Modalis Therapeutics : Notice Regarding a Patent Allowance for ‘Method for treating muscular dystrophy by targeting utrophin gene’ in the USA

This document has been translated from the Japanese original for reference purposes only.

In the event of any discrepancy between this translated document and the Japanese original, the original shall prevail. The Company assumes no responsibility for this translation or for direct, indirect or any other forms of damages arising from the translation.

June 22, 2022

Company name: Modalis Therapeutics Corporation

Stock exchange listing: Tokyo Stock Exchange

Code number: 4883

URL: https://www.modalistx.com/en/

Representative: Haruhiko Morita

Notice Regarding a Patent Allowance

for 'Method for treating muscular dystrophy by targeting utrophin gene' in the USA

Modalis Therapeutics Corporation (hereafter, "the Company") hereby announces that the Company has received the following notice of a patent allowance in the United States regarding 'Method for treating muscular dystrophy by targeting utrophin gene', the Company and Astellas Pharma Inc. have jointly filed.

Name	： METHOD FOR TREATING MUSCULAR DYSTROPHY
	BY TARGETING UTROPHIN GENE
Country of application	： USA
Application number	： No 17/320,643
Applicant	： Modalis Therapeutics Corporation、Astellas Pharma Inc.

The patent has already been registered in Japan, but the Company has also received a notice of a patent allowance this time in the USA, which is the base of research and development. By this allowance followed by a registration, products which are developed based on this patent will be protected in US. The Company has been also working toward the establishment of the patent in other countries.

This patent is for a therapeutic concept and molecules that uses CRISPR-GNDM® to treat DMD*. The treatment is based on a mechanism that supplements mis-functioning Dystrophin gene in patients by upregulating the expression of the fetal/juvenile form of the gene, Utrophin. The cDNA length of the Dystrophin gene is 14 kbp, too large to be carried in an AAV vector, and this is a target that takes advantage of the CRISPR-GNDM®, which is gene size agnostic. No disclosure is made for the collaboration program as to this patent correspond to which collaboration program with Astellas.

The impact of this matter on the Company's consolidated financial results for the fiscal year ending December 31, 2022, is expected to be minimal.

• DMD: Duchenne muscular dystrophy. A genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin.