RegulationBreakthrough Therapy (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Breakthrough Therapy (China), Priority Review (Canada), Priority Review (United States), PRIME (European Union)

Structure/Sequence

Sequence Code 1324438509

Source: *****

Clinical Trials associated with Brexucabtagene Autoleucel

NCT06553872

/ RecruitingPhase 2IIT

A Phase 2, Open-Label, Randomized Study of Pirtobrutinib and Brexucabtagene Autoleucel in Patients With Relapsed or Refractory Mantle Cell Lymphoma

This is a phase 2, open-label, randomized, multicenter clinical trial in patients with relapsed/refractory mantle cell lymphoma (R/R MCL) who meet the criteria for standard-of-care FDA label for CD19 CAR T-cell therapy with brexucabtagene autoleucel (brexu-cel).

Start Date23 Aug 2024

Sponsor / Collaborator

H. Lee Moffitt Cancer Center & Research Institute, Inc.

[+1]

NCT06253663

/ Active, not recruitingPhase 2

A Phase 2 Multicenter Study Evaluating the Safety and the Efficacy of KTE-X19 in Adult Japanese Subjects With Relapsed/Refractory Mantle Cell Lymphoma or Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia

The goal of this clinical study is to learn more about KTE-X19, and how safe and effective it is in adult Japanese participants with relapsed/refractory (r/r) Mantle Cell Lymphoma (MCL) or r/r B-precursor Acute Lymphoblastic Leukemia (B-ALL).
The primary objectives of this study are to evaluate the efficacy of KTE-X19, as measured by:
* Objective response rate (ORR) per investigator assessment, in adult Japanese participants with r/r MCL
* Overall complete remission (OCR) defined as complete remission (CR) and complete remission with incomplete hematologic recovery (CRi) per investigator assessment, in adult Japanese participants with r/r ALL

Start Date18 Mar 2024

Sponsor / Collaborator

Kite Pharma, Inc.

NCT06482684

/ RecruitingPhase 2

Early Treatment Intensification in Patients With High Risk Mantle Cell Lymphoma Using CAR-T-cell Treatment After an Abbreviated Induction Therapy With Rituximab and Ibrutinib and 6 Months Ibrutinib Maintenance (Arm A) as Compared to Standard of Care Induction and Maintenance (Arm B)

First-line CAR-T-cell consolidation after an abbreviated induction with 2 cycles of Rituximab and Ibrutinib prior to CAR-T-cell treatment and followed by 6 months of maintenance with Ibrutinib in patients with high risk MCL.

Start Date15 Feb 2024

Sponsor / Collaborator

Ludwig-Maximilians-Universität München

[+1]

100 Clinical Results associated with Brexucabtagene Autoleucel

100 Translational Medicine associated with Brexucabtagene Autoleucel

100 Patents (Medical) associated with Brexucabtagene Autoleucel

148

Literatures (Medical) associated with Brexucabtagene Autoleucel

01 Nov 2025·Drug information journal

Post-marketing Safety Assessment of CAR T-cell Therapies: Analysis of Individual Case Safety Reports in the VigiBase

Article

Author: AlFadel, Nouf S ; AlRasheed, Meshael M ; AlOrf, Nora N ; AlHawas, Solaiman M ; AlHarbi, Fawaz F

PURPOSE:

Chimeric Antigen Receptor (CAR)-T cell therapies have emerged as potential therapy for hematological malignancies, however, their safety profiles should be monitored after approval. Therefore, we aimed in this study to explore and analyze individual case safety report (ICSRs) associated with CAR-T cell therapies and reported to the World Health Organization (WHO) global database (VigiBase).

METHODS:

A retrospective pharmacovigilance study was conducted to describe and characterize Adverse drug reactions (ADRs) reported to Vigibase from inception to March 31st, 2024 and associated with use of the following CAR-T cell therapies: Tisagenlecleucel, Axicabtagene ciloleucel, Brexucabtagene autoleucel, Lisocabtagene maraleucel, Idecabtagene vicleucel, and Ciltacabtagene autoleucel.

RESULTS:

A total of 11,693 ICSRs were identified with the use of CAR-T cell therapies in VigiBase (Axicabtagene ciloleucel (N = 5668, 48.5%), Tisagenlecleucel (N = 3364, 28.8%), Brexucabtagene autoleucal (N = 1027, 8.8%), Lisocabtagene maraleucel (N = 304, 2.6%), Idecabtagene vicleucel (N = 579, 4.9%), and Ciltacabtagene autoleucel (N = 751, 6.4%)). ICSRs completeness score was averaged between 0.2 and 0.57 among all included products and the majority of reported ADRs were serious (67-91%). Among serious ADRs, death was reported with an average percentage of (8.8-21.5%). The majority of ADR reports with fatal outcome occurred in accordance with their approved indications. About 18% of fatal events reported with Tisagenlecleucel as the suspected drug were in the pediatric population.

