ToolGen Completes U.S. Patent Registration for Core CRISPR Cas9 RNP Technology; Files U.S. Lawsuit Related to CASGEVY®

- U.S. patent granted for CRISPR-Cas9 RNP platform technology in the world's largest biotech market
- Next-generation platform that enhances editing efficiency and accuracy strengthens ToolGen's global patent portfolio
- Following actions in Europe, ToolGen initiates U.S. litigation concerning CASGEVY®, seeking fair recognition of foundational technology

SEOUL, South Korea, Nov. 19, 2025 /PRNewswire/ -- ToolGen (KOSDAQ: 199800; CEO Jong-sang Ryu), a leader in genome editing technology, announced that the United States Patent and Trademark Office has issued U.S. Patent No. 12,473,559 related to ToolGen's core foundational technology—direct intracellular delivery of the CRISPR-Cas9 ribonucleoprotein (RNP) complex.

Concurrently with this U.S. patent registration, ToolGen filed a patent-infringement lawsuit in the United States concerning the production and sale of CASGEVY®. ToolGen believes that CASGEVY®, the world's first CRISPR-based genome-editing therapy, practices technology claimed in the newly issued patent.

This grant secures rights to a next-generation platform technology that significantly improves genome-editing efficiency and accuracy in the U.S. market. ToolGen has already secured key CRISPR-Cas9 RNP-related patents in South Korea, Europe, Japan, Hong Kong, and Australia. With the U.S. grant, ToolGen further consolidates a robust global patent portfolio.

Traditional approaches commonly introduced Cas9 via DNA vectors or mRNA, requiring intracellular expression time and raising safety concerns, including off-target editing. ToolGen's patented approach delivers Cas9 directly as a protein complexed with guide RNA (RNP), avoiding DNA/mRNA-related cytotoxicity, eliminating the risk of foreign DNA insertion, and improving safety through reduced off-target activity. CRISPR RNP delivery is widely used in therapeutic development as well as in animal and plant genome editing.

ToolGen's U.S. patent covers methods of directly delivering into cells a CRISPR RNP complex formed by combining Cas9 protein and guide RNA at a predetermined molar ratio. ToolGen believes the defendants use this technology in the manufacture of CASGEVY®.

CASGEVY® is the first approved CRISPR-based genome-editing therapy and is indicated for patients with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). It has received approvals and has been commercialized in major regions, including the U.S. and Europe.

"This U.S. patent registration reaffirms, in the world's largest market, the importance of ToolGen's CRISPR-Cas9 RNP technology," said Jong Sang Ryu, CEO of ToolGen. "Together with our actions in Europe, we intend to obtain fair recognition for the value of ToolGen's intellectual property in the United States and to help build a healthy and sustainable industry ecosystem."

ToolGen remains open to constructive engagement with Vertex Pharmaceuticals and continues to prefer collaborative pathways where appropriate. ToolGen's aim is not to limit or delay patient access to CASGEVY® or other genome-editing therapies, but to ensure appropriate recognition and compensation for the use of ToolGen's foundational technology—whether through licensing or other suitable means—thereby supporting continued innovation and responsible patient access.

About ToolGen

ToolGen (KOSDAQ: 199800) is a biotechnology company focused on developing and commercializing gene-editing platform technologies and applications across human therapeutics, agriculture, and industrial biotechnology.

CASGEVY® is a registered trademark of its owner.

This press release contains forward-looking statements that are subject to risks and uncertainties. Actual outcomes may differ materially from those expressed or implied. ToolGen undertakes no obligation to update forward-looking statements, except as required by law.

SOURCE ToolGen