Crispr Therapeutics Ag · NASDAQ:CRSP
Crispr Therapeutics — Corporate Update (2024)
26 slides from Crispr Therapeutics's corporate update, Nov 17, 2024
Slide 1: Creating transformative gene-based medicines for serious diseases
Creating transformative gene-based medicines for serious diseases Corporate Overview Q4 2024 © 2024 CRISPR Therapeutics | 1
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 a
Slide 3: CRISPR Therapeutics Highlights
CRISPR Therapeutics Highlights Leading gene editing company with a strong and diversified pipeline, efficient operating model, and proven track record of execution Best-in-class technology, strategy, and execution culminating Hemoglobinopathies in the historic approval of CASGEVY Advancing multiple
Slide 4: Broad and Diversified Pipeline
Broad and Diversified Pipeline Program Disease Research IND-enabling Clinical Approved Partner Structure Severe sickle cell disease (SCD) CASGEVY1 Collaboration Transfusion-dependent β-thalassemia (TDT) Heme Next-generation conditioning Various Wholly owned2 In vivo editing of HSCs Various Wholly ow
Slide 5: CASGEVY: Historic First Approval of a CRISPR-Based Medicine
CASGEVY: Historic First Approval of a CRISPR-Based Medicine Unparalleled speed and execution Well-positioned for commercial success to a landmark approval1 Strong commercial partner with Vertex Foundational Year in 2024 Significant Large addressable market ex-U.S., market in U.S. and including >23,0
Slide 6: Groundbreaking Data Across >100 Patients
Groundbreaking Data Across >100 Patients TDT: Transfusion independence achieved out to 59.4 months SCD: VOC-free and no in-patient hospitalizations for VOCs achieved out to 54.8 months Locatelli et al. EHA 2024 Frangoul et al. EHA 2024 Exa-cel treatment resulted in early and sustained increases in H
Slide 7: CASGEVY Has a Large Addressable Market
CASGEVY Has a Large Addressable Market Significant opportunity in US and Europe, with potential to expand further with targeted conditioning β-thalassemia Sickle Cell Disease Exa-cel addressable market with 5,000 standard of care conditioning 30,000 Exa-cel potential market with 16,000+ targeted con
Slide 8: Expanding the Addressable Patient Population
Expanding the Addressable Patient Population Near-Term: Targeted conditioning using Antibody-drug conjugate against c-Kit to expand addressable population for CASGEVY High on-target potency Proprietary GMP monoclonal antibody Optimal Attributes with short half-life targeting c-Kit Low off-target & s
Slide 9: Three Additional Franchises Beyond CASGEVY
Three Additional Franchises Beyond CASGEVY CAR T In Vivo Type 1 Diabetes I/O and Autoimmune Common and Rare Disease World’s most Established sophisticated CAR T First-in-class edited mRNA-LNP cell candidates, beta cell therapies platform for manufactured for T1D liver editing internally Multiple opp
Slide 10: Our Gene-Edited Allogeneic CAR T Franchise
Our Gene-Edited Allogeneic CAR T Franchise Advancing the most sophisticated CAR T cell candidates in the clinic against multiple opportunities across heme and solid cancers, plus autoimmune indications First-generation: CTX110 and CTX130 Next-generation: CTX112 and CTX131 Additional indications and
Slide 11: CTX112 and CTX131 Incorporate Novel Potency Edits
CTX112 and CTX131 Incorporate Novel Potency Edits Next-generation CRISPR gene-edited allogeneic CAR T chassis TGFBR2 CAR Novel Potency Edits disruption TCR MHC I disruption • Regnase-1 KO: Increase disruption • TCR KO: Prevent GvHD functional persistence, • CAR KI: Site-specific cytokine secretion a
Slide 12: CTX112 and CTX131 Build Upon First Gen CAR Ts in Oncology
CTX112 and CTX131 Build Upon First Gen CAR T's in Oncology First-generation CAR Ts, CTX110 & CTX130 Proof-of-concept that our allogeneic CAR T cells can produce durable remissions following a standard LD regimen in hematological malignancies as well as solid tumors Potency edits in CTX112 lead to ex
Slide 13: CTX112: Significant Opportunity in Autoimmune Diseases
CTX112: Significant Opportunity in Autoimmune Diseases B cell depletion following CTX110 infusion among patients with detectable B cells at baseline (N=9) ▪ Allogeneic CAR T cells produce deep B cell depletion 1000 in B cell malignancies, as observed with CTX110 500 ▪ CD19-directed autologous CAR T
