News

The investigator-initiated, single-arm, open-label trial will follow 20 patients with high-risk LBCL for 2 years, with a primary outcome measure of complete response rate per Lugano classification.

Catch up with some of CGTLive’s top news articles from the annual WORLDSymposium.

In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest expert insights in gene therapies for hemophilia, which is among the most common of these disorders.

GenSight Biologics reported that 73% of patients treated bilaterally with Lumevoq improved at least +15 ETDRS letters relative to their worst recorded BCVA.

The full dataset, published in the New England Journal of Medicine, reveals the newly approved hemophilia B gene therapy reduced annualized bleeding rates and improved the need for factor IX infusions.

Matthew B. Harms, MD, associate professor of neurology at Columbia University, and medical consultant and care center director at the MDA, discussed the session he will be chairing at the conference.

Catch up with some of CGTLive’s top conversations from the annual ASTCT and CIBMTR meeting.

Alison Betof Warner, MD, PhD, assistant attending physician, Memorial Sloan Kettering Cancer Center, discussed the solid tumor session she chaired at the 2023 Tandem Meetings.

Serious adverse events of reduced visual acuity and eye inflammation were observed in 2 clinical trials.

The study of VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy, is already ongoing in Canada. It is expected to kick off in the United States in the first half of 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.

The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed working with the FDA toward a first disease-modifying therapy approval in Duchenne.

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.

Samiah Al-Zaidy, MD, vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the latest results from the Imagine-01 clinical trial evaluating the investigational treatment PBGM01.

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

Three patients treated with Genethon’s GNT-003 were able to cease treatment with standard of care phototherapy for at least 1 year.

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 associate professor of neurology at Columbia University spoke about the session he will be chairing at MDA’s 2023 conference.

NR082 is being evaluated in a phase 1/2/3 clinical trial in patients with Leber hereditary optical neuropathy.

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

SNK01 is primarily being assessed in combination and as a monotherapy for treating solid tumors.

Competitors REGENXBIO and Capricor Therapeutics both have gene therapy candidates in clinical trials.

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

AskBio’s AB-1003 was previously granted fast track designation by the FDA.

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