On January 13, 2026 Vivere Oncotherapies, a UC Berkeley spin-out developing cancer therapies that activate the immune system to detect and destroy cancer cells in immunologically cold tumors, reported over $10M in funding from YK Bioventures, Pillar, Berkeley Frontier Fund, Freeflow Ventures and The National Cancer Institute. Leveraging technology that enables engineering of targeted immunotherapies, Vivere will develop transformative treatments for difficult-to-treat cancers that otherwise evade immune system detection and for which there are few effective treatments.

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Solid tumors such as colorectal and ovarian are among the most difficult cancers to treat. These tumors are characterized by their lack of immune-cell infiltration, making them resistant to conventional immunotherapies like checkpoint inhibitors. Despite decades of research, there has been limited success in cracking the code on cold tumors, leaving patients with few options and poor prognosis.

"Vivere’s goal is to empower the patients’ immune systems to fight off cancer. Our team is united by a belief that cold tumors are not inherently untreatable, just misunderstood," said Melissa Kotterman, Ph.D., CEO of Vivere Oncotherapies. "We’ve spent years building a platform capable of breaking through the immunological invisibility of these tumors. With the support of our investors, we’re now poised to bring a new generation of targeted immunotherapies to patients who currently have no effective options."

"Building Vivere’s platform has been about rethinking what’s possible in cancer therapy. From its early days, the vision was to build a platform that could finally unmask cold tumors to the immune system through improved delivery of targeted and safe therapies," said David Schaffer, Ph.D., Co-founder of Vivere Oncotherapies and Professor of Chemical and Biomolecular Engineering, Bioengineering, and Molecular and Cell Biology at UC Berkeley and Director of QB3 and Bakar Labs. "Our team’s rare combination of engineering, immunology, and translational expertise paired with our experience in building clinical-stage biotech companies gives us the tools to tackle problems others have deemed intractable."

(Press release, Vivere Oncotherapies, JAN 13, 2026, View Source [SID1234662034])

On January 13, 2026 BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported it will outline the pillars of its growing global oncology leadership during its presentation at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco.

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John V. Oyler, Co-Founder, Chairman, and CEO at BeOne, will highlight the Company’s transformative leadership in treating B-cell malignancies. The presentation will feature BRUKINSA, the global leader among Bruton’s tyrosine kinase (BTK) inhibitors, as well as foundational hematology assets: sonrotoclax, a next-generation and potentially best-in-class B-cell lymphoma 2 (BCL2) inhibitor that received its first global regulatory approvals on December 30, and BGB-16673, a potentially first-in-class and best-in-class BTK chimeric degradation activation compound (CDAC). In addition, Mr. Oyler will share further information on BeOne’s global development superhighway, which encompasses global, wholly internal, and competitively advantaged clinical capabilities that can greatly reduce the cost and time to develop a new medicine, thereby increasing the speed of access to innovation for patients around the world.

"BeOne is emerging as the world’s leading oncology company with established leadership in the treatment of B-cell malignancies, an industry-leading pipeline and a unique set of internal capabilities to address tremendous unmet patient need around the world," Mr. Oyler said. "BRUKINSA has entrenched itself as the best-in-class, foundational BTK inhibitor and global revenue leader with an unparallelled long-term efficacy and safety profile across all approved indications. The investigational combination of BRUKINSA and BCL2 inhibitor sonrotoclax has the potential to change the fixed-duration treatment landscape for CLL with best-in-class rates and kinetics of minimal residual disease. BGB-16673 is the most advanced BTK degrader in the clinic and continues to emerge as a potential first-in-class and best-in-class treatment. Combining one of the industry’s most innovative research teams with our vertically integrated clinical development capabilities puts us in a strong position for our next phase of growth with a solid financial profile and a growing global footprint."

Key themes from BeOne’s presentation (7:30 a.m. PT, Tuesday, Jan. 13, 2026) at the J.P. Morgan Healthcare Conference include:

BeOne is the only company with potentially best-in-class, foundational medicines in three key MOAs in CLL

BeOne is the leading company in the treatment of chronic lymphocytic leukemia (CLL) with three approved or clinical-stage foundational medicines addressing all patient subtypes.

