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AAVivo Advances In Vivo CAR-T Platform with PACE Technology for Precision Gene Delivery

CAR-TPrecision MedicineOncology
  • AAVivo's PACE platform transforms non-targeted AAV vectors into precise, cell-specific gene delivery systems for in vivo CAR-T therapy development.
  • The company's IntelligentAAV platform enables rapid development of precision biotherapies produced directly by patients' bodies for long-term disease control.
  • AAVivo's pipeline includes AVO-100 for B cell malignancies and AVO-200/AVO-300 for solid tumors, positioning the company as a leader in gene therapy innovation.

A2 Bio to Present Early Clinical Data from Logic-Gated CAR-T Therapy Trials at SITC 2025

CAR-TPrecision Medicine
  • A2 Biotherapeutics will present early safety and efficacy data from its EVEREST-2 phase 1/2 study evaluating A2B694, a logic-gated CAR-T therapy targeting mesothelin-positive solid tumors with HLA-A*02 loss.
  • The company will also provide enrollment updates for the DENALI-1 study testing A2B395, an allogeneic logic-gated CAR-T therapy targeting EGFR-expressing solid tumors.
  • Four abstracts accepted for SITC 2025 showcase A2 Bio's proprietary Tmod™ platform, which uses dual-receptor design to selectively target tumor cells while protecting normal cells.
  • The presentations will detail approaches to enhance potency and preserve selectivity of the company's precision cell therapies for multiple solid tumor types.

A Study to Evaluate the Safety and Efficacy of A2B694, a Logic-gated CAR T, in Participants with Solid Tumors That Express MSLN and Have Lost HLA-A*02 Expression

NCT06051695RecruitingPhase 1
A2 Biotherapeutics Inc.
Posted 4/3/2024

Solid Tumor Analysis for HLA Loss of Heterozygosity (LOH) and Apheresis for CAR T- Cell Manufacturing

NCT04981119Recruiting
A2 Biotherapeutics Inc.
Posted 10/29/2021

A Study to Evaluate the Safety and Efficacy of A2B395, an Allogeneic Logic-gated CAR T, in Participants With Solid Tumors That Express EGFR and Have Lost HLA-A*02 Expression

NCT06682793RecruitingPhase 1
A2 Biotherapeutics Inc.
Posted 5/22/2025

Legend Biotech Explores Dual Listing Strategy to Accelerate CAR-T Therapy Expansion

CAR-TOncology
  • Legend Biotech is considering a second listing outside the US, potentially on Hong Kong Stock Exchange or NASDAQ, to diversify its investor base and access new capital sources.
  • The company reported strong revenue growth of 134.73% driven by its flagship CAR-T therapy CARVYKTI, though profitability challenges persist with a net margin of 14.09%.
  • Analysts estimate the company's fair value at $44.9 per share, representing a 23.9% premium to current trading levels, with 12-month price targets averaging $75.8.
  • The dual listing strategy aligns with industry trends, as cross-border listings accounted for 62% of US IPOs in the first half of 2025.

MD Anderson and Phoenix SENOLYTIX Forge Strategic Cross-Licensing Agreement to Advance Injectable Safety Switch Technology for Cell and Gene Therapies

CAR-T
  • The University of Texas MD Anderson Cancer Center and Phoenix SENOLYTIX announced a global cross-licensing agreement to enhance inducible safety switch technologies for cell and gene therapies using rimiducid.
  • Phoenix will develop a proprietary injectable formulation of rimiducid for intramuscular or subcutaneous administration, replacing intravenous infusion to improve patient accessibility and compliance.
  • MD Anderson receives exclusive rights to use Phoenix's new rimiducid formulation with its CaspaCIDe safety switches in ex vivo cell therapy platforms, including CAR-NK and CAR-T cell programs.
  • The collaboration aims to enhance therapeutic safety by enabling rapid elimination of genetically modified cells in case of adverse reactions like cytokine release syndrome.

Florida Cancer Specialists Presents Real-World Evidence Studies on Blood Cancers at SOHO 2025

Real World EvidenceCAR-TMonoclonal Antibody
  • Florida Cancer Specialists & Research Institute presented four abstracts at the Society for Hematologic Oncology 2025 annual meeting, showcasing real-world evidence studies on myelofibrosis and myelodysplastic syndromes.
  • The research includes a retrospective analysis of baseline clinical factors impacting overall survival in myelofibrosis patients and treatment patterns for luspatercept in lower-risk myelodysplastic syndromes.
  • FCS operates more than 150 active clinical trials and has participated in studies for the majority of new cancer drugs approved in the U.S. prior to FDA approval.
  • The organization leverages proprietary real-world datasets and advanced data science to transform clinical data into insights that improve clinical decision-making and patient outcomes.

