Antisense Oligonucleotide Therapy in Chronic Hepatitis B: Clinical Progress and Phase 3 Outcomes
When Sheree was 26, she kept wondering why she was always so tired. Though she was a mom and a nurse working multiple double shifts, she was still young and knew she shouldn’t be this fatigued. Then the nausea and abdominal pain began. Soon, the ...
Al3Cas12f RKK: A New, Enhanced Compact Editor that is Highly Efficient
CRISPR-based gene-editing tools have transformed the biomedical field, but delivering these systems directly into human tissues — known as in vivo gene editing — remains a major challenge. Many therapeutic CRISPR approaches still rely on ex vivo editing, where the patient’s cells are removed, ...
OTS Member Highlight – Keith T. Gagnon, PhD
As a child, before Keith Gagnon knew anything about science, he told his mom he wanted to be a scientist when he grew up. Now, decades later, Dr. Keith Gagnon is a biochemist and molecular biologist specializing in RNA biology, nucleic acid therapeutics, repeat ...
Interesting Findings on the Impact of Antisense Oligonucleotides on Genome Stability and DNA-Repair Enzyme Activation
Antisense oligonucleotides (ASOs) have emerged as promising drug candidates for the treatment of genetic diseases. Because of their ability to target virtually any disease-related gene product, several ASO drugs have been approved, and many more are in development (1). While they remain among the most ...
Beyond the “Too Rare” Barrier: Platforms for Rare Mutations Leverage New Regulatory Tool
In November, Food and Drug Administration (FDA) officials introduced the Plausible Mechanism Pathway, a regulatory tool designed to make it easier to approve treatments — specifically investigational gene therapies — for rare conditions. An article published in The New England Journal of Medicine (1) ...
Bioanalysis of Oligonucleotide Therapeutics via Nucleic Acid Nanorobotic Platform
Date: May 21, 2026 Time: 11-12pm EDT / 5-6pm CEST Title: Bioanalysis of Oligonucleotide Therapeutics via Nucleic Acid Nanorobotic Platform Description: Dynamic DNA nanotechnology enables the isothermal and enzyme-free exchange of nucleic acid strands. The underlying mechanism of action in this ...
Phase 3 Trials of Intellia’s Nex-z CRISPR Therapy Resume after the Clinical Holds are Lifted
Intellia Therapeutics recently announced that the U.S. Food and Drug Administration (FDA) has lifted the hold on its clinical trial for the investigational CRISPR-based gene-editing therapy, nexiguran ziclumeran, also known as nex-z or NTLA-2001. The four-month hold was implemented after a patient in the ...
OTS Member Highlight – Dr. Timothy Yu
The sometimes twisty, sometimes intuitive, sometimes amazingly elegant hidden logic of the natural world has always fascinated Dr. Timothy Yu. Knowledge we now take for granted — the genetic code, machinery of transcription, or the layered biological logic of developmental patterning — were once ...
FDA’s Plausible Mechanism Pathway for Personalized Therapies to Treat Rare Diseases
The clock was ticking as soon as baby KJ was born in the summer of 2024. Within two days of his birth, he was lethargic and struggled to breathe. His blood test results showed elevated ammonia levels, leading to a diagnosis of carbamoyl-phosphate synthetase ...
Immunogenicity Risk Assessment for Nucleic Acid Therapeutics
Date: October 1, 2026 Time: 11-12pm EDT / 5-6pm CEST Register Here Title: Immunogenicity Risk Assessment for Nucleic Acid Therapeutics: A Comprehensive Evaluation for ASO, siRNA, and Nonvaccine mRNA/LNP Therapies Description: Nucleic acid therapeutics require new immunogenicity evaluation frameworks, as the safety and ...
