Modalis Therapeutics Corporation announced that it has decided to pursue development of MDL-105 as In-house pipelines. MDL-105 is a novel gene therapy to treat dilated cardiomyopathy (DCM) by upregulating the Titin gene. Titin is the largest human protein, which consists of ~35,000 amino acids coded by 17kbp cDNA and is the third most abundant protein in muscle.

It has been difficult to upregulate the expression of such large proteins with conventional technologies, but the company able to achieve this with CRISPR-GNDM® technology. Historically, cardiomyopathy has been classified as a cardiovascular disease, but mechanistically it can be considered a muscular disease, and the company believes that it is a plug-and-play indication to which accumulated technologies and know-how in the area of muscular diseases can be directly applied. MDL-105 is a pipeline that the company been researching in-house, and due to the scale of the disease, The company already received inquiries from several biotech companies and major pharmaceutical companies to consider partnering with even though the company still in the early stages of development.

The timing of the licensing agreement has not yet been determined. In addition to the filing of a patent application, a reasonable evaluation of the business feasibility is required before the Company decides to include a newly developed product in In-house pipeline, which was approved by the Board of Directors. With the addition of MDL-105, the company now has five products in In-house pipeline, and together with the three pipelines in Collaboration, the company now has a total of eight products in pipeline.