Shielding gene-targeting drugs to make them safer and longer-lasting
Making Oligonucleotides Better Biopharmaceuticals by Steric Protection
['FUNDING_R01'] · NORTHEASTERN UNIVERSITY · NIH-11308242
A new polymer 'shield' aims to make antisense gene-targeting medicines safer, longer-lasting, and better at reaching tissues like skin, muscle, and heart.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | NORTHEASTERN UNIVERSITY (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11308242 on ClinicalTrials.gov |
What this research studies
Researchers are attaching antisense oligonucleotides to a dense 'bottlebrush' polymer called pacDNA that acts like an entropic shield around the drug. The shield reduces unwanted interactions with blood proteins (which can cause coagulopathy and immune activation), improves stability against enzymes, and keeps the drug in the bloodstream and target tissues longer. Early lab and animal work shows improved potency, tissue deposition in places such as skin, muscle, and heart, and avoidance of anti-carrier immune responses. The team will use a combinatorial polymer library to optimize the pacDNA backbone and properties for safer, more effective delivery.
Who could benefit from this research
Good fit: People who may be interested include those eligible for antisense oligonucleotide therapies—such as certain blood coagulation disorders or genetic diseases affecting skin, muscle, or heart—or patients willing to donate samples for related research.
Not a fit: Patients with conditions unrelated to gene-targeting medicines or those needing immediate clinical treatment are unlikely to receive direct benefit from this preclinical work.
Why it matters
Potential benefit: If successful, this could reduce side effects, allow less frequent dosing, and enable antisense treatments to reach tissues that are currently hard to treat.
How similar studies have performed: Antisense drugs and other delivery systems (for example lipid nanoparticles) have had clinical success, but this high-density bottlebrush polymer shielding approach is novel and remains at preclinical stages.
Where this research is happening
BOSTON, UNITED STATES
- NORTHEASTERN UNIVERSITY — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ZHANG, KE — NORTHEASTERN UNIVERSITY
- Study coordinator: ZHANG, KE
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.
Conditions: Blood Coagulation Disorders