Crispr Therapeutics Ag · NASDAQ:CRSP

Crispr Therapeutics — Q3 2026 Earnings Presentation

43 slides from Crispr Therapeutics's earnings presentation, Aug 3, 2026

1. Slide 1: CRISPR Q3 Corporate Update

   CRISPR Corporate Update Q3 Corporate Update August 3rd, 2026

2. Slide 2: Forward-Looking Statements

   Forward-Looking Statements​ This presentation and related materials may contain statements regarding matters that are not historical facts and are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and

3. Slide 3: Executing Our Vision Across Four Therapeutic Franchises

   Executing Our Vision Across Four Therapeutic Franchises Our vision is to develop cures for people suffering from serious diseases through transformative gene-based medicines Heme In Vivo CAR-T Regen Med Commercial / • CASGEVY®, with multi- • CTX310 Phase Ia update at • Best-in-class allogeneic • All

4. Slide 4: Broad & Diversified Pipeline

   Broad & Diversified Pipeline Program Disease(s) Research IND-Enabling Clinical Approved Partner CASGEVY1 SCD and TDT Heme In vivo HSC editing2 SCD, TDT, and others Autoimmune Zugocabtagene geleucel Autoimmune: SLE, SSc, IIM, ITP, WAIHA Anti-CD19 allogeneic CAR-T Oncology: B cell malignancies CAR-T I

5. Slide 5: © 2026 CRISPR Therapeutics

   © 2026 CRISPR Therapeutics 5

6. Slide 6: CASGEVY: Acceleration Towards Multi-$B Potential

   CASGEVY: Acceleration Towards Multi-$B Potential 2Q2026 CASGEVY revenue: $76M Regulatory expansion: • Approval for ages 2 to <12 years in U.S. • Regulatory submissions for 5 to <12 complete in KSA and U.K. Strong trends in Germany following access agreement in SCD and TDT patients 12 years and older

7. Slide 7: Continue to Build on the Momentum for CASGEVY in 2026

   Continue to Build on the Momentum for CASGEVY in 2026 Potential expansion into pediatric severe SCD and CASGEVY included on list of programs eligible for FDA’s TDT patients CNPV program Data presented at ASH 25, from pivotal studies in children ages 5-11, FDA NEWS RELEASE demonstrated the transforma

8. Slide 8: Serial Innovation in Enabling Technologies Will Broaden Access

   Serial Innovation in Enabling Technologies Will Broaden Access Targeted Conditioning In vivo Editing of HSCs Antibody–drug conjugate (ADC) for specific depletion of hematopoietic Delivery Editing stem cells (HSCs) with no off-target or bystander toxicity Proprietary GMP monoclonal antibody with shor

9. Slide 9: In vivo Editing: Achieved >50% Editing in HSCs, Durable >6 months

   In vivo Editing: Achieved >50% Editing in HSCs, Durable >6 months After a Single Dose in NHPs In Vivo HSC Editing Platform Editing in HSCs1 following a single IV dose of LNP-mRNA 60 • Proprietary antibody- conjugated LNP delivery 50 systems for in vivo HSC editing 40 • NHP studies provide % Editing

10. Slide 10: © 2026 CRISPR Therapeutics

    © 2026 CRISPR Therapeutics 10

11. Slide 11: Transformative Data with Our First In Vivo Program – CTX310

    Transformative Data with Our First In Vivo Program – CTX310 ~50% mean LDL and ~55% mean triglyceride reductions with well-tolerated safety profile in Phase I basket study for CTX310 targeting ANGPTL3 CRISPR to provide late-breaking update on CTX310 clinical trial at ESC Congress in August 2026 © 202

12. Slide 12: CTX310: CRISPR-Cas9 Editing of ANGPTL3

    CTX310: CRISPR-Cas9 Editing of ANGPTL3 Intravenous delivery Reduced atherogenic CRISPR/Cas9-based editing of ANGPTL3 targeting the liver lipoprotein concentrations CTX310 Endothelial lipase ANGPTL3 LDL-C Endocytosis Lipoprotein lipase and TG Endosomal escape DNA cleavage and repair Nuclear transport

13. Slide 13: ANGPTL3 Associated with Low Lipids and Low CV Risk

    ANGPTL3 Associated with Low Lipids and Low CV Risk Human Genetic Proof-Of-Concept Demonstrating Protective Effect of ANGPTL3 Loss-of-Function Variants Study 1: “The Long-Lived Village” Study 2: Familial Hypolipidemia • Residents of Campodimele, Italy are generally known to live • Nonsense mutations

