Hematology

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

The planned clinical trial of EB103 has received clearance to include patients with HIV-associated lymphoma and primary and secondary central nervous system lymphoma.

In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news in gene therapies for hemophilia—one of the most common of these disorders.

Mesoblast’s allogeneic off-the-shelf therapy showed long-term benefit in supporting data among children with steroid-refractory acute graft versus host disease. The FDA has set a PDUFA action date of August 2, 2023, for its review.

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

As investigational cell therapies influx the clinical trial landscape, especially for oncologic indications, some companies are ceding the race to their competitors.

The extension follows the submission of additional phase 3 data from the GENEr8-1 clinical trial that constitutes a major amendment to the biologics license application. The new deadline for review is June 30, 2023.

The company is back to the drawing board after discontinuing its last clinical-stage program at the end of 2022.

The company has halted phase 3 plans for BIVV003 in favor of therapies for Fabry and immune rejection in kidney transplantation.

Review top news and interview highlights from the week ending March 3, 2023.

The associate professor of medicine at Sylvester Comprehensive Cancer Center discussed updated data from the phase 3 ALLELE study.

Intellia is also seeking an IND for phase 2 US sites of its other lead candidate, NTLA-2001 for ATTR amyloidosis.

The vice chair for cellular therapies at Roswell Park Comprehensive Cancer Center discussed new research from the center presented at the 2023 Tandem Meetings.

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

The MD, PhD candidate at Baylor College of Medicine discussed findings from a new study presented at the 2023 Tandem Meetings.

CGTLive takes a look at some upcoming FDA decisions for Rare Disease Day.

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%.