Base Editing in Clinical Trials to Treat Acute Lymphoblastic Leukemia
Base editing has experienced a rapid rise in use since it first came on the scene in 2016, with multiple trials underway, testing its ability to treat conditions with precise, single-letter changes to DNA. The technique presents a potentially more accurate and safer method compared to nuclease-based approaches; however, it’s not without its risks. Currently, researchers are investigating how ...
2023 Annual Meeting Highlights
We are pleased to note that this year’s annual Oligonucleotide Therapeutics Society meeting in Barcelona, Spain had the highest involvement of any previous meeting, with 800 in-person attendees, 297 abstract submissions, 260 posters, 47 travel grants, 36 Sponsors, and 29 Exhibitors. We hope all who attended in person or virtually enjoyed their time and will carry the passion and ...
Fanzor: A Programmable RNA-Guided System In Eukaryotes Similar to CRISPR
Researchers have uncovered the first programmable RNA-guided system in eukaryotes that could be even more precise than CRISPR gene-editing. The discovery — led by Feng Zhang at the McGovern Institute for Brain Research at MIT and the Broad Institute of MIT and Harvard — demonstrates that DNA-modifying proteins exist across all kingdoms of life (1). In a recently published study ...
RNAi: The Persistence of a Few Leads to Success
On August 10, 2018, the U.S. Food and Drug Administration (FDA) approved the first RNAi therapy. The approval marked a significant milestone not just for the drug but the entire field of RNA interference (RNAi). The success of the technology was never guaranteed, and a tumultuous period where Big Pharma companies started selling off their RNAi assets heralded what ...
From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis
On April 25 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval to tofersen (trade name Qalsody) for the treatment of Superoxide Dismutase 1 (SOD1) associated amyotrophic lateral sclerosis (ALS) [1]. The clinical trial failed to meet its primary endpoint; however, approval was granted based on a biomarker response: reduced plasma levels of neurofilament light protein [2]. ...
Investigating the Biology and Potential of CircRNA
In the past few years, mRNA has become a mainstream word with the swift creation of mRNA vaccines to treat SARS-CoV-2. This new technology was already in development, placing it in the perfect position to meet an urgent global need. Yet, there are still challenges to be met to further improve mRNA vaccines and other mRNA therapeutics. Another RNA ...
Base Editing in Clinical Trials to Treat Acute Lymphoblastic Leukemia
Base editing has experienced a rapid rise in use since it first came on the ...
2023 Annual Meeting Highlights
We are pleased to note that this year’s annual Oligonucleotide Therapeutics Society meeting in Barcelona, ...
Fanzor: A Programmable RNA-Guided System In Eukaryotes Similar to CRISPR
Researchers have uncovered the first programmable RNA-guided system in eukaryotes that could be even more precise ...
RNAi: The Persistence of a Few Leads to Success
On August 10, 2018, the U.S. Food and Drug Administration (FDA) approved the first RNAi ...
From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis
On April 25 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval to ...
Investigating the Biology and Potential of CircRNA
In the past few years, mRNA has become a mainstream word with the swift creation ...