Slide 14: CTX112 is Positively Differentiated From Other CD19 Therapies
CTX112 is Positively Differentiated From Other CD19 Therapies • Off-the-shelf benefits are critical in context of autoimmune patients (e.g., removing requirement for apheresis allows patients to continue use of concurrent medications) CTX112 vs. Autologous CAR T • Potential to expand addressable pat
Slide 15: Owning Manufacturing Gives Us Flexibility
Owning Manufacturing Gives Us Flexibility Manufacturing CTX112 and CTX131 at our internal GMP facility These candidates exhibit increased manufacturing robustness, with a higher and more consistent number of CAR T cells produced per batch Potential for significantly lower COGS and greater scalabilit
Slide 16: Advancing Plug-and-Play In Vivo Platform Across Multiple Diseases
Advancing Plug-and-Play In Vivo Platform Across Multiple Diseases Established plug-and-play LNP/ Potential to transform the treatment Advancing in vivo programs in mRNA platform for in vivo gene paradigm for CV with additional rare diseases disruption, starting in the liver CTX310, CTX320, CTX330 an
Slide 17: CTX310 and CTX320 Could Transform the Treatment Paradigm for ASCVD
CTX310 and CTX320 Could Transform the Treatment Paradigm for ASCVD The chronic care model A new treatment paradigm: one-time Daily Weekly Multiple infusions Surgical CRISPR-based therapies with the potential to… medications injections annually interventions Recapitulate the proven benefit of targets
Slide 18: CTX310 and CTX320: A One-Time Dose to Treat CV Diseases
CTX310 and CTX320: A One-Time Dose to Treat CV Diseases Intravenous delivery Reduced atherogenic CRISPR/Cas9-based editing targeting the liver lipoprotein concentrations CTX310 or CTX320 Endothelial lipase LDL-C ANGPTL3 Endocytosis and TG (CTX310) Lipoprotein lipase Endosomal escape Lp(a) DNA cleava
Slide 19: CTX310 Targeting ANGPTL3 for Cardiovascular Disease
CTX310 Targeting ANGPTL3 for Cardiovascular Disease Natural loss-of-function mutations in ANGPTL3 are associated A one-time, CRISPR-based therapy could recapitulate the with reduced LDL-C, triglycerides (TG), and ASCVD risk protective effect of naturally occurring loss-of-function without any negati
Slide 20: CTX320 Targeting Lp(a), an Independent Risk Factor for ASCVD
CTX320 Targeting Lp(a), an Independent Risk Factor for ASCVD Elevated lipoprotein(a) [Lp(a)] levels Up to 20% of the global population A one-time, CRISPR-based therapy increase ASCVD risk, as observed has elevated Lp(a)6,7, primarily could recapitulate the protective across numerous studies1,2,3,4,5
Slide 21: CTX340 Targeting AGT For Refractory Hypertension
CTX340 Targeting AGT For Refractory Hypertension Hypertension is the leading cause of Refractory hypertension defined as uncontrolled A one-time, CRISPR-based therapy cardiovascular morbidity and blood pressure despite use of ≥5 or more targeting angiotensinogen (AGT) mortality worldwide1,2 antihype
Slide 22: CTX450 Targeting ALAS1 for AHP
CTX450 Targeting ALAS1 for AHP Acute hepatic porphyrias (AHP) are inherited metabolic disorders caused by deficiencies of specific enzymes in the A one-time, CRISPR-based therapy could durably reduce heme biosynthesis pathway.1,2 The rate-limiting enzyme of the disease-associated biomarkers, potenti
Slide 23: 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 Deviceless approach Non-exclusive license with Vertex First-in-class edited beta-cell U
Slide 24: 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 one editing approach will gene-editing technologies to the needed to make the required edit dominate – each disease will clinic has just begun in the required location have i
Slide 25: Several Upcoming Catalysts Across Portfolio
Several Upcoming Catalysts Across Portfolio Program Disease Status Heme CASGEVY SCD and TDT Ongoing launch – 2024 is a foundational year B-cell malignancies Trial ongoing – update in 2024 Autoimmune CTX112 - allo CD19 CAR T SLE Trial ongoing I/O & Solid tumors Trial in RCC ongoing – update in 2025 C
Slide 26: Building an Industry-Leading Company
Building an Industry-Leading Company BEST-IN-CLASS COLLABORATIVE & EXPERIENCED platform and ENTREPRENEURIAL management team capabilities culture INTERNAL ~$1.9 BILLION MANUFACTURING cash balance as of at state-of-the-art September 30, 2024 GMP facility CRISPR Therapeutics | www.crisprtx.com © 2024 C