~90k US prevalence ~140k EU prevalence ~29k Japan prevalence ~220k China prevalence MG, myasthenia gravis 1. Vor metanalysis and estimates; excludes Chinese
MG Market Rapidly Expanding But
Lacks Disease-Modifying Treatments MG, myasthenia gravis; gMG: generalized myasthenia gravis; 1. EvaluatePharma and Global Data sales and consensus High growth market with underserved patient segments gMG US Biologic Sales ~90,000 Diagnosed MG
patients in the US 60% Potential expansion of branded medicines in eligible gMG patients 62% CAGR increase since 2018 ~$3.7B1 ~$10.7B1 Projected Market Size (2030)
Biologic use rising (~35% of gMG
patients1) with choices driven by efficacy, convenience, phenotype, and coverage FcRn inhibitors dominate but ~20-50% of patients experience insufficient treatment responses2 Complement inhibitors limited by safety, black box warning, and
vaccination requirements3 B cell depleting and cell therapies face challenges with efficacy, safety, and logistical limits despite disease-modifying potential4 Current Myasthenia Gravis Therapies Target Symptoms, Not Disease AChEI,
acetylcholinesterase inhibitors; AChR, acetylcholine receptor; FcRn, neonatal Fc receptor; IVIG, intravenous immune globulin; MuSK, muscle-specific tyrosine kinase; PLEX, plasma exchange; RTX, rituximab; ISx, immunosuppressants. Even with the
availability of biologics, unmet need for new therapies remain 1. Wedbush Neurologist Surveys 2. doi: 10.1136/jnnp-2024-334404 3. ULTOMIRIS, SOLIRIS label 4. doi: 10.1007/s40259-020-00443-w AChR+ MG AChEi Steroids ISx MuSK+ MG Steroids
RTX RTX FcRn Antagonists C5 Inhibitors, IVIG, PLEX IVIG, PLEX
Phase 3 Trial in Generalized
Myasthenia Gravis Completed in China MG-ADL; Myasthenia Gravis Activities of Daily Living; QMG, Quantitative Myasthenia Gravis; QW, per week. ClinicalTrials.gov. NCT05737160. Updated May 5, 2025. Accessed August 19, 2025.
https://clinicaltrials.gov/study/NCT05737160 Best-in-disease profile in China; randomized, double-blind, placebo-controlled study gMG R 1:1 114 Adults With gMG Double-Blind Treatment Period (24 Weeks) Open-Label Extension Period (24 Weeks)
Telitacicept (240mg QW) Placebo Telitacicept (240mg QW) Primary Endpoint Change from baseline in MG-ADL at 24 weeks Secondary Endpoints Change from baseline in MG-ADL at 12, 36, and 48 weeks Change from baseline in QMG at 12, 24, 36, and 48 weeks
Number of patients with 3 point decrease in MG-ADL, 5 point decrease in QMG at 24 and 48 weeks RemeGen-sponsored trial
Well Balanced Baseline
Characteristics Consistent with recent global Phase 3 populations AE, adverse event; AChR, acetylcholine receptor; EP, efficacy population; MG-ADL, Myasthenia Gravis-Activities of Daily Living; MGFA, Myasthenia Gravis Foundation of America; MuSK,
muscle-specific tyrosine kinase; QMG, Quantitative Myasthenia Gravis; SD, standard deviation; RS, randomized set; SS, safety set Telitacicept (N=57) Placebo (N=57) Age (yr), mean SD 49.1 14.69 49.6 15.03 Sex Male, n
(%) 30 (52.6) 21 (36.8) Female, n (%) 27 (47.4) 36 (63.2) Disease duration (month), mean SD 83.09 84.507 76.05 87.817 MGFA classification Class IIa, n (%) 3 (5.3) 12 (21.1) Class IIb, n (%) 14 (24.6) 9 (15.8) Class IIIa, n
(%) 25 (43.9) 23 (40.4) Class IIIb, n (%) 11 (19.3) 12 (21.1) Class IVa, n (%) 4 (7.0) 1 (1.8) Baseline MG-ADL score, mean SD 10.0 2.60 9.9 2.62 Baseline QMG score, mean SD 17.9 3.43 18.8 3.65
Antibody-positive at screening AChR, n (%) 55 (96.5) 55 (96.5) MuSK, n (%) 2 (3.6) 2 (3.5) Standard-of-care therapy Anticholinesterase inhibitors, n (%) 53 (93.0) 52 (91.2) Steroids, n (%) 36 (63.2) 34 (59.6) Immunosuppressants, n (%) 34 (59.6) 34
(59.6) Screening (n=148) Randomized (n=114) Received Double-Blind Treatment (n=114) Telitacicept (n=57) Placebo (n=57) Completed Treatment (n=54) Completed Study (n=54) Completed Treatment (n=50) Completed Study (n=52) RS (n=57) EP (n=57) SS (n=57)
RS (n=57) EP (n=57) SS (n=57) Discontinued Treatment (n=3) Discontinued Study (n=3) Discontinued Treatment (n=7) Discontinued Study (n=5) gMG RemeGen-sponsored trial
MG-ADL Responders - Primary
