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LogicBio Therapeutics Announces Strategic Collaboration and Option Agreement with CANbridge Pharmaceuticals Leveraging Gene Editing and Gene Delivery Platforms

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- LogicBio grants exclusive option to license LB-001 in Greater China; investigational treatment for methylmalonic acidemia based on GeneRide™ platform

- Deal also grants worldwide license for AAV sL65, the first capsid based on LogicBio proprietary sAAVyTM platform, to support development of CANbridge gene therapy programs for Fabry disease, Pompe disease and options for two additional indications

- Agreement includes upfront payment in addition to up to $581 million in options and milestones payments, and up to double-digit royalties

LEXINGTON, Mass., April 27, 2021 /PRNewswire/ -- LogicBio Therapeutics, Inc. (LogicBio - Nasdaq: LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced a strategic collaboration and option agreement with CANbridge Pharmaceuticals, Inc., a biopharmaceutical company delivering life-changing therapeutics built upon a foundation in China.

"We are very pleased to be collaborating with CANbridge whose breadth of experience in developing and commercializing innovative drugs will allow us to help more patients around the world including those in China, which is a large and widely underserved market," said Frederic Chereau, president and chief executive officer of LogicBio. "This collaboration further validates the significant potential of our GeneRide and sAAVy platforms and their unique approaches to gene insertion and gene delivery."

Under the terms of the agreement, CANbridge is granted an option to an exclusive license for LB-001, an investigational in-vivo gene editing technology based on LogicBio's GeneRideTM platform for the potential treatment of methylmalonic acidemia (MMA) in Greater China (China, Taiwan, Hong Kong and Macau).

CANbridge would assume responsibility and costs for all future development, regulatory, commercial, and potentially manufacturing activities in the territory upon exercise of said option.The agreement also grants CANbridge a worldwide license for the adeno-associated virus (AAV) sL65, the first capsid produced from LogicBio's sAAVyTM platform, and development support on gene therapy candidates for the treatment of Fabry and Pompe disease plus two optional indications.

LogicBio is eligible to receive an upfront payment of $10 million. This payment triggers the development of two gene therapy candidates for Fabry and Pompe disease based on AAV sL65. In addition, it grants options for a LB-001 license in Greater China and two additional undisclosed gene therapy programs. The agreement also includes options payments, clinical, regulatory and commercial milestone payments of up to $581 million and up to double-digit royalties on net sales.

"We look forward to advancing our rare disease program with LogicBio's next generation, best-in-class capsid and gene editing technologies as we build our gene therapy capability and develop new rare disease treatments in Greater China and worldwide," said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals, Inc. "Partnering with LogicBio is a vital part of our strategy to build a world class rare disease program."

"It is an exciting time to be involved in genetic medicines, as the sector is rapidly evolving and more innovative therapeutic approaches are emerging. LogicBio's next generation capsid with its ability to precisely target liver tissue, its potential to overcome a risk of limited potency and immunogenicity, and its more efficient manufacturing yield is well positioned to overcome challenges of older-generation medicines. Through this collaboration we look forward to advancing CANbridge gene therapy programs," said Mariana Nacht, Ph.D., chief scientific officer of LogicBio.

"Together both of our companies aim to develop and commercialize rare disease treatments, which are desperately needed in China and the rest of the world. This new partnership positions us well to reach patients who often have no or limited treatment options available including patients affected by MMA," said Daniel Gruskin, M.D., senior vice president and head of clinical development at LogicBio.

About LogicBio Therapeutics, Inc.

LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood. The company's proprietary GeneRide™ platform is a new approach to precise gene insertion that harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. LogicBio's cutting-edge sAAVyTM capsid development platform is designed to support development of treatments in a broad range of indications and tissues. The company is based in Lexington, MA. For more information, visit https://www.logicbio.com/.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. is a biopharmaceutical company accelerating development and commercialization of treatments for orphan diseases and rare cancers to address unmet medical needs.

CANbridge has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases. In greater China, where it is a recognized leader in rare diseases, CANbridge has an exclusive licensing agreement to commercialize Hunterase®, an enzyme replacement therapy for the treatment of Hunter syndrome (also known as mucopolysaccharidosis type II), developed by GC Pharma and marketed in more than 11 countries worldwide. CANbridge also has a collaborative agreement with the Horae Gene Therapy Center at UMass Medical School for the research and development of gene therapies to treat rare genetic diseases.

For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com

Forward-Looking Statements

This press release contains "forward-looking" statements within the meaning of the federal securities laws, including with respect to the Company's research and development efforts under its collaboration with CANbridge and the potential of the GeneRide and sAAVy capsid platforms. These are not statements of historical facts and are based on management's beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Company's plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Company's current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Company's filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Company's Annual Report on Form 10-K filed on March 16, 2020, the Company's Quarterly Report on Form 10-Q filed on May 11, 2020, and the Company's subsequent filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Media Contacts:

Bill Berry
Berry & Company Public Relations
W: 212-253-8881
C: 917-846-3862
bberry@berrypr.com

Jenna Urban
Berry & Company Public Relations
W: 212-253-8881
C: 203-218-9180
jurban@berrypr.com

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SOURCE LogicBio Therapeutics, Inc.