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University College London has initiated the single site trial at Great Ormond Street Hospital, London.

The associate professor at Fred Hutch Cancer Center discussed data from a retrospective analysis of patients treated with tisa-cel, axi-cel, or liso-cel.

There was 1 serious AE of pneumonia which was found to be possibly related to 4D-710.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The clinical associate professor at Stanford Medicine also discussed ongoing trends in sickle cell disease research.

In terms of safety, Descartes-08 was characterized as well-tolerated during the study and long-term follow-up period.

The postdoctoral researcher at Laboratory for Translational Cancer Immunology, Ludwig-Maximilians-Universität München, discussed further research his group is conducting with cell therapy.

Altogether, there was a 91% reduction in median serum TTR at day 28.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive™ team.

Alexis Thompson, MD, MPH, the chief of hematology at Children’s Hospital of Philadelphia, discussed the need to determine whether the recently approved sickle cell disease gene therapies could benefit younger patients.

Exa-cel was recently approved for the treatment of SCD in the US, but data from the pivotal clinical trial continues to be collected.

The Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed his experience with and reaction to the approval of Casgevy.

The trial has completed enrollment and all 14 patients have demonstrated islet cell engraftment and endogenous insulin production.

The clearance continues a recent trend of IND clearances for CAR-T therapies in autoimmune disease.

The chief hematology/oncology fellow at University of Chicago discussed real-world experience data and new analyses of patients with B-cell acute lymphoblastic leukemia treated with brexu-cel.

Alexis Thompson, MD, MPH, the chief of hematology at Children’s Hospital of Philadelphia, discussed an analysis of follow-up data for bluebird bio’s beti-cel she presented at ASH’s 2023 conference.

No unifying characteristics were found in a small subset of 6 patients from the BENEGENE-2 trial.

The clinical associate professor at Stanford Medicine discussed new research on patients who have received HCT or gene therapy for SCD.

Review top news and interview highlights from the week ending January 5, 2024.

The internal medicine resident physician at University of Kansas Medical Center discussed research conducted into gender authorship rates of 13 pivotal trials published between 2017 and 2022.

The FDA and EMA have accepted marketing authorization applications and decisions are pending.

Senti Bio anticipates the launch of a multicenter clinical trial in hematological malignancies this year, with the first patient expected to receive the therapy within 2024’s second quarter.

The AAV-SLB101 capsid used in SGT-003 may yield increased transgene expression and improved safety compared with first-generation AAV gene therapies.

The cofounder, executive vice president, and chief medical officer, CLL Society, discussed updates in CLL research in the field in general and presented at the ASH 2023 meeting.

The approval of exa-cel, the first gene editing therapy, may herald a new era in developing gene editing strategies.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The chief of hematology at Children’s Hospital of Philadelphia discussed dual approval of the 2 gene therapies that coincided with ASH’s 2023 conference.

Ocugen and the FDA have agreed on trial design aspects including end point, patient enrollment strategy, and study duration.

CGTLive spoke with Donald Wood, PhD, president and CEO of the MDA while looking ahead to its March conference.

The associate professor at Peter MacCallum Cancer Centre discussed further research and questions that need to be investigated with the cell therapy.