14. Slide 14: Phase I Study Evaluating the Safety and Efficacy of CTX310

    Phase I Study Evaluating the Safety and Efficacy of CTX310 Open-label, multicenter, Phase Ia/Ib study evaluating the safety and efficacy of CTX310 in homozygous familial hypercholesterolemia (HoFH), heterozygous familial hypercholesterolemia (HeFH), severe hypertriglyceridemia (sHTG), or mixed dysli

15. Slide 15: CTX310 Baseline Participant Characteristics

    CTX310 Baseline Participant Characteristics © 2026 CRISPR Therapeutics 15

16. Slide 16: CTX310: Mean Percent Change from Baseline in ANGPTL3

    CTX310: Mean Percent Change from Baseline in ANGPTL3 N = 15 patients 9.6% 9.4% Percent change from baseline (%) -32.7% -73.2% -79.7% 0.1 mg/kg 0.3 mg/kg 0.6 mg/kg 0.7 mg/kg 0.8 mg/kg (N=3) (N=3) (N=3) (N=2) (N=4) Data presented at American Heart Association Meeting in Nov 2025 Note: Mean percent cha

17. Slide 17: CTX310: Mean Percent Change from Baseline in LDL-C and Triglycerides

    CTX310: Mean Percent Change from Baseline in LDL-C and Triglycerides LDL Cholesterol Triglycerides 46.7% 15.4% 38.8% Percent change from baseline (%) Percent change from baseline (%) 4.2% -21.0% -19.2% -39.2% -48.9% -55.2% -62.0% 0.1 0.3 0.6 0.7 0.8 0.1 0.3 0.6 0.7 0.8 mg/kg mg/kg mg/kg mg/kg mg/kg

18. Slide 18: CTX310: Adverse Events & Safety Findings

    CTX310: Adverse Events & Safety Findings N (%) Any serious adverse events 2 (13) Serious adverse events related to CTX310 0 (0) Participants with any investigator-reported adverse event 14 (93) Adverse event deemed related to CTX310 7 (47) Adverse event of special interest 4 (27) • Allergic or local

19. Slide 19: CTX310 Has Multi-Billion Commercial Opportunity

    CTX310 Has Multi-Billion Commercial Opportunity Addressable US Patient Populations Across sHTG, HoFH, HeFH, and non-FH sHTG HoFH HeFH + non-FH2 3M ~1,300 ~1M Prevalence 0.9M ~1,200 150k – 220k CTX310 Addressable Patient population ~30% High-risk ~90% Refractory to ~20% Refractory to AP1 patients PCS

20. Slide 20: Lp(a) Gene Editing Program is a Key Focus for the Company

    Lp(a) Gene Editing Program is a Key Focus for the Company Lp(a) is an independent risk factor for atherosclerotic cardiovascular CTX321: New Lp(a) gRNA has potency on par with CTX310 disease (ASCVD), affecting ~ one-fifth of global population Elevated LP(a) is high risk for ASCVD1 Lp(a) levels in ge

21. Slide 21: Additional In Vivo Programs have entered Phase I Clinical Trials

    Additional In Vivo Programs have entered Phase I Clinical Trials CTX340 Targeting AGT For Refractory Hypertension (rHTN) CTX460 Targeting SERPINA1 for AATD • Hypertension is the leading cause of cardiovascular morbidity and • Alpha-1 antitrypsin deficiency (AATD) is caused by mutations in the mortal

22. Slide 22: CTX611 is a Factor XI siRNA with Best-in-Class Potential

    CTX611 is a Factor XI siRNA with Best-in-Class Potential FXI: Interrupting thrombosis while maintaining hemostatic clotting Unmet Need • Current anti-coagulation market is ~$20B per year Traditional clotting factors Factor XI • Patients with bleeding risk or certain conditions, such as (e.g., FXa /

23. Slide 23: CTX611 Phase 1 Data: Shows Deep, Durable Reductions in FXI Activity

    CTX611 Phase 1 Data: Shows Deep, Durable Reductions in FXI Activity CTX611 Phase I Clinical Results: Dose-dependent pharmacodynamic response to therapy sustained through Month 6 FXI Antigen FXI Activity 95.0% peak reduction in FXI antigen 93.9% peak reduction in FXI activity Clinical trials executed

