Duyuru • Apr 04
CRISPR Therapeutics AG, Annual General Meeting, Jun 04, 2026 CRISPR Therapeutics AG, Annual General Meeting, Jun 04, 2026, at 08:00 W. Europe Standard Time. Location: at the offices of walder wyss ltd, seefeldstrasse 123, 8008 switzerland, zurich Switzerland Duyuru • Dec 24
CRISPR Therapeutics Provides Broad Update on Zugocaptagene Geleucel in Autoimmune Disease and Hematologic Malignancies CRISPR Therapeutics provided updates on zugocaptagene geleucel (zugo-cel), formerly known as CTX112™?, its investigational allogeneic CAR T targeting CD19, in development for autoimmune disease and hematologic malignancies. Autoimmune Disease: Zugo-cel, targeting CD19, is in an ongoing Phase 1 basket trial in autoimmune rheumatologic diseases, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis. Patients in the study may have active SLE (with or without renal involvement), SSc, or idiopathic inflammatory myopathy (IIM) despite the use of standard therapies. Preliminary clinical data from the Phase 1 study has been encouraging, and zugo-cel has been well tolerated to date. No Grade 3 ICANS or CRS has been observed at the 100 million cell dose, which is the dose currently being studied in the autoimmune basket trials. The Phase 1/2 clinical trial in R/R B-cell malignancies is ongoing. The Company expects to provide additional updates in the second half of 2026. CRISPR Therapeutics has also established a new collaboration and clinical supply agreement with Lilly to evaluate zugo-cel together with pirtobrutinib in aggressive B-cell lymphomas, further expanding the program's development in oncology. Duyuru • Nov 09
CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310 CRISPR Therapeutics announced positive Phase 1 data from its ongoing clinical trial evaluating CTX310, an investigational, in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3. A single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum -89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%) and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose. These data were presented during a late-breaking session at the American Heart Association (AHA) Scientific Sessions and published simultaneously in The New England Journal of Medicine (NEJM) in a peer-reviewed article entitled "Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3." Phase 1 Clinical Trial Design The Phase 1, open label, dose-escalation trial evaluated single-course IV doses of CTX310 ranging from 0.1 to 0.8 mg/kg (lean body weight) targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous Family hypercholesterolemia (HeFH), or mixed dyslipidemias (elevated TG and LDL). Overall, CTX310 demonstrated a well-tolerated safety and tolerability profile that supports continued advancement of the program. These new results build upon previously disclosed top-line data from 12 participants across the first four potential cohorts, corresponding to 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). Results from the Phase 1 clinical trial highlight the potential of CTX310 to safely and durably lower both TG and LDL following a single-course IV administration. CRISPR Therapeutics is advancing CTX310 into Phase 1b clinical trials, prioritizing development in sHTG and mixed dyslipidemia. CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholester Holemia, mixed dyslipidemias, or severe dyslipidemias (e elevated TG and LDL). Overall,CTX310 demonstrated a well-Tolerated safety and tolerability profiles that supports continued advancement of the programs. These new results build upon previous disclosed top-line data from12 participants across the first four potential cohort, corresponding to lean body weight -based doses of DL1 [ 0.1 mg/kg], DL2 [0.3 u/kg, DL3 [ 0.6mg/kg] and DL4[0.8 mg/kg]. Next steps: Results from the Phase 1 clinical trials highlight the potential of CTX 310 to safely and durable both TG and LDL following a one- course IV administration. CRISPRTherapeutics is advancing CTX 310 into Phase 1b clinical trial, prioritizing development in s HTG and mixed dyslipidemias. CTX320 are in current clinical trials in patients with heter heterozygous familial hyperchurchaseolemia, homozygous familial hyperchurchasemia, mixed dyslipidemia, or severe dyslipidemia, and in patients with elevated lipoprotein(a), respectively. Duyuru • Oct 10
CRISPR Therapeutics Presents New Preclinical Data for CTX460™? Demonstrating in Vivo Gene Correction of Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase™? Editing Platform CRISPR Therapeutics reported new preclinical data from its novel SyNTase™? gene editing platform for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The data are featured in an oral presentation titled "Single-dose in vivo gene correction of AATD via LNP-delivered SyNTase editors" at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress. The AATD program, designated CTX460™?, is the first investigational candidate to use SyNTase editing and is expected to enter the clinic in mid-2026. "Alpha-1 antitrypsin deficiency remains an area of significant unmet need, with current treatments unable to address the underlying genetic cause. The