CONCLUSION:

Our study provides an overall exploration of the post-marketing safety profiles of currently approved CAR-T cell therapies. The significant proportion of fatalities occurred in accordance with approved indications, emphasizes the need for ongoing investigation into ADRs with fatal outcomes, particularly in the pediatric population.

13 Oct 2025·Oncoscience

Targeted therapies and resistance mechanisms in lymphoma: Current landscape and emerging solutions

Review

Author: Afshan, Roshan ; Sridhar, Shruthi ; Tiwari, Bishal

Lymphomas represent a diverse group of hematologic malignancies with variable clinical behavior and underlying biology. The fifth edition of the WHO classification (WHO-HAEM5, 2022) provides an updated, lineage-based framework to categorize lymphoid neoplasms, integrating immunophenotypic, genetic, and clinical features. With advancements in molecular profiling and immunotherapy, targeted treatments have transformed the therapeutic landscape of both Hodgkin and non-Hodgkin lymphomas. This review delineates the critical role of cell surface and intracellular receptors-including CD19, CD20, CD30, PD-1, and CCR4-in lymphoma pathogenesis and as therapeutic targets. We comprehensively evaluate FDA-approved targeted agents, including monoclonal antibodies (rituximab, brentuximab vedotin, obinutuzumab, mogamulizumab), immune checkpoint inhibitors (nivolumab, pembrolizumab), CAR T-cell therapies (axi-cel, tisa-cel, liso-cel, brexu-cel), bispecific T-cell engagers (mosunetuzumab, epcoritamab), and small-molecule inhibitors (ibrutinib, idelalisib, venetoclax). Each class is appraised for mechanism of action, efficacy, and safety in key lymphoma subtypes. Despite significant progress, therapeutic resistance remains a major obstacle. We categorize resistance mechanisms as antigen loss or modulation, pathway reactivation, immune microenvironment adaptation, and genetic/epigenetic evolution. Examples include CD19 antigen loss post-CAR-T therapy, BTK mutations conferring ibrutinib resistance, and immune checkpoint upregulation impairing T-cell function. Emerging strategies to counteract resistance include rational combination therapies, dual-targeted CAR constructs, next-generation bispecific antibodies, and precision-guided immunotherapy. Integration of biomarker profiling, real-time resistance monitoring, and novel immune-engineering approaches offers potential to overcome current therapeutic limitations. In conclusion, understanding the molecular basis of lymphoma and resistance mechanisms is critical to optimizing targeted therapy. This review synthesizes current evidence to inform clinical decision-making and outlines future directions for durable, personalized lymphoma care.

26 Aug 2025·Blood Advances

Outcomes of brexucabtagene autoleucel in patients with relapsed/refractory acute lymphoblastic leukemia with CNS involvement

Article

Author: Roloff, Gregory W. ; Guzowski, Caitlin ; Muffly, Lori S. ; Connor, Matthew ; Mathews, John ; Gupta, Vishal K. ; Malik, Shahbaz A. ; Leonard, Jessica T. ; Shah, Bijal D. ; Kumaran, Muthu ; Dykes, Kaitlyn C. ; Cassaday, Ryan D. ; Dekker, Simone E. ; O'Connor, Timothy E. ; Lee, Catherine J. ; Koura, Divya ; Battiwalla, Minoo ; Muhsen, Ibrahim N. ; Solh, Melhem M. ; Valtis, Yannis ; Tsai, Stephanie B. ; Hill, LaQuisa C. ; Faramand, Rawan ; Park, Jae H. ; Othman, Tamer ; Oliai, Caspian H. ; Sutherland, Katherine C. ; Mountjoy, Luke ; Kopmar, Noam E. ; Miller, Katharine ; Logan, Aaron C. ; Majhail, Navneet ; Frey, Noelle ; Schwartz, Marc ; Sasine, Joshua P. ; Zhang, Amy ; Mercadal, Santiago ; Ahmed, Mohamed ; Shaughnessy, Paul ; Jeyakumar, Nikeshan ; Aldoss, Ibrahim

Abstract:

Patients with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL) with central nervous system (CNS) involvement (CNS B-ALL) have poor outcomes and were frequently excluded from CD19-targeting chimeric antigen receptor (CAR) T-cell clinical trials. The efficacy and safety of brexucabtagene autoleucel (brexu-cel) in adults with R/R B-ALL was established by the ZUMA-3 trial, which excluded patients with advanced or symptomatic CNS involvement. In this retrospective multicenter analysis, we investigated the safety and efficacy of brexu-cel in patients with CNS B-ALL using data from the ROCCA (Real-World Outcomes Collaborative for CAR T in ALL) consortium. Of 189 patients who received infusion, 31 had CNS-2 (presence of blasts in cerebrospinal fluid with <5 white blood cells [WBCs] per μL) or CNS-3 (presence of blasts with >5 WBCs per μL and/or clinical signs/symptoms) disease before apheresis and are the focus of this report. The median age was 46.5 years (range, 24-76), and 58.1% were male. Most (87.1%) received bridging therapy. After brexu-cel, 21 of 24 patients with CNS restaging (87.5%) achieved CNS-1. Additionally, 28 of 30 evaluable patients achieved marrow complete remission; 25 were measurable residual disease negative. No statistically significant differences were seen in progression-free survival or overall survival after brexu-cel among patients with or without CNS involvement. Similarly, grade 3/4 immune effector cell–associated neurotoxicity syndrome occurred similarly in patients with (35.5%) and without (30%) CNS disease. In conclusion, our data suggest that brexu-cel results in high response rates in patients with CNS B-ALL, with toxicity comparable with that in patients without CNS involvement.

