A phase I randomized, double-blinded, placebo-controlled study assessing the safety and pharmacokinetics of RIPK1 inhibitor GFH312 in healthy subjects.

Receptor-interacting protein kinase 1 (RIPK1) mediates necroptosis and inflammation in various pathophysiologies, emerging as a pharmacological target for neurodegenerative and inflammatory indications. This phase I, first-in-human, placebo-controlled study evaluated the safety, pharmacokinetics (PKs), and pharmacodynamics (PDs) of GFH312, an RIPK1 inhibitor, in healthy adults. Subjects received GFH312 as a single ascending dose up to 500 mg (part I) or once-daily repeated doses up to 200 mg for 14 days (part II).

Publisher Correction: Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancer.

An amendment to this paper has been published and can be accessed via a link at the top of the paper.

Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancer.

Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 editing of immune checkpoint genes could improve the efficacy of T cell therapy, but the first necessary undertaking is to understand the safety and feasibility. Here, we report results from a first-in-human phase I clinical trial of CRISPR-Cas9 PD-1-edited T cells in patients with advanced non-small-cell lung cancer (ClinicalTrials.gov NCT02793856).

Three-year outcomes of individualized ranibizumab treatment in patients with diabetic macular edema: the RESTORE extension study.

Objective: To evaluate long-term efficacy and safety profiles during 3 years of individualized ranibizumab treatment in patients with visual impairment due to diabetic macular edema (DME).

Design: Phase IIIb, multicenter, 12-month, randomized core study and 24-month open-label extension study.

Participants: Of the 303 patients who completed the randomized RESTORE 12-month core study, 240 entered the extension study.