Hematology

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Betibeglogene autotemcel is being withdrawn from the German market following a breakdown in reimbursement conversations, which has resulted in bluebird reducing and reshaping its European workforce.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

RP-L301 demonstrated improvements in hemoglobin at 3- and 6-months for the first 2 patients treated in a phase 1 study for pyruvate kinase deficiency.

AB-205 showed an encouraging safety profile along with a robust ability to eliminate oral and gastrointestinal severe regimen-related toxicities in patients with systemic lymphoma who were undergoing high-dose therapy and autologous hematopoietic stem cell transplantation.

Omidubicel significantly improved median time to neutrophil engraftment compared with standard umbilical cord blood transplantation in patients with hematologic malignancies.

Axicabtagene ciloleucel elicited a high objective response rate of 85%, with a complete response rate of 74% when used as a first-line therapy in patients with high-risk large B-cell lymphoma.

A risk-adapted CD19 CAR T-cell therapy dosing approach allowed pediatric patients with relapsed/refractory acute lymphocytic leukemia to maintain a high rate of remission, while reducing the risk of severe toxicities among those with a high initial disease burden

The lentiviral vector used to create LentiGlobin was deemed a very unlikely cause of AML in a patient with sickle cell disease.

The FDA has granted an RMAT designation to RP-L201 as a potential treatment for patients with leukocyte adhesion deficiency-I.

The FDA has granted an RMAT designation to valoctocogene roxaparvovec for hemophilia A.

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.

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.