233

News (Medical) associated with Brexucabtagene Autoleucel

29 Dec 2025

·www.prnewswire.com

Cell and Gene Therapy Market to Exceed US$105B by 2033, Driving Strategic Value Across Healthcare Portfolios | According to DataM Intelligence

AUSTIN, Texas and TOKYO, Dec. 29, 2025 /PRNewswire/ -- According to DataM Intelligence, the global cell and gene therapy market reached a value of US$13.90 billion in 2024 and is projected to expand significantly to US$105.83 billion by 2033, registering a robust compound annual growth rate (CAGR) of 21.5% during the forecast period from 2025 to 2033. This exceptional growth trajectory reflects the accelerating transition of cell and gene therapies from experimental and early-stage clinical research into commercialized, life-altering treatments. Increasing regulatory approvals, expanding clinical pipelines, and rising investments in advanced therapeutic platforms are positioning cell and gene therapy as one of the most transformative segments within the global biopharmaceutical industry. Request Executive Sample | Cell and Gene Therapy Market Intelligence: Market Overview Cell and gene therapies represent a fundamental shift in how diseases are treated by addressing conditions at their biological root rather than managing symptoms. These approaches can be deployed independently or combined to create gene-modified cell therapies, offering unprecedented therapeutic potential. In combined approaches, a patient's cells are genetically modified through gene therapy techniques and then reintroduced into the body as a cell therapy. One of the most prominent examples is CAR-T cell therapy, where a patient's T cells are engineered to recognize and destroy cancer cells before being reinfused. This approach has demonstrated remarkable success in oncology and continues to expand into additional disease indications. Both cell and gene therapies are increasingly being explored for chronic, inherited, and life-threatening diseases that have limited or no effective treatment options. Their ability to provide long-term or potentially curative outcomes has significantly increased interest from healthcare providers, regulators, and investors worldwide. Market demand is being fueled by a growing number of therapy approvals, a rapidly expanding clinical development pipeline, and rising recognition of the unmet medical needs these therapies can address. Clinical Pipeline Momentum According to the American Society of Cell and Gene Therapy, the sector maintained strong momentum into early 2024, with several notable therapy approvals and sustained growth across all stages of clinical development. Currently, more than 4,000 gene, cell, and RNA-based treatments are in research and development globally. The clinical pipeline has expanded significantly across all phases, with Phase I trials experiencing the highest growth, increasing by approximately 11%. This surge highlights the industry's strong innovation engine and underscores the long-term commercial potential of next-generation therapies entering early-stage development. Market Dynamics Drivers: Advancements in Delivery Vectors One of the most significant growth drivers in the cell and gene therapy market is the increasing utilization of advanced delivery vectors. These vectors are essential for transporting genetic material-DNA or RNA-into target cells safely and efficiently. Traditional delivery systems have limitations related to targeting accuracy, immune response, and dosage efficiency. As a result, next-generation viral and non-viral vectors are being developed to improve therapeutic outcomes while reducing adverse immune reactions. Enhanced delivery systems can also lower dosage requirements, improving both safety and cost-effectiveness. In May 2023, AGC Biologics introduced its BravoAAV and ProntoLVV viral vector platforms, offering faster, scalable, and reproducible production under Good Manufacturing Practice (GMP) conditions. These platforms are designed to support both clinical and commercial-scale manufacturing. Similarly, in May 2024, Charles River Laboratories International, Inc. launched reference materials for adeno-associated virus (AAV) and lentiviral vector (LVV) portfolios to streamline cell and gene therapy research and enable smoother transitions to GMP-quality production. Restraints: High Development and Manufacturing Costs Despite strong growth prospects, high development costs and complex manufacturing processes remain significant restraints for