Aug 2026· Nucleic Acid Insights· Vol 3, pp. 479–482· 0 citations
Abstract
June and July 2026 saw continued clinical, regulatory, and strategic momentum across nucleic acid therapeutic modalities, with notable clustering in antisense approaches to ALS and RNA-based approaches to obesity and cardiometabolic disease. On the regulatory front, Praxis Precision Medicines secured FDA Breakthrough Therapy Designation for the ASO elsunersen in SCN2A developmental and epileptic encephalopathy, and Atrium Therapeutics received IND clearance for siRNA ATR-1072 in PRKAG2 syndrome. In the clinic, Wave Life Sciences and Resalis Therapeutics advanced siRNA and ASO candidates in obesity, while Leal Therapeutics and Trace Neuroscience initiated ASO trials in ALS. Preclinical highlights included CDW Holding’s spleen-targeted LNP-LANFA delivery platform and Confluence Genetics’ Cas-CLEAR CRISPR oncology platform. Strategic activity featured a Ribo–Madrigal siRNA milestone in MASH and a Vect-Horus–Servier agreement to develop brain-targeted oligonucleotides for rare CNS disorders.
Late July to mid-September 2026 saw continued regulatory, clinical, and strategic momentum across nucleic acid therapeutic modalities, with notable clustering in rare neurological and neuromuscular disorders. On the regulatory front, the US FDA approved Ionis’ antisense oligonucleotide Zanvastro™ (zilganersen) for Alex...
Neurodegenerative disorders have diverse etiologies from monogenic mechanisms such as toxic gain-of-function mutation in subsets of amyotrophic lateral sclerosis (ALS) to multifactorial cascades exemplified by Alzheimer’s disease (AD), which feature proteostasis failure, neuroinflammation, mitochondrial dysfunction, or...
Masahiro Ohara, S. Ishigaki, Jonathan K. Watts et al.· Molecular Neurodegeneration...· 0 citations
This review surveys recent advances in engineering LNPs that traverse the blood–brain barrier (BBB), evade innate immune surveillance, and achieve cell‐selective expression in the ageing brain and outlines a translational roadmap for brain applications.
A. Belaidi, Denzil Furtado, Francesca M. Alves et al.· Exploration· 1 citation
Onpattro, the first FDA-approved RNAi therapy, is highlighted, Onpattro’s clinical development is highlighted, compares delivery platforms, discusses translational challenges, and emerging technologies that will guide the next generation of RNAi nanomedicines in personalized therapy are examined.
Dilpreet Singh, Satvir Singh, Nitin Tandon et al.· Iranian Journal of Basic Med...· 1 citation
A multidimensional framework is proposed in which gene therapy platforms are evaluated according to three intrinsic properties—genetic precision, temporal control, and dosage tunability—while delivery, clinical maturity, and disease context act as major translational constraints.
Jared Wieland, Peyton Jackson, William Penrod et al.· Cells· 0 citations
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