News

Binod Dhakal, MD, discusses the potential impact of ide-cel, orva-cel, and cilta-cel in multiple myeloma therapy.

AbbVie and Caribou Biosciences, Inc have entered into a collaboration and license agreement for the research and development of chimeric antigen receptor T-cell therapeutics.

ADP-A2M4CD8, a novel T-cell therapy, is being investigated in a range of tumor types that express MAGEA4, an antigen expressed in solid tumors that investigators say represents a promising target for cellular immunotherapy.

FDA advises against submission of NurOwn as a treatment for ALS, based on a lack of statistical significance in phase 3 study.

An independent board has determined that ofranergene obadenovec is safe allowing the phase 3 OVAL study to continue.

Bijan Nejadnik, MD, chief medical officer, SanBio, also thanked neurological experts that collaborated on the phase 2 STEMTRA trial.

Masahito Kawabori, MD, PhD, associate professor, Hokkaido University, discussed results of the phase 2 STEMTRA trial.

Findings presented at the 2021 Transplant & Cellular Therapy Meetings indicate that patients with B-cell non-Hodgkin lymphoma may benefit from a type of natural killer immunotherapy added to chemotherapy and transplant.

Novartis and the Bill & Melinda Gates Foundation have entered a new agreement to discover and develop a single-administration gene therapy to cure sickle cell disease in countries with limited healthcare infrastructure.

Novartis is teaming up with the Gates Foundation to develop a gene therapy for sickle cell disease; the World Health Organization reports a drop in global cases of coronavirus disease 2019 (COVID-19); storms hinder vaccine rollout across the United States.

On this episode of Managed Care Cast, we highlight a new feature on AJMC.com, the website of The American Journal of Managed Care®, called Clinical Spotlight, where our editors provide a series of interviews with leading experts in certain specialties.

The suspension is due to recently reported diagnoses of AML and myelodysplastic syndrome in 2 treated patients.

No approved treatment for leber hereditary optic neuropathy is currently available in the United States.

CAR T-cell therapy has shown robust responses and curative potential in patients with diffuse large B-cell lymphoma, regardless of cytogenetics or age.

February 15, 2021 - Betibeglogene autotemcel, a one-time gene therapy, enabled durable transfusion independence in most patients with transfusion-dependent β-thalassemia who were treated across 4 clinical studies.

Both therapeutic programs utilize AAD2-GDNF gene therapy targeted to brain structures vulnerable to Parkinson disease and multiple system atrophy.

Descriptive findings from the phase 1/2 study allude to promise for the gene therapy in the rare pediatric disorder.

New phase 2/3 findings meet investigators' hypotheses on the gene therapy's mixed benefits for heparan sulfate reduction.

The AAV-based gene therapy was well-tolerated and showed enough promising effect to warrant further investigation, researchers reported.

Investigators observed sustained, dose-dependent benefits for young patients at 24 months.

Treatment with the first-generation precision cell therapy Orca-T led to a significant reduction in cases of graft-versus-host disease, an impressive GVHD relapse-free survival rate, a lack of treatment-related mortalities, while showcasing scalability potential.

Study examining the role of IL-17A in patients with diabetes.

The HSPC therapy showed fetal hemoglobin production in transfusion-dependent patients in a matter of months—with sustained results over 1 year.

Treatment with the investigative gene cell therapy betibeglogene autotemcel led to durable transfusion independence in 87% of pediatric patients less than 18 years of age with transfusion-dependent beta-thalassemia with a median average hemoglobin of 11.3 g/dL.

Investigators focus on biophysical method to study protein-protein interactions .

Responsiveness to treatment received immediately prior to CAR T-cell therapy may not be associated with post–CAR T outcomes in patients with relapsed/refractory diffuse large B-cell lymphoma who receive axicabtagene ciloleucel.

The investigators found the primary efficacy endpoints occurred in 20 evaluable patients (87%).

FLT201 is a combination of AAVS3 capsid and a liver-specific promotor to drive the expression of GCasevar85.