News

Topline data from the phase 3 REVEAL 1 study show VGX-3100 led to higher rates of high-grade squamous intraepithelial lesions regression and HPV-16 and/or HPV-18 clearance compared with placebo.

UniQure plans to file etranacogene dezaparvovec with the FDA for hemophilia B in March, pending a positive resolution to a clinical hold placed in December.

More than 2 years after treatment, some of the patients with relapsed/refractory multiple myeloma have yet to see a relapse. An FDA decision on the therapy is expected within a month.

High-dose chemotherapy and transplant remains the second-line standard of care for the majority of patients with relapsed/refractory diffuse large B-cell lymphoma; however, CAR T-cell therapy and other novel agents, which have transformed the third-line setting, may offer alternative and more personalized second-line options in the coming years.

Rocket Pharmaceuticals has summarized multiple major milestones for its 5 gene therapies from 2020 and provided a forecast and outlook for what is expected to be a banner year for the organization.

CD19-targeted CAR T-cell therapies have yielded durable remissions in approximately half of all patients with aggressive relapsed/refractory B-cell lymphomas.

Preliminary evidence pinpoints classical causes for AML in patient treated with LentiGlobin in a phase 1/2 study for sickle cell disease.

Treatment with chimeric antigen receptor T cells has induced unprecedented overall response rates and complete response rates in patients with relapsed/refractory B cell malignancies.

Since the publication of the pivotal ELIANA trial in pediatric patients with acute lymphoblastic leukemia, the field of CAR T-cell therapy has grown significantly and left providers better equipped to understand and manage treatment-related adverse effects, such as cytokine release syndrome.

Advancements in gene therapy approaches have opened the door for a glimpse into the future of care for a number of diseases.

February 24, 2021 - The CAR T-cell product KTE-X19 continues to demonstrate durable clinical benefit in patients with relapsed/refractory mantle cell lymphoma, with an overall response rate of 92%.

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.

Yi Lin, MD, PhD, discusses promising data with CAR T-cell therapy in multiple myeloma.

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.