Endpoint Met MG-ADL, Myasthenia Gravis-Activities of Daily Living; MMRM, mixed models for repeated measures. Statistically significant and clinically meaningful improvement in activities of daily living -4.8 -5.74 -0.91 Change from Baseline in
MG-ADL at Week 24 points Placebo-adjusted decrease in MG-ADL: The primary estimate was analyzed using MMRM. Intercurrent event was predefined, Missing data was imputed. gMG RemeGen-sponsored trial
QMG Responders - Secondary Endpoint
Met QMG, Quantitative Myasthenia Gravis; MMRM, mixed models for repeated measures. Statistically significant and clinically meaningful improvement in physician-assessed measure of muscle strength -6.4 -8.66 -2.27 Change from Baseline in QMG at Week
24 points Placebo-adjusted decrease in QMG: The data was analyzed using MMRM. Missing data was imputed, and as observed (AO) analysis was performed. gMG RemeGen-sponsored trial
Telitacicept: Potential
Best-In-Disease Efficacy Globally Statistically significant and clinically meaningful improvement in MG-ADL score -4.8 FcRn Inhibitors Complement Inhibitors Anti-CD19 mAb PBO-adjusted LSM MG-ADL Change from Baseline gMG RemeGen-sponsored trial
MG-ADL, Myasthenia Gravis-Activities of Daily Living; MMRM, mixed models for repeated measures. Based on historical clinical data; not a head-to-head trial Efgartigimod - ADAPT; Nipocalimab - Vivacity-MG3; Rozanolixizumab - MycarinG;
Ravulizumab - CHAMPION-MG; Eculizumab - REGAIN; Zilucoplan - RAISE; Inebilizumab - MINT
Telitacicept Significantly Improved
MG-ADL and QMG Scores gMG Missing data imputed as non-response. MG-ADL, Myasthenia Gravis - Activities of Daily Living; QMG, Quantitative Myasthenia Gravis. Mean Change in QMG Score -8.66 -2.27 Mean Change in MG-ADL Score -5.74
-0.91 Proportion of patients with a 3 point reduction in MG-ADL score from baseline over time 12.0% 98.1% Proportion of patients with a 5 point reduction in QMG score from baseline over time 16.0% 87.0% RemeGen-sponsored
Telitacicept Provides Sustained
Disease Modification While FcRn Inhibitors Require Repeat Cycles Missing data imputed as non-response. MG-ADL, Myasthenia Gravis - Activities of Daily Living REMEGEN Phase 3 China Trial -5.74 -0.91 Argenx Phase 3 Adapt Trial Cross trial
comparison = Mean Change in Total MG-ADL From Cycle 1 Baseline Over Time in AChR-Ab Positive Patients (mITT Analysis Set) Mean Change in MG-ADL Score RemeGen-sponsored trial Based on historical clinical data; not a head-to-head
Consistent Reduction in IgG, IgA,
IgM, and B Cells gMG IgG -32.89% 0.69% IgA -59.57% 0.00% IGM -68.93% 0.53% B cell -24.37% 2.61% RemeGen-sponsored trial
Favorable Safety Profile AE,
adverse event; pSD, primary Sjogren's disease; SLE, systemic lupus erythematosus; RA, rheumatoid arthritis; IgAN, IgA nephropathy Consistent with data from clinical trials in SLE, RA, pSD, and IgAN, and post-marketing data gMG Telitacicept
Placebo (n=57) (n=57) n (%) Events n (%) Events Infection-Associated AEs in >5% of patients Infections and infestations 26 (45.6) 46 34 (59.6) 50 Upper respiratory tract infection 12 (21.1) 17 20 (35.1) 24 Urinary tract infection 9 (15.8) 11 6
(10.5) 6 Pneumonia 1 (1.8) 1 6 (10.5) 6 Respiratory tract infection 1 (1.8) 1 2 (3.5) 2 Influenza 0 (0) 0 3 (5.3) 3 Number of Serious AEs 4 (7.0) 4 6 (10.5) 7 Pneumonia 1 (1.8) 1 4 (7.0) 4 COVID-19 pneumonia 1 (1.8) 1 0 (0) 0 Influenza 0 (0) 0 1
(1.8) 1 Upper respiratory tract infection 0 (0) 0 1 (1.8) 1 Open fracture 0 (0) 0 1 (1.8) 1 Pneumonitis 1 (1.8) 1 0 (0) 0 Accidental death 1 (1.8) 1 0 (0) 0 RemeGen-sponsored trial
Telitacicept Targets What Matters

Frequently Asked Questions

What is Vor Biopharma known for?

Vor Biopharma focuses on developing telitacicept for autoimmune diseases.

What are the potential benefits of telitacicept?

Telitacicept may provide disease modification and has blockbuster potential.

What autoimmune conditions does telitacicept target?

It targets conditions like myasthenia gravis and primary Sjögren's disease.

What is the safety profile of telitacicept?

Telitacicept shows a favorable and predictable safety profile in trials.

How many patients have been treated with telitacicept in China?

Over 70,000 patients have been treated commercially with telitacicept.

Last updated: Sep 2, 2025