News

Review top news and interview highlights from the week ending November 17, 2023.

The CAR-T previously was cleared for separate trials in systemic lupus erythematosus, myositis, and systemic sclerosis.

The chairman, chief executive officer, and cofounder of Ocugen discussed the recent data update on the company’s OCU400 program in inherited retinal diseases.

The professor of medicine at University of California San Diego discussed promising preclinical findings she presented at the American Heart Association’s Scientific Sessions 2023.

CGTLive takes a look at MPSIII therapies in the pipeline for World Sanfilippo Awareness Day on November 16.

Vertex and CRISPR’s Exa-cel, which remains an investigational treatment in the United States, is the first CRISPR-based gene therapy to be approved in the UK, where it will be marketed as Casgevy.

The company most recently reported data from a trial in retinitis pigmentosa and Leber congenital amaurosis demonstrating OCU400’s gene-agnostic mechanism of action.

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

For World Cord Blood Day, the chairman and president of StemCyte discussed his thoughts on the importance of cord blood stem cells for the healthcare community.

The medical oncologist at Moffitt Cancer Center spoke about A2 Bio’s A2B530 as part of a larger discussion of novel cell therapy approaches in solid tumors.

Data on zilebesiran from the KARDIA-2 trial, in combination with other hypertension medicines, are expected in 2024.

The postdoctoral research fellow at Cedars Sinai Medical Center discussed preclinical research he presented at the American Heart Association’s Scientific Sessions 2023.

The therapy is already being evaluated in patients with lupus nephritis and diffuse cutaneous systemic sclerosis.

The director of the Mass General Brigham Gene and Cell Therapy Institute discussed the history and future of gene therapy in cardiology at the American Heart Association’s Scientific Sessions 2023.

One serious AE of myocardial infarction may have been related to VERVE-101 therapy while other AEs of fatal cardiac arrest and tachycardia were determined to be unrelated to the therapy.

Milind Desai, MD, MBA, an investigator on the MyPeak-1 trial and the director of the Hypertrophic Cardiomyopathy Center and the vice chair of the Heart Vascular Thoracic Institute at the Cleveland Clinic, discussed Tenaya Therapeutics’ TN-201.

The trial will follow a phase 1/2/3 design and will evaluate GS-100 in pediatric patients with NGLY1 deficiency.

AVB-101 was simultaneously granted fast track designation by the agency. The company plans initiate US activities in 2024.

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed unmet needs that remain, chiefly concerning widening the criteria for therapies and trials.

The median progression-free survival was 11 months and the SCG101 was well-tolerated.

Ben Creelan, MD, a medical oncologist at Moffitt Cancer Center, discussed several different approaches to cell therapy that could lead to new treatment options in the field.

Among 20 patients who received MDR-101, 12 have completed participation in the trial and have not taken their immunosuppression therapy for 2 years.

The therapy was previously granted priority review in May 2023.

The medical oncologist at Moffitt Cancer Center discussed 3 different approaches to cell therapy that could lead to new treatment options in the field.

Microsatellite instability (MSI) tumors are frequently found in people with Lynch syndrome, one of the most common hereditary colorectal cancers.

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

BRL Medicine is also enrolling a phase 1/2 multicenter trial evaluating BRL-201 at lower dose ranges.

The chief scientific officer of CureDuchenne discussed research he is looking forward to seeing in 2024.

The BASECAMP-1 screening study has now identified 28 patients whose solid tumors are positive for HLA-A*02 LOH.

David Rawlings, MD, the director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute, discussed findings from several preclinical studies that could help push engineered B-cell therapies towards clinical trials.