BRUKINSA is now the global revenue leader in the BTKi class and the only BTKi to demonstrate superior progression-free survival (PFS) and cardiac safety profile versus ibrutinib in a Phase 3 head-to-head trial. At the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December, the Company presented landmark six-year results from the global, Phase 3 SEQUOIA trial of BRUKINSA versus bendustamine plus rituximab (BR) in treatment-naïve CLL or small lymphocytic lymphoma with an estimated 74% PFS compared with 32% PFS for BR. The overall survival (OS) at 72 months was 84% for BRUKINSA and 80% with BR. After adjusting for COVID-19, the OS rates were 88% and 82%, respectively.

BeOne is rapidly advancing the investigational combination of BRUKINSA and sonrotoclax as a potential best-in-class fixed-duration regimen for the treatment of first-line CLL with clinical results showing potential best-in-class rates of undetectable minimal residual disease (uMRD) at the earliest time as exploratory endpoints. The Company anticipates uMRD results from the Phase 3 CELESTIAL trial of zanubrutinib plus sonrotoclax (ZS) versus venetoclax plus obinutuzumab (VO) in 2026.

In addition, the Company continues to advance BGB-16673 as a potentially first-in-class and best-in-class targeted degrader of BTK with Phase 1 trial results in heavily pretreated CLL patients at a median follow-up of 18 months demonstrating an overall response rate of 86% and 12-month progression-free survival of 79%. The Company anticipates potentially pivotal Phase 2 trial results for BGB-16673 in R/R CLL in 2026.

BeOne’s peerless global development superhighway combined with an industry-leading oncology R&D team drives faster development, accelerated regulatory registrations, and broader global patient access

BeOne is leveraging one of the largest oncology research teams in the industry alongside nearly 6,000 clinical development and manufacturing colleagues supporting the Company’s wholly internal, strategically advantaged global development superhighway to rapidly bring our medicines to cancer patients around the world.

The Company’s dedicated team of research scientists holds deep expertise in designing innovative small-molecule inhibitors, biologics, targeted protein degraders, multispecific antibodies and antibody drug conjugates. In the past 18 months, the Company has advanced 15 New Molecular Entities (NME) into the clinic across a broad range of modalities and expects to deliver an additional eight to 10 NMEs per year into the clinic starting in 2026. BeOne plans to share updates for the following programs, each of which has achieved clinical proof of concept and presents a significant market opportunity: CDK4 inhibitor, B7-H4 ADC, PRMT5 inhibitor, GPC3x41BB bispecific antibody, and CEA ADC.

A strong financial profile enables investment for BeOne’s future growth

BeOne’s strong financial position enables the Company to achieve greater global scale while maintaining profitability in 2026 and beyond.

The Company generated more than $350 million in free cash flow in the third quarter of 2025 with more than $4 billion in cash on hand. BeOne will continue to invest in driving innovation across its hematology and solid tumor pipelines to maximize long-term value for patients and shareholders while pursuing value-creating business development, including opportunities to leverage the Company’s global development superhighway.

Live webcast of BeOne’s presentation can be accessed from the investors section of the Company’s website at View Source Archived replays will be available on the Company’s website.

(Press release, BeOne Medicines, JAN 13, 2026, View Source [SID1234662020])

On January 13, 2026 Daiichi Sankyo reported the first patient has been dosed in the TROPIONLung17 phase 3 trial evaluating DATROWAY (datopotamab deruxtecan) compared to docetaxel in patients with TROP2 NMR positive locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) without actionable genomic alterations previously treated with immunotherapy and platinum-based chemotherapy.

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DATROWAY is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (LSE/STO/Nasdaq: AZN).

The current standard first-line treatment for advanced NSCLC without actionable genomic alterations is immunotherapy with or without platinum-based chemotherapy. 1 However, most patients will eventually experience disease progression and traditional chemotherapy remains the current standard of care in the second-line and beyond settings. 2,3,4,5 While TROP2 is a protein broadly expressed on the surface and inside of NSCLC cells, there is no established predictive biomarker that can identify patients who may benefit from a TROP2 directed antibody drug conjugate. 6,7 TROPION-Lung17 is the first phase 3 clinical trial to prospectively enroll patients with tumors that test positively for TROP2 NMR, a biomarker measured using Quantitative Continuous Scoring (QCS), which is AstraZeneca’s computational pathology platform.

