Crispr Therapeutics Ag · NASDAQ:CRSP
Crispr Therapeutics — Investor Presentation (July 2025)
33 slides from Crispr Therapeutics's investor presentation, July 2025
Slide 1: Creating transformative gene-based medicines for serious diseases
Creating transformative gene-based medicines for serious diseases Corporate Overview Q2 2025 CRISPR THERAPEUTICS – CONFIDENTIAL © 2025 CRISPR Therapeutics | 11
Slide 2: Forward-Looking Statements
Forward-Looking Statements Statements contained in this presentation and other related materials regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks
Slide 3: CRISPR Therapeutics Today
CRISPR Therapeutics Today Our vision is to develop cures for people suffering from serious diseases through transformative gene-based medicines 1 approved 5 clinical 10 preclinical therapy programs programs CASGEVY® for severe sickle cell Expanded portfolio into both Establish a sustainable disease
Slide 4: Executing on Our Vision Across Four Therapeutic Franchises
Executing on Our Vision Across Four Therapeutic Franchises Heme CAR T In vivo T1D Best-in-class allogeneic cell Establishing a differentiated LNP- Partnered with Vertex on global therapies with novel potency edits mRNA platform, initially focused Utilizing gene editing to develop an launch of CASGEV
Slide 5: Broad and Diversified Pipeline
Broad and Diversified Pipeline Program Disease(s) Research IND-enabling Clinical Approved Partner Structure Severe sickle cell disease (SCD) CASGEVY1 Heme Transfusion-dependent β-thalassemia (TDT) Collaboration CD117 ADC / In vivo HSC editing SCD, TDT and others B cell malignancies CTX112 Wholly own
Slide 6: Entering a Critical Phase of Our Growth Journey
Entering a Critical Phase of Our Growth Journey Sector-Leading Biotech Inflection Year ▪ CASGEVY revenue provides a path to Foundational Years a sustainable biotech company ▪ Strong launch trajectory for CASGEVY ▪ Relentless focus to bring CASGEVY to globally with favorable market access ▪ Clinical
Slide 7: Hemoglobinopathies
Hemoglobinopathies CRISPR THERAPEUTICS – CONFIDENTIAL 7
Slide 8: 2024: A Foundational Year for CASGEVY
2024: A Foundational Year for CASGEVY Addressable Market2 Unparalleled speed and execution to a landmark approval1 ~60,000 Severe patients in approved territories eligible for treatment Investments made to meet global demand for disease-modifying therapy 1 Approved by the U.S. FDA for treatment of p
Slide 9: 2025: Focused on Execution and Expansion of Opportunity
2025: Focused on Execution and Expansion of Opportunity Continued Progress in U.S. to Expanding into untapped Middle Manufacturing Expansion to Serve Significant Unmet Need East and ex-U.S. Markets Support Launch1 New CMMI model to improve access and First GCC patient reimbursed at ~$2M; Manufacturi
Slide 10: Serial Innovation in Enabling Technologies to Broaden Access
Serial Innovation in Enabling Technologies to Broaden Access Targeted conditioning In vivo editing of HSCs DELIVERY EDITING cKit (CD117) antibody-drug conjugate (ADC) for specific depletion of hematopoietic stem cells (HSCs) and no off-target/bystander toxicity Proprietary GMP monoclonal antibody wi
Slide 11: CAR T
CAR T CRISPR THERAPEUTICS – CONFIDENTIAL © 2025 CRISPR Therapeutics | 11 11
Slide 12: Best-in-Class Cell Therapy Platform for Treating Cancer and Autoimmune Disease
Best-in-Class Cell Therapy Platform for Treating Cancer and Autoimmune Disease CTX112 CTX131 Autologous GPC3 Currently in Phase I/II trial in r/r NHL, plus Currently in Phase I/II trial in RCC, plus Ongoing preclinical work with anti-GPC3 Phase I trial in SLE/SSc/IIM Phase I/II trial in TCL autologo
