Exegenesis Bio and Modalis Therapeutics have entered a research collaboration and license agreement to advance MDL-201, an experimental treatment candidate for Duchenne muscular dystrophy (DMD).
The agreement, which takes effect on September 14, 2026, combines Exegenesis Bio’s muscle-targeting AAV delivery technology with Modalis’ CRISPR-GNDM epigenome editing platform. The companies aim to accelerate the development of MDL-201 and move the program toward clinical testing.
DMD is a rare genetic disease caused by a lack of dystrophin, a protein that helps maintain muscle cells. The condition causes progressive weakness and affects skeletal, heart and respiratory muscles. Although several treatment approaches have emerged in recent years, developing therapies that can provide long-term control of the disease across a broad patient population remains a challenge.
MDL-201 is being developed to activate utrophin in muscle tissue. Utrophin can perform some functions similar to dystrophin, making it a potential target for DMD treatment. Modalis’ CRISPR-GNDM technology is designed to regulate gene activity without cutting both strands of DNA.
Under the agreement, Modalis will gain rights to use EMC181, an engineered AAV capsid developed by Exegenesis Bio, for the MDL-201 program. EMC181 is designed to preferentially target muscle tissue while reducing delivery to the liver.
The companies believe this combination could improve the delivery of the CRISPR-GNDM payload to muscle while limiting exposure to other organs. They are also pursuing MDL-201 as a mutation-agnostic approach, which could potentially allow it to address a broad range of DMD patients regardless of their specific genetic mutation.
The collaboration brings together Exegenesis Bio’s AAV capsid development capabilities and Modalis’ epigenome editing technology. Modalis will contribute its CRISPR-GNDM platform and development experience, including work from its MDL-101 program.
Zhenhua Wu, CEO of Exegenesis Bio, said the company believes combining EMC181 with Modalis’ CRISPR-GNDM technology could offer a promising approach to muscle targeting in DMD.
Haruhiko Morita, CEO of Modalis Therapeutics, said the partnership brings together precise activation of utrophin with muscle-focused delivery and could help move MDL-201 toward clinical development.
The companies plan to continue research and development of MDL-201, with the next stages expected to include nonclinical development before potential clinical testing.
Modalis said the financial impact of the collaboration on its results for the current fiscal year is expected to be immaterial. The company also said it does not plan to change its current earnings forecast.