China’s National Medical Products Administration (NMPA) has accepted for review Carsgen Therapeutics Holdings Ltd.’s NDA for satricabtagene autoleucel (satri-cel, CT-041), an autologous CAR T candidate targeting Claudin18.2 for treating Claudin18.2-positive advanced gastric/gastroesophageal junction adenocarcinoma (G/GEJA) in patients who have failed at least two prior lines of therapy. Just one day earlier, Carsgen announced that it had submitted the satri-cel NDA to the NMPA.
Abbvie Inc. is shelling out up to $2.1 billion to acquire CAR T player Capstan Therapeutics Inc., gaining rights to a phase I-stage program targeting CD19 as well as an in vivo cell engineering platform. The announcement comes on the heels of recently published data detailing Capstan’s delivery approach using targeted lipid nanoparticles (tLNPs) and marks Abbvie’s latest foray into the CAR T space.
China’s National Medical Products Administration (NMPA) has accepted for review Carsgen Therapeutics Holdings Ltd.’s NDA for satricabtagene autoleucel (satri-cel, CT-041), an autologous CAR T candidate targeting Claudin18.2 for treating Claudin18.2-positive advanced gastric/gastroesophageal junction adenocarcinoma (G/GEJA) in patients who have failed at least two prior lines of therapy. Just one day earlier, Carsgen announced that it had submitted the satri-cel NDA to the NMPA.
Chimeric antigen receptor (CAR) T-cell therapy has been a game changer in the treatment of B-cell malignancies, although their manufacturing process is complex and needs lymphodepletion, thus limiting their use. Researchers from Capstan Therapeutics Inc. have recently published data regarding an in vivo engineering approach to generate CAR T cells using targeted lipid nanoparticles (LNPs) for mRNA delivery to specific T-cell populations for the treatment of cancer and B-cell-mediated autoimmune diseases.
A study has demonstrated the potential of a novel ligand-based CAR T-cell therapy for targeting CD7-positive T-cell malignancies, including T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphomas. The receptor CD7 is a prominent target antigen, being expressed in around 95% of T-ALL, 50% of peripheral T-cell lymphomas and 10% of acute myeloid leukemias.
Newco Brink Therapeutics SAS is poised to work on the next chapter in genome editing after raising €3.5 million (US$4 million) in seed funding to discover and develop programmable recombinase enzymes.
Although CAR T-cell therapies have reached significant clinical success in hematological malignancies, their utility in solid tumors remains limited. One of the main challenges is the scarcity of truly cancer-specific antigens for precise targeting of solid tumors. The use of engineered small, specific antigen-binding domains, such as nanobodies, could be a potential strategy to improve the specificity and efficacy of CAR T cells against solid tumors.
The first clinical data from an FDA-approved trial of a CAR T-cell therapy in the treatment of multiple sclerosis (MS) show the cells cause complete B-cell depletion, leading on to a reset of the immune system that is accompanied by an improvement in symptoms.
The U.S. FDA is to temper the alert it put out in November 2023 pointing to a potential risk of CAR T therapies causing de novo malignancies. “There was this issue of possible safety concerns with T-cell lymphomas, with these CAR T cells. I think this year, we are feeling reassured in this regard,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), told the Alliance for Regenerative Medicine briefing at the J. P. Morgan Healthcare Conference on Jan. 13.
Autolus Therapuetics plc has been granted FDA approval for Aucatzyl (obecabtagene autoleucel) for the treatment of acute lymphoblastic leukemia in adults, becoming the first marketed CAR T therapy that does not have a risk evaluation and mitigation strategy attached to its label. The approval of Aucatzyl was based on results of the Felix clinical trial in relapsing/remitting ALL, which showed a strong safety profile compared to existing CAR T-cell therapies. The conduct of the trial was dogged by the COVID-19 pandemic, but of the 65 patients from an initial dosed cohort of 95 for whom efficacy was evaluated by the FDA, 63% achieved overall complete remission.
