Exosomes for the targeted delivery of CRISPR/Cas9 in human gene editing

This technology utilizes exosomes or extracellular vesicles to deliver the CRISPR/Cas9 gene editing system for the treatment of human diseases.

Unmet Need: Safe modalities for CRISPR/Cas9 gene editing in human patients

Current methods to deliver CRISPR/Cas9 to cells and organs in the human body include electroporation, microinjection, and viral delivery. However, the therapeutic applicability of these delivery systems is hindered by numerous technical limitations including non-specificity, cell damage, low efficiency, and off-target effects. In view of this, a safer, more efficient, and highly targeted method for CRISPR/Cas9 delivery will be needed to successfully leverage this molecular system for gene therapy applications.

The Technology: Targeted human gene editing through exosome-mediated CRISPR/Cas9 delivery

This technology utilizes exosomes or extracellular vesicles (EVs) as vehicles for targeted, safe, and effective in vivo delivery of the CRISPR/Cas9 gene editing system. The vesicles are engineered to express targeting moieties on their surfaces for highly tissue-specific delivery. Furthermore, the vesicles are devoid of endogenous nucleic acids for minimization of off-target effects. As such, this technology may improve the suitability, efficacy, and scalability of CRISPR/Cas9 system for human gene therapies.

Applications:

• Therapies for genetic diseases
• Therapies for cancer, cystic fibrosis, autoimmune, and inflammatory diseases
• Gene editing tool for life science research

Advantages:

• Avoids cytotoxicity or immune activation
• Minimizes side effects
• Increases scalability and efficacy
• Cost-effective
• Biocompatible

Lead Inventor:

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

Patent Information:

Patent Status

Tech Ventures Reference:

• IR CU20006

• Licensing Contact: Joan Martinez