An RNA interference (RNAi) molecule that selectively targets the KRAS G12V mutation could represent a key advance in the treatment of cancer associated with this oncogenic variant. Researchers at the University of North Carolina (UNC) at Chapel Hill, in collaboration with Enfuego Therapeutics Inc., have developed a new RNAi designed to enter cells through the epidermal growth factor receptor (EGFR), which is commonly overexpressed in tumor cells. This targeted entry pathway could minimize the side effects associated with therapies that affect KRAS.

Rona Therapeutics Co. Ltd. has reported preclinical data for its RNA interference (RNAi) therapy RN-026 targeting lipoprotein(a) (Lp[a]).

A development deal with Biogen Inc. could eventually bring City Therapeutics Inc. about $1 billion in milestone payments. It’s a step in the direction the company had been going: looking for the right partners, including those from big pharma. Privately held City Therapeutics is getting $46 million in the deal. That includes $16 million as an up-front payment and an investment of $30 million in exchange for a City Therapeutics convertible note.

N4 Pharma plc has successfully completed the first in vivo study of orally delivered Nuvec using its lead program N4 101, an orally administered anti-inflammatory treatment for inflammatory bowel disease (IBD).

Sirius Therapeutics Inc. raised nearly $50 million in a series B2 financing round May 9 to support its pipeline of small interfering RNA (siRNA) molecules for cardiovascular disease indications. SRSD-107, a long-acting Factor XI anticoagulant for thromboembolic disorders, is its lead candidate waiting on EMA clearance to begin a phase II study in Europe. 

Sirius Therapeutics Inc. raised nearly $50 million in a series B2 financing round May 9 to support its pipeline of small interfering RNA (siRNA) molecules for cardiovascular disease indications. SRSD-107, a long-acting Factor XI anticoagulant for thromboembolic disorders, is its lead candidate waiting on EMA clearance to begin a phase II study in Europe. 

Rona Therapeutics Co. Ltd. has obtained IND clearance from China′s National Medical Products Administration (NMPA) for RN-1871, a small interfering RNA (siRNA) drug targeting angiotensinogen (AGT) for hypertension.

Previous work uncovered the role of the complement system in neuroinflammation and synaptic loss in neurodegenerative disorders such as Alzheimer’s disease (AD). The third component, C3, central in all complement activation pathways, has been proposed as a potential therapeutic target in AD.

Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors.