News

The FDA has granted Rare Pediatric Disease Designation to Myonexus Therapeutics for its MYO-101, which is an AAV-based gene therapy for the treatment of limb girdle muscular dystrophy (LGMD) type 2E.

CMS's initial payment codes for CAR T-cell therapies are opposed by the medical community on the basis that they would be cumbersome to implement and wouldn't reflect the full amount of care delivered to each patient.

The US Food and Drug Administration (FDA) has accepted the Bioverativ's Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease.

The investigational new drug application allows the initiation of phase 1/2 clinical trial to assess the safety of BIVV003 in adults.

Agilis Biotherapeutics, Inc. has announced that the company's gene therapy for AADC deficiency results in de novo dopamine production and supports durable Improvement in major motor milestones.

New data being presented at ASGCT detail patients (1.67 to 8.42 years of age) enrolled in a study evaluating the investigational gene therapy treatment, AGIL-AADC.

The first patient has been dosed in aphase 1/2 study (BMN 270-203) evaluating the investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.

A new case study found that an acute myeloid leukemia patient has remained cancer free for 9 months following treatment with the chimeric antigen receptor (CAR) T-cell treatment, CYAD-01, and a bone marrow transplant.

The approval includes adults with R/R DLBCL after two or more lines of prior systemic therapy, high-grade B-cell lymphoma, and DLBCL arising from FL.

A first-time study of chimeric antigen receptor T-cell therapy in youths with Down syndrome-associated relapsed/refractory (r/r) acute lymphoblastic leukemia produced high remission rates and toxicity results that were similar to those observed in patients with r/r ALL.

Results from a recent study may show why some patients with chronic lymphocytic leukemia are resistant to tisagenlecleucel, while potentially offering a pathway to enhance patient response.

Orchard Therapeutics announced that its gene therapy candidate, OTL-200, has been granted Rare Pediatric Disease designation for the treatment of metachromatic leukodystrophy.

CAR T-cell therapy can induce next generation sequencing negativity in patients with relapsed/refractory acute lymphoblastic leukemia, suggesting a "synergistic" relationship with hematopoietic cell transplant.

Tisagenlecleucel, sold as Kymriah, has gained its second indication following the FDA's approval of the chimeric antigen receptor T-cell therapy for the treatment of adult patients with relapsed or refractory large B-cell lymphoma, the most common form of non-Hodgkin lymphoma.

REGENXBIO Inc. announced that the U.S. FDA has granted Fast Track designation to RGX-131, a novel, one-time investigational treatment for mucopolysaccharidosis type II (MPS II).

The chimeric antigen receptor T-cell therapy tisagenlecleucel has been approved for a second type of blood cancer; the National Institutes of Health has started recruiting individuals for a database that will include data on more than 1 million people; Kansas’ request to impose a 3-year lifetime limit on Medicaid benefits is testing just how open the Trump administration is to allowing states flexibility.

The FDA has approved tisagenlecleucel (Kymriah) for use in adult patients with relapsed/refractory large B-cell lymphoma—including diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma—after 2 or more lines of systemic therapy.

Alectinib offered significantly improved outcomes over standard chemotherapy in patients with ALK-positive non–small-cell lung cancer who had progressed on crizotinib therapy.

A fully automated artificial intelligence-based system could effectively classify function and potency of cell therapy.

Keith Stewart, MB, ChB, discusses the role of stem cell transplant, minimal residual disease testing, and maintenance therapy in the treatment of patients with multiple myeloma.

Children receiving AVXS-101 have shown such improvement that researchers are calling for an update to the CHOP-INTEND scale used to track their progress.

Maung Myo Htut, MD, discusses the use of CAR T-cell therapy in multiple myeloma and the potential infusion of immunotherapy into treatment.

Drug company bluebird bio, Inc, has announced the publication of positive interim data for its gene therapy to eliminate or reduce chronic blood transfusions in patients with transfusion-dependent β-thalassemia.

Frederick L. Locke, MD, discusses the ongoing ZUMA-2 trial investigating axi-cel for patients with mantle cell lymphoma.

The U.S. FDA has cleared the Investigational New Drug application for DTX401 for the treatment of glycogen storage disease type Ia (GSDIa).

City of Hope is currently the only institution in Southern California to offer both FDA-approved CAR T-cell therapies.

With chimeric antigen receptor (CAR) T-cell therapy being so new, there is going to be a learning curve as providers become more educated about the treatments, the manufacturing process, and the toxicities, Houston Holmes, MD, MBA, FACP, a medical oncologist with Texas Oncology, explained at the Community Oncology Alliance’s (COA) 2018 Community Oncology Conference.

Reimbursement models, novel gene therapy–based treatments, and price transparency: these were some of the topics covered by presenters and panelists at the annual meeting of the Community Oncology Alliance.

C. Ola Landgren, MD, PhD, shares his insight on CAR T-cell therapy, sequencing, and triplet and quadruplet regimens in myeloma.

A new class of injectable monoclonal antibodies directed against calcitonin gene related peptide (CGRP) or its receptor may offer hope to migraineurs who have not found effective prophylactic therapy.