Axicabtagene Ciloleucel

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

Issued on behalf of GT Biopharma, Inc. VANCOUVER, BC, Dec. 19, 2025 /PRNewswire/ -- USA News Group News Commentary – The recent FDA approval in November of the first bispecific antibody combination for second-line blood cancer treatment marks the departure from traditional chemotherapy that oncologists have been anticipating for decades[1]. Engineered cell therapies and bispecific antibodies are now achieving response rates exceeding 90% in patients with relapsed and refractory hematologic malignancies, demonstrating the transformative power of immunotherapy in blood cancers[2]. These breakthroughs position companies like GT Biopharma, Inc. (NASDAQ: GTBP), Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN), Genmab A/S (NASDAQ: GMAB), Autolus Therapeutics plc (NASDAQ: AUTL), and Lyell Immunopharma, Inc. (NASDAQ: LYEL). Market forecasters project the CAR-T cell therapy market will surge from $3.87 billion in 2024 to $13.25 billion by 2030, driven by unprecedented efficacy in relapsed and refractory patient populations[3]. GT Biopharma, Inc. (NASDAQ: GTBP), a clinical-stage immuno-oncology company, recently reported advancing its Phase 1 clinical trial of GTB-3650 to Cohort 4, where patients now receive 10μg/kg/day dosing. The company specializes in developing next-generation immunotherapy treatments targeting some of the world's most resistant cancer types through its proprietary natural killer cell engager TriKE platform. GT Biopharma's Phase 1 dose escalation trial evaluates GTB-3650 in patients facing relapsed or refractory blood cancers expressing CD33, particularly acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). These cancers represent exceptionally challenging cases where conventional treatments have either failed completely or provided only temporary benefit before disease returned. The therapy harnesses the patient's natural killer cells, an immune cell type that instinctively identifies and eliminates abnormal cells, directing them to attack cancer specifically. Treatment delivery follows a continuous infusion schedule structured as two-week treatment periods followed by two-week rest intervals, with cycles continuing up to four months depending on individual patient response. "We are highly encouraged by the continued progress of our Phase 1 clinical trial evaluating GTB-3650 in cancer patients, which has now advanced to Cohort 4 at a dose level of 10 µg/kg/day," said Michael Breen, Executive Chairman and CEO. "We look forward to assessing higher doses, as we are now approaching the efficacy range predicted by preclinical in vivo leukemia models, and we plan to share the next trial update in the first quarter of 2026." The six patients enrolled throughout Cohorts 1, 2, and 3 have completed GTB-3650 treatment successfully, establishing the therapy's safety profile across escalating dose concentrations. GT Biopharma indicates the current Cohort 4 dose of 10μg/kg/day represents a threshold where clinical efficacy becomes more likely, supported by encouraging immunological biomarker trends and zero dose-limiting toxicities across all completed cohorts. The first-in-human Phase 1 protocol plans for approximately 14 patients distributed across seven cohorts, with each cohort enrolling two patients at progressively increasing doses starting from 1.25μg/kg/day in Cohort 1 and potentially reaching 100μg/kg/day in Cohort 7 if warranted. Following Cohort 4, three additional escalation levels remain: Cohort 5 testing 25μg/kg/day, Cohort 6 evaluating 50μg/kg/day, and Cohort 7 examining the maximum protocol dose of 100μg/kg/day. The company anticipates providing its next comprehensive trial update during first quarter 2026. Beyond hematologic malignancies, GT Biopharma is advancing GTB-5550, targeting B7H3, a protein prevalent across multiple solid tumor categories including breast, lung, ovarian, pancreatic, bladder, and prostate cancers. Regulatory submission to initiate GTB-5550 human trials is expected in late December 2025 or January 2026. Designed as a subcutaneous injection, GTB-5550 offers the potential for eventual self-administration at home, significantly reducing patient burden. Both therapeutic candidates leverage GT Biopharma's proprietary TriKE platform utilizing specialized antibody fragments initially discovered in camels and llamas. These molecules provide distinct advantages compared to traditional antibodies through their compact size and enhanced stability. GT Biopharma maintains exclusive worldwide licensing rights from the University of Minnesota for this technology. CONTINUED… Read this and more news for GT Biopharma, Inc. at: Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) presented data from the Phase 1/2 LINKER-MM4 trial showing Lynozyfic monotherapy achieved very good partial response or better of ≥70% across all three dose groups in newly diagnosed multiple myeloma patients despite limited follow-up. The first-ever BCMAxCD3 bispecific monotherapy trial in this setting demonstrated that 95% of evaluable responders achieved minimal residual disease negative status at 10⁻⁵ sensitivity. "Lynozyfic monotherapy is already achieving MRD negativity rates comparable to quadruplet regimens but earlier in the treatment course, and these compelling results are expected to deepen with longer follow up," said Robert Orlowski, M.D., Ph.D., Deputy Chair, Professor of Medicine, and Director of Translational Myeloma Research in the Departments of Lymphoma/Myeloma and Experimental Therapeutics at The University of Texas MD Anderson