Sanofi’s Venglustat shows breakthrough results in rare neurological Gaucher disease

Venglustat, a glucosylceramide synthase inhibitor (GCSi), works by reducing the abnormal buildup of sugar-and-fat molecules in cells and organs

Onchilles Pharma gets FDA green light for first-in-human trials of breakthrough cancer therapy

N17350 is the company’s first-in-class tumor-directed therapeutic, designed to harness the ELANE pathway

Pfizer’s monthly obesity drug shows breakthrough weight loss in Phase 2b trial

The study tested whether PF’3944 could maintain efficacy when switching from weekly to monthly injections and remain safe and well-tolerated

Cirena launches to revolutionize long RNA production for CRISPR & gene editing

As CRISPR and prime editing applications advance, researchers increasingly rely on longer guide RNAs to boost editing efficiency and reduce off-target effects

Epigenica launches EpiFinder Analysis Service to accelerate Epigenomic research

The new service delivers end-to-end support, managing every stage of an epigenomics project

Shilpa Medicare & mAbTree Biologics win FDA nod for rare blood cancer therapy

The pioneering monoclonal antibody is for Essential Thrombocythemia (ET) and Polycythemia Vera (PV), rare chronic blood cancers with limited treatment options

Moderna teams up with Recordati to commercialize rare disease therapy

Fides launches imaging CRO focused on early-phase clinical decision-making

Fid?s brings core strengths across imaging applications and human biology, supporting work in digestive disease, GI oncology, and select solid tumors

Insilico Medicine’s AI-designed Parkinson’s drug cleared for human trials by FDA

The upcoming Phase I trial will test the safety, tolerability, and pharmacokinetics of ISM8969 in healthy volunteers and determine the optimal dosing for future studies

Eyestem moves closer to blindness breakthrough with phase 2 trial green light

The early-stage trial demonstrated an excellent safety profile alongside clinically meaningful improvements in visual acuity