News

Ultragenyx Pharmaceutical, Inc. released positive long-term safety and efficacy data from the first dose cohort of the Phase 1/2 study of DTX301. The investigational AAV gene therapy, is intended for the treatment of OTC deficiency.

In this interview, Dr. Frederick Locke discusses the promise of CAR T-cell therapy for lymphomas, and how this gene therapy could offer hope for patients who don't respond to standard treatments.

Ian W. Flinn, MD, PhD, discusses the impact of CAR T-cell therapy on the field of lymphoma and highlighted emerging options for the treatment of patients with MCL.

One month after Fibrocell submitted an Investigational New Drug application with the U.S. FDA for FCX-013, the application for the gene therapy to treat scleroderma was granted allowance.

Alexander Perl, MD, discusses the implementation of CAR T-cell therapy in ALL and other advances with this unique treatment across the spectrum of hematologic malignancies.

Researchers have identified a molecular target that could allow chimeric antigen receptor (CAR) T-cell therapy to be used in treating patients with glioblastoma. Although the heterogeneous expression of tumor-associated antigens limits the efficacy for CAR-redirected T cells for the treatment of glioblastoma, chondroitin sulfate proteoglycan 4 (CSPG4), a cell surface type 1 transmembrane protein, is highly expressed in a majority of glioblastoma specimens with limited heterogeneity.

The use of chimeric antigen receptor (CAR) T-cell therapies for the treatment of hematologic malignancies is still in its early stages, but when the FDA approved tisagenlecleucel and axicabtagene ciloleucel in 2017, this gave hope to oncologists and patients with some types of leukemia and lymphoma who have exhausted all other options.

A panel of experts discusses the current state of CAR T-cell therapies and sheds light on the future directions of this therapeutic approach.

Coverage of the Keynote Address from Patient-Centered Oncology Care, November 16-17, 2017.

Dr. Jeffrey Weber of NYU Langone Health’s Perlmutter Cancer Center and Dr. Hope Rugo of UCSF Helen Diller Comprehensive Cancer Center are among the faculty featured in Evidence-Based Oncology™, a publication of The American Journal of Managed Care®.

UW Carbone Cancer Center will begin treating adults with a “living drug” that employs their own immune cells to fight a common type of aggressive blood cancer.

CAR T- cell therapies are among the most expensive ever invented. For now, there’s a lot of uncertainty, as both government and commercial insurers, and a handful of the nation’s leading cancer centers, navigate a reimbursement structure that truly has no precedent.

CAR T-cell therapies tisagenlecleucel (Kymriah, Novartis) and axicabtagene ciloleucel (Yescarta, Kite Pharma/Gilead) may come with hefty price tags, but the cost-effectiveness of both therapies fell below or within commonly cited thresholds of $50,000 to $150,000 per quality-adjusted life years, according to a report by the Institute for Clinical and Economic Review.

The collaboration intends to combine AbbVie’s clinical resources in monoclonal antibody develop with Voyager’s gene therapy platform.

Maintenance therapy with pazopanib significantly prolonged progression-free survival in patients with extensive-disease small-cell lung cancer, but with toxicity.

The US FDA has granted Priority Review designation for tisagenlecleucel (Kymriah) for treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma who are ineligible for, or have relapsed after, ASCT.

Abeona Therapeutics, Inc. announced that the FDA has granted Orphan Drug Designation to its ABO-202 program for the treatment of infantile Batten disease.

Adjuvant therapy with sunitinib after nephrectomy was associated with increased mortality among older women with renal cell carcinoma, according to a subgroup analysis of data from the ASSURE trial.

Fibrocell Science has submitted an Investigational New Drug Application (IND) with the FDA for a gene therapy candidate to treat moderate to severe localized scleroderma.

Research aiming to elucidate the underlying factors for oxidative damage to the trabecular meshwork in glaucoma has led to studies of alterations in gene expression that may ultimately guide the development of genetic therapy for glaucoma, said Carla J. Siegfried, MD, recipient of the 2018 Shaffer Prize for Innovative Glaucoma Research.

Research aiming to elucidate the underlying factors for oxidative damage to the trabecular meshwork in glaucoma has led to studies of alterations in gene expression that may ultimately guide the development of genetic therapy for glaucoma, said Carla J. Siegfried, MD, recipient of the 2018 Shaffer Prize for Innovative Glaucoma Research.

A long-term follow-up analyzing the toxic effects and results from a phase 1 clinical trial of adult patients with relapsed B-cell acute lymphoblastic leukemia (ALL) who were treated with CD19-specific chimeric antigen receptor (CAR) T cells found patients with low disease burden had a longer medial overall survival and a lower incidence of toxicity.

From 2002 to 2015, CD4 cell counts at the start of combination antiretroviral therapy (cART) increased, and the proportion of individuals with severe immunodeficiency at the start of cART decreased among all income groups, according to a study published in Clinical Infectious Diseases.

A single infusion of the anti-CD19 chimeric antigen receptor T-cell therapy tisagenlecleucel produced durable remissions in pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic lymphoma.

The U.S. FDA has granted Capricor Therapeutics RMAT designation for its lead investigational cell therapy for the treatment of Duchenne muscular dystrophy, CAP-1002.

Fibrocell Science announced the submission of an Investigational New Drug Application with the U.S. FDA for FCX-013, a gene therapy candidate for the treatment of moderate to severe localized scleroderma.

Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).

Rare pediatric disease designation was granted by the FDA to MeiraGTx’s A002 (ZZV2/8-hCARp.hCNGB3) for achromatopsia.