Cancer Center and the lead investigator for the LINKER-MM4 trial. "These results underscore Lynozyfic's potential as a foundational component of frontline treatment regimens for multiple myeloma – or even a monotherapy regimen – for both transplant-eligible and transplant-ineligible patients." The broad clinical development program includes ongoing Phase 2 expansion at the recommended 200mg dose and the LINKER-MM6 trial evaluating combination therapy in transplant-ineligible newly diagnosed patients. Regeneron will host a virtual Regeneron Roundtable investor event on December 10 to discuss its multiple myeloma development program. Genmab A/S (NASDAQ: GMAB) announced new data from its Phase 1b/2 EPCORE CLL-1 trial demonstrating epcoritamab's effectiveness as both monotherapy and in combination regimens for patients with Richter transformation, a rare and aggressive evolution of chronic lymphocytic leukemia. In first-line monotherapy, patients achieved a 57% overall response rate with 52% complete response, while 95% of evaluable responders achieved minimal residual disease negative status across all dose groups. "The results from these trials demonstrate the potential of epcoritamab as a monotherapy, and in combination, in patients with Richter transformation, a rare, often fatal, transformation of chronic lymphocytic leukemia into an aggressive lymphoma, mostly diffuse large B-cell lymphoma," said Dr. Judith Klimovsky, Executive Vice President and Chief Development Officer of Genmab. "We are deeply committed to exploring epcoritamab as a potential core therapy across a range of B-cell malignancies, both as an initial treatment and as a later line of therapy." Genmab continues expanding epcoritamab's development across hematologic malignancies through multiple ongoing Phase 3 trials in collaboration with AbbVie. Autolus Therapeutics plc (NASDAQ: AUTL) presented initial clinical data from its CATULUS Phase 1 trial demonstrating obe-cel achieved a 95.5% overall response rate with 90.9% complete response in pediatric patients with relapsed/refractory B-cell acute lymphoblastic leukemia. The safety profile was consistent with the adult experience, showing low rates of high-grade cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome (both 8.7%), with 20 of 21 responding patients remaining in ongoing remission at data cutoff. "Pediatric patients with r/r B-ALL have a poor prognosis, particularly those who relapse early," said Dr. Matthias Will, Chief Development Officer of Autolus. "We were pleased to share the first data from the Phase 1 CATULUS trial showing obe-cel can produce high remission rates in this pediatric patient population, including in patients with high-risk relapse and patients with primary CNS relapse. Consistent with our experience in the adult population, data show low rates of severe CRS and ICANS." Autolus also presented post-hoc analyses from the FELIX pivotal trial demonstrating that CAR T-cell persistence at three months post-infusion may serve as a marker for predicting long-term outcomes in adult r/r B-ALL patients. Planning for the Phase 2 expansion of the CATULUS trial is underway to further explore obe-cel in pediatric relapsed/refractory B-ALL patients. Lyell Immunopharma, Inc. (NASDAQ: LYEL) presented new clinical data from its ongoing trial of rondecabtagene autoleucel showing 93% overall response and 76% complete response rates with median progression-free survival of 18 months in patients with relapsed/refractory large B-cell lymphoma in the third-or-later-line setting at the 67th American Society of Hematology Annual Meeting. Patients evaluated in the second-line setting, comprised predominantly of those with primary refractory disease, achieved an 83% overall response rate and 61% complete response rate, with 70% of complete responders remaining in complete response at six months or longer. "These data from the ongoing clinical trial showing high rates of durable complete responses along with a manageable safety profile in patients with high-risk large B-cell lymphoma represent the potential of ronde-cel to improve patient outcomes," said Sarah M. Larson, MD, Associate Professor at the David Geffen School of Medicine, University of California, Los Angeles. "The two pivotal trials underway, including the first-of-its kind head-to-head CAR T-cell trial, are expected to provide a comprehensive and robust evaluation of the potential for ronde-cel to demonstrate differentiated benefit over approved CD19 CAR T-cell therapies." Ronde-cel demonstrated a manageable safety profile appropriate for outpatient administration with no high-grade cytokine release syndrome and five percent or less of patients experiencing Grade 3 or greater immune effector cell-associated neurotoxicity syndrome following dexamethasone prophylaxis. Lyell has initiated two pivotal clinical trials, PiNACLE-H2H, a Phase 3 head-to-head randomized controlled trial versus lisocabtagene maraleucel or axicabtagene ciloleucel in the second-line setting, and PiNACLE, a single-arm registration trial enrolling up to 120 patients in the third-or-later-line setting. Article Sources: CONTACT: USA NEWS GROUP [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. This article is being distributed by USA News Group on behalf of Market IQ Media Group Inc. ("MIQ"). MIQ has been paid a fee for GT Biopharma, Inc. advertising and digital media from Creative Digital Media Group ("CDMG"). There may be 3rd parties who may have shares of GT Biopharma, Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. 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