the cell and gene therapy market. Production requires specialized cleanroom facilities, highly skilled personnel, and strict adherence to regulatory standards. Many therapies-particularly autologous treatments such as CAR-T-must be customized for each individual patient, substantially increasing production time and cost. Manufacturing costs for CAR-T therapies can exceed US$100,000 per patient, while certain gene therapies, such as one-time treatments for rare genetic diseases, can cost over US$2 million per patient. These high costs create challenges related to reimbursement, scalability, and patient access. In addition, the production of viral vectors and specialized raw materials is both capital- and time-intensive, with limited suppliers further increasing expenses. According to simulations by the National Institutes of Health (NIH), annual spending on gene therapies could reach approximately US$20.4 billion, even under conservative assumptions. Purchase Corporate License | Cell and Gene Therapy Market Intelligence: Market Segmentation Analysis By Therapy Type The global cell and gene therapy market is segmented into cell therapy and gene therapy, with cell therapy accounting for the dominant share. In 2024, the cell therapy segment held approximately 61.2% of the global market share. The segment demonstrated strong growth, reaching US$13.39 billion in 2022 and increasing further to US$14.66 billion in 2023. Cell therapies have gained widespread adoption due to their strong clinical success, particularly in oncology and regenerative medicine. CAR-T therapies and stem cell-based treatments have shown high efficacy, accelerating approvals and market uptake. A notable milestone occurred in April 2024, when India launched its first indigenously developed CAR-T cell therapy, developed by the Indian Institute of Technology (IIT) Bombay and Tata Memorial Centre. The therapy is being offered at nearly one-tenth of the global cost, significantly improving accessibility and highlighting the role of innovation in cost reduction. Substantial funding from governments and research institutions continues to support cell therapy development. For example, researchers at the University of California, San Francisco (UCSF) initiated a clinical trial for a next-generation CAR-T therapy targeting glioblastoma, supported by an US$11 million grant from the California Institute for Regenerative Medicine (CIRM). Geographical Analysis North America North America dominates the global cell and gene therapy market, accounting for approximately 43.7% of the total market share. The region recorded a market size of US$10.94 billion in 2022, which expanded to US$11.97 billion in 2023. The region's leadership is supported by a strong biotechnology ecosystem, advanced healthcare infrastructure, and the presence of leading academic research institutions. The United States, in particular, benefits from proactive regulatory frameworks that accelerate development and commercialization. The U.S. Food and Drug Administration (FDA) has introduced specialized pathways such as the Regenerative Medicine Advanced Therapy (RMAT) designation, which expedites the review of promising therapies. In August 2024, Ocugen, Inc. received FDA clearance to initiate an expanded access program for OCU400, a modifier gene therapy for retinitis pigmentosa, highlighting continued regulatory support for innovative treatments. Competitive Landscape The cell and gene therapy market features a combination of established pharmaceutical companies and innovative biotechnology firms. Leading players focus on expanding clinical pipelines, improving manufacturing capabilities, and forming strategic collaborations. Key Market Players Emerging players such as Editas Medicine, Intellia Therapeutics, Rocket Pharmaceuticals, Regenxbio, and Affinia Therapeutics are contributing to innovation through next-generation gene-editing and vector technologies. Novartis AG Key CGT products: Kymriah (CAR-T), Zolgensma (AAV gene therapy) Technology focus: CAR-T cell therapy, in-vivo AAV gene therapy Competitive positioning: Novartis is a first-mover and scale leader in CGT, with strong manufacturing capabilities, regulatory experience, and global commercialization across oncology and rare genetic diseases. Primary markets served: US, Europe, Japan, China Gilead Sciences, Inc. Key CGT products: Yescarta, Tecartus (CAR-T therapies) Technology focus: Autologous CAR-T cell therapy (via Kite Pharma) Competitive positioning: