News

The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.

Motor and pulmonary function have stabilized or improved in participants treated with SGT-001.

Findings from a preclinical study, which are in contrast with the current biphasic recovery model, have implications for both HSC transplantation and gene therapy.

Investigators from Seattle Children’s Hospital found that engineered T-cell regulatory products demonstrated significant suppression of effector T cells.

The trial met its primary efficacy end point, and participants reported improvements in quality-of-life factors such as outlook on the future and feelings about having hemophilia.

The interim study results presented at ASGCT 2022 likely represent the longest follow-up data to date for the largest cohort of infants with XSCID treated with lentiviral vector gene therapy.

Safety and efficacy data suggest this to be a feasible and clinically meaningful treatment for a patient population with high disease burden.

Catch up on the latest news and announcements in cell and gene therapies presented at the annual ASGCT meeting.

Multiple insertions were observed in affected participants, including insertions in the MECOM and PRDM16 genes.

Data from 2 patients dosed in the Imagine-1 clinical trial were presented in a late breaking session at ASGCT 2022.

Investigators are evaluating the safety, tolerability and preliminary efficacy of CT-0508 in patients with solid tumors and HER2 overexpression.

Investigators found a dose-dependent increase in CAART cell persistence.

Trial sponsors Pfizer, Sarepta, Genethon, and Solid Biosciences organized a group of experts to investigate shared serious AEs.

Investigators in Ukraine share data on the safety and efficacy of autologous mesenchymal stem cell administration for type 2 diabetes complications under the hypothesis that success is dependent on the administration route and quality of the autologous MSC population.

In a late-breaking presentation at ASGCT 2022, investigators shared preclinical results of a gene-editing strategy that seeks to reactivate developmentally silenced fetal hemoglobin (HbF, α2γ2) in order to replace defective sickle hemoglobin (HbS, α2βS2).

Study sponsor Pfizer recently announced the launch of a phase 3 trial in patients with Duchenne muscular dystrophy.

Interim data from the phase 2 Skyline trial demonstrated a 62.5% response rate.

Further data from Mustang Bio’s phase 1/2 trial will be presented at the EHA 2022 Congress.

Principal investigator Barry Greenberg, MD, FHFSA, shares extended results from the phase 1 trial and also preliminary pediatric safety results in a presentation at ASGCT 2022.

The 3 patients in cohort 1 have exhibited clinically meaningful improvements and no safety concerns. Follow-up for the 5 patients in cohort 2 is ongoing.

At ASCGT 2022, Italian investigators report updated safety and efficacy data from a phase 1/2 trial, in which all evaluable subjects achieved platelet transfusion independence and reported improved median platelet counts.

Review top news and interview highlights from the week ending May 13, 2022.

The locally injected gene therapy product is the subject of an ongoing phase 2a clinical trial.

The investigational therapy is also currently being evaluated in a clinical trial for sickle cell disease.

Caribou will present the initial data at the upcoming European Hematology Association (EHA) meeting in June.

Late-breaking interim data were presented at the 14th Myasthenia Gravis Foundation of America (MGFA) International Conference on Myasthenia and Related Disorders.

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

The cell therapy is the first to come out of the company’s proprietary ARC-SparX platform.