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.

Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York.

Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York. The agency is trying to set up cell and gene companies for success and that’s a very different agency than what it was years ago, said Paul Bresge, CEO of Ray Therapeutics Inc.

Alongside the release of abstracts related to the American Society of Hematology meeting next month in San Diego, and as part of the firm’s third-quarter update, Beam Therapeutics Inc. disclosed that one patient died in the phase I/II trial testing BEAM-101 in sickle cell disease (SCD).

Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York.

Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York. The agency is trying to set up cell and gene companies for success and that’s a very different agency than what it was years ago, said Paul Bresge, CEO of  Ray Therapeutics Inc.

The Japanese government, industry and academia are deliberating health care policies and initiatives to boost Japan’s role in the future of regenerative medicine, experts at Bio Japan 2024 said, as the fruits of cell and gene therapy research come to fruition with new approvals.

The Japanese government, industry and academia are deliberating health care policies and initiatives to boost Japan’s role in the future of regenerative medicine, experts at Bio Japan 2024 said, as the fruits of cell and gene therapy research come to fruition with new approvals.

U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies.