Latest Conference Coverage

The assistant professor of oncology at UNC School of Medicine discussed the first-in-human study being conducted with CT-0508.

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

The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.

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.

One patient with metastatic pancreatic cancer remains alive on maintenance chemotherapy 3 years after treatment.

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.

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.

Investigators presented up to 2 years of post-marketing data on the first-ever gene therapy.

Updated data at ARVO show that AGTC-501 had sustained efficacy and safety at 18 months for patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Veeral S. Sheth, MD, MBA, Director of Clinical Research at the University of Retina and Macula Associates, discusses advanced therapies for retinal diseases.

New data from cohort 4 showed outer retinal restoration following treatment with OpRegen.

The post-hoc analysis of the OPTIC trial explored the effects of neutralizing antibodies on patients treated with ADVM-002.

There was a continued trend toward improvement of overall survival with omidubicel at 73% compared with UCBT at 60%.

The analysis of data from ZUMA-7 demonstrated significant efficacy for axi-cel over second-line standard treatment.

Tisa-cel was associated with a more favorable safety profile in the analysis.

In this population, median progression-free survival was nearly 40 months.

A phase 1/2 multicenter study is underway following MB-106’s IND approval.