ROI 2022-063, 2022-091, 2025-022, 2025-023, 2025-024
Market Need
Oligonucleotide drugs—such as siRNA and other nucleic acid therapeutics—are powerful but face major challenges: they are unstable in the body, are rapidly degraded, struggle to cross biological barriers, and often fail to reach the tissues where they are needed.
Technology Overview
Five patent families protecting a platform of dendritic conjugates engineered to enhance the delivery of therapeutic oligonucleotides to multiple tissues—including the lungs, tumors, skin, and brain—by chemically linking oligonucleotides to dendrons that improve stability, targeting, and intracellular uptake.
The lung-targeted conjugates employ dendrons bearing hydrophilic end groups, phosphate groups, and/or hydrophobic chains to optimize distribution and therapeutic performance in pulmonary tissue.
Similarly, tumor-delivery systems use dendritic oligonucleotide conjugates designed to modulate pharmacokinetics and biological interactions, enabling more effective accumulation and activity in tumor environments.
For dermatologic applications, dendritic conjugates facilitate topical or transdermal penetration of oligonucleotides by enhancing absorption, retention, and cutaneous tissue interaction. Complementing these, a microneedle-based topical delivery system provides an alternative physical method to increase skin penetration and stability of therapeutic oligonucleotides for the treatment of skin-related conditions.
Finally, dendritic conjugates engineered for brain delivery address challenges such as the blood–brain barrier by improving the stability, uptake, and distribution of oligonucleotides—such as siRNA—within neural tissue. Together, these inventions present a versatile, tunable dendritic delivery platform capable of improving nucleic acid therapeutic performance across diverse biological barriers and target tissues.
Commercial Advantages
- Tissue-specific, tunable delivery across multiple biological barriers
- Improved stability, pharmacokinetics, and intracellular uptake
Additional Information
- Researcher: Hanadi Sleiman
- Patents:
- United States US 18/592,676,
- United States US 18/609,299 PCT/US2024/020535 converted to US, Europe, Canada, Australia and Japan
- PCT/US2024/018007 converted to US, Korea, Japan, Europe, China, and Australia
- PCT/US2024/018052 converted to US, Korea, China, Japan, Europe, and Australia
- Publications of work:
- Fakih, H.H., et al. Intradermal delivery of lipophilic siRNAs enables prolonged skin retention and sustained gene silencing in a porcine model. Nat Commun (2026). https://doi.org/10.1038/s41467-026-68993-1
- Fakih, Hassan H. et al. Albumin-binding dendritic siRNA improves delivery and efficacy to solid tumors in a melanoma model. Molecular Therapy Nucleic Acids, (2025). https://doi.org/10.1016/j.omtn.2025.102579
- Sarli, S.L., et al. Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo. Nucleic Acids Research (2024). https://doi.org/10.1093/nar/gkae260
- Fakih, H.H., et al. Dendritic amphiphilic siRNA : Selective albumin binding, in vivo efficacy, and low toxicity. Molecular Therapy Nucleic Acids. (2023). https://doi.org/10.1016/j.omtn.2023.102080
- Keywords: Drug Delivery, Therapeutic, Neuroscience, Oncology
