HORSHAM, PA — Exegenesis Bio will supply its muscle-targeting AAV delivery technology to Modalis Therapeutics Corp. (TOKYO: 4883) under a research and licensing agreement aimed at advancing an experimental Duchenne muscular dystrophy therapy toward nonclinical and clinical development.
The agreement gives Modalis rights to use Exegenesis Bio’s EMC181 engineered AAV capsid with MDL-201, a therapeutic candidate being developed using Modalis’ CRISPR-GNDM epigenome-editing technology.
EMC181 is designed to target muscle tissue while reducing exposure to the liver. The companies plan to combine the capsid with Modalis’ gene-regulation payload to improve delivery of MDL-201 to skeletal and other muscle tissue.
MDL-201 is designed to selectively increase expression of utrophin, a protein that may compensate for some functions of dystrophin. Duchenne muscular dystrophy results from a deficiency of dystrophin and causes progressive deterioration of skeletal, cardiac and respiratory muscle function.
Unlike gene-editing approaches that cut double-stranded DNA, Modalis’ CRISPR-GNDM technology is designed to regulate gene expression through epigenome editing. The companies are pursuing MDL-201 as a mutation-agnostic approach, potentially allowing it to address patients across different underlying DMD mutations.
Exegenesis is contributing EMC181 and its gene-therapy development platform to the collaboration. Modalis will provide the CRISPR-GNDM payload and development capabilities built through programs including MDL-101.
“EMC181 was developed to enable efficient muscle targeting while reducing liver exposure,” Exegenesis Bio Chief Executive Officer Zhenhua Wu said. He said combining the capsid with Modalis’ payload fits the company’s strategy of applying its AAV technology to differentiated gene therapies.
Modalis Chief Executive Officer Haruhiko Morita said the combination is intended to pair sustained utrophin activation with muscle-targeted delivery as the company works to move MDL-201 toward the clinic.
Modalis said the collaboration is expected to have an immaterial effect on its results for the current fiscal year and does not require a revision to its earnings forecast.
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