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 ...
The Man Behind the Molecules: John Maraganore’s RNAi Legacy
The 2024 OTS lifetime achievement winner, John Maraganore, is best known for his pioneering role in making RNA interference (RNAi) a successful therapeutic modality while CEO at Alnylam Pharmaceuticals. However, before joining and after leaving Alnylam, he was and continues to be influential in ...
Mastering the Art of Reviewing Scientific Papers
Date: December 12, 2024 Time: 8am PST / 11am EST / 5pm CET Title: Mastering the Art of Reviewing Scientific Papers Description: Join our webinar on mastering the art of reviewing scientific papers, presented by Prof. Dr. Annemieke Aartsma-Rus, co-editor in ...
Bridge Editing: A Novel Technique for Large-Scale DNA Modifications
Link Source: Hiraizumi, M., Perry, N.T., Durrant, M.G. et al. Structural mechanism of bridge RNA-guided recombination. Nature 630, 994–1002 (2024). Gene editing has swiftly advanced since CRISPR was discovered, with CRISPR being developed not only to provide therapeutic gene editing treatments for humans but also for uses ...
