Hematology

Review top news and interview highlights from the week ending April 26, 2024.

The decision was supported by the phase 3 BENEGENE-2 trial, which suggested that the Pfizer gene therapy was superior to standard of care FIX prophylaxis.

The medical director of the Center for Inherited Blood Disorders discussed the entrance of BioMarin’s val-rox into his center’s practice.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The EC’s decision comes several weeks after the FDA approved cilta-cel for a similar expanded indication.

The Assistant Professor of Medicine at Weill Cornell Medical College discussed the changing cell transplant landscape.

The principal investigator at the Gene Therapy Program of Dana-Farber/Boston Children’s Cancer and Blood Disorder Center discussed innovative uses of gene editing technology.

The attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital also discussed hurdles to accessibility for SCD gene therapy.

Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.

The medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center discussed results from a phase 1b trial for allo-HCT alternative Orca-T that he presented at Tandem’s 2024 conference.

Review top news and interview highlights from the week ending April 19, 2024.

The FDA has requested that black box warnings related to secondary cancer risks be added to all 6 CAR-T therapies currently on the market.

The infectious diseases specialist at Memorial Sloan Kettering Cancer Center discussed the session she chaired for the Infectious Diseases Track at Tandem’s 2024 Meeting.

For World Hemophilia Day, CGTLive takes a look at how the hemophilia field has adapted to the introduction of gene therapy to the treatment landscape.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Aimee C. Talleur, MD, a physician at St. Jude Children’s Research Hospital, discussed obstacles to understanding late effects of CAR-T.

In light of the IND clearance, Tr1X anticipates initiating a phase 1 clinical trial within the second quarter of this year.

The attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital discussed recently approved and still-upcoming gene therapy options for SCD.

Amar Kelkar, MD, a stem cell transplantation physician at the Dana-Farber Cancer Institute, discussed his research on the value of advanced therapeutics.

The director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine discussed novel innovations that lie on the horizon.

Review top news and interview highlights from the week ending April 12, 2024.

Shalini Shenoy, MD, MBBS, discussed when the choice should be made to transition from symptom management to curative therapies.

The best response was very good partial response in 3 of 5 participants.

The associate professor in the Translational Science and Therapeutics Division at Fred Hutch Cancer Center discussed her lab’s research on making TCR T-cell therapies more effective.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The stem cell transplantation physician at the Dana-Farber Cancer Institute discussed how to look at the value therapies provide and how to improve the cost-effectiveness of their use.

The director of Cell Therapy and Transplant at Penn Medicine discussed his outlook on CAR T-cell therapy research and the future.

Haydar Frangoul, MD, discussed advantages and disadvantages of haploidentical bone marrow transplant and the 2 new gene therapies for SCD.

Nirav Shah, MD, an associate professor of medicine at Medical College of Wisconsin, discussed LV20.19, a bispecific CD19/CD20-targeted CAR-T being evaluated for chronic lymphocytic leukemia and Richter’s transformation.

The FDA’s decision was based on data from the phase 3 CARTITUDE-4 clinical trial (NCT04181827).