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AbbVie Acquires Capstan Therapeutics for $2.1 Billion to Advance In Vivo CAR-T Technology for Autoimmune Diseases

CAR-T
  • AbbVie has agreed to acquire Capstan Therapeutics for up to $2.1 billion to gain access to CPTX2309, a potential first-in-class treatment for autoimmune diseases currently in Phase 1 trials.
  • The acquisition centers on Capstan's innovative in vivo CAR-T technology that uses lipid nanoparticles to deliver mRNA directly into the body to reprogram CD8 T cells without requiring complex ex vivo manufacturing.
  • CPTX2309 is designed to engineer T cells to express anti-CD19 chimeric antigen receptors that destroy B cells involved in autoimmune disease, offering an off-the-shelf approach that eliminates the need for preconditioning chemotherapy.
  • The deal provides AbbVie with Capstan's targeted lipid nanoparticle delivery system, which could potentially be applied to other diseases beyond autoimmune conditions.

AbbVie Acquires In Vivo CAR-T Developer Capstan Therapeutics for Up to $2.1 Billion

CAR-T
  • AbbVie has agreed to acquire cell therapy developer Capstan Therapeutics in a deal worth up to $2.1 billion in cash, gaining access to innovative in vivo CAR-T technology.
  • Capstan's lead candidate CPTX2309 recently dosed its first patient in a Phase 1 trial for B cell-mediated autoimmune diseases, targeting CD19-expressing B cells.
  • The acquisition brings AbbVie a novel platform that uses lipid nanoparticles and mRNA to engineer T cells directly in the body, potentially transforming autoimmune disease treatment.
  • Major pharmaceutical companies including Pfizer, Bayer, Eli Lilly, Bristol Myers Squibb, Novartis and Johnson & Johnson previously invested in Capstan through private funding rounds totaling $340 million.

CAR-T Therapy Linked to 8.9-Fold Increased Risk of Secondary T-Cell Lymphoma in Global Study

CAR-T
  • A comprehensive global study analyzing 607 cases from pharmacovigilance databases reveals CAR-T therapy recipients face an 8.9-fold elevated risk of developing T-cell lymphoma and 3.5-fold higher risk of myelodysplastic syndromes.
  • Secondary malignancies emerge significantly earlier in CAR-T patients with a median onset of 282 days compared to 526 days in controls, with pediatric patients showing extraordinarily rapid development within just 35 days.
  • The FDA now mandates lifelong monitoring of CAR-T recipients, while experts recommend enhanced surveillance protocols and informed consent discussions to address these emerging safety concerns.
  • Multiple mechanisms contribute to secondary cancer risk including insertional mutagenesis from viral vectors, impaired immune surveillance, and clonal hematopoietic expansion following lymphodepleting chemotherapy.

BCMA-Targeted CAR-T Therapy Achieves 96.3% Response Rate in Relapsed Multiple Myeloma Study

CAR-T
  • A novel dual nanobody CAR-T therapy targeting BCMA demonstrated a remarkable 96.3% overall response rate in 27 patients with relapsed or refractory multiple myeloma, including many with high-risk features.
  • The treatment showed a median duration of remission of 11 months with a range of 2-36 months, indicating sustained therapeutic benefit in this challenging patient population.
  • One-year survival outcomes revealed an overall survival rate of 61.1% and progression-free survival of 57.2%, representing significant clinical activity in heavily pretreated myeloma patients.
  • Real-world data on belantamab mafodotin showed more modest results with 43% overall response rate and 3.8 months median progression-free survival in relapsed/refractory multiple myeloma patients.

FDA Removes Regulatory Barriers for Bristol Myers Squibb's CAR T-Cell Therapies, Expanding Patient Access

CAR-T
  • The FDA eliminated Risk Evaluation and Mitigation Strategy (REMS) programs and reduced post-treatment monitoring from four weeks to two weeks for Bristol Myers Squibb's CAR T-cell therapies Breyanzi and Abecma.
  • The regulatory changes are based on real-world safety data from over 30,000 patients, showing that critical risks like cytokine release syndrome typically manifest within the first two weeks post-infusion.
  • These modifications enable community cancer centers to offer CAR T therapies, potentially doubling or tripling the patient pool as currently only 20% of eligible patients receive these treatments.
  • Bristol Myers Squibb is positioned to capture a larger share of the $15 billion CAR T-cell therapy market, which is projected to grow at 20.9% CAGR through 2030.

FDA Approves Streamlined Access for Bristol Myers Squibb CAR T Cell Therapies

CAR-TDrug Approval
  • The FDA has approved label updates for Bristol Myers Squibb's CAR T cell therapies Breyanzi and Abecma, reducing patient monitoring requirements and removing Risk Evaluation and Mitigation Strategy (REMS) programs.
  • Key changes include reducing driving restrictions from 8 weeks to 2 weeks post-treatment and decreasing the requirement to stay near healthcare facilities from 4 weeks to 2 weeks following infusion.
  • These updates reflect growing real-world evidence from over 30,000 patients treated with CAR T cell therapy, with studies showing most serious adverse events occur within the first two weeks of infusion.
  • Currently only about 2 in 10 eligible patients receive cell therapy treatment due to logistical and geographic barriers, which these label changes aim to address.

