American Society for Gene & Cell Therapy (ASGCT)

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.

Findings from an ongoing phase 1 trial were presented at the 2021 ASGCT meeting.

Early safety and efficacy data support the use of natural killer T (NKT) cells in patients with stage IV relapsed/refractory neuroblastoma.

The CEO of Avamab Pharma discussed how funding and research changes directions.

The CEO of Avamab Pharma discussed how the field of gene therapy has shifted due to the COVID-19 pandemic.

The head of neuroscience at Sangamo Therapeutics discussed advances in understanding disease biology that allow for more specific gene targeting.

The head of neuroscience at Sangamo Therapeutics discussed the potential of ZFPs in central nervous system disorders.

Flexion Therapeutics believes that local administration of a gene therapy may not only improve pain management but potentially slow disease progression.

Birth control guidance provided to women taking rheumatoid arthritis therapies is lacking, and other rheumatology news reported across MJH Life Sciences™.

No adverse effects on weight, general activity, or survival in knockout mice and wild type littermates were observed throughout the study.

The CEO of Locanabio shares details of 2 presentations the company is making this week at the 24th Annual Meeting of the American Society of Gene & Cell Therapy.

Following 2 unexpected serious adverse events, the trial was put on clinical hold as of February 2021.

The therapy was generally well-tolerated and offered substantial pain relief in patients.