Noah Stansfield

Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia, discussed follow-up data of up to 6 years with investigations of fidanacogene elaparvovec.

Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital, discussed findings from the open-label extension of the ATLAS studies at ASH 2024.

The product will be marketed under the name Ryoncil, and is indicated for patients aged 2 months and older.

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

The agency agreed that data from the ongoing phase 1/2 studies compared to external control natural history data will be sufficient to support a BLA.

Hosted by Joseph Fraietta, PhD, ImmunoLogic will be tailored for an audience fluent in the language of medicine and biotechnology, offering data-driven insights while maintaining accessibility.

The trial for the exon-51 skipping therapy is already active in the United Kingdom.

CGTLive took a closer look at the design of the Eli Lilly subsidiary’s trial in PD for the AAV vector-based gene therapy.

The clinical director of lymphoma services at the University of Colorado discussed 5-year follow-up data from the TRANSCEND-NHL-001 clinical trial.

Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, discussed the latest data update from the CLIMB SCD-121 trial evaluating exa-cel.

The results come from patients with lenalidomide-refractory MM treated in the CARTITUDE-4 trial after 1 to 3 lines of prior therapy.

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

With regard to safety, there were no dose-limiting toxicities, no cases of GvHD, and no cases of TAK-007-related ICANS.

Protocol-defined transfusion independence (TI) was achieved by 52 of the 63 patients in long-term follow-up study LTF-303.

Alongside the updated data, Cartesian announced that it plans to launch a phase 3 clinical trial, dubbed AURORA, in the first half of next year.

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

The phase 2 trial will assess Hansa’s imlifidase as a pretreatment for Genethon’s GNT-0003.

The company also announced updated efficacy data from the trial’s phase 1/2 portion.

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

Clinical responses were reported in patients with lupus and myositis.

David Barrett, JD, the chief executive officer of ASGCT, discussed likely future trends extrapolated from the organization’s Q3 Landscape Report.

The patient’s dosing took place at the University of California, San Francisco, although the multicenter study is expected to eventually dose patients at other locations in the United States, United Kingdom, and Europe.

The FDA’s decision was based on data from 3 different sources.

David Barrett, JD, the chief executive officer of ASGCT, discussed the latest trends in the field of cell and gene therapy captured in the organization's quarterly Landscape Report.

All 3 patients who received the high dose showed stabilization of motor function, as assessed by the 34-point North Star Ambulatory Assessment.

Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg, discussed findings from 2 early studies of CD19 CAR T-cell therapy.

The chief executive officer of ASGCT discussed likely future trends extrapolated from the organization’s Q3 Landscape Report.

Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center, discussed interim data from the phase 1/1b clinical trial evaluating Poseida's CAR-T.

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

The chief executive officer of ASGCT discussed the latest trends in the field of cell and gene therapy.