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 ...
Oligonucleotide Treatment Advances Offer Relief for Patients with Familial Chylomicronemia Syndrome
At just three weeks old, Darlene was sent to the emergency room after her mom had brought her to a pediatrician. The baby wouldn't stop crying and wasn't eating. Concerningly, her triglyceride levels were in the 20,000's, far over the normal 150 level. Darlene ...
Trainee Spotlight Series: Poster Winners #OTS24 Session 3
Date: March 13, 2025 Time: 12pm EDT / 5pm CET Join us for this Trainee Spotlight webinar featuring trainees Jessica Bennett, and Chris Saab, all of whom won poster awards at the 2024 OTS Annual Meeting. Title: Reprogramming DNA with a ...
Trainee Spotlight Series: Poster Winners #OTS24 Session 2
Date: February 13, 2025 Time: 11-12pm EST Join us for this Trainee Spotlight webinar featuring trainees Edwin van Oosten, Josh Robinson, and Trishalina Das, all of whom won poster awards at the 2024 OTS Annual Meeting. Title: Exploring Nucleic ...
Trainee Spotlight Series: Poster Winners #OTS24 Session 1
Date: January 23, 2025 Time: 11-12pm EST Title: Antisense oligonucleotides targeting linked-SNPs provide allele-specific knockdown of a dominant-negative SPTAN1 pathogenic variant Description: Our first webinar of the year features trainees Christiana Wang, Elaine Kang, and Sinan Faiad, all of whom won poster ...