Gilead is a dominant CAR-T oncology player, focusing on hematological malignancies with continuous innovation in manufacturing efficiency and next-generation CAR designs. Primary markets served: US, Europe Bristol Myers Squibb Company Key CGT products: Breyanzi, Abecma (CAR-T therapies) Technology focus: CAR-T cell therapies for blood cancers Competitive positioning: BMS has built a robust CAR-T franchise through strategic acquisitions and partnerships, emphasizing differentiated safety profiles and broader patient eligibility. Primary markets served: US, Europe, Japan Vertex Pharmaceuticals Incorporated Key CGT products: Casgevy (exagamglogene autotemcel; gene-edited therapy, partnered) Technology focus: CRISPR/Cas9 gene editing (functional cure approach) Competitive positioning: Vertex is redefining CGT with curative, one-time gene-editing therapies, particularly in hemoglobinopathies, moving the market beyond symptomatic treatment. Primary markets served: US, Europe, select global markets Orchard Therapeutics Key CGT products: Libmeldy (ex-vivo gene therapy for metachromatic leukodystrophy) Technology focus: Ex-vivo autologous hematopoietic stem cell gene therapy Competitive positioning: Orchard is a pure-play rare pediatric gene therapy innovator, specializing in ultra-rare, life-threatening genetic disorders. Primary markets served: Europe, United States Krystal Biotech, Inc. Key CGT products: Vyjuvek (topical gene therapy for dystrophic epidermolysis bullosa) Technology focus: Redosable, non-invasive gene therapy platforms Competitive positioning: Krystal stands out for non-systemic, localized gene therapy delivery, significantly lowering safety and manufacturing complexity compared with systemic CGTs. Primary markets served: United States, Europe Request Customized Intelligence Aligned to Your Business Strategy: Recent Industry Developments The market continues to evolve through technological advancements and strategic initiatives: September 2024: CPC (Colder Products Company) launched a new aseptic micro-connector designed specifically for freeze cassettes used in cell and gene therapy processing. April 2024: Walgreens announced plans to work directly with drug manufacturers to expand patient access to cell and gene therapies through its specialty pharmacy division, including AllianceRx. May 2024: ProPharma, in partnership with Italy-based PBL, launched the Cell Factory Box (CF Box)-a fully automated system enabling decentralized CGT manufacturing in controlled environments. Conclusion The global cell and gene therapy market is entering a period of unprecedented growth, driven by rapid innovation, expanding clinical pipelines, and increasing regulatory support. While challenges related to cost, scalability, and manufacturing complexity persist, ongoing advancements in delivery vectors, automation, and decentralized production are expected to address many of these barriers. With strong momentum across research, development, and commercialization, cell and gene therapies are poised to redefine treatment paradigms and deliver long-term value to healthcare systems worldwide through 2033 and beyond. Related Report: Cell and Gene Therapy Manufacturing Services Market How Biopharma Is Scaling Viral Vector Capacity and Reducing Time-to-Market Gene Editing Tools Market Investment Opportunities Across CRISPR, TALEN, and Next-Generation Editing Platforms Gene Therapy Market Commercialization Challenges, Pricing Pressures, and Growth Opportunities Through 2033 Cancer Gene Therapy Market Pipeline Trends, Clinical Success Rates, and Competitive Positioning Japan Gene Therapy Market Regulatory Pathways, Market Entry Strategies, and Growth Potential Japan Cell Therapy Market Demand Drivers, Manufacturing Landscape, and Strategic Opportunities About DataM Intelligence DataM Intelligence is a renowned provider of market research, delivering deep insights through pricing analysis, market share breakdowns, and competitive intelligence. The company specializes in strategic reports that guide businesses in high-growth sectors such as nutraceuticals and AI-driven health innovations. To find out more, visit or follow us on Twitter, LinkedIn, and Facebook. Contact: Sai Kiran DataM Intelligence 4market Research LLP Ground floor, DSL Abacus IT Park, Industrial Development Area Uppal, Hyderabad, Telangana 500039 USA: +1 877-441-4866 Email: [email protected] Logo: SOURCE DataM Intelligence 4 Market Research LLP 21% more press release views with Request a Demo

