Co-delivery platform for enhanced nucleic acid therapeutics

This technology describes compositions which enhance the potency and stability of nucleic acid therapeutics for the treatment of diseases or conditions.

Unmet Need: Effective nucleic acid therapeutics for disease treatment

In recent years, numerous nucleic acid therapeutics have been described for the treatment of a myriad of diseases and conditions. Several of these drugs have been approved by the FDA, including the first small interfering RNA (siRNA) and micro RNA (miRNA)-based drugs. However, the success of these nucleic acid therapeutics remains limited by factors including off target effects, the limited half-life of RNA molecules, and unstable payload expression and release.

The Technology: Compositions to improve the performance of nucleic acid therapeutics

This technology describes several compositions which improve the performance of miRNA-based therapeutics using a co-delivery platform in which the therapeutic is paired with a stabilizing or enhancing element. This element may be an RNA induced silencing complex (RISC) protein, to stabilize payloads within target cells, thereby maximizing therapeutic effectiveness. The technology uses exosomes or other co-delivery vehicles which are engineered to minimize endogenous nucleic acid loading, to reduce off-target effects and enable targeted therapeutic delivery.

This technology has been validated with human cancer cell lines and in mouse models.

Applications:

• Nucleic acid treatment of disease or disorders, including cancer and diseases of the liver
• Animal models to understand miRNA processing in vivo
• Delivery vehicle to enhance other therapeutics

Advantages:

• Broadly compatible with many nucleic acid therapeutics
• Reduces off-target or adverse effects
• Stable preparations

Lead Inventor:

Fatemeh Momen-Heravi, DDS, MPH, PhD, MS

Patent Information:

Patent Pending

Related Publications:

• Momen-Heravi F, Catalano D, Talis A, Szabo G, Bala S. “Protective effect of LNA-anti-miR-132 therapy on liver fibrosis in mice” Mol Ther Nucleic Acids. 2021 May 14; 25: 155-167.

• Wu X, Cheng YL, Matthen M, Yoon A, Schwartz GK, Bala S, Taylor AM, Momen-Heravi F. “Down-regulation of the tumor suppressor miR-34a contributes to head and neck cancer by up-regulating the MET oncogene and modulating tumor immune evasion” J Exp Clin Cancer Res. 2021 Feb 17; 40(1): 70.

Tech Ventures Reference:

• IR CU21087, CU20006

• Licensing Contact: Joan Martinez