2024 FDA Approvals: A Wave of Innovation in Treating Serious Diseases

February 25th, 2025|Categories: Perspectives on Current Science|

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

February 12th, 2025|Categories: Perspectives on Current Science|

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

February 5th, 2025|Categories: Perspectives on Current Science|

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 1

January 17th, 2025|Categories: Past Webinars|

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

January 9th, 2025|Categories: Perspectives on Current Science|

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 ...

Bridge Editing: A Novel Technique for Large-Scale DNA Modifications

December 3rd, 2024|Categories: Perspectives on Current Science|

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 ...

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