CAR T - Cell Therapy - Competitive landscape, 2023

This “CAR T - Cell Therapy- Competitive landscape, 2023” report provides comprehensive insights about 250+ companies and 500+ drugs in CAR T - Cell Therapy Competitive landscape. It covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

CAR T - Cell Therapy: Understanding

CAR T - Cell Therapy: Overview

CAR T-cells are the fusion proteins of a selected single-chain fragment variable from a specific monoclonal antibody and one or more T-cell receptor intracellular signaling domains. A CAR combines antigen-binding domains-most commonly, a single-chain variable fragment (scFv) derived from the variable domains of antibodies with the signalling domains of the TCR chain and additional costimulatory domains from receptors, such as CD28, OX40, and CD137. Since the initial development of CARs in 1989, CAR T-cells can be divided into four generations, according to the intracellular domain structure. First-generation CARs comprised of the ζ (zeta) chain of complex TCR/CD3 (CD3ζ), Second-generation CARs are characterized by the dual signal for T-cell activation: one triggered by the antigen recognition and another produced by a co-stimulatory molecule, such as CD28/B7, which promotes the IL-2 synthesis to complete the activation of T-cells and avoid apoptosis, Third-generation CARs combine sequences of co-stimulatory signals, such as OX40 (CD134), CD28, 4-1BB (CD137), CD27, DAP10, or other molecules, in combination with CD3ζ. The combination of multiple co-stimulatory signals may enhance CAR T-cell function via increased cytokine production, T-cell proliferation, and killing in the setting of recursive exposure to antigen and Fourth Generation CARs: Additionally, further optimized design of CARs, such as CAR T-cells redirected for universal cytokine killing (TRUCK), has also been suggested by many researchers. TRUCK cells produce and then release a transgenic product, such as IL-12 or IFN-γ. Advantages of CAR-T cells: HLA (Human Leukocyte Antigen) independent antigen recognition, Minimal risk of generating undesired autoimmunity or GvHD (Graft-versus-host disease), Significant quantities of tumor-specific T-cells are rapidly generated, CARs active in both CD4 + and CD8 + T-cells and Target antigens include proteins, carbohydrates, and glycolipids. One of the most challenging limitations of CAR-T cell therapy is the development of tumor resistance to single antigen targeting CAR constructs. Although initially single antigen targeting CAR-T cells can deliver high response rates, the malignant cells of a significant portion of patients treated with these CAR-T cells display either partial or complete loss of target antigen expression. One of the challenges in targeting solid tumor antigens is that solid tumor antigens are often also expressed on normal tissues at varying levels. Compared to hematological malignancies, solid tumor CAR-T cell therapy is limited by the ability of CAR-T cells to traffic to and infiltrate solid tumors as the immunosuppressive tumor microenvironment and physical tumor barriers such as the tumor stroma limit the penetration and mobility of CAR-T cells.

Report Highlights

• In January 2023, CARsgen Therapeutics had announced a collaboration with Huadong Medicine to commercialize zevorcabtagene autoleucel (zevor-cel), CT053, in mainland China. Under the collaboration, Huadong Medicine will have the exclusive right to commercialize CARsgen's CT053 in mainland China. According to the deal, CARsgen will receive $29.7m (RMB200m) in upfront payment and is also eligible for up to $152.4m (RMB1,025m) in regulatory and commercial milestones.
• In December 2022, Arcellx, Inc. announced a global strategic collaboration to co-develop and co-commercialize Arcellx's lead late-stage product candidate, CART-ddBCMA, for the treatment of patients with relapsed or refractory multiple myeloma. Currently in Phase 2 clinical development, CART-ddBCMA is an investigational cell therapy product comprising autologous T cells that have been genetically modified to target multiple myeloma. CART-ddBCMA utilizes Arcellx's novel D-Domain binder. Kite and Arcellx will jointly advance the CART-ddBMCA asset.
• In December 2022, CARsgen Therapeutics Holdings Limited announced that at the 2022 American Society of Hematology (the "ASH") Annual Meeting, the Company presented a poster with the results of the phase I/II LUMMICAR STUDY 1 clinical trial of zevorcabtagene autoleucel. Results for the 14 subjects treated in phase I of LUMMICAR STUDY 1 showed a well-tolerated safety profile, plus deep and durable responses with an objective response rate (ORR) of 100% and a complete response/stringent complete response (CR/sCR) rate of 78.6%.
• In October 2022, CARsgen Therapeutics Holdings Limited announced that the National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) for zevorcabtagene autoleucel for the treatment of relapsed and/or refractory multiple myeloma (R/R MM). The acceptance of the NDA was based on data from an open-label, single arm Phase I/II clinical trial (LUMMICAR STUDY 1 [Protocol number CT053-MM-01]) in China. Study results showed that zevor-cel has excellent safety and efficacy profiles. Zevor-cel also represents a promising treatment option for patients with high-risk disease.
• In August 2022, Poseida Therapeutics, Inc. announced it has entered into a broad strategic collaboration and license agreement with Roche, focused on developing allogeneic CAR-T therapies directed to hematologic malignancies. The global collaboration covers the research and development of multiple existing and novel "off-the-shelf" cell therapies against targets in multiple myeloma, B-cell lymphomas and other hematologic indications.

