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FDA Adds Boxed Warning to Carvykti for Fatal Gastrointestinal Complications

CAR-T
  • The FDA has approved labeling changes for Johnson & Johnson and Legend Biotech's CAR-T therapy Carvykti to include a boxed warning for immune effector cell-associated enterocolitis (IEC-EC), a potentially fatal gastrointestinal condition.
  • IEC-EC symptoms include persistent diarrhea, abdominal pain, and weight loss occurring weeks to months after treatment, with some cases leading to life-threatening complications like bowel perforation and sepsis.
  • Despite the new safety concerns, the FDA determined that Carvykti's overall benefits continue to outweigh the risks, citing statistically significant overall survival improvements in late-stage trial data.
  • The therapy remains approved for adults with relapsed or refractory multiple myeloma who have received at least one prior line of therapy.

Bristol Myers Squibb Acquires Orbital Therapeutics for $1.5 Billion to Advance In Vivo CAR-T Cell Therapy

CAR-T
  • Bristol Myers Squibb announced the acquisition of private startup Orbital Therapeutics for $1.5 billion in cash to strengthen its cell therapy portfolio.
  • The deal provides access to Orbital's in vivo CAR-T technology, which could eliminate the need for costly manufacturing and high-dose chemotherapy required by current CAR-T treatments.
  • Orbital's lead candidate OTX-201 is currently in the investigational new drug (IND)-enabling stage, utilizing advanced RNA engineering and AI design.
  • The acquisition represents Bristol Myers' strategic move in the competitive race to develop safer and more scalable cell therapies for cancer and autoimmune disorders.

CD276 Emerges as Promising Cancer Target with Major Licensing Deals and FDA Breakthrough Designations

CAR-T
  • Minghui Pharmaceutical and Qilu Pharmaceutical signed a global rights deal worth over $200 million in 2024 for CD276-targeted ADC MHB-088C, demonstrating significant commercial confidence in this emerging target.
  • BrainChild Bio received both RMAT and Breakthrough designations from the FDA for its CD276 CAR T-cell therapy BCB-276, particularly for treating diffuse intrinsic pontine glioma (DIPG) where no approved treatments exist.
  • Advanced ADC technology platforms including DXd and SuperTopoiT are enabling improved specificity and reduced off-target toxicity in CD276-targeted therapies, with bispecific formats also being explored.
  • While cancer remains the primary focus, preclinical studies suggest CD276's immunomodulatory activity may have future applications in autoimmune and inflammatory diseases.

Cabaletta Bio's CAR-T Therapy Shows Promise Without Preconditioning in Pemphigus Vulgaris Trial

CAR-T
  • Cabaletta Bio's rese-cel CAR-T therapy achieved complete B cell depletion and meaningful clinical responses in pemphigus vulgaris patients without requiring preconditioning chemotherapy.
  • Two of three patients experienced near-complete resolution of clinical symptoms, with all patients remaining off immunomodulators since treatment infusion.
  • The simplified no-preconditioning regimen could expand treatment access while maintaining similar CAR-T cell expansion compared to over 30 patients who received preconditioning in other trials.
  • Results support continued exploration of the approach in pemphigus vulgaris and potential evaluation in other autoimmune diseases within the RESET program.

BBG Advanced Therapies and CELLforCURE Form Transatlantic Partnership to Expand ATMP Manufacturing Capacity

CAR-T
  • BBG Advanced Therapies and CELLforCURE have announced a strategic partnership to provide transatlantic manufacturing solutions for Advanced Therapy Medicinal Products (ATMPs).
  • The collaboration enables U.S. companies to access CELLforCURE's European manufacturing capabilities while European companies can utilize BBG Advanced Therapies' U.S. production capacities.
  • Combined facilities offer over 3,500 square meters of GMP-certified manufacturing space across multiple cleanrooms, supporting development from Phase I through commercial production.
  • The partnership creates enhanced global CDMO capacity to accelerate the journey from concept to commercialization for cell and gene therapy developers.

