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PolTREG Establishes U.S. Subsidiary Immuthera and Receives Positive EMA Opinion for Type 1 Diabetes Cell Therapy

CAR-T
  • PolTREG established Immuthera as a U.S. subsidiary in Delaware and submitted a Pre-IND application to the FDA to advance its T-regulatory cell therapies in the American market.
  • The European Medicines Agency's Paediatric Committee issued a positive opinion on PolTREG's Pediatric Investigation Plan for PTG-007, an autologous Treg therapy for preventing symptomatic type 1 diabetes in children.
  • The EMA committee recommended expanding the eligible patient population from ages 6-16 years to 3-18 years, suggesting the therapy demonstrates exceptional safety profile.
  • PolTREG operates one of Europe's largest GMP-certified manufacturing facilities with over 2,100 sqm of laboratory space and is the first company worldwide to administer Treg therapies to patients.

CAR-T Cell Therapies Show Promise for Severe Autoimmune Diseases at EULAR 2025

CAR-T
  • Multiple CAR-T cell therapies targeting CD19-positive B cells demonstrated clinical efficacy across severe autoimmune diseases including systemic lupus erythematosus, systemic sclerosis, and rheumatoid arthritis at EULAR 2025.
  • Resecabtagene autoleucel achieved deep B cell depletion and clinical responses in patients with systemic sclerosis, idiopathic inflammatory myopathy, and lupus, with patients remaining free of immunosuppressive therapy.
  • Novel approaches including dual CD19/BCMA targeting CAR-T cells and off-the-shelf iPSC-derived CAR-T therapies showed favorable safety profiles with no severe cytokine release syndrome or neurotoxicity.
  • Early data suggest CAR-T therapy can reset the immune system in multiple rheumatic and musculoskeletal diseases, allowing patients to achieve sustained remission without ongoing immunosuppression.

Johnson & Johnson's Dual-Targeting CAR-T Therapy Achieves 80% Complete Response Rate in Lymphoma Trial

CAR-TOncology
  • Johnson & Johnson's investigational dual-targeting CAR-T therapy JNJ-4496 demonstrated 75-80% complete response rates in patients with relapsed or refractory large B-cell lymphoma at the recommended Phase 2 dose.
  • The therapy targets both CD19 and CD20 antigens on malignant B-cells, potentially addressing resistance mechanisms that limit current single-antigen CAR-T treatments to 40% long-term remission rates.
  • Safety data showed no Grade 3 or 4 cytokine release syndrome cases, with neutropenia being the most common severe adverse event affecting 72% of patients.
  • The Phase 1b study results were presented at the 2025 European Hematology Association Congress, marking a significant advancement in treating the most common aggressive lymphoma type.

A Study of C-CAR039 in Subjects With Relapsed and/or Refractory B Cell Non-Hodgkin's Lymphoma

NCT05149391RecruitingPhase 1
Peking University
Posted 7/20/2021

A Study of JNJ-90014496 in Participants With B-Cell Non-Hodgkin Lymphoma

NCT05421663RecruitingPhase 1
Janssen Research & Development, LLC
Posted 8/12/2022

FDA Approves First-in-Human Trial for Novel CAR-T Therapy Targeting Neuroblastoma

CAR-TDrug Approval
  • The FDA has approved Myrio Therapeutics' IND application for PHOX2B PC-CAR T, marking the first human trial for a therapy targeting the PHOX2B protein in neuroblastoma cells.
  • The novel CAR-T therapy breaks HLA restriction by recognizing peptides across multiple HLA allotypes, potentially expanding treatment to a broader patient population.
  • Neuroblastoma affects approximately 800 children annually in the US and accounts for 15% of pediatric cancer deaths, with current high-risk treatments showing low response rates.
  • The Phase 1 trial will be led by Prof. John Maris at a leading Philadelphia children's hospital, with first patient enrollment anticipated for mid-2025.

BioNTech to Acquire CureVac for $1.25 Billion to Strengthen mRNA Cancer Immunotherapy Pipeline

CAR-T
  • BioNTech announced a $1.25 billion all-stock acquisition of CureVac through a public exchange offer, offering $5.46 per CureVac share representing a 55% premium to strengthen its mRNA-based cancer immunotherapy capabilities.
  • The strategic transaction will combine complementary mRNA technologies in design, delivery formulations, and manufacturing to accelerate development of transformative cancer treatments and establish new standards of care.
  • The deal has secured support from shareholders representing 50.08% of CureVac shares and is expected to close in 2025, marking a key milestone in BioNTech's oncology strategy focused on pan-tumor programs and bispecific antibody candidates.

