Onco3R Completes Phase 1 First Cohort for SIK3 Inhibitor
Phase 1 topline: Onco3R Therapeutics completed dosing of the first cohort (n=6) in its first-in-human study of O3R-5671 with...
Dye-Driven Precision: Targeting Chemo Directly to Tumors
Lyle Small leads a biotech startup pursuing a bold new approach: dye-drug conjugates that carry chemotherapy directly to cancer...
Functional Improvement Seen in Dyne’s One-Year DM1 Trial Data
One-year data from six adults with myotonic dystrophy type 1 (DM1) treated with zeleciment basivarsen at 6.8 mg/kg every...
Rheumatologists’ Powerful Hope: Cell Therapy for Sclerosis
Survey data from 104 US rheumatologists point to guarded enthusiasm for cell therapy in autoimmune disease: most hold favorable...
Nilo Therapeutics Launches with $101M to Target Immune Disease
Nilo Therapeutics launched with a $101 million Series A led by The Column Group, DCVC Bio, and Lux Capital,...
Pioneering Orphan Drug Updates from Sharp Therapeutics in 2025
No human data were disclosed. Sharp Therapeutics plans to present a Phase 1–ready preclinical package for its small‑molecule candidate...
Nurexone Shows Vision Recovery in Preclinical Glaucoma Model
Preclinical data from an optic nerve crush model showed that ExoPTEN, an exosome-delivered PTEN siRNA, produced a reproducible, dose-dependent...
Aardvark Gets FDA Alignment for Prader-Willi Phase 3 Trial
Aardvark Therapeutics lowered the minimum age of eligibility in its Phase 3 HERO trial of ARD-101 for hyperphagia in...
FDA Clears Promising Schizophrenia Drug for Phase 3 Trial
In a 189‑patient Phase 2 study, Celon Pharma’s PDE10A inhibitor CPL’36 delivered statistically significant symptom reductions at four weeks...
New Parkinson’s Drug GT-02287 Shows Promising Phase 1b Data
Early interim data from Gain Therapeutics’ Phase 1b study of GT-02287 in Parkinson’s disease point to stability and improvements...