Slide 13: CTX112: An Allogeneic CAR T Optimized for Potency
CTX112: An Allogeneic CAR T Optimized for Potency CTX112 Novel Potency Edits (TGFBR2, Regnase-1) Other CTX112 Competitive Advantages TGFBR2 Anti-CD19 disruption CAR Ability to multiplex gene edits precisely TCR and efficiently disruption MHC I disruption Comprehensive and FDA-validated genomic analy
Slide 14: CTX112 Phase I Immuno-Oncology Clinical Trial Design
CTX112 Phase I Immuno-Oncology Clinical Trial Design Open-label, multicenter, Phase I/II study evaluating the safety and efficacy of CTX112 in relapsed or refractory B-cell malignancies Patients could receive an additional infusion of CTX112 CTX112 infusion with LD chemotherapy if they achieved a pa
Slide 15: Initial Efficacy Data On Par with Auto CAR T
Initial Efficacy Data On Par with Auto CAR T CTX112 Initial Efficacy Data (N=12) High risk patient population (58% primary refractory; 67% >3 prior therapies; 50% with tumor SPD > 4000 mm2) CTX112 demonstrated tolerability with no CRS, ICANS or infections Grade ≥3 Patient-level data Aggregated data
Slide 16: Updated CTX112 Data Shows PK in Line with Auto CAR T
Updated CTX112 Data Shows PK in Line with Auto CAR T Analysis from subsequent data cut on Dec 20, 2024 (N=25) CTX112 Cell Expansion Data CAR T Cell Expansion Comparison 100,000 DL1/2 DL3/4 10,000 [CAR] (Copies/μg) Other 1,000 CTX112 Autologous Allogeneic (DL3/4) CAR T CAR T 100 Mean Cmax 45,000- Apx
Slide 17: Updated CTX112 Data Shows Efficacy in post-TCE Subset
Updated CTX112 Data Shows Efficacy in post-TCE Subset Analysis from subsequent data cut on Dec 20, 2024 # TCE CTX112 Histology Prior Prior Bispecific T Cell Engager (TCE) Best Overall Response Best Overall Response Lines FL 7 5L: Mosunetuzumab PR PR Dose level 3 LBCL 2 2L: R-ICE & Epcoritamab PD PR
Slide 18: CTX112 is Positively Differentiated From Other CD19 Therapies
CTX112 is Positively Differentiated From Other CD19 Therapies • Safety benefits are critical in context of larger patient populations and in community hospital settings CTX112 vs. • Improved patient experience with no apheresis; enables rapid enrollment to dosing without the need Autologous CAR T to
Slide 19: Next-Generation CAR T for Solid Tumors
Next-Generation CAR T for Solid Tumors CTX131 Next-Generation CAR T Chassis: Solid Tumor CAR T pipeline Most sophisticated allogeneic CAR T candidate in the clinic Program Indications Anti- TGFBR2 CD70 ▪ Phase I trial in RCC and other solid disruption CAR TCR tumors ongoing; update in 2025 MHC I dis
Slide 20: In Vivo
In Vivo CRISPR THERAPEUTICS – CONFIDENTIAL 20
Slide 21: Plug-and-play LNP/mRNA Platform for Gene Disruption
Plug-and-play LNP/mRNA Platform for Gene Disruption CTX310 CTX320 CTX340/450 ANGPTL3 targeted asset with Potential to address large patient Additional preclinical programs potential across multiple indications populations with elevated Lp(a) targeting AGT and ALAS1 respectively Update in 2H 2025 Upd
Slide 22: CTX310: A One-Time Therapy to Silence Expression of ANGPTL3
CTX310: A One-Time Therapy to Silence Expression 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 rep
Slide 23: 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 24: Initial Results for CTX310 Phase 1 Dose Escalation Trial