A Study to Evaluate the Efficacy and Safety of JCAR017 in Adult Subjects With Relapsed or Refractory Indolent B-cell Non-Hodgkin Lymphoma (NHL)

NCT04245839RecruitingPhase 2
Celgene
Posted 7/14/2020

A Study to Compare the Efficacy and Safety of JCAR017 to Standard of Care in Adult Subjects With High-risk, Transplant-eligible Relapsed or Refractory Aggressive B-cell Non-Hodgkin Lymphomas

NCT03575351CompletedPhase 3
Celgene
Posted 10/23/2018

Study Evaluating Safety and Efficacy of JCAR017 in Subjects With Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL)

NCT03331198RecruitingPhase 1
Juno Therapeutics, a Subsidiary of Celgene
Posted 11/27/2017

Lisocabtagene Maraleucel (JCAR017) as Second-Line Therapy (TRANSCEND-PILOT-017006)

NCT03483103CompletedPhase 2
Juno Therapeutics, a Subsidiary of Celgene
Posted 7/27/2018

EVA Pharma Establishes Eighth Global Office in Hangzhou's BioPharma Town to Advance Cell and Gene Therapy Innovation

CAR-TGene Editing
  • EVA Pharma signed a strategic cooperation agreement with Qiantang District's BioPharma Town in Hangzhou, establishing its eighth global office to focus on pharmaceuticals, vaccines, and biologics R&D.
  • The partnership leverages Hangzhou's position as a global leader in Cell and Gene Therapy, with specialized GMP platforms and companies focused on mRNA vaccines, CAR-T therapy, and gene editing.
  • EVA Pharma will contribute its R&D expertise and regulatory knowledge to explore joint projects in vaccine technology, mRNA applications, and therapeutic biologics while accessing local capabilities.
  • The collaboration strengthens EVA Pharma's global expansion strategy, building on its existing portfolio across twelve therapeutic areas and operations in over 70 countries worldwide.

Priothera Secures €1.7 Million Government Funding to Enhance CAR-T Cell Therapy with Novel S1P Receptor Modulator

CAR-T
  • Priothera received €1.7 million in non-dilutive funding from France's i-Nov innovation competition to support the MOCART clinical program evaluating mocravimod as an adjunct to CAR-T cell therapy.
  • The novel oral S1P receptor modulator mocravimod aims to address the significant toxicity challenges of CAR-T therapy, where 40-60% of patients experience high-grade side effects including cytokine release syndrome and neurological toxicity.
  • Mocravimod's dual mechanism of action is designed to reduce the incidence and severity of CAR-T-related toxicities while potentially improving response rates and treatment durability.
  • The funding represents an expansion of Priothera's clinical development beyond their ongoing Phase 3 MO-TRANS trial in acute myeloid leukemia patients undergoing allogeneic stem cell transplantation.

Vector BioMed Partners with Indian Cancer Hospital to Deliver Affordable CAR-T Therapy to Rural Populations

CAR-T
  • Vector BioMed has formed a strategic partnership with Kailash Cancer Hospital in rural Gujarat, India, to provide affordable CAR-T cell therapy starting in Q1 2026.
  • The collaboration will make KCHRC the first site in Gujarat and among the first in India to deliver advanced CAR-T treatments outside major metropolitan areas.
  • Vector BioMed's LENTIVERSE™ platform enables treatment of one patient per day at significantly reduced costs compared to proprietary systems, making it ideal for resource-limited settings.
  • The partnership aims to expand access to life-saving cancer therapies for underserved populations, with KCHRC serving over 70,000 outpatients annually and providing reduced or no-cost care to more than 60% of patients.

CARMIL2 Gain-of-Function Mutation Shows Promise for Next-Generation Cancer Immunotherapies

CAR-T
  • A landmark preclinical study published in the Journal of Experimental Medicine reveals that a gain-of-function mutation in the CARMIL2 gene unlocks powerful T cell activity and offers a promising new avenue for advanced immunotherapies.
  • The CARMIL2 mutation creates a novel CD28-independent activation pathway that bypasses tumor immune evasion mechanisms and renders T cells resistant to PD-1 and CTLA-4 checkpoint inhibition.
  • French biotech Carmil Therapeutics, founded in 2024, holds exclusive global rights to develop therapeutic applications of CARMIL2-GOF variants for enhancing CAR-T, TCR-T, and TIL therapies.
  • The research positions CARMIL2-GOF as a powerful new approach for adoptive cell therapy, particularly for hard-to-treat solid tumors and refractory cancers.

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