Exegenesis Bio licenses EMC181 capsid to Modalis Therapeutics for DMD gene therapy

USA—Exegenesis Bio and Modalis Therapeutics have entered into a research partnership and licensing agreement to advance MDL 201, an investigational therapy for Duchenne muscular dystrophy (DMD).

The collaboration will combine Modalis’ CRISPR GNDM (Guide Nucleotide Directed Modulation) technology with Exegenesis Bio’s engineered muscle tropic adeno-associated virus (AAV) capsid, EMC181.

The agreement is expected to take effect on September 14, 2026, and will give Modalis rights to use EMC181 to develop MDL 201.

Exegenesis Bio designed EMC181 to improve the delivery of genetic medicines to muscle tissue while limiting exposure to the liver.

The company plans to use the capsid to deliver the CRISPR GNDM-based therapy to muscles affected by DMD.

Targeting utrophin expression

MDL 201 is designed to treat DMD by selectively activating utrophin expression in muscle tissue.

Utrophin is a protein that can perform functions similar to dystrophin, the protein affected by mutations that cause DMD.

By targeting utrophin rather than correcting individual mutations in the dystrophin gene, the therapy could potentially be applicable across different genetic forms of DMD.

Modalis is developing CRISPR GNDM as a gene regulation approach that can alter gene activity without directly changing the underlying DNA sequence.

The companies said combining the technology with EMC181 could improve delivery to skeletal muscle while reducing exposure to organs such as the liver.

They expect the liver detargeting properties of EMC181 to support the safety profile of MDL 201 as development progresses.

DMD is a progressive genetic disorder that causes increasing muscle weakness and loss of muscle function.

Because patients can carry different mutations and disease progression remains a long-term challenge, researchers continue to investigate approaches that could complement existing treatment options.

Companies outline partnership

Exegenesis Bio CEO Zhenhua Wu said the company was pleased to work with Modalis to advance MDL 201.

He said EMC181 was developed to improve muscle targeting while reducing liver exposure and that combining the capsid with Modalis’ CRISPR GNDM technology could offer a new approach to DMD treatment.

Wu added that the partnership forms part of Exegenesis Bio’s strategy of applying its AAV capsid platform to gene therapies targeting diseases with significant unmet medical needs.

Modalis said the partnership is not expected to have a material financial impact during the current fiscal year.

The company also said it had not changed its full-year earnings forecast following the agreement.

In September 2024, the US Food and Drug Administration granted Modalis Therapeutics a rare pediatric disease designation for its investigational gene therapy for congenital muscular dystrophy type 1A, a condition associated with mutations in the LAMA2 gene.

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