Alnylam Pharmaceuticals

City Therapeutics aims to lead RNAi-based medicine by improving siRNA engineering, with a $135 million Series A funding and a clinical development plan starting in 2025, led by RNAi pioneers.

Intellia Therapeutics initiates Phase 3 HAELO trial for NTLA-2002, a CRISPR gene editing treatment targeting hereditary angioedema, aiming to reduce attacks and kallikrein protein levels. The trial will involve 60 adults, with a 2-to-1 randomization to NTLA-2002 or placebo, followed by a crossover phase. Intellia seeks to compete with Pfizer, BridgeBio Pharma, and Alnylam in rare disease treatments.

Judo Bio, a biotech firm with $100 million and an RNA drug platform, aims to tackle kidney diseases, leveraging a technology similar to Alnylam's RNA interference. Judo's ligand-siRNA conjugates target kidney cells via megalin receptors, potentially addressing systemic diseases like diabetes and hypertension, as well as kidney-specific conditions.

Transthyretin amyloidosis cardiomyopathy drug development, including Pfizer's Vyndamax and BridgeBio's acoramidis, is a major focus. FDA decisions on acoramidis, AstraZeneca's dato-dxd for lung cancer, Vertex's vanza triple for cystic fibrosis, PTC's Upstaza for AADC deficiency, and Bristol Myers' subcutaneous Opdivo for solid tumors are imminent, with significant market implications.

Alnylam presents HELIOS-B study data on vutrisiran for ATTR-CM at HFSA 2024, showing significant improvements in cardiac structure, systolic and diastolic function, and stability of biomarkers NT-proBNP and Troponin-I over 30 months.

CoherentMI's report on the RNA Therapeutics Market projects it to grow from US$2.5M in 2024 to US$948M by 2031, at a CAGR of 68%. The market leverages RNA interference and antisense technologies for gene expression modulation, targeting hereditary disorders, cancer, and infectious diseases. Key players include Alphavax, Arcturus Therapeutics, Atyr Pharma, and Gritstone Bio. The market is driven by increasing R&D investments and demand for precision medicine.

Novartis' Phase III V-MONO trial of Leqvio met primary endpoints, showing significant LDL-C reduction in low/moderate ASCVD risk patients. The trial compared Leqvio monotherapy against ezetimibe and placebo, with findings to be discussed with regulatory authorities.