RNA Interference News and Research

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Kidney Week 2026 features late-breaking clinical trials in kidney care

The American Society of Nephrology, has selected the following late breaking clinical trials to be presented in-person at Kidney Week 2026 October 22–25.

24 Oct 2026

Undecimal Bio launches with $18 million oversubscribed seed financing to address ‘liver trap’ in siRNA therapeutic delivery

Undecimal Bio, a platform biotechnology company advancing targeted delivery of siRNA therapeutics, today announced an oversubscribed $18 million USD seed financing.

From UndecimalBio 13 Oct 2026

Long non-coding RNAs offer new pathways to overcome cancer treatment resistance

Scientists are highlighting long non-coding RNAs (lncRNAs) as promising targets in the effort to overcome one of cancer treatment's most persistent challenges: therapeutic resistance.

12 Oct 2026

Scientists find new way to overcome cell’s natural defenses against self-amplifying mRNA vaccines

The innovation could enable protection at lower doses, meaning vaccines could be rolled out further and faster than was previously imaginable, and opens new possibilities in gene therapy, cancer immunotherapy, and protein replacement therapies.

28 Sep 2026

Advances in chemistry and delivery are taking oligonucleotide medicines beyond rare diseases

A review in Cell Reports Medicine examines how advances in oligonucleotide chemistry and delivery have established ASOs and RNA interference therapies as a class of genetic medicines, with 21 oligonucleotides approved at the time of writing. It also identifies extrahepatic delivery, safety, target validation, individualized treatment, and data quality as priorities as the field expands into common diseases and explores AI-assisted development.

24 Sep 2026

Advancing intracellular delivery with LumiPore®: A new approach to cell transfection and drug discovery

Discover how LumiPore improves intracellular delivery with gentle, high-efficiency transfection for drug discovery and cell therapy research.

From Trince 8 Sep 2026

SHASTA trials show substantial triglyceride reductions with plozasiran

Plozasiran significantly reduced triglyceride levels in severe hypertriglyceridemia patients, showing promise in preventing acute pancreatitis after one year.

31 Aug 2026

Blocking the Axl receptor may offer a new way to promote bone formation

Osteoporosis is one of the most common age-related bone diseases, affecting millions of people worldwide and increasing the risk of fractures, disability, and reduced quality of life.

28 Jul 2026

AI-powered analytics and experimental validation: Transforming target identification in drug discovery

Discover how AI-powered analytics and experimental validation combine to improve target identification and accelerate drug discovery success.

From Axxam S.p.A 23 Jul 2026

Scientists capture atomic images to improve RNA interference drug design

RNA interference is a natural mechanism for living cells to control whether specific genes are being used or not.

29 Jun 2026

Cells detect and silence invading transposons through abnormal RNA signals

Transposons, DNA sequences that can self-replicate and move (jump) throughout the genome, are widespread and can affect cell survival if left unchecked. Cells control these "jumping genes" by silencing them, but little was known about how cells recognize and defend themselves from invading transposons.

26 May 2026

Ohio State study unlocks the long-standing mystery of RNA interference

While scientists have known for over two decades that all cells use a strategy called RNA interference to regulate gene expression, a new study is the first to describe how a specific protein manages the step-by-step process of assembling the molecular complex that performs the regulatory job.

26 May 2026

Twice-yearly blood pressure treatment could reshape hypertension care, but doctors warn against a “fire-and-forget” approach

Long-acting RNA interference therapy for hypertension could shift blood pressure control from daily pill-taking to scheduled, system-led protection. But the authors warn that this “vaccine-like” model must preserve lifestyle support, monitoring, and follow-up to avoid clinical disengagement.

27 Apr 2026

CRISPR-Cas3: A safer gene-editing tool shows promise for transthyretin amyloidosis treatment

Genetic disorders occur due to alterations in the primary genetic material, deoxyribonucleic acid (DNA), of an organism. Transthyretin amyloidosis (ATTR) is a progressive disorder involving amyloid deposits of misfolded transthyretin (TTR) proteins.

14 Jan 2026

Fruit fly study identifies genes regulating dopamine and sleep

Dopamine in the brain influences movement, learning, motivation and sleep. In humans, problems with dopamine are linked to conditions like Parkinson's disease, depression and sleep disorders.

13 Jan 2026

Artificial intelligence unlocks new frontiers in RNA drug design

In the realm of modern medicine, RNA-based therapies have emerged as a promising avenue, with significant advancements in metabolic diseases, oncology, and preventive vaccines. A recent article published in Engineering titled "The Future of AI-Driven RNA Drug Development" by Yilin Yan, Tianyu Wu, Honglin Li, Yang Tang, and Feng Qian, explores how artificial intelligence (AI) can revolutionize RNA drug development, addressing current limitations and offering new opportunities for innovation.

31 Dec 2025

Novel biotechnical vector integrates viral and synthetic features for targeted cancer therapy

Gene therapy offers the possibility of addressing cancer at its molecular roots by targeting disease-causing genes rather than relying solely on surgery, chemotherapy, or radiotherapy.

16 Sep 2025

Phase II trial finds zilebesiran lowers systolic blood pressure in patients with uncontrolled hypertension

Among individuals with cardiovascular disease or high cardiovascular risk and uncontrolled hypertension on multiple anti-hypertensive agents, a single subcutaneous dose of zilebesiran 300 mg led to a 5-mmHg reduction in systolic blood pressure at Month 3 compared with placebo, a difference that did not reach statistical significance.

1 Sep 2025

Breakthroughs in diagnosis and therapy for transthyretin amyloidosis

Transthyretin amyloidosis (ATTR) is caused by the misfolding and aggregation of the transthyretin (TTR) protein, leading to multi-organ damage.

21 Aug 2025

'Two-in-one' RNA molecule can simultaneously turn off two difficult-to-target cancer-related genes

University of North Carolina Lineberger Comprehensive Cancer Center researchers have developed a "two-in-one" molecule that can simultaneously turn off two notoriously difficult-to-target cancer-related genes, KRAS and MYC, as well as directly deliver drugs to tumors that express these genes.

4 Aug 2025