Hematology

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.

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

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.

The suspension is due to recently reported diagnoses of AML and myelodysplastic syndrome in 2 treated patients.

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.

Treatment with the first-generation precision cell therapy Orca-T led to a significant reduction in cases of graft-versus-host disease, an impressive GVHD relapse-free survival rate, a lack of treatment-related mortalities, while showcasing scalability potential.

The HSPC therapy showed fetal hemoglobin production in transfusion-dependent patients in a matter of months—with sustained results over 1 year.

Treatment with the investigative gene cell therapy betibeglogene autotemcel led to durable transfusion independence in 87% of pediatric patients less than 18 years of age with transfusion-dependent beta-thalassemia with a median average hemoglobin of 11.3 g/dL.

Responsiveness to treatment received immediately prior to CAR T-cell therapy may not be associated with post–CAR T outcomes in patients with relapsed/refractory diffuse large B-cell lymphoma who receive axicabtagene ciloleucel.

Patients with refractory large B-cell lymphoma have less to gain from axicabtagene ciloleucel if they have never achieved a complete response to any line of prior therapy.

Data from the ongoing HGB-206 study reports reduced hemolysis, increased total hemoglobin, and a complete resolution of vaso-occlusive crises among patients.

February 5, 2021 - Following stem cell transplant or treatment with CAR T-cell therapies, patients with hematologic malignancies and coronavirus disease 2019 tend to have favorable outcomes, especially if they are diagnosed in complete remission and further out from their cell infusion.