Cell TherapyGene TherapyAcquisition

05 Dec 2025

·www.fiercepharma.com

BMS scores 5th indication for Breyanzi with FDA marginal zone lymphoma nod

Bristol Myers Squibb's Breyanzi has scored an FDA approval in its fifth cancer type, marginal zone lymphoma. With FDA approval to treat marginal zone lymphoma (MZL), Bristol Myers Squibb’s Breyanzi has become the first CAR-T therapy in the indication, as well as the first CAR-T to reach the market for five different types of blood cancer.The U.S. regulator has endorsed the one-time infused CD19-directed treatment for adult patients with relapsed or refractory MZL who have received at least two lines of prior therapy.Breyanzi, which broke through in 2021 with a nod for large B-cell lymphoma, has also been blessed by the U.S. regulator to treat mantle cell lymphoma (MCL) and follicular lymphoma (FL), as well as chronic lymphocytic lymphoma (CLL) and small lymphocytic lymphoma (SLL).The latest endorsement gives BMS a leg-up on Gilead’s CD19-directed duo of Yescarta and Tecartus, which have been approved for a combined four blood cancer indications, with the latter CAR-T owning a unique nod in B-cell acute lymphoblastic leukemia.As for MZL, which accounts for roughly 7% of all non-Hodgkin's lymphoma cases, the slow-growing disorder develops when white blood cells clump together in the lymph nodes or organs. Remission is often accomplished with initial therapy, but relapse is common and can occur often and over the span of several years. Some MZL cases can progress into large B-cell lymphoma, which is more aggressive.“Today’s approval represents a major advancement in precision medicine, essentially turning the patient’s immune system into a more effective tool to treat MZL,” Vinay Prasad, M.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER), said in a release.Setting Breyanzi up for approval included results from the phase 2 Transcend FL trial, which enrolled 66 MZL patients and reported a 95% overall response rate, with 62% of participants achieving a complete response. Of the responders, 90% maintained their response through 24 months.With MZL, there is a “pressing need for new treatment options with durable outcomes,” according to study investigator M. Lia Palomba, M.D., a lymphoma and cell therapy specialist at the Memorial Sloan Kettering Cancer Center.Palomba characterized the approval as “a significant advancement in redefining the treatment landscape and providing patients with an option that has demonstrated high rates of responses with an established safety profile.” In the MZL cohort of the study, any grade cytokine release syndrome (CRS) occurred in 76% of patients. Any grade nervous system disorders included headache (21%), tremor (21%), encephalopathy (21%), dizziness (16%), and aphasia (10%), BMS said.With Breyanzi, BMS is playing catch-up with Gilead, which scored its first CAR-T approval with Yescarta in 2017. The treatment became a blockbuster in 2022 and achieved sales of $1.6 billion last year, in addition to sales of $403 million for Tecartus, which reached the market in 2020. Gilead’s revenues from the two CAR-Ts are now in decline, however, with third-quarter sales down for Yescarta by 10% and for Tecartus by 15%. Their combined sales were $432 million for the period.Meanwhile, BMS reported revenues from Breyanzi were up 58% to $359 million in Q3. With sales of $907 million so far this year, Breyanzi is set to become a blockbuster for the first time.BMS gained Breyanzi with its $74 billion acquisition of Celgene in 2019. The deal came less than a year after Celgene gained the treatment with its $9 billion buyout of Juno Therapeutics.

