CGTLive took a closer look at the design of the Eli Lilly subsidiary’s trial in PD for the AAV vector-based gene therapy.
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Cutting-edge cell and gene therapies have sparked ideas for new payment models that could reshape affordability and access to life-changing care.
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Real-world data showed ide-cel was active in patients with central nervous system manifestations of multiple myeloma.
The head of the Referral Center for Sickle Cell Disease and Clinical Research Department at Hôpital Intercommunal de Créteil of the Université Paris Cité discussed the Drepagreffe-1 and 2 studies and improvements seen over 10 years of follow-up.
Review top news and interview highlights from the week ending December 13, 2024.
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.
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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.
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The 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.
A long-term follow-up to the DREPAGREFFE-1 trial suggest that children with sickle cell anemia may benefit long-term on risk of cerebral injury, cognitive functions, and quality of life over standard care transfusions.
Among those who had undetectable minimal residual disease, autologous hematopoietic cell transplantation showed signs of benefit only for those who remained MRD-positive following induction therapy.
The CAR-T, marketed as Kymriah, showed a 4-year overall survival rate of 79.3% and a median progression-free survival of 53.3 months.
With regard to safety, there were no dose-limiting toxicities, no cases of GvHD, and no cases of TAK-007-related ICANS.
The Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed the latest data update from the CLIMB SCD-121 trial evaluating exa-cel.
Protocol-defined transfusion independence (TI) was achieved by 52 of the 63 patients in long-term follow-up study LTF-303.
Allo-HSCT showed good 2-year survival data, with matched sibling donors showing superior outcomes to alternative donors.
In the wake of fludarabine shortages, lemphodepletion with bendamustine was found to be an effective alternative compared for patients with large B-cell lymphoma being treated with a CD19-directed CAR T-cell therapy.
With an FDA deadline pending in January, tabelecleucel remained safe and effective with longer follow-up for Epstein–Barr virus-associated post-transplant lymphoproliferative disease.
Zamtocabtagene autoleucel showed promising early complete response rates and survival outcomes in patients with relapsed/refractory diffuse large B-cell lymphoma.
Rapcabtagene autoleucel showed high rates of durable complete remissions and a favorable safety profile for patients with relapsed/refractory diffuse large B-cell lymphoma.
Review top news and interview highlights from the week ending December 6, 2024.
In observance of HIV/AIDS Awareness Month, held annually in December, we took a look back at the past year's news and expert insights in gene therapy and gene editing for HIV indications.
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.
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
Sana Biotechnology’s SC291 chimeric antigen receptor T-cell therapy is being developed to treat relapsed/refractory systemic lupus erythematosus, including extrarenal lupus and lupus nephritis.
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.
