Hematology

Tisagenlecleucel led to high complete response rates that appear durable for patients with follicular lymphoma, prompting a regulatory submission later this year.

The FDA has lifted clinical holds placed on several BB305-based gene therapy programs from bluebird bio.

Results of a phase 1/2 trial show the feasibility of an approach targeting CLL1 in pediatric patients with relapsed or refractory acute myeloid leukemia.

Ciltacabtagene autoleucel elicited a response in nearly all patients with relapsed/refractory multiple myeloma previously treated with 1 to 3 prior treatments.

A single infusion of KTE-X19, a CAR T-cell therapy, demonstrated robust and durable responses in heavily pretreated patients with relapsed/refractory B-cell acute lymphoblastic leukemia.

The CAR T-cell therapy idecabtagene vicleucel continues to demonstrate improved survival among heavily pretreated patients with relapsed/refractory multiple myeloma.

An off-the-shelf natural killer cell therapy demonstrated high response rates and limited toxicity for patients with relapsed/refractory B-cell lymphoma.

Ross Macdonald, PhD, managing director and CEO of Cynata Therapeutics, discusses the biotech company's approach to overcoming reproducibility challenges with mesenchymal stem cells.

Maria A. Croyle, PhD, discusses the development of a novel film matrix that can safely and effectively store and transport AAV-based gene therapies.

A discussion of the course of gene therapy compared with stem cell therapy.

Idecabtagene vicleucel elicited a median overall survival of 24.8 months with a 51% event-free rate at 24 months in patients with heavily pretreated, relapsed/refractory multiple myeloma.

Maria A. Croyle, PhD, discusses the manufacturing challenges facing the gene therapy space and her inspiration for developing a novel preservation method for viral vector-based therapies.

FIX activity was similar in participants without and with pre-existing neutralizing antibodies.

Following 2 unexpected serious adverse events, the trial was put on clinical hold as of February 2021.

Researchers observed 20-25% frequencies of this 4.9 kb deletion in HSPCs prior to xenotransplantation and at 17 weeks post-engraftment.

Early results from the MOMENTUM study show that treated patients experienced fewer vaso-occlusive episodes and related hospital admissions.

The FDA has lifted a clinical hold on the development of etranacogene dezaparvovec for patients with hemophilia B.

After a more than 35-year pursuit, the licensing of a gene therapy product for treating hemophilia may be available within 2 years.

Vertex Pharmaceuticals will take the lead on worldwide development, manufacturing, and commercializing of CTX001, with payments and split economics to CRISPR Therapeutics.

Betibeglogene autotemcel is being withdrawn from the German market following a breakdown in reimbursement conversations, which has resulted in bluebird reducing and reshaping its European workforce.

In a phase 1 study of the innate cell engager AFM13 pre-complexed with NK cells, all 4 patients with CD30-positive, relapsed/refractory Hodgkin lymphoma treated with the therapy achieved at least a partial response.

RP-L201 continued to show promising preliminary efficacy for patients with severe LAD-I after additional follow-up in a phase 1/2 study.

A budget impact analysis suggest the burgeoning drug class may not be as available as it could be for sickle cell patients.

Etranacogene dezaparvovec was "highly unlikely" to be the cause of hepatocellular carcinoma in a patient with hemophilia B enrolled in the phase 3 HOPE-B trial, according to an independent investigation.

The FDA has approved idecabtagene vicleucel as the first BCMA-directed CAR T-cell therapy for patients with relapsed/refractory multiple myeloma after 4 or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.