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news.Exegenesis Bio and Modalis Therapeutics have entered a research partnership and licence agreement to progress MDL-201, a therapeutic candidate for Duchenne muscular dystrophy (DMD).
EMC181 aims to address DMD by activating utrophin in muscle using CRISPR-GNDM. Credit: MY STOCKERS / Shutterstock.com.
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The agreement involves the use of Modalis’ CRISPR-GNDM (Guide Nucleotide-Directed Modulation) technology in combination with Exegenesis Bio’s engineered muscle-tropic adeno-associated virus (AAV) capsid, EMC181.
EMC181 is designed for enhanced delivery to muscle tissue.
The arrangement is set to become effective on 14 September 2026.
Under the agreement, Modalis will be granted rights to utilise EMC181 in the development of MDL-201.
The therapy aims to address DMD by selectively and sustainably activating utrophin expression in muscle using CRISPR-GNDM, potentially providing an approach independent of the specific genetic mutation in the dystrophin gene.
The companies intend for this combination to facilitate efficient targeting of muscle tissues and reduce exposure to off-target organs such as the liver.
Employing EMC181 as the delivery vector is expected to enhance the safety profile of MDL-201 due to its liver-detargeting properties.
In addition to DMD’s progressive muscle decline, the disease presents a challenge for long-term disease control in all patients, so alternative approaches are sought to support or complement existing therapies.
Exegenesis Bio CEO Zhenhua Wu said: “We are pleased to collaborate with Modalis to advance MDL-201. EMC181 was developed to enable efficient muscle targeting while reducing liver exposure, and we believe its combination with Modalis’ innovative CRISPR-GNDM payload represents a compelling approach for DMD.
“This collaboration reflects our strategy of applying our next-generation AAV capsid platform to differentiated gene therapies with the potential to address significant unmet medical needs.”
Modalis stated that the financial effects of this partnership are expected to be immaterial for the current fiscal year and no changes to the full-year earnings forecast have been made.
In September 2024, Modalis Therapeutics was awarded a rare paediatric disease designation by the US Food and Drug Administration for its congenital muscular dystrophy type 1a (LAMA2-CMD) gene therapy.