Kernal Bio Secures $48 Million ARPA-H Grant to Develop Revolutionary In Vivo CAR-T Cell Therapies

CAR-T
  • Kernal Bio received up to $48 million from ARPA-H to advance its in vivo CAR-T cell therapy program KR-402, targeting multiple sclerosis and B-cell malignancies including acute lymphoblastic leukemia and large B-cell lymphoma.
  • The company's mRNA 2.0 platform uses selective mRNA and targeted lipid nanoparticles to reprogram T cells directly inside the body, potentially reducing manufacturing costs by 100-fold compared to traditional ex vivo CAR-T therapies.
  • The in vivo approach eliminates the need for toxic lymphodepletion procedures and reduces the three-week vein-to-vein turnaround time associated with current CAR-T treatments.
  • Kernal Bio will collaborate with Stanford University School of Medicine, Dana-Farber Cancer Institute, and The Jackson Laboratory to develop this next-generation cell therapy technology.

Cellectis Advances Non-Viral Gene Therapy with Circular DNA Templates and TALE Base Editor Safety Data

CAR-T
  • Cellectis presents breakthrough research on circular single-stranded DNA (CssDNA) templates that achieved high gene insertion frequency in hematopoietic stem cells and showed superior engraftment compared to AAV-edited cells.
  • The company's TALE base editors (TALEB) demonstrated strong safety profile with no evidence of off-target editing biases in nuclear genome analysis of primary T cells.
  • These findings support the potential of non-viral gene therapy approaches and base editing technologies for therapeutic applications in cell and gene therapy development.

Arovella's CLDN18.2 CAR-iNKT Therapy Shows Potent Activity Against Pancreatic Cancer Cells

CAR-T
  • Arovella Therapeutics' proprietary CLDN18.2 chimeric antigen receptor demonstrated potent activity against pancreatic adenocarcinoma cells in preclinical testing at the University of North Carolina.
  • The CAR-T cells engineered with Arovella's patent-protected CLDN18.2 sequence performed comparably to CARsgen Therapeutics' leading construct currently under regulatory review in China.
  • The company plans to integrate its CLDN18.2 CAR into its invariant natural killer T (iNKT) cell platform, which may outperform conventional CAR-T cells in treating solid tumors.
  • Arovella is positioning itself as one of only a handful of companies pursuing CLDN18.2 CAR-iNKT therapies for pancreatic and gastric cancers.

OmniaBio Partners with BrainChild Bio to Manufacture CAR-T Therapy for Fatal Pediatric Brain Cancer

CAR-TRare Disease
  • OmniaBio Inc. and BrainChild Bio announced a manufacturing collaboration for BCB-276, an autologous CAR-T therapy targeting pediatric diffuse intrinsic pontine glioma (DIPG).
  • BCB-276 addresses a critical unmet need in DIPG, a universally fatal brain cancer with median overall survival of less than one year.
  • The partnership will support BrainChild Bio's Phase 2 pivotal registration trial aimed at securing FDA approval for treating children and young adults with DIPG.
  • Manufacturing will utilize OmniaBio's advanced robotics and AI-powered facility in Hamilton, Ontario, emphasizing scalable production and patient access.

FDA Grants Fast Track Designation to UB-VV111, First In Vivo CAR-T Therapy for Relapsed B-Cell Malignancies

CAR-T
  • The FDA has awarded fast track designation to UB-VV111 for treating relapsed/refractory large B-cell lymphoma and chronic lymphocytic leukemia after at least two prior therapies.
  • UB-VV111 represents the first in vivo CAR-T therapy to receive FDA clearance, generating CD19-targeted CAR-T cells directly in patients rather than through traditional ex vivo manufacturing.
  • The investigational therapy aims to address limitations of current autologous CAR-T treatments including high costs, long wait times, and limited patient access.
  • A Phase 1 clinical trial is currently evaluating the safety and efficacy of UB-VV111 in patients with CD19-positive B-cell malignancies.

A Phase 1 Study of UB-VV111 With and Without Rapamycin in Relapsed/Refractory CD19+ B-cell Malignancies

NCT06528301RecruitingPhase 1
Umoja Biopharma
Posted 3/10/2025

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