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CARsgen Secures European Patent Protection for GPC3-Targeted CAR-T Therapy Following Successful Defense at EPO

CAR-T
  • CARsgen Therapeutics successfully defended its European patent EP3445407 covering GPC3-targeted CAR-T cell therapy after the sole remaining appellant withdrew its challenge on July 3, 2025.
  • The patent protects key claims for GPC3 CAR-T therapy with cyclophosphamide and fludarabine pretreatment across multiple solid tumor types including liver, lung, ovarian, breast, gastric, and thyroid cancers.
  • The EPO Opposition Division's decision to maintain the patent in amended form is now final and binding, preventing any future challenges to the intellectual property.
  • This outcome strengthens CARsgen's competitive position in the emerging field of CAR-T therapies for solid tumors, an area with significant unmet medical needs.

University of Cincinnati Joins International Phase 1 Trial Testing CAR-T Cell Therapy for Multiple Sclerosis

CAR-T
  • The University of Cincinnati Gardner Neuroscience Institute has become one of the first U.S. sites participating in an international Phase 1 clinical trial testing CAR-T cell therapy for multiple sclerosis patients.
  • The innovative treatment involves genetically modifying patients' own T cells to recognize and eliminate malfunctioning immune cells that drive MS, potentially reaching areas current therapies cannot access effectively.
  • Unlike current MS therapies that mainly target inflammatory cells in the bloodstream, engineered CAR-T cells are designed to find and eliminate harmful immune cells throughout the body, including in the brain and other tissues.
  • The trial includes both relapsing and progressive MS patient cohorts, with researchers having already successfully screened their first patient in the progressive MS group.

Concentra Biosciences Acquires Cargo Therapeutics for $202M Following CAR-T Trial Failure

CAR-TOncology
  • Cargo Therapeutics agreed to be acquired by Concentra Biosciences for approximately $202 million upfront after halting development of its lead CAR-T therapy firicabtagene autoleucel due to poor durability results.
  • The company's Phase II trial showed firi-cel achieved a 77% response rate but only 18% durability at three months, leading to rapid patient relapse and termination of the mid-stage study in January.
  • Concentra, backed by Tang Capital Partners, continues its acquisition strategy targeting distressed biotechs, having completed five buyouts in 2024 including Elevation Oncology and Kronos Bio.
  • The deal provides Cargo shareholders with 100% of net cash above $217.5 million at closing plus 80% of proceeds from asset sales within two years, effectively winding down the company that raised $200 million in 2023.

CERo Therapeutics Advances Novel CER-T Cell Therapy for AML with Invivoscribe Diagnostic Support

CAR-T
  • CERo Therapeutics has initiated a Phase 1 clinical trial for CER-1236, a novel Chimeric Engulfment Receptor T-cell (CER-T) therapy targeting acute myeloid leukemia patients who are relapsed/refractory, in remission with MRD, or newly diagnosed with TP53-mutated MDS/AML.
  • The company's proprietary CER-T platform integrates innate and adaptive immunity characteristics to employ phagocytic mechanisms for tumor destruction, potentially offering broader therapeutic applications than current CAR-T therapies across both hematological malignancies and solid tumors.
  • Invivoscribe's LabPMM laboratories provide critical diagnostic support through customized multiparametric flow cytometry services and validated AML MRD assays to ensure product purity and assess therapeutic response.
  • CER-1236 recently received FDA Orphan Drug Designation for AML treatment, highlighting the clinical potential of this next-generation engineered T-cell therapeutic approach.

FDA Grants Orphan Drug Designation to Mustang Bio's MB-101 CAR-T Therapy for Brain Cancer Treatment

CAR-T
  • The FDA has granted Orphan Drug Designation to Mustang Bio's MB-101 CAR-T therapy for treating astrocytomas and glioblastoma, providing seven years of market exclusivity and development incentives.
  • Phase 1 trial results published in Nature Medicine showed 50% of patients achieved stable disease or better, with two complete responses lasting 7.5 and 66+ months respectively.
  • Mustang Bio is developing MB-109, a combination therapy pairing MB-101 with MB-108 oncolytic virus, designed to convert "cold" tumors into "hot" ones for enhanced CAR-T efficacy.
  • The designation covers a broader indication than originally proposed, expanding the addressable market for this rare brain cancer treatment with significant unmet medical need.

