Hematology

The FDA’s decision to approve this first gene therapy in hemophilia A was based on data from the phase 3 GENEr8-1 clinical trial.

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

Updated data are from a total of 36 patients treated in a phase 1/2 study as of June 2023.

In addition to the BLA acceptance, Pfizer noted that a European MAA for the gene therapy has been accepted by the EMA.

Susan Bal, MD, assistant professor of medicine at University of Alabama – Birmingham, discussed new data on BMS-986393.

Among 4 patients who were treated at the highest dose level evaluated using a manufacturing process referred to as Process A, all 4 patients achieved responses.

Jason Westin, MD, FASCP, the director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center, discussed factors that limit patient access to treatment and a potential solution.

The new data from the TRANSCEND FL and TRANSCEND NHL 001 studies are the latest in a series of positive updates regarding liso-cel that have been announced by Bristol Myers Squibb in recent months.

The director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center discussed axi-cel's safety profile and an important factor for improving access to CAR-T therapies.

Review top news and interview highlights from the week ending June 23, 2023.

The associate professor from Dana Farber Cancer Center and Harvard Medical School discussed continued efficacy and safety data presented at the 2023 EHA Meeting.

Jason Westin, MD, FASCP, the director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center, discussed the implications of data he presented at ASCO’s 2023 conference.

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

The assistant professor in the Division of Lymphoma at City of Hope discussed the potential implications of real-world data she presented at ASCO’s 2023 conference.

The company believes that limitations on bridging therapies were a contributing factor to the death.

The director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center discussed recent and ongoing studies that are upending the paradigm in LBCL care.

More work remains to be done to improve accessibility to populations and countries that have the highest unmet need with SCD treatment.

The assistant professor of medicine at University of Alabama – Birmingham discussed efficacy findings on BMS-986983 and more research to be done.

Two of 6 patients in the WM cohort achieved a complete response and 16 of 20 patients in the FL cohort achieved a complete response.

Review top news and interview highlights from the week ending June 16, 2023.

The assistant professor of medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy at Stanford University discussed potential methods for optimizing CAR-T efficacy that could be explored in the future.

The phase 1/2 Euplagia-1 trial is currently ongoing in Europe.

The assistant professor of medicine at University of Alabama – Birmingham discussed new data from the first-in-human trial of the CAR T-cell therapy.

New data from 3 patients were published in the New England Journal of Medicine.

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

The assistant professor of medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy at Stanford University discussed the safety and efficacy results he presented at EHA’s 2023 congress.

Seattle Children’s and 2seventy bio, which are collaborating on the development of SC-DARIC33, are currently investigating the cause of the grade 5 serious adverse event.

The associate professor at Medical College of Wisconsin discussed new findings from the CARTITUDE-4 study.

The objective response rate among patients treated at DL2 and DL3 who were evaluable for efficacy was 57%.

The assistant professor in the Division of Hematologic Malignancies and Cellular Therapeutics at the University of Kansas Medical Center discussed real-world safety outcomes for brexu-cel in patients with r/r MCL.