"We initiated TROPION-Lung17 to further evaluate DATROWAY in this patient population, building on results from retrospective analyses of several clinical trials, including TROPION-Lung01, which showed a correlation between TROP2 NMR positivity and outcomes for patients with lung cancer," said Abderrahmane Laadem, MD, Head, Late-Stage Oncology Clinical Development, Daiichi Sankyo.

"TROPION-Lung17 is the first phase 3 trial to prospectively enroll patients using a TROP2 biomarker to determine whether DATROWAY can improve survival compared to current standard of care chemotherapy."

"TROPION-Lung17 aspires to bring a precision medicine approach to patients with advanced lung cancer in the second-line setting, where traditional chemotherapy remains the standard of care," said Leora Horn, MD, MSC, FRCPC, Senior Vice President, Late Development Oncology, AstraZeneca. "Research has shown DATROWAY has the potential to improve outcomes in this setting, and we are confident the TROP2 NMR biomarker and its investigational AI-powered companion diagnostic – previously granted a Breakthrough Device Designation in the U.S. – can help us bring this medicine to the patients most likely to benefit."

TROPION-Lung17 is the ninth phase 3 trial evaluating DATROWAY in NSCLC. Ongoing trials in advanced NSCLC include four phase 3 trials in the first-line metastatic setting evaluating DATROWAY in combination with immunotherapy in tumors without actionable genomic alterations (AVANZAR, TROPION-Lung07, TROPION-Lung08 and TROPION-Lung10) and one phase 3 trial of DATROWAY in combination with osimertinib, AstraZeneca’s EGFR tyrosine kinase inhibitor (TKI), (TROPION-Lung14) in EGFR-mutated NSCLC. Additional later-line phase 3 trials include evaluating DATROWAY with or without osimertinib (TROPION-Lung15) and DATROWAY alone (TROPION-Lung17).

About TROPION-Lung17

TROPION-Lung17 is a global, multicenter, randomized, open-label, phase 3 trial evaluating the efficacy and safety of DATROWAY (6 mg/kg) versus docetaxel in patients with TROP2 NMR positive locally advanced or metastatic nonsquamous NSCLC without actionable genomic alterations previously treated with PD-1/PDL1 inhibitor therapy and platinum-based chemotherapy. All patients will undergo prospective and central tumor testing for TROP2 NMR using the investigational VENTANA TROP2 (EPR20043) RxDx Device (comprised of the companion diagnostic assay and computational pathology algorithm) and those with TROP2 NMR positive tumors will be randomized in a 1:1 ratio to receive either DATROWAY or docetaxel.

The dual primary endpoints of TROPION-Lung17 are progression-free survival as assessed by blinded independent central review and overall survival. Key secondary endpoints include overall response rate, duration of response and safety.

TROPION-Lung17 will enroll approximately 400 patients across multiple sites in Asia, Europe and North America. For more information visit ClinicalTrials.gov.

About TROP2 NMR

TROP2 is a protein broadly expressed in NSCLC tumors.6,7 TROP2 expression on the surface of tumor cells, as measurable with conventional pathology methods, has not been predictive of patient responses to TROP2 directed therapies.8,9 Normalized membrane ratio (NMR) refers to the QCS enabled measure of a tumor cell’s surface TROP2 expression, relative to its surface and cytoplasm TROP2 expression. NSCLC is considered TROP2 NMR positive if at least 75% of tumor cells have a greater proportion of TROP2 in the cytoplasm. TROP2 NMR is being developed to identify patients with NSCLC most likely to benefit from treatment with DATROWAY

Exploratory analyses of the TROPION-Lung01 phase 3, TROPION-Lung02 phase 2 and TROPIONPanTumor02 phase 2 trials have shown a correlation between TROP2 NMR positivity and improved clinical outcomes in patients with NSCLC treated with DATROWAY

Daiichi Sankyo and AstraZeneca are collaborating with Roche Tissue Diagnostics to co-develop and commercialize the VENTANA TROP2 (EPR20043) RxDx Device as a novel AI-powered companion diagnostic for DATROWAY in lung cancer. The device was granted Breakthrough Device Designation by the U.S. Food and Drug Administration. In addition to TROPION-Lung17, it is being used to analyze tissue samples in the TROPION-Lung10 and AVANZAR phase 3 trials.

