Developing and testing new oligonucleotides for medical use
MANUFACTURE AND PRECLINICAL TESTING OF SITE BLOCKING OLIGONUCLEOTIDES (SBO)
This study is working on creating new types of medicines that target specific genes, which could lead to better treatments for patients with various health conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Advanced Bioscience Laboratories, INC. NIH-funded |
| Lab location | 1 site (Kensington, United States) |
| Project ID | NIH-11211981 on NIH RePORTER |
What this research studies
This research focuses on the manufacture and preclinical testing of site-blocking oligonucleotides, which are specialized molecules designed to interact with specific genetic sequences. The approach involves producing and qualifying master cell banks and viral stocks, developing analytical methods, and ensuring that the manufacturing process meets stringent quality standards. Patients may benefit from advancements in drug formulations that could lead to more effective treatments for various conditions. The research emphasizes aseptic processing to ensure the safety and efficacy of the drug products being developed.
Who could benefit from this research
Good fit: Ideal candidates for this research would be patients with conditions that could be treated with oligonucleotide therapies.
Not a fit: Patients who do not have genetic conditions or who are not candidates for oligonucleotide treatments may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the development of more effective and targeted therapies for patients with specific genetic conditions.
How similar studies have performed: Other research has shown promise in the development of oligonucleotide therapies, indicating a potential for success in this area.
Where this research is happening
Kensington, United States
- Advanced Bioscience Laboratories, INC. — Kensington, United States (Active)
Researchers
- Principal investigator: Retterer, Cary — Advanced Bioscience Laboratories, INC.
- Study coordinator: Retterer, Cary
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.