Gene therapy for metabolic dysfunction-associated steatohepatitis (MASH)

Unmet Need: Therapeutic treatment options for MASH

Metabolic dysfunction-associated steatohepatitis (MASH) can lead to cirrhosis and is the leading cause of liver transplants in the United States. Currently, there is only one FDA-approved treatment on the market for MASH, the daily oral medication resmetirom; other treatment options include lifestyle changes for weight reduction, such as diet and exercise, leaving a huge gap in treatment options for MASH patients.

The Technology: Long-term gene-based therapy for MASH

This technology is a gene-based therapy for metabolic dysfunction-associated steatohepatitis (MASH) that restores retromer protein complex activity, a pathway impaired in MASH that influences RhoA signaling, protein sorting, and lipid metabolism. By enhancing retromer function, downstream molecules such as EHBP1 and SORL1 are stabilized, which leads to restored lipid uptake. This, in turn, helps mitigate the effects of MASH and fibrosis. Compared to daily oral medications, this technology offers long-term benefits in maintaining fat homeostasis in the liver, serving as a potential long-lasting treatment for MASH and other related diseases.

Applications:

Treatment for MASH and other liver diseases
Treatment for neurodegenerative diseases
Research tool for studying retromer dysfunction
Research tool for studying cellular trafficking
Drug discovery screening platform

Advantages:

Cell-type specific targeting
Synergistic with existing therapies
Long-term therapeutic effects

Lead Inventor:

Scott A. Small, M.D.

Patent Information:

Patent Pending

Related Publications:

Tech Ventures Reference:

IR CU25230
Licensing Contact: Jerry Kokoshka