About Non-Small Cell Lung Cancer

Lung cancer is the most common cancer globally and is the leading cause of cancer-related death in both men and women.10 More than 2.48 million lung cancer cases were diagnosed in 2022, with 1.8 million deaths globally. NSCLC is the most common type of lung cancer, accounting for approximately 85% of cases.

For patients with advanced NSCLC without actionable genomic alterations, immunotherapy with or without platinum-based chemotherapy is the standard first-line treatment.1 While these medicines have improved outcomes in the first-line metastatic setting, most patients experience disease progression and traditional chemotherapy remains the standard of care in the second-line and beyond setting.

About DATROWAY

DATROWAY (datopotamab deruxtecan; datopotamab deruxtecan-dlnk in the U.S. only) is a TROP2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, DATROWAY is one of six DXd ADCs in the oncology pipeline of Daiichi Sankyo, and one of the most advanced programs in AstraZeneca’s ADC scientific platform. DATROWAY is comprised of a humanized anti-TROP2 IgG1 monoclonal antibody, developed in collaboration with Sapporo Medical University, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

DATROWAY (6 mg/kg) is approved in more than 40 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HR positive, HER2 negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease based on the results from the TROPION-Breast01 trial.

DATROWAY (6 mg/kg) is approved in Russia and the U.S. for the treatment of adult patients with locally advanced or metastatic EGFR-mutated NSCLC who have received prior EGFR-directed therapy and platinum-based chemotherapy based on the results from the TROPION-Lung05 and TROPION-Lung01 trials. Continued approval for this indication in the U.S. may be contingent upon verification and description of clinical benefit in the confirmatory trial.

(Press release, Daiichi Sankyo, JAN 13, 2026, View Source [SID1234662021])

On January 12, 2026 Bio‑Sourcing and Zerion Pharma A/S reported that their joint project to develop an oral formulation of Trastuzumab, the pioneer monoclonal antibody used to treat breast cancer, has been awarded funding under EUREKA Eurostars. This is Bio‑Sourcing’s second Eurostars success and, together with Zerion’s breakthrough Dispersome technology, it will support the development of an oral formulation of an anti‑HER2 antibody for the treatment of HER2‑positive breast cancer.

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The program brings together Bio‑Sourcing’s disruptive BioMilk platform—which leverages natural milk components to protect and transport biologics through the gastrointestinal tract and has already been used to produce TrastuzOral —and Zerion’s solid oral‑dosage capabilities using its protein‑based Dispersome technology. Over 36 months, the consortium will optimize an orally deliverable trastuzumab biobetter produced in goat’s milk and progress it through preclinical validation, with the ambition to prepare for entry into Phase I clinical studies around 2030. The total project budget is €1.3 million, with Bio‑Sourcing as lead and Zerion as partner.

An effective oral therapy for HER2 breast cancer has the potential to move care beyond infusion centers, improving patient convenience and adherence while reducing pressure on healthcare resources. The differentiated oral route offers a compelling value proposition in a mature trastuzumab market and creates a platform opportunity to expand oral delivery to other biologics. With the global trastuzumab market estimated at $4.1 billion by 2030, an orally delivered formulation offers clear differentiation and the potential to both capture a significant share of this demand as well as increase overall market size.

Bertrand Mérot, CEO of Bio‑Sourcing: "We are delighted and very proud of Eurostars’ confirmation of the relevance of our unique strategy for oral administration of monoclonal antibodies. I am confident that our partnership with Zerion will further accelerate the development and commercialization of this disruptive formulation for breast cancer. Bio-Sourcing is thus continuing its strategy of developing oral administration of monoclonal antibodies, which has already led to the production of OralimuMab and TrastuzOral in a synergistic partnership with Zerion to further enhance this paradigm shift. The development of this breakthrough in the systemic oral administration of monoclonal antibodies will not only greatly improve patient comfort but also increase access to these innovative treatments, thereby significantly expanding the biopharmaceutical markets."

Ole Wiborg, CEO of Zerion Pharma A/S: "We are excited and very pleased to obtain this Eurostar grant together with Bio-Sourcing. I believe that our joint project holds a significant promise for improving the treatment of breast cancer patients and will further open up a plethora of additional opportunities for switching injectables to orally administered drugs. This blue stamping from EUREKA is also another recognition of our Dispersome technology and our excellent team of scientists that has relentlessly transformed great academic science into a commercially successful technology. I am grateful for that."