Initial Results for CTX310 Phase 1 Dose Escalation Trial N=10; data cutoff April 16, 2025 Efficacy Highlights Mean % Change from Baseline at Day 30 post- ▪ 0.8 mg/kg (DL4) patient with sHTG had infusion an 82% reduction in triglycerides from (+/- SEM) a baseline of 1073 mg/dL at day 30 ▪ 0.6 mg/kg (
Slide 25: Large Addressable Patient Population for CTX310
Large Addressable Patient Population for CTX310 HoFH HeFH Mixed dyslipidemias sHTG ▪ LDL-C can reach >400 mg/dL ▪ LDL-C can reach >190 mg/dL ▪ Adults with high LDL-C and TG ▪ Adults with ≥500 mg/dL fasting 150-499 mg/dL triglyceride levels ~1.5K U.S. Patients1 ~1M U.S. Patients2 >40M U.S. Patients3
Slide 26: Lp(a): An Emerging Key Target to Potentially Reduce CV Events
Lp(a): An Emerging Key Target to Potentially Reduce CV Events Lp(a) is an independent risk factor for atherosclerotic Comparison of atherogenicity of Lp(a) and LDL cardiovascular disease (ASCVD) • Lp(a) is an LDL-like lipoprotein synthesized and secreted by hepatocytes • Epidemiologic, Mendelian ran
Slide 27: Single CTX320 Dose Resulted in Durable Lp(a) Reduction (NHP)
Single CTX320 Dose Resulted in Durable Lp(a) Reduction (NHP) ~70% editing of LPA1 at 1 year ~95% reduction in plasma Lp(a) sustained at 2 years 80 0 Change from Baseline (%) -20 60 Liver Editing (%) -40 40 -60 20 -80 0 -100 1 2 mg/kg 0 8 16 24 32 40 48 56 64 72 80 88 96 104 Study Week Updated NHP da
Slide 28: Phase I Study Evaluating the Safety and Efficacy of CTX320
Phase I Study Evaluating the Safety and Efficacy of CTX320 CTX320 infusion Acute toxicity monitoring 12-month total follow-up Long-term Screening follow-up Day 1 Day 30 Phase I: Single ascending dose escalation to identify optimal biological dose Phase II Key eligibility criteria Dose level 4 ▪ Age
Slide 29: CTX320 Has Potential to Address Population with Elevated Lp(a)
CTX320 Has Potential to Address Population with Elevated Lp(a) Elevated Lp(a) is considered the most common genetically inherited risk factor for cardiovascular disease (CVD)1 Distribution of Lp(a) levels in Lifetime risk of major cardiovascular events general population2 with increasing Lp(a) level
Slide 30: Two Additional Programs Advancing Toward Clinic
Two Additional Programs Advancing Toward Clinic CTX340 Targeting AGT For Refractory Hypertension CTX450 Targeting ALAS1 for AHPs • Hypertension is the leading cause of cardiovascular morbidity • Acute hepatic porphyrias (AHP) are caused by deficiencies of and mortality worldwide1,2 specific enzymes
Slide 31: Three Parallel Efforts in Type 1 Diabetes (T1D)
Three Parallel Efforts in Type 1 Diabetes (T1D) Gene editing is key to achieving the goal of developing a beta-cell replacement product to treat diabetes without requiring long-term immunosuppression 1 2 3 CTX211 CTX213 Non-exclusive license with Vertex First-in-class edited beta-cell Deviceless, iP
Slide 32: Next-Generation Editing and Proprietary LNP Platform
Next-Generation Editing and Proprietary LNP Platform The race to bring next-generation Both editing and delivery expertise No single editing approach will gene-editing technologies to the are needed to make the required edit dominate; each disease will require clinic has only just begun at the requi
Slide 33: 2025: A Year of Significant Value Creation
2025: A Year of Significant Value Creation ▪ Ongoing launch of CASGEVY with investments driven by strong patient demand ▪ Catalyst rich year with several data readouts expected across our pipeline: - CTX112 update in Oncology and Autoimmune diseases - CTX310 update in the second half of 2025 - Addit
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