24. Slide 24: CTX611 Phase II Trials: TKA Top-Line Data Expected in 2H 2026

    CTX611 Phase II Trials: TKA Top-Line Data Expected in 2H 2026 CTX611-201 Phase II Clinical Program: TKA Top-line Data Expected in 2H 2026 CTX611-202 Phase II CV Study Design Objective: Establish PoC efficacy in TKA-VTE participant population Objective: Establish PK-PD in chronic “arterial” populatio

25. Slide 25: CTX611 Has Significant Potential Across Multiple Indications

    CTX611 Has Significant Potential Across Multiple Indications US Potential Market Opportunity for FXI Anticoagulation Agent ~2M Number of Patients ~850k ~800k ~800k ~250k ~300k Cancer Total ESRD Patients with Atrial Fibrillation - Secondary Stroke PAD post Associated VTE Knee Arthroplasty catheter DO

26. Slide 26: © 2026 CRISPR Therapeutics

    © 2026 CRISPR Therapeutics 26

27. Slide 27: CRISPR Tx Cell Therapy Strategy

    CRISPR Tx Cell Therapy Strategy CRISPR Focus CAR-T Landscape Ex vivo In vivo Allogeneic Allogeneic In vivo In vivo Autologous (Healthy donor) (iPSC derived) (Viral) (Non-viral) CRISPR Ex Vivo Cell Therapy Focus CRISPR In Vivo Cell Therapy Focus • CRISPR built allogeneic cell therapy platform on heal

28. Slide 28: Zugo-cel is an Allogeneic CAR-T Optimized for Potency

    Zugo-cel is an Allogeneic CAR-T Optimized for Potency Zugo-cel Novel Potency Edits (TGFBR2, Regnase-1) Several Competitive Advantages TGFBR2 disruption Anti-CD19 CAR Ability to multiplex gene edits precisely TCR disruption and efficiently MHC I disruption Comprehensive and FDA-validated genomic anal

29. Slide 29: Zugo-cel Phase I Autoimmune Basket Clinical Trial Ongoing

    Zugo-cel Phase I Autoimmune Basket Clinical Trial Ongoing Open-label, Phase 1 clinical trial evaluating the safety and efficacy of zugo-cel in adult subjects with refractory autoimmune disease Zugo-cel infusion Trial Design: Screening Standard Lymphodepletion D28 assessment and follow-up Fludarabine

30. Slide 30: Zugo-cel Clinical Data Shows Comparable PK and PD in AID and NHL

    Zugo-cel Clinical Data Shows Comparable PK and PD in AID and NHL Cell Expansion Summary1 B Cell2 Depletion 1,000 8000 SLE 100M (n=2) SLE (n=2) 6000 NHL (n=44) NHL 100M (n=3) 4000 [CAR] (copies/µg) B Cells (Cells/μL) 2000 100 300 200 10 LLOQ LOD 100 1 0 n 4 8 12 16 20 24 28 14 28 M 2 15 2 3 6 M9 on i

31. Slide 31: Case Study of SLE Patients Treated with Zugo-cel

    Case Study of SLE Patients Treated with Zugo-cel 2 SLE patients treated and have discontinued treatment with immunomodulators Baseline Characteristics SLE-1 SLE-2 Clinical Efficacy Results Dose DL1 (100M cells) DL1 (100M cells) Age/Sex 33 yo F 30 yo F Disease Duration (years) 10 11 DORIS Clinical Re

32. Slide 32: Zugo-cel Autoimmune Program Expansion

    Zugo-cel Autoimmune Program Expansion Indication Expansion Summary Geographic Expansion Summary Heme AID CNS AID expansion expansion Scleroderma ITP PPMS Myositis WAIHA NMOSD Lupus MOGAD NMDAR / LGI1 AIE SPS Cleared IND application for Phase I trial in several Enrollment initiated in the EU autoimmu

33. Slide 33: Zugo-cel Phase I Immuno-Oncology Clinical Trial Design

    Zugo-cel Phase I Immuno-Oncology Clinical Trial Design Open-label, multicenter, Phase I/II study evaluating the safety and efficacy of zugo-cel in relapsed or refractory B-cell malignancies Patients could receive an additional infusion of zugo-cel Zugo-cel infusion with LD chemotherapy if they achie

