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Safety, efficacy, and immunogenicity of a novel IgG degrading enzyme (KJ103): results from two randomised, blinded, phase 1 clinical trials.
Gene Ther
January 2025
Shanghai Bao Pharmaceuticals Co., Ltd., No. 28 Luoxin Road, Baoshan, Shanghai, China.
The approved intravenous adeno-associated virus (AAV) therapies are limited by the widespread prevalence of pre-existing anti-AAV antibodies in the general population, which are known to restrict patients' ability to receive gene therapy and limit transfection efficacy in vivo. To address this challenge, we have developed a novel recombinant human immunoglobulin G degrading enzyme KJ103, characterized by low immunogenicity and clinical value for the elimination of anti-AAV antibodies in gene transfer. Herein, we conducted two randomized, blinded, placebo-controlled, single ascending dose Phase I studies in China and New Zealand, to evaluate the pharmacokinetics, pharmacodynamics, safety and immunogenicity of KJ103 in healthy volunteers.
View Article and Find Full Text PDFObjective: Presenting at academic conferences is an important means of disseminating research, networking, and building a professional reputation, but the quality of presentations at conferences is often suboptimal. This project describes the design, implementation, and evaluation of a presentation coaching program offered by an academic surgical society to presenters at its annual meeting.
Design: Oral presenters were paired with a coach and encouraged to meet independently, yet coaching was unstructured.
No QT interval prolongation effect of sepiapterin: a concentration-QTc analysis of pooled data from phase 1 and phase 3 studies in healthy volunteers and patients with phenylketonuria.
J Pharmacokinet Pharmacodyn
January 2025
PTC Therapeutics, Warren, NJ, USA.
Sepiapterin is an exogenously synthesized new chemical entity that is structurally equivalent to endogenous sepiapterin, a biological precursor of tetrahydrobiopterin (BH), which is a cofactor for phenylalanine hydroxylase. Sepiapterin is being developed for the treatment of hyperphenylalaninemia in pediatric and adult patients with phenylketonuria (PKU). This study employed concentration-QT interval analysis to assess QT prolongation risk following sepiapterin treatment.
View Article and Find Full Text PDFFactors associated with weight loss response to GLP-1 analogues for obesity treatment: a retrospective cohort analysis.
BMJ Open
January 2025
University of British Columbia, Vancouver, British Columbia, Canada.
Objectives: The response to glucagon-like peptide-1 (GLP-1) analogues for weight loss varies significantly. We investigated the anthropometric, demographic and clinical characteristics associated with total body weight loss (TBWL) from subcutaneous GLP-1 analogue therapy in patients with obesity in a real-world setting.
Design: Retrospective cohort analysis.
Differential expressions of exosomal miRNAs in patients with chronic heart failure and hyperuricemia: diagnostic values of miR-27a-5p and miR-139-3p.
Nan Fang Yi Ke Da Xue Xue Bao
January 2025
Department of Cardiology, Nanjing Hospital of Chinese Medicine Affiliated to Nanjing University of Chinese Medicine, Nanjing 210022, China.
Objectives: To analyze the differentially expressed exosomal miRNAs in patients with chronic heart failure (CHF) complicated by hyperuricemia (HUA) and explore their potential as novel diagnostic molecular markers and their target genes.
Methods: This study was conducted among 30 CHF patients with HUA (observation group) and 30 healthy volunteers (control group) enrolled between September, 2020 and September, 2023. Peripheral blood samples were collected from 6 CHF patients with HUA for analyzing exosomal miRNAs by high-throughput sequencing, and the results were validated in the remaining 24 patients using qRT-PCR.
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