Eurostars forms part of the Horizon Europe framework and supports innovative SMEs and project partners—including universities and research organizations—through funding for international collaborative R&D and innovation projects. It is managed by EUREKA, an intergovernmental network covering 37 countries.

(Press release, Zerion, JAN 12, 2026, View Source [SID1234662004])

On January 12, 2026 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported its strategic priorities and anticipated 2026 milestones.

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"Entering 2026, CRISPR Therapeutics is well positioned, with CASGEVY gaining momentum, and multiple programs with encouraging data advancing rapidly through clinical trials across a diverse set of therapeutic areas," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "Across the portfolio, clinical data demonstrated the transformative potential of CTX310 for patients suffering from severe cardiovascular disease, as well as zugo-cel in oncology and autoimmune diseases. We also added a siRNA pillar through our partnership with Sirius and are advancing our lead siRNA asset, CTX611 targeting Factor XI through the clinic. Beyond these clinical stage programs, we have multiple additional assets in preclinical development that leverage our best-in-class advanced editing platform. Together, our broad portfolio, strong balance sheet and effective operating model reinforce our confidence as we move into the next phase."

Anticipated Key Milestones in 2026
CRISPR Therapeutics anticipates several key milestones across its portfolio:

Continued global commercialization and adoption of CASGEVY, with ongoing quarterly updates.
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.
Top-line Phase 2 clinical data from CTX611 in patients undergoing total knee arthroplasty (TKA) are expected in the second half of 2026.
Updates across autoimmune disease and immuno-oncology for zugocabtagene geleucel (zugo-cel; formerly CTX112) are expected in the second half of 2026.
The Company expects to initiate a clinical trial for CTX460 in alpha-1 antitrypsin deficiency (AATD) in mid-2026.
The Company expects to initiate a clinical trial for CTX340 in refractory hypertension in the first half of 2026.

Portfolio Highlights and Outlook

Hemoglobinopathies and CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY now approved in the U.S., the United Kingdom, the EU, the Kingdom of Saudi Arabia, the Kingdom of Bahrain, Qatar, Canada, Switzerland, the United Arab Emirates, and Kuwait for patients 12 years and older with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).
CASGEVY exceeded $100M in revenue in 2025, reflecting more than 60 patients receiving CASGEVY infusions.
Achieved a nearly three-fold increase in patient initiations and first cell collections in 2025 compared to 2024.
Regulatory submissions for CASGEVY in patients aged 5-11 years with SCD and TDT are expected in the first half of 2026.
CASGEVY awarded a Commissioner’s National Priority Voucher for pediatric submissions in SCD and TDT, enabling accelerated regulatory review once submissions are complete.
Continued development of next-generation conditioning approaches with the potential to expand addressable patient populations for SCD and TDT with CASGEVY.
Continued development of a lipid nanoparticle (LNP) based in vivo approach for editing hematopoietic stem cells (HSCs), with the potential to address a broader patient population in SCD and TDT.

In Vivo Liver Editing

CRISPR Therapeutics continues to advance a diversified portfolio of in vivo gene editing programs leveraging its proprietary LNP delivery platform.

In 2025, CRISPR Therapeutics presented data for CTX310, demonstrating deep and durable reductions of triglycerides (TG) and low-density lipoprotein (LDL) following a single-course intravenous infusion, with a well-tolerated safety profile. Based on the positive Phase 1 results, the Company has advanced CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), into Phase 1b clinical trials, prioritizing development in severe hypertriglyceridemia (sHTG) and refractory hypercholesterolemia. The Company expects to provide an update in the second half of 2026.
CTX320, targeting LPA, has demonstrated reductions of up to 73% in the dose escalation phase of the clinical trial. In parallel, the Company is advancing a next-generation LPA program, CTX321, incorporating an updated guide RNA that demonstrates approximately two-fold greater potency in preclinical testing, while utilizing the same LNP delivery system. CTX321 is currently in IND/CTA-enabling studies, with an Lp(a) program update expected in 2026.
In addition to its clinical-stage programs, CRISPR Therapeutics continues to advance several preclinical in vivo gene editing candidates, including:
CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), is the first investigational candidate to emerge from the Company’s SyNTase editing platform. The Company expects to initiate a clinical trial for CTX460 in mid-2026.
CTX340, targeting angiotensinogen (AGT) for refractory hypertension, is currently in IND/CTA-enabling studies. The Company expects to initiate a clinical trial for CTX340 in the first half of 2026.

siRNA-based Programs
CRISPR Therapeutics’ small interfering RNA (siRNA)-based portfolio includes clinical-stage programs in cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics.