Clinical ResultDrug ApprovalImmunotherapyAcquisitionCell Therapy

25 Nov 2025

·www.businesswire.com

BostonGene to Showcase Transformative Impact of AI-Driven Profiling with Six Abstracts at the 67th American Society of Hematology Annual Meeting & Exposition

WALTHAM, Mass.--(BUSINESS WIRE)--BostonGene, a leading provider of AI-driven molecular and immune profiling solutions, today announced the selection of two abstracts for oral presentation, three abstracts for poster presentation and one abstract for online publication at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, which will be held December 6-9, 2025, in Orlando, Florida. BostonGene will exhibit in booth #1581. Our AI-powered tools, such as the Lymphly classifier and our B-cell-based marker provide the molecular precision required to de-risk clinical programs and ensure the right drug reaches the right patient subpopulation. “The research we are presenting highlights the critical need for advanced patient stratification in hematology. Our AI-powered tools, such as the Lymphly classifier and our B-cell-based marker provide the molecular precision required to de-risk clinical programs and ensure the right drug reaches the right patient subpopulation. This is about moving beyond conventional diagnostics to build a foundational, integrated approach for effective companion diagnostics and unified monitoring in drug studies,” said Nathan Fowler, MD, Chief Medical Officer at BostonGene. Details of the presentations are below: Oral presentations: Abstract number: 14219 Title: Acalabrutinib plus rituximab followed by brexucabtagene autoleucel for frontline treatment of high-risk mantle cell lymphoma: The WINDOW-3 clinical trial Date & time: December 7 — 5:45 PM – 6:00 PM Location: Tangerine Ballroom F2 Speaker: Preetesh Jain, MD, DM, PhD, The University of Texas MD Anderson Cancer Center The WINDOW-3 clinical trial evaluating acalabrutinib plus rituximab followed by brexucabtagene autoleucel CART in patients with high-risk mantle cell lymphoma (MCL) demonstrated strong efficacy and encouraging survival outcomes. BostonGene’s AI-based immunoprofiling revealed CART therapy in high-risk MCL led to drastic immune remodeling, with specific CART-cell types and memory subsets correlating with toxicity, indicating a key role in immune system monitoring for treatment-related toxicities. Research conducted in collaboration with MD Anderson Cancer Center Abstract number: 7242 Title: A phase 1b trial of the EZH2 inhibitor tazemetostat combined with CAR T cell therapy in B cell lymphomas Date & time: December 8 — 5:00 PM – 5:15 PM Location: OCCC - Tangerine Ballroom F3-4 Speaker: Samuel Yamshon, MD, Weill Cornell Medical College Based on the premise that inhibition of EZH2 prevents T cell exhaustion and modulates T cell activity, the phase I study administered tazemetostat with CAR-T in patients with B-cell lymphoma. The combination resulted in an impressive 100% response rate with no signal of additional toxicity. BostonGene’s novel immunophenotyping is being performed to demonstrate the immune modulatory effects of combination therapy, providing mechanistic rationale for further combination approaches. Research conducted in collaboration with Weill Cornell Medical Center Poster presentations: Abstract number: 1837 Title: Refining diffuse large B-cell lymphoma subtyping using Lymphly, an evidence-based classifier Date & time: December 6 — 5:30 PM - 7:30 PM Speaker: Nikita Kotlov, MS, BostonGene BostonGene developed Lymphly, an AI hierarchical classifier for diffuse large B-cell lymphoma (DLBCL), to resolve genetically heterogeneous and atypical tumors often missed by existing classifications. By integrating pathway-relevant genomic alterations into a transparent framework, Lymphly distinguishes both established and emerging subtypes, including high-risk TP53+ and MYC+ groups, offering refined molecular stratification for clinical trial design, treatment selection and targeted therapy development. Abstract number: 2692 Title: A genetic comparison of Epstein-Barr virus-associated polymorphic lymphoproliferative disorder and diffuse large B cell lymphoma Date & time: December 6 — 5:30 PM - 7:30 PM Speaker: Jennifer Chapman, MD, University of Miami and Sylvester Comprehensive Cancer Center BostonGene’s multimodal platform with integrated genomic, transcriptomic and immune data was used to analyze and compare Epstein-Barr virus (EBV)-positive lymphoproliferative disorder and diffuse large B-cell lymphoma. This study underscored the complexity of EBV-driven diseases and highlighted the need for improved classification strategies, demonstrating the potential of BostonGene’s AI-powered platform to advance precision diagnostics and improve patient outcomes. Research done in collaboration with the University of Miami Sylvester Cancer Institute Abstract number: 12996 Title: An immunometabolic companion biomarker to enhance FDG-PET interpretation and guide frontline therapy in follicular lymphoma Date & time: December 8 — 6:00 PM - 8:00 PM Speaker: Joshua W.D. Tobin, MD, MSc, Princess Alexandra Hospital, Mater Research Institute In collaboration with MD Anderson and several hospitals and academic institutions in Australia, BostonGene leveraged its multimodal pipeline to design a B cell-based candidate biomarker in patients with follicular lymphoma treated with bendamustine-based immunochemotherapy (ICT). Represented by centroblast (CB) scores, this biomarker identifies patients with high-grade disease who may respond to bendamustine-based ICT. Independent of and additive to pre-treatment PET, the CB score was highly prognostic suggesting a potential role for patient stratification. Research done in collaboration with the University of Queensland Online only Title: Replacing FISH with a comprehensive integrated approach for tumor genotyping and immune monitoring in multiple myeloma BostonGene’s integrated platform, combining whole exome and RNA sequencing of tumor (CD138⁺ plasma cells) and peripheral blood samples, reproduced FISH-detectable abnormalities (e.g., del17p/TP53 deletion or t(11;14) translocations) and revealed additional mutations, structural variants and immune signatures in multiple myeloma patients, including low-tumor-content samples. By overcoming limitations of conventional FISH, BostonGene’s approach may provide a scalable solution and broader diagnostic capabilities for unified genomic and immune monitoring in clinical trials and patient care. Research done in collaboration with the University of Miami Sylvester Cancer Institute In addition to the poster presentations, the abstracts are published online in the November supplemental issue of Blood. About BostonGene Corporation BostonGene is redefining cancer patient care and drug development through the integration of omnimodal data and artificial intelligence. Built and validated through an extensive real-world clinical testing network, BostonGene’s Foundation Model of cancer and the immune system integrates genomic, transcriptomic, and immune data with clinical outcomes to generate biologically grounded, actionable insights. These insights enable biopharma partners to design and de-risk trials, identify novel targets, and optimize therapeutic response prediction across all stages of development while simultaneously improving patient care through clinically integrated innovation. For more information, visit www.BostonGene.com.

