CTX310 / CRISPR Therap - LARVOL DELTA

Durability of Effects of CTX310, a CRISPR-Cas9 Gene Editing Targeting ANGPTL3 (ESC 2026) - No abstract available

Late-breaking abstract • Cardiovascular • ANGPTL3

A Safety and Tolerability Study Evaluating CTX310 in Participants With Refractory Dyslipidemias (ANZCTR) - P1 | N=69 | Recruiting | Sponsor: CRISPR Therapeutics AG | N=24 ➔ 69

Enrollment change • First-in-human • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Heterozygous Familial Hypercholesterolemia • Homozygous Familial Hypercholesterolemia • Hypertriglyceridemia • Metabolic Disorders • Mixed Hyperlipidemia • Severe Hypertriglyceridemia

Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310. (PubMed, N Engl J Med) - No abstract available

Journal • ANGPTL3

Promise of ANGPTL3 as a therapeutic target for controlling cholesterol levels. (PubMed, Expert Opin Ther Targets) - "These observations rapidly catalyzed the development of pharmacologic strategies to inhibit ANGPTL3 using monoclonal antibodies (e.g. evinacumab), antisense oligonucleotides (e.g. vupanorsen), small interfering ribonucleic acid (e.g. zodasiran and solbinsiran), and most recently genome-editing approaches (e.g. VERVE-201 and CTX310). The degree of hypertriglyceridemia in the patient's baseline lipid profile appears to be an important determinant of drug response. Drawing on genetic, mechanistic, and clinical trial data, the promise and limitations of ANGPTL3 inhibition are considered and its potential place in future lipid-lowering strategies is outlined."

Journal • Review • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Hypertriglyceridemia • Metabolic Disorders • ANGPTL3

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias (clinicaltrials.gov) - P1 | N=90 | Recruiting | Sponsor: CRISPR Therapeutics AG

First-in-human • New P1 trial • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Heterozygous Familial Hypercholesterolemia • Homozygous Familial Hypercholesterolemia • Hypertriglyceridemia • Metabolic Disorders • Mixed Hyperlipidemia • Severe Hypertriglyceridemia

CRISPR Therapeutics anticipates several key milestones across its portfolio (GlobeNewswire) - "Global regulatory submissions for CASGEVY in patients ages 5 – 11 are expected to be initiated by Vertex in the first half of 2026; Updates from CTX310 are expected in the second half of 2026; Updates from the Lp(a) program are expected in 2026; Updates across autoimmune disease and immuno-oncology for zugocabtagene geleucel (zugo-cel; formerly CTX112) are expected in the second half of 2026."

Filing • Trial status • Autoimmune Hemolytic Anemia • Beta-Thalassemia • Hematological Disorders • Hematological Malignancies • Hypertriglyceridemia • Immune Thrombocytopenic Purpura • Immunology • Myositis • Severe Hypertriglyceridemia • Sickle Cell Disease • Systemic Lupus Erythematosus • Systemic Sclerosis

Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3. (PubMed, N Engl J Med) - P1 | "Editing of ANGPTL3 was associated with few adverse events and resulted in reductions from baseline in ANGPTL3 levels. (Funded by CRISPR Therapeutics; Australia New Zealand Clinical Trials Registry number, ACTRN12623000809639.)."

Journal • P1 data • Atherosclerosis • Cardiovascular • CNS Disorders • Dyslipidemia • Hypertriglyceridemia • Metabolic Disorders • Mixed Hyperlipidemia • ANGPTL3

First-in-Human Phase 1 Clinical Trial of a CRISPR-Cas9 Gene Editing Therapy Targeting ANGPTL3 (AHA 2025) - "At the time of the data cutoff, all patients will have completed a minimum of 30 days of safety follow-up.Secondary Endpoints: Secondary endpoints include CTX310 pharmacokinetics, pharmacodynamics, and efficacy data. Efficacy data will be assessed by changes in lipid biomarkers, including LDL-C, serum triglycerides, non-HDL-C, and apolipoprotein B.Outcome(s): To be presented at conference."

Clinical • First-in-human • Late-breaking abstract • P1 data • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Heterozygous Familial Hypercholesterolemia • Homozygous Familial Hypercholesterolemia • Hypertriglyceridemia • Metabolic Disorders • Mixed Hyperlipidemia • Severe Hypertriglyceridemia • ANGPTL3 • APOB • LPL

CTX310, targeting ANGPTL3 (GlobeNewswire) - P1 | N=24 | ACTRN12623000809639 | Sponsor: CRISPR Therapeutics AG | "These new results build upon previously disclosed results from the first 10 patients across the first four cohorts (lean body weight-based doses of DL1 [0.1 mg/kg], DL2 [0.3 mg/kg], DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg]) with at least 30 days of follow-up for each participant. As dose-range finding continues, data to date demonstrate peak reductions of up to 82% in TG and LDL reductions of up to 86% at DL4 without any clinically significant changes in liver enzymes and a safety and tolerability profile consistent with previous findings; The Company anticipates presenting the complete Phase 1 data for CTX310 at a medical meeting in the second half of 2025."

P1 data • Dyslipidemia • Heterozygous Familial Hypercholesterolemia • Homozygous Familial Hypercholesterolemia • Severe Hypertriglyceridemia

CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2024 Financial Results (GlobeNewswire) - "CTX112 is also in an ongoing Phase 1 clinical trial in systemic lupus erythematosus (SLE), systemic sclerosis and inflammatory myositis....Based on favorable oncology data, CRISPR Therapeutics expanded the CTX112 trial in SLE to include patients with systemic sclerosis and inflammatory myositis in a basket study, with updates expected in mid-2025....CTX310 is in an ongoing Phase 1 clinical trial targeting ANGPTL3 in patients with homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (SHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias...Dose escalation is ongoing, with an update expected in the first half of 2025."

P1 data • Familial Hypercholesterolemia • Immunology • Myositis • Severe Hypertriglyceridemia • Systemic Lupus Erythematosus • Systemic Sclerosis

A Safety and Tolerability Study Evaluating CTX310 in Subjects With Refractory Dyslipidemias (ANZCTR) - P1 | N=24 | Recruiting | Sponsor: CRISPR Therapeutics AG | Not yet recruiting ➔ Recruiting

Enrollment open • Dyslipidemia • Metabolic Disorders • ANGPTL3 • APOB

CTX310: An Investigational in vivo CRISPR-Based Therapy Efficiently and Durably Reduces ANGPTL3 Protein and Triglyceride Levels in Non-Human Primates After a Single Dose (AHA 2023) - "Conclusion CTX310 was well-tolerated in NHPs and led to significant and durable reductions in circulating ANGPTL3 and triglycerides. This data suggests that CTX310 could be used to treat dyslipidemias in humans."

Preclinical • Atherosclerosis • Cardiovascular • Dyslipidemia • Metabolic Disorders • ANGPTL3

A Safety and Tolerability Study Evaluating CTX310 in Subjects With Refractory Dyslipidemias (ANZCTR) - P1 | N=24 | Not yet recruiting | Sponsor: CRISPR Therapeutics AG

New P1 trial • Dyslipidemia • Metabolic Disorders • ANGPTL3 • APOB