Exegenesis Bio and Modalis Therapeutics have entered into a research collaboration and licence agreement to advance MDL-201, an investigational treatment for Duchenne muscular dystrophy (DMD). 

Under the agreement, Modalis will receive rights to use EMC181, an engineered muscle-targeting adeno-associated virus (AAV) capsid developed by Exegenesis Bio, for MDL-201. The collaboration combines Exegenesis Bio's delivery technology with Modalis' CRISPR-GNDM® (Guide Nucleotide-Directed Modulation) epigenome editing technology.

DMD is a rare genetic disorder caused by a lack of dystrophin, a protein that helps maintain muscle fibre structure and function. The condition causes progressive weakness and affects skeletal, cardiac and respiratory muscles. While several treatment approaches, including nucleic acid-based medicines, have expanded treatment options, providing long-term disease control across a broad range of patients remains challenging.

MDL-201 is being developed to increase the production of utrophin in muscle tissue. Utrophin is a naturally occurring protein that has structural and functional similarities to dystrophin. Modalis aims to activate the body's own utrophin gene using CRISPR-GNDM® without cutting both strands of DNA.

The approach is intended to provide sustained and selective activation of utrophin in muscle tissue. As utrophin is produced from a gene separate from the dystrophin gene, targeting its expression could offer a treatment approach that is not dependent on the specific dystrophin mutation.

Exegenesis Bio's EMC181 is an engineered AAV capsid designed to target muscle tissue while reducing delivery to the liver. Combining the capsid with the CRISPR-GNDM® payload is intended to improve delivery to muscle and reduce exposure to non-target tissues.

The companies will work together to advance MDL-201 through research and development, with the aim of progressing towards non-clinical and clinical development. The programme is intended to explore a mutation-agnostic approach that could potentially be applicable to a broad range of people with DMD.

The collaboration brings together Exegenesis Bio's AAV capsid development capabilities and Modalis' CRISPR-GNDM® epigenome editing technology. Modalis will contribute its development experience from programmes including MDL-101, while Exegenesis Bio will provide its muscle-targeting and liver-detargeting capsid technology.

The agreement will become effective on 14 September 2026.

 

Source: businesswire.com

Report Abuse