REGENXBIO Enters Strategic Partnership with Nippon Shinyaku for Development of Gene Therapies

On January 14, 2025, REGENXBIO Inc. (NASDAQ: RGNX) disclosed in an 8-K filing with the Securities and Exchange Commission (SEC) that the company has established a Collaboration and License Agreement with Nippon Shinyaku Co., Ltd. This collaboration focuses on the development and commercialization of gene therapies for Mucopolysaccharidosis II (MPS II) and Mucopolysaccharidosis I (MPS I).

Under the terms of the agreement, REGENXBIO is set to receive $110 million upfront with the potential to gain up to an additional $700 million based on the achievement of certain milestones. These include $40 million earmarked for development and regulatory milestones and $660 million tied to sales milestones. Additionally, REGENXBIO will benefit from significant double-digit royalties on net sales in the U.S. and select Asian countries. The company will lead the manufacturing of the gene therapies for clinical and commercial distribution in the Licensed Territory.

The collaboration delineates that Nippon Shinyaku will handle the commercialization of the licensed products in the Licensed Territory, while future clinical development will be spearheaded by REGENXBIO in the U.S. and by Nippon Shinyaku in other areas of the Licensed Territory. Noteworthy is that REGENXBIO will maintain exclusive rights to, and keep 100 percent of proceeds from, the sale of the Priority Review Voucher for RGX-121 in the event of potential approval.

Regarding termination clauses, the agreement allows for mutual termination under specified conditions, such as material breach, challenges to licensed patents, or changes in control of one of the parties. Additionally, standard provisions for representation and warranties, indemnification obligations, and intellectual property protocols are included.

This transaction is expected to formally close by the end of the first quarter of 2025, subject to regulatory greenlights and other customary closing conditions. The full agreement is set to be included in REGENXBIO’s Quarterly Report on Form 10-Q for the quarter ending March 31, 2025.

In a press release, REGENXBIO President and CEO Curran M. Simpson expressed confidence in the partnership, highlighting the potential impact of the gene therapies on patients. The novel treatments, RGX-121 and RGX-111, are poised to transform the landscape of MPS diseases, with RGX-121 potentially securing FDA approval by late 2025.

Investors and industry observers alike are keen to see the outcomes of this collaboration between REGENXBIO and Nippon Shinyaku, especially as it paves the way for innovative gene therapies in the treatment of rare genetic disorders.

This news underscores REGENXBIO’s commitment to advancing gene therapies and underscores the company’s strategic endeavors to introduce life-changing treatments to patients in need.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read REGENXBIO’s 8K filing here.

About REGENXBIO

REGENXBIO Inc, a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company’s products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase I/II clinical trial for the treatment of Duchenne muscular dystrophy.