34. Slide 34: Zugo-cel Baseline Characteristics (LBCL N=10, DL4)

    Zugo-cel Baseline Characteristics (LBCL N=10, DL4) Baseline Characteristics Total N = 10 Age > 65, n (%) 7 (70) Sex (Female), n (%) 6 (60) Prognostic Score at Baseline3 • Intermediate or High Risk, n (%) 7 (70) LBCL Subtype, n (%) • DLBCL NOS 4 (40) • Transformed Follicular Lymphoma 3 (30) • Transfo

35. Slide 35: Zugo-cel Ph1 Data Suggest Durability in LBCL at RP2D (600M)

    Zugo-cel Ph1 Data Suggest Durability in LBCL at RP2D (600M) (N=10 with D28 response assessment1) CR PR PD LBCL Pt 1 Second zugo-cel infusion LBCL Pt 2 Response assessment LBCL Pt 3 LBCL Pt 4 • ORR 90%, CRR 70% at DL4 (600M cell dose) LBCL Pt 5 • 2 CRs ongoing at 12-mo visit LBCL Pt 6 • 83% ORR (5/6)

36. Slide 36: Collaboration to Study Zugo-cel Combined with Pirtobrutinib

    Collaboration to Study Zugo-cel Combined with Pirtobrutinib Potential for deep, durable responses with an off-the-shelf therapy for aggressive B-cell lymphomas Zugo-cel Pirtobrutinib (allogeneic CD19 CAR-T) (non-covalent BTKi) • Zugo-cel has demonstrated overall and complete response rates comparabl

37. Slide 37: CRISPR's in vivo CAR-T Platform Overview

    CRISPR’s in vivo CAR-T Platform Overview Best-in-class Platform for Transient and Integrated in vivo CAR-Ts Targeted Delivery Extended Durability Permanent CAR approaches T cell specific delivery and liver Proprietary mRNA optimization for best- Non-viral delivery, site-specific HDR- de-targeting wi

38. Slide 38: CRISPR's mRNA-LNPs Generate CAR+ T Cells In Vivo

    CRISPR’s mRNA-LNPs Generate CAR+ T Cells In Vivo CDX1 binder delivers to primarily CD8+ T cells and CDX2 binder delivers both CD4+ and CD8+ T cells Study design Spleen Bone marrow CAR-LNP (0.5mg/kg) CD4+ T cells CD8+ T cells CD4+ T cells CD8+ T cells 100 100 76.3 80 80 % CAR+ 60 % CAR+ PBMC-hNSG 60

39. Slide 39: CDX1-Targeted eGFP mRNA-LNPs Showed Robust Delivery to CD8 T cells

    CRISPR’s CDX1-Targeted eGFP mRNA-LNPs Showed Robust Delivery to CD8 T cells with Durable Expression up to 14 days CDX1 Targeted eGFP mRNA-LNP study in Cynomolgus Monkey, Peripheral Blood readout (0.5mg/kg eGFP mRNA-LNP, mRNA Format v1) Study Design % GFP positive cells % GFP positive cells 80 60 CD4

40. Slide 40: Multi Dose LNP-CDX1 Binder Led to Deep B-cell Depletion

    Multi Dose of 0.5 mg/kg LNP-CDX1 Binder 2 and Binder 3 Led to Deep B-cell depletion in Blood, Bone Marrow, Lymph Nodes and Spleen Blood Bone Marrow Lymph Nodes Spleen (Up to Day 7) (Day 8) (Day 8) (Day 10) % Remaining B Cells % Remaining B Cells % Remaining B Cells Peripheral CD20+ B Cell Count Dosi

41. Slide 41: CTX213: Deviceless Hypoimmune Islet Cell Therapy for T1D

    CTX213: Deviceless Hypoimmune Islet Cell Therapy for T1D CTX211, a gene-edited, stem cell–derived encapsulated cell CTX213, an unencapsulated iPSC-derived islet cell product product for the treatment of type 1 diabetes utilizing the same edits as CTX211 • Ph1 completed in n=5 patients • Well-tolerat

42. Slide 42: Anticipated Key Milestones in 2026

    Anticipated Key Milestones in 2026 Program Timing Details Quarterly Commercial launch updates Heme In vivo HSC 2H 2026 Preclinical data update CTX310 2H 2026 Phase Ia CV trial clinical data CTX611 2H 2026 Phase II TKA study top-line data In Vivo CTX340 1H 2026 Phase I rHTN trial initiated CTX460 Mid

43. Slide 43: © 2026 CRISPR Therapeutics

    © 2026 CRISPR Therapeutics 43

CRISPR Therapeutics — Q3 2026 Earnings Presentation on 6ix