Rna Interference

CHICAGO — In this Healio video exclusive, Dipti Itchhaporia, MD, FACC, FESC, discusses some of the late-breaking science presented at the American Heart Association Scientific Sessions.

Medesis Pharma has received a grant of 300,000 euros from AFM-Téléthon to fund the first phases of preclinical development of its RNA interference treatment for Huntington’s disease, the company announced.

A novel therapeutic approach targeting transforming growth factor-B1 and cyclooxygenase-2 demonstrated safety and efficacy in a small cohort of patients with cutaneous in situ squamous cell carcinoma, according to study findings.

Novartis announced the FDA approved inclisiran, its small-interfering RNA therapy for LDL lowering.

Lumasiran, an RNA interference therapeutic that suppresses glycolate oxidase and reduces hepatic oxalate production, exhibited an acceptable safety and efficacy profile in patients with primary hyperoxaluria type 1, according to a study.

Adding to its resources on rare diseases, The American Kidney Fund has launched an education and awareness campaign on primary hyperoxaluria.

The FDA has approved Alnylam Pharmaceuticals Inc.’s lumasiran, known as Oxlumo, for the treatment of primary hyperoxaluria type 1 in both adults and children.

Phase 3 results from the Illuminate-B study suggest lumasiran, an investigational RNA interference therapeutic, may be safe and effective for treating pediatric patients who have primary hyperoxaluria type 1.

Dicerna Pharmaceuticals and Novo Nordisk announced an agreement to develop RNA interference therapies for liver-related cardiometabolic diseases, according to a press release.

PHILADELPHIA — Two novel RNA interference agents, one targeting apolipoprotein C-III and one targeting angiopoietin-like protein 3, improved various lipid parameters in early studies with healthy volunteers, according to new data presented at the American Heart Association Scientific Sessions.