ImmunotherapyClinical ResultPhase 1Cell TherapyASH

100 Deals associated with Brexucabtagene Autoleucel

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Precursor B-Cell Lymphoblastic Leukemia-Lymphoma	Australia	Gilead Sciences Pty Ltd.	30 Sep 2022
Mantle cell lymphoma recurrent	European Union	Kite Pharma EU BV	14 Dec 2020
Mantle cell lymphoma recurrent	Iceland	Kite Pharma EU BV	14 Dec 2020
Mantle cell lymphoma recurrent	Liechtenstein	Kite Pharma EU BV	14 Dec 2020
Mantle cell lymphoma recurrent	Norway	Kite Pharma EU BV	14 Dec 2020
Mantle cell lymphoma refractory	European Union	Kite Pharma EU BV	14 Dec 2020
Mantle cell lymphoma refractory	Iceland	Kite Pharma EU BV	14 Dec 2020
Mantle cell lymphoma refractory	Liechtenstein	Kite Pharma EU BV	14 Dec 2020
Mantle cell lymphoma refractory	Norway	Kite Pharma EU BV	14 Dec 2020
Acute Lymphoblastic Leukemia	United States	Kite Pharma, Inc.	24 Jul 2020
Mantle-Cell Lymphoma	United States	Kite Pharma, Inc.	24 Jul 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Burkitt Lymphoma	Phase 2	United States	Kite Pharma, Inc.	01 Nov 2022
Burkitt Lymphoma	Phase 2	Austria	Kite Pharma, Inc.	01 Nov 2022
Burkitt Lymphoma	Phase 2	France	Kite Pharma, Inc.	01 Nov 2022
Burkitt Lymphoma	Phase 2	Germany	Kite Pharma, Inc.	01 Nov 2022
Burkitt Lymphoma	Phase 2	Italy	Kite Pharma, Inc.	01 Nov 2022
Burkitt Lymphoma	Phase 2	Netherlands	Kite Pharma, Inc.	01 Nov 2022
Burkitt Lymphoma	Phase 2	Spain	Kite Pharma, Inc.	01 Nov 2022
Burkitt Lymphoma	Phase 2	Switzerland	Kite Pharma, Inc.	01 Nov 2022
Hairy Cell Leukemia	Phase 2	United States	Kite Pharma, Inc.	01 Nov 2022
Hairy Cell Leukemia	Phase 2	Austria	Kite Pharma, Inc.	01 Nov 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ChiCTR2300073872 (ASH2025) Manual	Phase 2	Precursor B-Cell Lymphoblastic Leukemia-Lymphoma	28	KTE-X19	yzrvrfgrmq(yowltmahnd) = ubgqhnhdpg judqvlieuu (trdlprlhpp, 62.0 - 90.2) View more	Positive	06 Dec 2025
NCT05495464 (WINDOW-3, ASH2025)	Phase 1	Mantle-Cell Lymphoma First line	20	Acalabrutinib plus rituximab	awrnplyfhf(odbukxvcvd) = only one patient had grade 3 adverse event (AE, skin rash) hrbhvfkiwg (nkynftheai ) View more	Positive	06 Dec 2025
NCT05537766 (ZUMA-25, ASH2025)	Phase 2	Burkitt Lymphoma	10	Brexucabtagene autoleucel (brexu-cel)	mqnwpksose(oyxrebopcs) = atxfdzhreb jmjnkqerjn (oifyxipdhh, 69 - 100) View more	Positive	06 Dec 2025
NCT04880434 (ZUMA-2 Cohort 3, ASH2025)	Phase 2	Mantle cell lymphoma refractory	86	Brexucabtagene autoleucel (Brexu-cel)	skehwqfqxv(gxncsabrsm) = vvkaecvgas iabfepkatq (tkijxpcwvk, 82.5 - 95.9) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Refractory Adult Acute Lymphoblastic Leukemia	134	Brexu-cel infusion	gexxruuiyr(lfszfiwkxs) = jtyokbmbay fjcdguhkxl (dymrtesaiw ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute lymphoblastic leukemia recurrent	92	Obecabtagene autoleucel (obe-cel)	wlapyvnsmr(svxfrzaezq) = pfjkzdylzr tpzgkmvccp (wtnlkadqod ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute lymphoblastic leukemia recurrent	92	Brexucabtagene autoleucel (brexu-cel)	wlapyvnsmr(hgaektpqqq) = qznjjnopfv euscgciybc (ysgnktwmee ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Philadelphia Chromosome Negative Acute Lymphoblastic Leukemia	165	Brexucabtagene autoleucel (Never HCT)	esxjojykrn(cspzrxcldk) = dttzaiysov uwfunwjqzb (bbsogqizbe ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable		165	Brexucabtagene autoleucel (Pre-CAR HCT)	esxjojykrn(cspzrxcldk) = ipjfsvnohl uwfunwjqzb (bbsogqizbe ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Large B-cell lymphoma \| Mantle-Cell Lymphoma \| Precursor B-Cell Lymphoblastic Leukemia-Lymphoma	430	Immunoglobulin replacement therapy (IGRT)	guheldtvtw(hggbohggta) = Infection was probable in 43 (18.1%). Of episodes with a pathogen identified, 58.3% were viral, 40.7% bacterial, 0.5% fungal and 0.5% other. The primary focus was respiratory in 75%. Infection was grade ≥3 in 44% including grade 5 in 6.8%. spvlztjnfb (jlowwzyykx ) View more	Positive	06 Dec 2025
ASCO2025	Not Applicable	CD19-positive B-cell acute lymphoblastic leukemia Consolidation CD19	46	Brexucabtagene autoleucel (Brexu-cel)	yiuvvvplyx(adivfllwlh) = Among the 3 pts with G3-4 CRS/ICANS, the peak CAR T expansion was 102, 1270 and 2222 cells/µL bfscdgnmnt (msvmlznetw )	-	30 May 2025
ZUMA-3 (EHA2025)	Phase 2	Precursor B-Cell Lymphoblastic Leukemia-Lymphoma CD19	78	Brexucabtagene Autoleucel (Brexu-Cel)	ezcanzqwuq(epuqcnqoxj) = llwettvhzx cvmszatcpl (rwwndwmwep, 28.4 - 51.3)	Positive	14 May 2025
ZUMA-3 (EHA2025)	Phase 2	Precursor B-Cell Lymphoblastic Leukemia-Lymphoma CD19	78	Prior Blinatumomab	ezcanzqwuq(epuqcnqoxj) = kzwhrzwckc cvmszatcpl (rwwndwmwep, 12.1 - 40.4)	Positive	14 May 2025

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