Hematology

In observance of Rare Disease Day, held annually on February 28, catch up on some of the latest data updates from clinical trials for rare diseases.

The assistant professor of BMT and cellular therapy discussed updated safety and efficacy data on CD22 CAR T-cell therapy.

Review top news and interview highlights from the week ending February 24, 2023.

The announcement of a “strategic realignment” comes less than 2 months after the company voluntarily paused the CEDAR clinical trial due to a serious, nula-cel-related adverse event.

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

The assistant professor of medicine and pediatrics at Stanford University discussed updated data from 2 studies of Orca-T cell therapy.

The gene therapy was approved in the US in the same indication in November 2022.

The assistant professor of medicine at Vanderbilt University Medical Center discussed results from a retrospective analysis presented at the 2023 Tandem Meetings.

It was found that ctDNA levels at 28 days post-treatment were significantly higher for patients who ultimately progressed by 90 days post-treatment.

The assistant professor of medicine, bone marrow transplantation and cellular therapy, Stanford, discussed favorable data from a real-world experience study.

The hematologist/oncologist at Cincinnati Children’s discussed unfavorable outcomes with tisa-cel reinfusion in patients with B-cell acute lymphocytic leukemia.

Median PFS was 13.3 months in the ide-cel arm, compared to 4.4 months for the SOC arm.

Patients with large B-cell lymphoma had an ORR of 68% and a CR rate of 53%.

Among the 14 patients with relapsed/refractory mantle cell lymphoma who were treated with LV20.19 CAR in the trial, the overall response at 28 days post-treatment was 100%.

Long-term hematologic stability has been observed in at least 6 out of 10 patients with at least 12 months of follow-up.

The associate professor of medicine at Medical College of Wisconsin discussed trials assessing the dual, CD19/CD20-targeting CAR T therapy.

Among 43 patients who had EBV+ PTLD following allogeneic hematopoietic cell transplant or solid organ transplant, the overall response rate was 51.2%.

Neither patient treated with RP-L301 required RBC transfusions at any point post-engraftment.

Antiviral responses increased until peaking at week 24.

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

A third price model to be tested will limit the price of generic drugs for chronic conditions to $2 under Medicare plan D.

CT103A recently demonstrated safety in a phase 1 trial treating participants with NMOSD.

Progression free survival and overall response rate were higher in patients with R/R MM treated with Abecma than those treated with standard of care.

Review top news and interview highlights from the week ending February 10, 2023.

The updated data included a median overall survival of 26 months after treatment with brexu-cel.