Genetically Modified T-cells in Treating Patients With Recurrent or Refractory Malignant Glioma

NCT02208362Active, Not RecruitingPhase 1
City of Hope Medical Center
Posted 5/18/2015

Everest Medicines Advances AI-Powered mRNA Platform with Cancer Vaccine Breakthroughs

Drug ApprovalCAR-TAI
  • Everest Medicines unveiled major breakthroughs in its proprietary AI-powered mRNA platform during its 2025 R&D Day, showcasing a fully integrated system spanning antigen design to manufacturing.
  • The company's personalized cancer vaccine EVM16 demonstrated robust immunogenicity and mutation-specific T-cell responses in early clinical trials, while off-the-shelf vaccine EVM14 received FDA IND clearance.
  • Everest's innovative in vivo CAR-T program achieved high T-cell transduction efficiency and effective B-cell clearance in preclinical studies, offering a scalable alternative to conventional CAR-T therapies.
  • The company is pursuing global partnerships with top 20 pharmaceutical companies to accelerate international deployment of its clinically validated mRNA assets.

Imugene Secures AU$5.87 Million R&D Tax Refund to Advance Immuno-Oncology Pipeline

OncologyCAR-T
  • Imugene Limited received AU$5.87 million in R&D tax refunds from the Australian Government's incentive program, providing up to 48.5% refundable offset for eligible research activities.
  • The funding will accelerate clinical development of the company's innovative immunotherapy pipeline, including allogeneic CAR T therapy azer-cel for blood cancers and CF33 oncolytic virus therapy for solid tumors.
  • Imugene continues advancing multiple oncology programs including B-cell vaccine candidates, leveraging cutting-edge technology to improve cancer outcomes and transform patient care.

NYU Engineers Develop First Immunocompetent Leukemia-on-a-Chip Device to Advance CAR-T Cell Therapy Testing

CAR-T
  • Researchers from NYU Tandon School of Engineering and University of Pennsylvania have created the first microscope slide-sized "leukemia-on-a-chip" device that combines bone marrow structure with a functioning human immune system.
  • The device enables real-time observation of CAR-T cell dynamics in an authentic microenvironment, revealing previously unseen interactions including a "bystander effect" where engineered cells stimulate non-targeted immune cells.
  • The platform can be assembled within half a day compared to months for animal models, opening possibilities for personalized medicine applications where patient-specific samples can be tested against multiple CAR-T cell designs.
  • Fourth-generation CAR-T cells demonstrated superior performance at lower dosages within the chip environment, suggesting potential for optimizing dose regimens while reducing toxic side effects.

Oncternal Therapeutics Sells ROR1-Targeting Programs to Ho'ola Therapeutics for $68 Million Deal

Monoclonal AntibodyCAR-T
  • Oncternal Therapeutics has sold its zilovertamab monoclonal antibody and ONCT-808 CAR-T cell therapy programs to Ho'ola Therapeutics for $3 million upfront and up to $65 million in milestone payments.
  • Both investigational therapies target ROR1 (Receptor Tyrosine Kinase-Like Orphan Receptor 1), with zilovertamab being a monoclonal antibody and ONCT-808 an autologous CAR-T therapy using zilovertamab's binding domain.
  • Following the asset sale completed on June 27, 2025, Oncternal has appointed Craig R. Jalbert to oversee the company's wind-down operations and future distribution of milestone payments.
  • The milestone structure includes up to $5 million for development achievements, $40 million for regulatory approvals, and $20 million for sales thresholds across major markets including the US, Europe, and Japan.

AvenCell Secures $40 Million AMED Grant to Advance Dual-Antigen Allogeneic CAR-T Therapy for B-Cell Lymphomas

CAR-TGene Editing
  • AvenCell Japan has been awarded up to $40 million in non-dilutive funding from Japan's AMED to support worldwide development of AVC203, a dual-antigen allogeneic CAR-T therapy targeting CD19 and CD20 for B-cell lymphomas.
  • The company's proprietary allogeneic technology uses multiple gene editing steps to prevent immune rejection while maintaining donor T-cell fitness and potency, addressing key challenges that have limited previous allogeneic CAR-T approaches.
  • Early clinical data from AvenCell's AVC201 program in relapsed and refractory AML patients demonstrate robust cell expansion and persistence beyond the typical one-month rejection period where other allogeneic candidates have failed.
  • The funding will enable AvenCell to collaborate with Japanese key opinion leaders and stakeholders to advance what the company describes as a paradigm-shifting therapeutic for patients with high unmet medical needs in lymphomas and leukemias.

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