Category: hematology

MD Anderson researchers find mechanisms behind CAR-T cells’ efficacy, toxicity

axicabtagene ciloleucel, BioPharma, biopharma nl, cancer, CAR-T, cell therapy, Daily, Gilead Sciences, hematology, Houston, lymphoma, MD Anderson Cancer Center, Oncology, SYN, Texas, Top Story, Yescarta / October 5, 2020 October 5, 2020

The findings may make it possible to identify a subset of patients who experience poorer outcomes or more serious side effects, especially neurotoxicity.

FDA rejects BioMarin’s hemophilia A gene therapy in ‘massive surprise’

BioMarin Pharmaceutical, BioPharma, biopharma nl, Complete Response Letter, CRL, Daily, FDA, Food and Drug Administration, gene therapy, hematology, hemophilia A, SYN, Top Story, valoctocogene roxaparvovec / August 19, 2020 August 19, 2020

In a CRL issued for valoctocogene roxaparvovec, the FDA told BioMarin that differences between its Phase I/II and Phase III studies limited the ability to rely on the earlier trial to support durability of effect, which an analyst said likely referred to a product comparability issue.

Novartis plans to seek approval for CAR-T in slow-growing lymphoma after Phase II success

Basel, BioPharma, biopharma nl, CAR-T, cell therapy, Daily, Follicular Lymphoma, hematology, Kymriah, Novartis, Oncology, Switzerland, SYN, tisagenlecleucel, Top Story / August 5, 2020 August 5, 2020

The company said the study of Kymriah in follicular lymphoma met its primary endpoint, though it did not disclose the trial data. It plans to submit approval applications to the FDA and EMA next year.

FDA approves MorphoSys, Incyte’s drug for vulnerable lymphoma population

BioPharma, biopharma nl, CD19, Daily, Delaware, diffuse large B-cell lymphoma, Germany, hematology, Incyte, Monjuvi, MorphoSys, Munich, Oncology, SYN, tafasitamab, Top Story, Wilmington / August 3, 2020 August 3, 2020

The FDA approved Monjuvi for second-line diffuse large B-cell lymphoma. Though the drug has the same antigen target as approved CAR-T cell therapies, it is designed for patients unable to tolerate such intensive treatments.

“When Blood Breaks Down”: It Can Break Your Heart

Book Review, Chadi Nabhan, hematology, Medical Practice, Mikkael Sekeres, Oncology, Physicians, When Blood Breaks Down / July 2, 2020 July 2, 2020

By CHADI NABHAN, MD, MBA, FACP “The goal for me and for my clinical and research colleagues is to put ourselves out of a job as quickly as possible”. This is how Mikkael Sekeres ends his book “When Blood Breaks Down” based on true stories of patients with leukemia. I share Mikkael’s sentiments and have …

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