Crispr Therapeutics Ag · NASDAQ:CRSP
Crispr Therapeutics — Corporate Update (February 2026)
43 slides from Crispr Therapeutics's corporate update, Feb 12, 2026
Slide 1: CRISPR Corporate Update
CRISPR Corporate Update Q1 Corporate Update February 12, 2026 1
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
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- • Transformative Phase 1 • Best-in-class allogeneic • Alloge
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
Slide 5: Hemoglobinopathies
Hemoglobinopathies
Slide 6: CASGEVY: Acceleration Towards Multi-$B Potential
CASGEVY: Acceleration Towards Multi-$B Potential Key Highlights Approved1 in 10 Countries2; At least 1 ATC in each region has initiated 20+ patients • Accelerating patient infusions Patients initiated 1st Cell collections Cell infusions via global ATC network • Broad coverage: ̶ ~90% Medicaid / Comm
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
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
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
Slide 10: In Vivo
In Vivo
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 Paradigm changing – Potential to disrupt the chronic care model with a “one and done” solution © 202
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
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
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
Slide 15: CTX310 Baseline Participant Characteristics
CTX310 Baseline Participant Characteristics © 2026 CRISPR Therapeutics 15
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
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
Slide 18: CTX310: Mean Percent Change in ApoB and Non-HDL-C
CTX310: Mean Percent Change in ApoB and Non-HDL-C Apolipoprotein B Non-HDL Cholesterol 14.3% 9.7% Percent change from baseline (%) Percent change from baseline (%) 13.3% 15.7% -23.7% -22.9% -33.4% -38.0% -44.6% -49.8% 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 mg/kg mg/kg
Slide 19: CTX310: Mean Percent Change in Apo CIII and Remnant Cholesterol
CTX310: Mean Percent Change in Apo CIII and Remnant Cholesterol Apolipoprotein CIII Remnant Cholesterol 31.7% 55.9% Percent change from baseline (%) Percent change from baseline (%) -3.0% -27.3% -33.3% -43.8% -42.2% -52.5% -58.3% -61.7% 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
Slide 20: 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
Slide 21: 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
Slide 22: 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
Slide 23: Additional In Vivo Programs Advancing Toward Clinical Trials
Additional In Vivo Programs Advancing Toward 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 mortality
Slide 24: 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 /
Slide 25: CTX611 Phase 1 Data: 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
Slide 26: 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
Slide 27: 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
Slide 28: CAR-T
CAR-T
Slide 29: 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
Slide 30: 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
Slide 31: 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
Slide 32: 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
Slide 33: Case Study of SLE Patients Treated with Zugo-cel
Case Study of SLE Patients Treated with Zugo-cel 2 SLE patients in remission; First SLE participant in drug-free remission maintained at Month 9 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 1
Slide 34: Zugo-cel Autoimmune Program Expansion
Zugo-cel Autoimmune Program Expansion Indication Expansion Summary Geographic Expansion Summary Heme AID expansion ITP WAIHA Scleroderma Scleroderma CTA approved in Germany Myositis Myositis Lupus Lupus IND approved for autoimmune hematologic disorders Expanding into the EU with approval of CTA in i
Slide 35: 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
Slide 36: 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
Slide 37: 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)
Slide 38: 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
Slide 39: Our In Vivo CAR-T Approach
Our In Vivo CAR-T Approach Transient, Re-dosable CAR-T Non-Viral, Integrating CAR-T Proprietary Antibody-LNP Platform • Targeted delivery to immune cells • Site-specific conjugation with robust manufacturability • Long-circulating LNPs minimizes off-target delivery (e.g., liver) • Proprietary binder
Slide 40: Targeted LNPs Showed Robust Delivery to T-cells
Targeted LNPs Showed Robust Delivery to T-cells with Durable Expression and Minimum Off-Target Expression in the Liver Cynomolgus Monkey, Peripheral Blood Humanized Mouse (0.5mg/kg eGFP mRNA-LNP) (0.5mg/kg eGFP mRNA-LNP, 24hr) % GFP positive cells Liver Immunohistochemistry (IHC) 60 CD8 T cells 40 2
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
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 Ib CV trial clinical data CTX611 2H 2026 Phase II TKA study top-line data In Vivo CTX340 1H 2026 Phase I rHTN trial initiation CTX460 Mi
Slide 43: CRISPR Therapeutics www.crisprtx.com
CRISPR Therapeutics www.crisprtx.com 43
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