CAR T - Cell Therapy: Company and Product Profiles (Marketed Therapies)

1. Company Overview: JW Therapeutics

JW Therapeutics is an innovative biotechnology company focusing on developing, manufacturing and commercializing cell immunotherapy products. Co-founded by Juno Therapeutics (a Bristol Myers Squibb company) and WuXi AppTec in 2016, JW Therapeutics is committed to becoming an innovation leader in cell immunotherapy. The company has built a top world-class platform for technology and product development in cell immunotherapy, as well as a promising product pipeline covering both hematologic malignancies and solid tumors, to bring the hope of a cure for Chinese and global patients, and to lead the healthy and standardized development of China's cell immunotherapy industry.

Product Description: Relmacabtagene autoleucel

Relmacabtagene autoleucel injection (abbreviated as Relma-cel, trade name: Carteyva®) is an autologous anti-CD19 CAR-T cell immunotherapy product independently developed by JW Therapeutics based on a CAR-T cell process platform of Juno Therapeutics (a Bristol Myers Squibb company). Being the first product of JW Therapeutics, Relma-cel was approved by the China National Medical Products Administration (NMPA) in September 2021 for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, making it the first CAR-T product approved as Category 1 biologics product in China. Currently, it is the only CAR-T product in China that has been simultaneously included in the National Significant New Drug Development Program, granted priority review and breakthrough therapy designations. Relma-cel is the first CAR-T product approved as a Category 1 biologics product in China, and sixth approved CAR-T product globally. Relma-cel, JW Therapeutics' first CAR-T product, was independently developed based on a CAR T cell process platform of Juno Therapeutics (a Bristol Myers Squibb company) to meet the needs of the Chinese market. Currently, it is the only approved CAR-T product in China that has been simultaneously included in the National Significant New Drug Development Program, granted priority review (in r/r LBCL) and breakthrough therapy designations (in follicular lymphoma). To date, over 100 patients have been dosed with Relma-cel in clinical studies, marking Relma-cel a most studied anti-CD19 CAR-T product in China.

2. Company Overview: Kite Pharma

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kite's singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. The goal is to fundamentally change the way people think about cancer treatment by pushing the boundaries of what is possible with cell therapy - using genetically modified immune cells to target tumors. Its industry-leading cell therapy technology uses the power of a person's own immune system to target and attack their tumors. This is accomplished through the use of genetically modified T cells that can increase the number of tumor-specific T cells and strengthen the body's ability to kill certain types of cancer cells.

Product Description: KTE-X19

KTE-X19 is an investigational, autologous, anti-CD19 CAR T cell therapy. KTE-X19 uses the XLP™ manufacturing process that includes T-cell selection and lymphocyte enrichment. Lymphocyte enrichment is a necessary step in certain B-cell malignancies with evidence of circulating lymphoblasts. KTE-X19 is currently in Phase I/II trials in acute lymphoblastic leukemia (ALL), mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL).In December 2020, Kite, announced that the European Commission has granted conditional marketing authorization for Tecartus (autologous, anti-CD19-transduced CD3+ cells; formerly KTE-X19). Tecartus is a chimeric antigen receptor (CAR) T cell therapy for adult patients with relapsed or refractory mantle cell lymphoma after two or more lines of systemic therapy including a Bruton's tyrosine kinase (BTK) inhibitor. In July 2020, the U.S. Food and Drug Administration (FDA) has granted accelerated approval to Tecartus (brexucabtagene autoleucel, formerly KTE-X19), the first and only approved chimeric antigen receptor (CAR) T cell therapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL).

