Hematology

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

The director of the Stem Cell Transplant & Cellular Therapy Program at St. Louis Children’s Hospital discussed when the choice should be made to transition from symptom management to curative therapies.

Val-rox was approved as Roctavian in June 2023.

The assistant professor at Mayo Clinic School of Medicine discussed plans for further research and a phase 2/3 study.

Judy Lieberman, MD, PhD, the endowed chair in cellular and molecular medicine at Boston Children’s Hospital, discussed how there is still much room for growth for RNA therapeutics despite progress so far.

Vivien Sheehan, MD, PhD, an associate professor of pediatrics at Emory University, discussed how patients should be able to choose to try the recently FDA-approved cell-based therapies for SCD when they are ready.

Review top news and interview highlights from the week ending March 29, 2024.

The clinical investigator in the Department of Bone Marrow Transplantation & Cellular Therapy at St. Jude Children’s Research Hospital discussed several areas of interest for research now that CAR-T is here to stay.

Judy Lieberman, MD, PhD, the endowed chair in cellular and molecular medicine at Boston Children’s Hospital, discussed her research on siRNA technology.

The transplant physician at St. David's South Austin Medical Center of the Sarah Cannon Transplant and Cell Therapy Network discussed a real-world study comparing bendamustine against fludarabine and cyclophosphamide.

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

The company noted that it plans to submit the BLA in the second quarter of 2024.

The associate professor of pediatrics at Emory University also discussed the need to empower patients and families to make their own treatment decisions.

In March 2024, the FDA approved the CAR T-cell therapy lisocabtagene maraleucel (Breyanzi) as a treatment for relapsed or resistant chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).

John Ligon, MD, an assistant professor in the department of pediatrics at the University of Florida College of Medicine, discussed his view of the main priorities for research in this area.

Review top news and interview highlights from the week ending March 22, 2024.

The endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed the rapid advancements in RNA-based treatments in the past 2 decades and potential advancements that remain on the horizon.

The professor from Cedars Sinai discussed how the cell therapy stands to revolutionize the treatment landscape.

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

Many surveyed participants were content with current treatments or wary of gene therapy’s adverse events and durability.

John Ligon, MD, an assistant professor in the department of pediatrics at the University of Florida College of Medicine, discussed his team’s early findings on CAR-T and fertility from CIBMTR centers.

Cita-cel, approved as Carvykti, is currently under review for an indication expansion as an earlier line of therapy, which the FDA will decide on by April 5, 2024.

Review top news and interview highlights from the week ending March 15, 2024.

Breyanzi was approved for the expanded indication under the accelerated approval pathway.

bluebird bio stated that it is working on additional agreements with more than 15 more Medicaid agencies.