current standard-of-care requires weekly intravenous (IV) infusion of purified functional M-AAT protein to mitigate lung disease symptoms, but no approved therapies address the underlying genetic cause. Better therapies are needed that can normalize AAT levels to minimize risk of lung disease progression. CTX460 is a SyNTase editing-based investigational candidate targeting the E342K mutation in SERPINA1, encapsulated in a de-risked, proprietary lipid nanoparticle (LNP). The preclinical data presented evaluated CTX460 in two preclinical models of AATD: the well-established NSG-PiZ mouse model, which carries human SERPINA1 Z alleles, and a novel humanized PiZ rat model in which the normal rat SERPINA1 gene is replaced with the human mutant SERPINA1 E342K variant. These models were used to evaluate gene and mRNA correction, serum protein levels, and durability of effect following a single dose of CTX460. CTX460 Key Preclinical Data Highlights: A single dose of CTX460 achieved significant, dose-dependent correction of liver DNA in both rat and mouse AATD models, with near saturating editing in hepatocytes at doses as low as 0.1 mg/kg. Furthermore, a single dose of CTX 460 was able to achieve >90% mRNA correction at a clinically relevant dose of 0.5 mg/kg in PiZ mice. Duyuru • Oct 01
CRISPR Therapeutics to Present Preclinical Data on Alpha-1 Antitrypsin Deficiency Utilizing Novel Syntase Gene Editing Technology At the European Society of Gene and Cell Therapy 2025 Annual Congress CRISPR Therapeutics announced the acceptance of an abstract for oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, taking place October 7-10, 2025. The presentation will introduce the Company's novel SyNTase gene editing technology and highlight its application in single-dose in vivo gene correction to treat Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disorder. CRISPR Therapeutics has developed SyNTase editing, a proprietary, next-generation, site-specific gene correction platform. SyNTase editors represent a significant advance over currently described prime editing systems by combining compact Cas9 proteins with a novel class of engineered polymerases. Together, these components enable gene editing with greater efficiency and precision, while also supporting scalable manufacturing. Using AI-guided structural modeling and large-scale screening, the polymerase was optimized to support gene correction activity based on synthetic nucleotide templates. When integrated with Cas9, SyNTase editors can utilize engineered templates with improved serum stability, enabling higher target correction efficiency. The abstract describes that SyNTase editing produces high levels of editing (up to 95%) in SERPINA1-E342K human hepatocyte cell models without any detectable (70% mRNA correction and >3-fold total serum AAT upregulation, exceeding the established clinically protective threshold. Together, these data provide proof-of-concept for a potentially best-in-class therapeutic modality to address the underlying cause of AATD and support SyNTase editing as a promising platform for the treatment of many monogenic disorders. Additional results beyond those included in the abstract will be presented at the conference. To accelerate and expand its efforts, CRISPR Therapeutics have formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR THERAPEUTICS®? standard character mark and design logo and SyNTase™? are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY®? and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. Duyuru • Sep 23
CRISPR Therapeutics and Sirius Therapeutics Announce First Patient Dosed in Phase 2 Trial of SRSD107 for Thromboembolic Disorders in Europe CRISPR Therapeutics and Sirius Therapeutics announced that the first patient has been dosed in a Phase 2 clinical trial of SRSD107, a next-generation, long-acting Factor XI (FXI) siRNA for the prevention of venous thromboembolism (VTE) in patients undergoing total knee arthroplasty (TKA). SRSD107 is being co-developed by CRISPR Therapeutics andSir Therapeutics as part of a strategic collaboration to advance innovative treatments for cardiovascular and clotting-related diseases. The ongoing Phase 2 clinical trial is a randomized, multicenter, global study evaluating the safety and efficacy of SRSD107 for the prevention of VTE in patients undergoing TKA. The trial will assess the anticoagulant effects and pharmacological profile of SRSD107 and help inform dose selection for future pivotal studies, with the goal of confirming its potential as a differentiated approach for reducing thrombotic risk in patients. SRSD107 is designed to selectively inhibit FXI, a key driver of pathological thrombosis, with minimal impact on normal hemostasis. In prior Phase 1 clinical trials conducted in Australia and China, single doses of SRSD107 were well tolerated and demonstrated strong, sustained pharmacodynamic effects, including reductions of over 93% in FXI levels, along with more than a twofold increase in activated partial thromboplastin time (aPTT) relative to baseline. These effects were sustained, with responses maintained for up to six months post-dosing. 
