NanoCas: A Newly Identified Compact Nuclease May Expand CRISPR’s Reach
The revolutionary CRISPR-Cas system has proven to be a precise method for genome editing, showing great promise in tackling genetic disorders. However, clinical trials for CRISPR-based treatments have faced delivery challenges due to the large size of the editing system. In the quest to ...
OTS Member Highlight – Holly Kordasiewicz, PhD
Holly Kordasiewicz was a curious, chatty child with a penchant for asking questions. At dinner, conversations were often about the fascinating things her mom, a registered nurse, learned at work, sparking Kordasiewicz's interest in medicine and biology. As her grandparents aged, and she witnessed ...
Perspectives on Oligonucleotide Therapeutics: A discussion with Rebecca Miles, incoming president of OTS
Date: June 12, 2025 Time: 11-12pm EDT / 5-6pm CEST Title: Perspectives on Oligonucleotide Therapeutics: A discussion with Rebecca Miles, incoming president of OTS Presentation Description: In this interview, Dr. Rebecca Miles, the president-elect of the Oligonucleotide Therapeutics Society, will discuss ...
Trainee Spotlight Series: Poster Winners #OTS24 Session 5
Date: May 15, 2025 Time: 11-12pm EDT / 5-6pm CEST Join us for this Trainee Spotlight webinar featuring trainee Daniel van Leeuwen, poster award winner at the 2024 OTS Annual Meeting. Title: Deciphering the impact of toxic antisense oligonucleotide (ASO) ...
OTS Member Highlight – Dr. Annemieke Aartsma-Rus
Growing up the oldest of four children in Leiden, Netherlands, Dr. Annemieke Aartsma-Rus became fascinated with medical research and genetics as a teenager. While her father's work as a physician made her realize she didn't want to be a doctor, it did teach her ...
Targeting the Kidney: The Promise of RNA-Based Therapeutics
When Yessenia Gutierrez was just nine years old, her kidneys failed; at 10 years old, she received her first kidney transplant. Gutierrez was born with polycystic kidney disease (PKD), a genetic condition that makes fluid-filled cysts grow on the kidney and can lead to ...
Trainee Spotlight Series: Poster Winners #OTS24 Session 4
Date: April 10, 2025 Time: 11am EDT / 5pm CEST Join us for the fourth Trainee Spotlight webinar of the year featuring trainees Faith Kivunga, Atish Wagh Ph.D, and Svenja Dudek, poster awards winners from the 2024 OTS Annual Meeting held last fall. ...
Meet Your New BOD Trainee Reps
Date: March 27, 2025 Time: 12pm EDT / 5pm CET Title: Developing Oligonucleotides to Modulate HTT mRNA isoforms Presentation Description: Huntington’s Disease is an autosomal dominant neurodegenerative disease caused by an expanded CAG repeat tract within exon 1 of the Huntingtin ...
2024 FDA Approvals: A Wave of Innovation in Treating Serious Diseases
In 2024, the Food and Drug Administration Center for Drug Evaluation (CDER) approved 50 new small molecules, biologics, and oligonucleotide therapies (1). The approvals — the second largest in 30 years — include an oligonucleotide therapy for blood cancer, an antisense oligonucleotide shown to ...
Transporting Therapeutics: A Novel Approach to Deliver ASOs to the Brain
Antisense oligonucleotide (ASO) therapies, which bind to RNA and modify protein expression, are promising drugs for treating neurological conditions. However, their inability to cross the blood-brain barrier (BBB) sometimes makes injection directly into the cerebral spinal fluid (via intrathecal injection) necessary, which is not ...