CTX611 (SRSD107), a long-acting siRNA targeting Factor XI (FXI), is in an ongoing Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). The Company expects to provide an update in the second half of 2026.

CRISPR Therapeutics will lead global Phase 3 development of CTX611, excluding Greater China. The program targets a range of thromboembolic and clotting-related indications and represents a multi-billion-dollar market opportunity, including arterial fibrillation (AF), venous thromboembolism (VTE), ischemic stroke, cancer-associated thrombosis (CAT), chronic kidney disease (CKD), peripheral vascular disease (PVD), chronic coronary artery disease (CAD).

Autoimmune Disease and Immuno-Oncology
Zugocabtagene geleucel (zugo-cel; formerly CTX112) continues to advance in both autoimmune disease and hematologic malignancies.

In autoimmune disease, Phase 1 clinical trials are ongoing across multiple indications, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis and a second Phase 1 trial in immune thrombocytopenia purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA). The first patient with SLE, refractory to 9 prior therapies with a baseline Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score of 8, has maintained drug-free DORIS clinical remission through month 9 following CAR-T therapy. The second SLE patient with a baseline SLEDAI-2K score of 8, has sustained B cell depletion with SLEDAI-2K score of 0 through month 2 following CAR-T therapy. The Company expects to provide updates in the second half of 2026.

The Phase 1/2 clinical trial of zugo-cel in B-cell malignancies is ongoing. The Company expects to provide updates in the second half of 2026. CRISPR Therapeutics has also established a 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.

Regenerative Medicine
CRISPR Therapeutics continues to advance its regenerative medicine portfolio, including its efforts in diabetes. Clinical data generated from CTX211 were promising, demonstrating detectable C-peptide levels 12 months after implantation. These data have informed the Company’s approach to hypoimmune cell engineering, supporting a transition to a next-generation candidate, CTX213. CTX213 has demonstrated compelling preclinical efficacy and is progressing towards the clinic. The Company expects to provide additional updates as development progresses.

About CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world.

About the CRISPR Therapeutics – Vertex Collaboration for CASGEVY

CRISPR Therapeutics and Vertex established a strategic research collaboration in 2015 to discover and develop therapies using CRISPR/Cas9 technology to address the underlying genetic causes of human disease. CASGEVY is the first approved therapy to emerge from this collaboration. Under an amended collaboration agreement, Vertex leads global development, manufacturing, and commercialization of CASGEVY and shares program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY.

About In Vivo Programs

CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase editing technologies. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are being developed for patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include: CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), CTX340, targeting AGT for the treatment of refractory hypertension and CTX321, targeting LPA for the treatment of patients with elevated lipoprotein(a).

About CTX611 (SRSD107)

CTX611 is a novel double-stranded siRNA, designed to target the human coagulation FXI messenger RNA and inhibit FXI protein expression. Through modulation of the intrinsic coagulation pathway, CTX611 is intended to provide anticoagulant and antithrombotic effects. Supported by clinical experience to date, CTX611 is being developed as a long-acting FXI inhibitor with the potential to support infrequent, including semi-annual, subcutaneous administration.

About Zugocabtagene Geleucel (zugo-cel; formerly CTX112)

Zugocabtagene geleucel (zugo-cel) is a wholly-owned, allogeneic chimeric antigen receptor (CAR) T cell therapy product candidate targeting Cluster of Differentiation 19 (CD19), in development for both autoimmune and immuno-oncology indications. Zugo-cel is an off-the-shelf allogeneic CAR-T that utilizes CRISPR Cas9 for targeted gene knockout and CAR insertion for immune evasion and enhanced T effector cell potency. Zugo-cel is given following a standard lymphodepletion regimen without the need for HLA matching. Zugo-cel is being investigated in ongoing clinical trials in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis and in adult patients with relapsed or refractory B-cell malignancies.

(Press release, CRISPR Therapeutics, JAN 12, 2026, View Source [SID1234661944])