3. Company Overview: Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company. Bristol Myers Squibb is inspired by a single vision - transforming patients' lives through science. The goal of the company's cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient's life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

Product Description: Abecma

Abecma recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells. Abecma is being jointly developed and commercialized in the U.S. as part of a Co-Development, Co-Promotion, and Profit Share Agreement between Bristol Myers Squibb and 2seventy bio. The companies' broad clinical development program for Abecma includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-9) in earlier lines of treatment for patients with multiple myeloma. Abecma is the first and only CAR T cell therapy approved that is directed to recognize and bind to BCMA, a protein that is nearly universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells. Abecma is delivered via a single infusion with a target dose of 420 x 106 CAR-positive viable T cells within a range of 260 to 500 x 106 CAR-positive viable T cells. Abecma is approved for use in all European Union (EU) member states. In August 2021, Bristol Myers Squibb announced that the European Commission (EC) has granted Conditional Marketing Authorization for Abecma, for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy. In March 2021, Bristol Myers Squibb and bluebird bio, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Abecma, for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.

CAR T - Cell Therapy: Company and Product Profiles (Pipeline Therapies)

1. Company Overview: CARsgen

CARsgen is a biopharmaceutical company with operations in China and the US and is focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. The Company has built an integrated cell therapy platform with in-house capabilities that span target discovery, antibody development, clinical trials, and commercial-scale manufacturing. CARsgen has internally developed novel technologies and a product pipeline with global rights to address major challenges of CAR T-cell therapies, such as improving the safety profile, enhancing the efficacy in treating solid tumors and reducing treatment costs. The Company's vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable.

Product Description: Zevorcabtagene autoleucel

Zevor-cel (CT053) is a fully human, autologous BCMA CAR T-cell product candidate for the treatment of R/R MM. CT053 uses a CAR construct with a fully human BCMA-specific single-chain variable fragment designed to have lower immunogenicity and increased stability. CARsgen is conducting a Phase I/II clinical trial to evaluate the safety and efficacy of zevor-cel for R/R Multiple myeloma. The Company also plans to conduct additional clinical trials to develop zevor-cel as an earlier line of treatment for multiple myeloma. CARsgen hopes the therapy will address challenges of T-cell exhaustion by reducing self-activation of CAR T-cells when tumor-associated targets are not present. The Company believes that zevor-cel is well positioned to potentially reshape the treatment paradigm for multiple myeloma and become a foundational treatment for multiple myeloma patients.

2. Company Overview: Cartesian Therapeutics

Founded in 2016 and with three assets currently in clinical trials, Cartesian is the leader in mRNA-engineered cell therapy. Cartesian's vision is to cure disease by mRNA engineering any cell, to deliver to any tissue, any combination of therapies. The company is pioneering mRNA cell therapies in and beyond oncology, with products in development for respiratory, autoimmune, and oncologic disorders. All investigational therapies are manufactured at Cartesian's wholly owned, state-of-the-art cGMP manufacturing facility in Gaithersburg, MD.

Product Description: Descartes 011

Descartes-11 is engineered with Cartesian's RNA Armory℠ platform to express its CAR molecules transiently instead of permanently, thereby reducing both short-term and long-term risks inherent with conventional CAR T-cell therapies. Descartes-11 is manufactured in-house at Cartesian's wholly owned, state-of-the-art cGMP manufacturing facility in Gaithersburg, MD. A Phase 1 study of Descartes-11 in patients with advanced myeloma showed no evidence of typical CAR T-cell toxicities such as cytokine release syndrome (CRS) of neurotoxicity. Tens of billions of cells are mRNA-engineered with the CAR molecule so that the full therapeutic dose can be administered with each infusion. This eliminates the need for preconditioning chemotherapy required by conventional CAR T-cell therapies. Currently the drug is in Phase II stage of Clinical trial evaluation for the treatment of Multiple Myeloma.

3. Company Overview: Autolus Therapeutics plc.

Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the Company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors.

Product Description: AUTO8

AUTO8 is our next-generation product candidate for multiple myeloma which comprises two independent CARs for the multiple myeloma targets, BCMA and CD19. Company have developed an optimized BCMA CAR which is designed for improved killing of target cell that express BCMA at low levels. This has been combined with fast off rate CD19 CAR from obe-cel. Autolus Therapeutics believe that the design of AUTO8 has the potential to induce deep and durable responses and extend the durability of effect over other BCMA CARs currently in development.

4. Company Overview: Sana Biotechnology

Sana Biotechnology, Inc. is focused on creating and delivering engineered cells as medicines for patients. We share a vision of repairing and controlling genes, replacing missing or damaged cells, and making our therapies broadly available to patients. We are a passionate group of people working together to create an enduring company that changes how the world treats disease. Sana has operations in Seattle, Cambridge, South San Francisco, and Rochester.

