Noah Stansfield

In 2024, CGTLive spoke with experts at various medical conferences to seek their insight on the data they presented and more.

Review top news and interview highlights from the weeks ending December 27, 2024, and January 3, 2025.

Cell therapy, particularly CAR-T, is expanding into autoimmune diseases like lupus and multiple sclerosis. While promising, safety, efficacy, and broad applicability remain uncertain.

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular endocrinology and dermatology stories in 2024.

The company requested priority review for the BLA.

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular lysosomal disorders stories in 2024.

Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed 5-year follow-up data from the TRANSCEND-NHL-001 clinical trial.

The trial is evaluating AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction.

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.