CRISPR Therapeutics Reports Breakthrough Results from In Vivo Gene Therapy CTX310 for Heart Disease

CAR-T
  • CRISPR Therapeutics reported initial top-line results from an early-stage study on CTX310, showing peak reductions of up to 82% in triglycerides and 81% in LDL cholesterol with a single dose.
  • The investigational in vivo CRISPR-based gene therapy targets ANGPTL3 for treating atherosclerotic heart disease, representing a significant advancement in direct gene delivery technology.
  • CTX310 results have generated excitement for the company's other in vivo candidate CTX320, targeting lipoprotein(a) for heart disease, with initial data expected by month's end.
  • The positive data provides momentum for CRISPR Therapeutics beyond its approved therapy Casgevy, as the company prepares for multiple pipeline readouts throughout 2025.

Toripalimab Emerges as Preferred PD-1 Inhibitor for Metastatic Nasopharyngeal Carcinoma Despite Access Challenges

CAR-TTargeted Therapy
  • Toripalimab has received NCCN category 1 recommendation for nasopharyngeal carcinoma based on positive phase 3 data in both first-line and second-line settings, unlike pembrolizumab which showed negative results in the KEYNOTE-122 trial.
  • Clinical practitioners report easier insurance coverage for pembrolizumab compared to toripalimab, leading many to use pembrolizumab off-label in combination with chemotherapy despite inferior clinical evidence.
  • PD-1 inhibitors combined with chemotherapy represent the established standard of care for systemic recurrent nasopharyngeal carcinoma, with EBV levels serving as useful biomarkers for monitoring treatment efficacy.
  • Future treatment approaches may focus on novel combinations including CAR-T cell therapy, tumor-infiltrating lymphocytes, and targeted therapies like EGFR inhibitors to improve outcomes while reducing toxicity.

PD-1 Blockade Combined With De-intensified Chemoradiotherapy Sparing Concurrent Cisplatin in Nasopharyngeal Carcinoma

NCT04907370Active, Not RecruitingPhase 3
Sun Yat-sen University
Posted 8/1/2021

Study of Pembrolizumab (MK-3475) in Platinum Pre-treated Recurrent/Metastatic Nasopharyngeal Cancer (MK-3475-122/KEYNOTE-122)

NCT02611960CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 4/18/2016

The Efficacy and Safety Study of TORIPALIMAB INJECTION Combined With Chemotherapy for Nasophapyngeal Cancer

NCT03581786Unknown StatusPhase 3
Shanghai Junshi Bioscience Co., Ltd.
Posted 10/18/2018

Cabaletta Bio Launches Public Offering to Fund CAR-T Cell Therapy Development for Autoimmune Diseases

CAR-T
  • Cabaletta Bio announced an underwritten public offering of common stock and warrants on June 11, 2025, with expected closing on June 12, 2025.
  • The clinical-stage biotechnology company is developing the first curative targeted cell therapies specifically designed for patients with autoimmune diseases.
  • The company's lead therapy rese-cel is a fully human CD19-CAR T cell investigational treatment being evaluated in the RESET clinical program.
  • The RESET program spans multiple therapeutic areas including rheumatology, neurology, and dermatology for autoimmune disease treatment.

Capstan Therapeutics Initiates First-in-Human Trial of Novel In Vivo CAR-T Therapy for Autoimmune Diseases

CAR-T
  • Capstan Therapeutics has successfully dosed the first participants in a Phase 1 trial of CPTX2309, an innovative in vivo CAR-T therapy targeting B cell-mediated autoimmune disorders.
  • CPTX2309 uses targeted lipid nanoparticles to deliver anti-CD19 CAR mRNA directly to CD8+ T cells, eliminating the need for chemotherapy, cell manipulation, or viral vectors.
  • The therapy demonstrated rapid, deep, and transient B cell depletion in preclinical studies, with B cell repopulation occurring predominantly with naïve B cells.
  • The Phase 1 trial in healthy volunteers will evaluate safety, tolerability, and pharmacodynamic activity to determine optimal dosing for future autoimmune disease studies.

MaxCyte and Ori Biotech Partner to Enhance CAR-T Cell Manufacturing Through Platform Integration

CAR-TGene Editing
  • MaxCyte and Ori Biotech announced a strategic collaboration integrating their ExPERT™ and IRO® platforms to improve gene-edited T cell yields and reduce manufacturing timelines.
  • The partnership will evaluate CD19 CAR expression via CRISPR knock-in in activated T cells as the initial test system to optimize manufacturing processes.
  • MaxCyte's Flow Electroporation® technology, used in over 19 active clinical and commercial programs, will be combined with Ori's automated manufacturing platform.
  • The collaboration aims to provide therapy developers with enhanced tools to achieve clinically relevant quantities of gene-edited T cells more rapidly and efficiently.

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