The company has already kicked off an open-label Phase I trial of ABX1100 in Canada following positive results from a study of healthy volunteers.

WVE-007 is a novel, long-acting GalNAc-siRNA targeting INHBE mRNA – a genetically validated target that provides a new approach for healthy, ...

CAMBRIDGE, Mass. - Wave Life Sciences Ltd . (NASDAQ:WVE), a clinical-stage biotechnology company with a market capitalization of $1.87 billion, has announced the commencement of its Phase 1 INLIGHT ...

Eli Lilly and insitro said the deals offers a “new paradigm” for collaborations between major pharmaceutical companies and smaller biotechs.

Atalanta Therapeutics has bagged $97 million, money the biotech plans to use to launch clinical trials for its small ...

The first siRNA drug, patisiran, was approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat a debilitating genetic disorder called hereditary transthyretin amyloidosis.

Reporting in Nature, Kumar and colleagues have now developed a method to target small-interfering RNA (siRNA) to the CNS, providing a safe and non-invasive approach for the delivery of this new ...

Registration Link for Presentation and live Q&A to take place, Tuesday February 4, 2025 at 2:00 pm ESTMarlborough, Massachusetts--(Newsfile ...

Novel programs will combine validated cell-targeted LNP (ctLNP) delivery with siRNA to selectively modulate T cells in vivo Programs to focus on silencing hard-to-drug targets of high therapeutic ...

In SNALPs, the backbone-modified siRNA is encapsulated within a closed shell of a cationic-zwitterionic lipid bilayer, furnished with an outer PEG shield (Figure 2A). The lipid bilayer contains a ...

Title: Efficacy and Safety of ETX-148 in Murine Models of Haemophilia A and B Format: Oral Presentation Presentation Number: OR10 Date: 7 th February 2025 Session: SLAM e-therapeutics plc ("ETX") ...

ALY-101 is an intradermal injectable JAK1 siRNA-lipid conjugate for the treatment of Alopecia Areata <li /> Significant progress made in ...