Charles River Laboratories Integrates Akadeum's Microbubble Technology into Cell Therapy Manufacturing Platform

CAR-T
  • Charles River Laboratories has integrated Akadeum Life Sciences' GMP-grade Human T Cell Leukopak Isolation Kit into its Cell Therapy Flex Program to enhance CAR-T and TCR-T therapy manufacturing.
  • The partnership introduces Akadeum's patented Microbubble technology, which uses buoyancy to isolate T cells by floating unwanted cells to the surface, improving cell viability and scalability.
  • The technology requires no additional equipment or laboratory space and can be performed directly in apheresis bags, streamlining workflows while maintaining GMP compliance.
  • The collaboration addresses key manufacturing challenges in cell therapy, including economics, processing times, and scalability barriers that have limited broader adoption.

UCLA Develops Off-the-Shelf CAR-NKT Cell Therapy for Metastatic Kidney Cancer

CAR-T
  • UCLA researchers have developed AlloCAR70-NKT, an innovative off-the-shelf immunotherapy that uses genetically engineered natural killer T cells to target CD70 protein on kidney cancer cells without requiring patient-specific customization.
  • The therapy demonstrated a multi-pronged attack mechanism in preclinical models, directly killing cancer cells, disrupting the tumor microenvironment, and eliminating immune cells that would normally reject donor cells.
  • This approach addresses critical limitations of traditional CAR-T therapies in solid tumors, offering potential benefits for metastatic renal cell carcinoma patients who face a five-year survival rate of just 12%.
  • The stem cell-derived therapy overcomes time delays and safety risks associated with current immunotherapies, particularly for patients with aggressive, late-stage disease.

Everest Medicines Reports Strong H1 2025 Growth with Advanced mRNA Platform and Pipeline Expansion

CAR-TDrug Approval
  • Everest Medicines achieved 48% year-over-year revenue growth to RMB 446 million in H1 2025, driven by strong performance of NEFECON® and XERAVA®.
  • The company's proprietary AI-enabled mRNA platform advanced significantly with EVM16 personalized cancer vaccine entering first-in-human trials and EVM14 receiving FDA IND clearance.
  • NEFECON® became the first etiological treatment for IgA nephropathy to receive full approval in China, with production expansion approved to meet growing demand.
  • EVER001, a next-generation BTK inhibitor, demonstrated encouraging Phase 1b/2a results in primary membranous nephropathy with sustained efficacy through 52 weeks.

Raina Biosciences Publishes Breakthrough AI Platform for mRNA Therapeutics in Science

AICAR-TDrug Discovery
  • Raina Biosciences announced publication of its GEMORNA platform data in Science, marking the world's first generative AI platform purpose-built for mRNA design and optimization.
  • GEMORNA-generated mRNAs demonstrated up to 150-fold increase in human erythropoietin expression and nearly 100% anti-tumor efficacy in primary human T cells compared to benchmarks.
  • The MIT spinout company closed a $5.7 million angel round and is collaborating with leading pharmaceutical and biotech companies to accelerate mRNA therapeutic development.
  • The platform addresses key limitations in mRNA therapeutics by designing sequences with enhanced expression levels and durability across diverse therapeutic applications.

Allogeneic CD19 CAR-T Cell Therapy Shows Promise for Refractory Lupus Nephritis in Phase 1 Trial

CAR-T
  • A novel allogeneic CD19-targeting T cell therapy called YTS109 demonstrated safety and efficacy in five patients with treatment-refractory systemic lupus erythematosus complicated by severe lupus nephritis.
  • All five patients achieved the primary endpoint of SLE responder index 4 response at month 3, with four patients showing sustained reduction in disease activity scores from a mean of 31.30 to 5.35 by month 6.
  • The CRISPR-engineered therapy overcomes limitations of autologous CAR-T treatments by eliminating personalized manufacturing requirements and reducing costs while achieving complete B cell depletion and renal structural restoration.

An Clinical Study of YTS109 Cell Injection in Subjects With Recurrent/Refractory Autoimmune Disease

NCT06379646RecruitingNot Applicable
China Immunotech (Beijing) Biotechnology Co., Ltd.
Posted 4/24/2024

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