Product Description: SG299

SG299 has the potential to generate CAR T cells in vivo (inside the patient), eliminating the need for conditioning chemotherapy and complex CAR T cell manufacturing. The company's goal is to file an IND this year to study this drug candidate in patients with B cell malignancies. In 2022, Sana transitioned to a new manufacturing process for SG295 and renamed the product SG299 in connection with that transition. SG299 has at least a 50X improvement in product potency, which Sana believes has the potential to translate into better efficacy, safety, and long-term manufacturability. The company plans to use this second-generation process for the first-in-human studies in patients with B cell malignancies.

CAR T - Cell Therapy Analytical Perspective

In-depth Commercial Assessment: CAR T - Cell Therapy Collaboration Analysis by Companies

The Report provides in-depth commercial assessment of drugs that have been included, which comprises collaboration, agreement, licensing and acquisition - deals values trends. The sub-segmentation is described in the report which provide company-company collaboration (licensing/partnering), company academic collaboration and acquisition analysis in tabulated form.

• CAR T - Cell Therapy Competitive Landscape

The report comprises of comparative assessment of Companies (by therapy, development stage, and technology).

CAR T - Cell Therapy Report Assessment

• Company Analysis
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions Answered

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing CAR T - Cell Therapy drugs?
• How many CAR T - Cell Therapy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of CAR T - Cell Therapy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the CAR T - Cell Therapy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for CAR T - Cell Therapy and their status?
• What are the key designations that have been granted to the emerging and approved drugs?

Key Players

• Cartesian Therapeutics
• CASI Pharmaceuticals/Juventas Cell Therapy
• Novartis
• Poseida Therapeutics
• Shanghai Unicar-Therapy Bio-medicine Technology
• JW Therapeutics
• Gilead Sciences
• Cellular Biomedicine Group
• Gracell Bio
• Mustang Bio
• Servier
• iCell Gene Therapeutics
• Kecellitics Biotech Company Ltd
• Gilead sciences
• Miltenyi Biotec
• Nanjing KAEDI Biotech
• Liminatus Pharma
• Yake Biotechnology
• AffyImmune Therapeutics
• Fundamenta Therapeutics
• Actinium Pharmaceuticals
• Allogene Therapeutics
• Celyad Oncology
• Maxcyte
• Sorrento Therapeutics
• Cellular Biomedicine Group
• Sorrento Therapeutics
• Shanghai GeneChem Co., Ltd.
• Sensei Biotherapeutics
• Obsidian Therapeutics
• Beijing Biohealthcare Biotechnology Co. Ltd.
• Eutilex/Utilities
• Precigen, Inc.
• Miltenyi Biomedicine
• Wugen
• WindMIL Therapeutics
• MiNK Therapeutics
• CiMaas
• Catamaran Bio
• Innate Pharma
• Healios
• Chimeric Therapeutics
• ONK Therapeutics
• Neukio Biotherapeutics
• Exacis Biotherapeutics
• Editas Medicine

Key Products

• Descartes-11
• CNCT19
• CTL119
• P-BCMA-101
• CD19/CD20/CD22/CD30 CAR T-cell therapy
• JWCAR029
• KTE-X19
• Ciltacabtagene autoleucel
• CD20-CD19 Bispecific CAR
• GC027
• MB-103
• UCART19
• BCMA-CD19 cCAR T cells therapy
• Anti CD19 CART
• Human anti-CD19
• CD20 CAR T cells
• Anti-CD19 Chimeric Antigen Receptor T cells (KD-019 CAR-T)
• GUCY2C-Targeted CAR-T Cells
• CD19 CAR T-cells
• AIC100
• ThisCART22
• Iomab-ACT Program
• ALLO-501
• CYAD-211
• MCY-M11
• PSMA CAR-T -cell immunotherapy
• Anti-CD20 CAR
• c-KIT CAR-T
• BCK - CART
• EGFRv III-CART
• SNS-723
• cytoDRiVE technology based therapy
• CD30-targeted Chimeric Antigen Receptor T cell therapy
• Solid cancer specialized CAR-T
• MVR CAR-T
• PRGN-3007
• PRGN-3008
• huBCMA
• MB-CART 2219.1
• WU-CART-002
• CAR-MILs
• Stromal target-CAR-iNKT
• CIM 301
• CAT-179
• IPH65
• IPH62
• IPH64
• HLCN 061
• CHM 0301
• ONKT105
• ONKT106

Research programme: NK cell therapeutics

• iPSC-CAR-NK
• ExaCAR-NK
• ExaNK
• ExaCAR-T
• EDIT 301

Research programme: allogeneic pluripotent stem cell therapeutics

Research programme: non alpha beta T cell therapies

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