CRISPR Therapeutics AG reports developments in gene-based medicines built around CRISPR/Cas9 gene editing, cell therapy, in vivo editing and siRNA programs. Recurring updates cover CASGEVY, developed with Vertex for severe sickle cell disease and transfusion-dependent beta thalassemia, as well as pipeline work in cardiovascular disease, autoimmune disease, oncology, rare disease and Type 1 diabetes.
Company news also includes financial results, business updates, investor conference presentations, manufacturing and platform disclosures, collaborations such as Sirius Therapeutics, and capital actions involving common shares or convertible senior notes.
CRISPR Therapeutics (Nasdaq: CRSP) announced a proposed private offering of $350 million aggregate principal amount of convertible senior notes due 2031, with an initial purchaser option of up to $52.5 million. Interest will accrue semiannually beginning September 1, 2026, and notes mature March 1, 2031. Upon conversion, the company will deliver common shares. The company intends to use net proceeds for general corporate purposes. Terms including interest rate and conversion rate will be set at pricing; the notes and shares will not be registered under the Securities Act.
CRISPR Therapeutics (Nasdaq: CRSP) reported Q4 and full‑year 2025 results and a broad business update on Feb 12, 2026. CASGEVY revenue was $54M in Q4 and $116M for 2025; 64 patients received infusions in 2025. Cash and equivalents totaled $1,975.8M at year‑end.
Pipeline progress includes in vivo liver editing (CTX310, CTX321), CTX460 planned trial mid‑2026, CTX340 planned H1 2026 start, siRNA collaboration updates in 2026, and zugo‑cel data advancing in autoimmune and oncology programs.
CRISPR Therapeutics (Nasdaq: CRSP) outlined 2026 strategic priorities and timing for multiple clinical and commercial milestones. The company enters 2026 with approximately $2 billion in cash, cash equivalents, and marketable securities and reported CASGEVY revenue exceeding $100M in 2025 with a nearly three-fold increase in patient initiations versus 2024. Key 2026 items include global CASGEVY commercialization and pediatric regulatory submissions in H1 2026, CTX310 and Lp(a) program updates in H2 2026, topline Phase 2 CTX611 data in H2 2026, and planned trial starts for CTX460 (mid-2026) and CTX340 (H1 2026). Multiple oncology, autoimmune, in vivo editing and siRNA programs advance through the clinic.
CRISPR Therapeutics (Nasdaq: CRSP) said members of its senior management will present at the 44th Annual J.P. Morgan Healthcare Conference on January 12, 2026 at 8:15 a.m. PT in San Francisco.
A live webcast will be available on the company’s Events & Presentations page at https://crisprtx.gcs-web.com/events, and a replay will be archived on the company website for 14 days following the presentation.
CRISPR Therapeutics (Nasdaq: CRSP) provided a broad update on zugocaptagene geleucel (zugo-cel), an allogeneic anti-CD19 CAR T, across autoimmune diseases and B-cell malignancies on Dec 22, 2025. Key clinical readouts include deep B-cell depletion and drug-free DORIS remission through Month 6 in an SLE patient at the 100 million cell dose, and in R/R large B-cell lymphoma an ORR 90% and CRR 70% at the 600 million cell RP2D. Safety findings: Grade 3 CRS and ICANS each at 17% and serious infections 8% at the RP2D. A Phase 1 basket study in ITP and wAIHA has started and a collaboration with Lilly will evaluate zugo-cel plus pirtobrutinib. Additional data expected H2 2026.
CRISPR Therapeutics (Nasdaq: CRSP) reported Q3 2025 results and a business update on Nov 10, 2025. Cash, cash equivalents, and marketable securities were $1,944.1M as of Sept 30, 2025. Net loss for Q3 was $106.4M. R&D expense decreased to $58.9M and collaboration expense increased to $57.1M for the quarter. Commercial momentum for CASGEVY continues: ~165 patients completed first cell collection and 39 patients received infusions to date; Vertex expects >$100M in CASGEVY revenue for 2025. Key clinical updates: positive Phase 1 CTX310 data published in NEJM, CTX460 preclinical correction >90%, pediatric exa-cel Phase 3 enrollment completed with data due Dec 6, 2025.
CRISPR Therapeutics (Nasdaq: CRSP) reported positive Phase 1 data for CTX310 on Nov 8, 2025, presented at AHA Scientific Sessions and published in NEJM. A single-course IV infusion produced dose-dependent, durable editing of ANGPTL3 with a mean reduction of -73% (max -89%). At the highest dose, mean triglycerides (TG) fell -55% (max -84%; mean -60% in patients with baseline TG >150 mg/dL) and mean LDL fell -49% (max -87%) by Day 60. CTX310 was generally well tolerated in 15 participants with no treatment-related serious adverse events and no ≥Grade 3 transaminase changes. CRISPR is advancing CTX310 into Phase 1b, prioritizing severe hypertriglyceridemia and mixed dyslipidemia.
CRISPR Therapeutics (NASDAQ: CRSP) reported preclinical CTX460 data using its SyNTase™ editing platform for Alpha-1 Antitrypsin Deficiency (AATD). Key results shown at ESGCT 2025 include >90% mRNA correction, a 5-fold increase in total serum AAT, and an >99% M-AAT:Z-AAT ratio in disease models after a single dose. Near-saturating hepatocyte editing occurred at doses as low as 0.1 mg/kg, with >90% mRNA correction at 0.5 mg/kg and durability of effect up to 7–9 weeks in rodent models.
CTX460 is formulated in a proprietary LNP and is expected to enter clinical testing in mid-2026, positioning the program as a potential best-in-class, mutation‑targeting approach for AATD.
Monash University, Australia's largest research institution, has announced the establishment of the Monash Boston Hub in Cambridge to strengthen its biotech partnerships in North America and Europe. The Hub will facilitate licensing, drug discovery, and clinical trial collaborations.
The university has completed over 160 license deals and created 30+ spinouts in the past five years, collectively raising nearly $1 billion. The new Hub, staffed by 5-7 professionals led by Nathan Elia, will be located at the Cambridge Innovation Center.
Notable partnerships include Moderna's mRNA manufacturing facility on Monash's campus and collaborations with CRISPR Therapeutics (NASDAQ: CRSP). The university's Monash Institute of Pharmaceutical Sciences (MIPS) has helped progress over 40 novel drug candidates into clinical development.
CRISPR Therapeutics (Nasdaq: CRSP) announced the upcoming presentation of its novel SyNTase gene editing technology at the ESGCT 2025 Annual Congress. The presentation will showcase preclinical data for treating Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disorder.
The SyNTase platform combines compact Cas9 proteins with engineered polymerases, achieving up to 95% editing efficiency in cell models with minimal off-target effects. In preclinical studies, a single intravenous dose (≤0.5 mg/kg) demonstrated high efficiency in humanized mouse models, while achieving >70% mRNA correction and >3-fold total serum AAT upregulation in humanized rat models of AATD.
The presentation is scheduled for October 10, 2025, at 11:00 a.m. CEST during the Gene Editing III session.