Exegenesis Bio and Modalis Partner on New Gene Therapy for Duchenne Muscular Dystrophy

Exegenesis Bio and Modalis Partner on New Gene Therapy for Duchenne Muscular Dystrophy

Exegenesis Bio and Modalis Therapeutics have entered into a research and licensing partnership to develop a new treatment for Duchenne muscular dystrophy (DMD).

DMD is a severe inherited disorder that causes progressive muscle weakness and usually begins in childhood.

The companies will work together on MDL-201, an experimental therapy designed to activate production of a protein called utrophin in muscle tissue.

Utrophin can help support muscle function and may partially compensate for dystrophin, the protein that is missing or does not function properly in people with DMD.

Modalis will use its CRISPR-GNDM gene-regulation technology, while Exegenesis Bio will provide EMC181, a specially engineered AAV vector designed to deliver the therapy more efficiently to muscle tissue.

An AAV vector is a delivery system used to carry therapeutic genetic material into cells.

According to the companies, EMC181 is designed to improve targeting of muscle tissue while reducing exposure to other organs, particularly the liver. This could potentially improve the safety profile of the treatment.

A key feature of the approach is that it may work independently of the specific genetic mutation causing DMD, which could make it relevant to a broader group of patients.

The agreement is expected to take effect on September 14, 2026.