Key Highlights OPGx-BEST1 targets BEST1-associated inherited retinal diseases, including BVMD and ARB AAV-based gene therapy approach designed to deliver a functional BEST1 gene directly to retinal pigment epithelium cells Five participants enrolled in Cohort 1, including three with BVMD and two with ARB Four participants have been dosed, with the fifth scheduled for dosing this month Dominant BEST disease participants underwent in vitro confirmation to assess whether their mutations are amenable to gene augmentation Early sentinel participant data showed positive tolerability and biological activity after subretinal administration Three-month topline Cohort 1 data expected in September 2026 For BEST1-associated diseases, careful patient selection, mutation-specific validation, and multi-endpoint retinal assessments may be key to advancing gene augmentation strategies
PTBP1, polypyridine tract-binding protein 1
Genome-wide association study of esophageal squamous cell carcinoma in Chinese subjects identifies susceptibility loci at PLCE1 and C20orf54
The tirzepatide dosing guide uses identical math with different peptide masses, demonstrating how transferable these skills are across compounds
Batch numbering systems that allow full traceability are now necessary to make sure that regulations are followed and that any quality issues that may come up after shipping are taken care of
I agree with the slightly sticky feeling for a while but it doesnt bother me as it doesnt last long and I layer other products in my routine