Hematology

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.

Two gene therapy techniques in separate, concurrently published trials demonstrate clinical success in mitigating monogenic hemoglobinopathies.

New one-year data makes history as the first to indicate the superiority of a one-injection gene therapy versus Factor VIII prophylaxis.

Gene-editing therapy in sickle cell disease and advances in multiple myeloma dominated hematology news in 2020.

The UCSF hematologist discusses the growing portfolio for the sickle cell therapy after ASH 2020.

The historic phase 3 HOPE-B trial shows all but 1 treated patient discontinued prophylaxis after a single dose of etranacogene deaparvovec.

The second process of ARU-1801 in a new patient resulted in up to 4 times greater engraftment of transduced hematopoietic stem cells and progenitors at 6 months.

What started out as a journey to better understand regulatory T cells has now led to an intriguing approach with an investigational cell therapy designed to prevent the risk of graft-versus-host disease and to improve relapse-free survival rates in patients undergoing hematopoietic stem cell transplantation.

An off-the-shelf CAR T-cell therapy that targets B-cell maturation antigen, ALLO-715, elicited responses in heavily pretreated patients with relapsed/refractory multiple myeloma in early findings from a first-in-human study presented at the 2020 ASH Meeting.

Treatment with the BCMA-targeted CAR T-cell therapy idecabtagene vicleucel was found to yield clinically meaningful improvements in the quality-of-life of triple-class exposed patients with relapsed/refractory multiple myeloma.

December 6, 2020 - Odronextamab, is a novel CD20xCD3 bispecific antibody, continues to show intriguing antitumor activity and an acceptable safety profile in patients with relapsed/refractory B-cell non-Hodgkin lymphoma, including those who have previously received chimeric antigen receptor T-cell therapy.

The furthest-progressed gene-edited therapy for a genetic disease reported historic efficacy data in ongoing phase 1/2 trials.

The complexities associated with the disease and lack of validated risk predictors makes such a model challenging to implement.

In adolescent patients with sickle cell disease, hydroxycarbamide therapy was associated with improvements in neurocognition variables such as working memory, verbal memory, and nonverbal IQ, according to study findings.

The Institute for Clinical and Economic Review (ICER) published a draft evidence report on crizanlizumab (Adakveo), voxelotor (Oxbryta), and L-glutamine (Endari) for sickle cell disease, as well as a draft scoping document on valoctocogene roxaparvovec, an investigational gene therapy, and emicizumab (Helimbra) for hemophilia.

Adult patients with beta thalassemia will now have an FDA-approved treatment available with luspatercept-aamt (Reblozyl). The therapy treats the rare inherited blood disorder, which requires patients to have regular red blood cell transfusions.

The only thing that should matter in these patients is their genetic code, but often it’s your zip code that really restrains your ability to access the healthcare you deserve, explained Ahmar Zaidi, MD, pediatric hematologist-oncologist, Comprehensive Sickle Cell Center, Children's Hospital of Michigan, when discussing the possibility of a gene therapy for sickle cell.