BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that it has entered into a research collaboration and license agreement with Scriptr Global, Inc., for the development of a gene therapy treatment targeting dystrophinopathies. CANbridge will gain exclusive worldwide rights to develop, manufacture and commercialize a gene therapy candidate for the treatment of dystrophinopathies, using Scriptr Global’s Stitchr™ platform, a proprietary ribozyme-mediated RNA assembly technology. Scriptr Global will be responsible for research, while CANbridge will assume all responsibilities for development, manufacturing, regulatory, and commercialization.

The financial terms of the agreement include an upfront payment, development and sales payments, upon hitting certain milestones, as well as royalties based on net sales.

“We are pleased to be aligning with Scriptr Global and the Stitchr technology platform, which we believe has the potential to revolutionize the dystrophinopathy gene therapy field,” said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals, Inc.

“The need for a transformative therapeutic approach for those individuals and families impacted with dystrophinopathies is great. Scriptr Global is delighted to undertake this important work with CANbridge utilizing Scriptr Global’s novel platform technology,” stated Keith Alkek, Co-Founder Chairman and CEO of Scriptr Global, Inc.

About dystrophinopathies

Dystrophinopathies are X-linked genetic muscular diseases which include Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and DMD-associated dilated cardiomyopathy (DCM). DMD usually presents in early childhood and is characterized by rapidly progressive muscle degeneration and weakness, leading to loss of ambulation by about 12 years of age. BMD is characterized by later-onset skeletal muscle weakness. Cardiomyopathy is a common cause of morbidity and death in both DMD and BMD patients. DCM is characterized by left ventricular dilation and congestive heart failure, usually with no clinical evidence of skeletal, or voluntary muscle involvement. The incidence of DMD is estimated to be 1/3,500 – 1/5,000 male births worldwide and 1/4,560 in China. The incidence of BMD is estimated to be 1/18500 - 1/30,000 male births, according to the National Organization for Rare Disease and published peer reviews. The prevalence of DCM is unknown.

About SCRIPTR GLOBAL

Scriptr Global, Inc., is a pre-clinical, biotechnology company committed to advancing novel therapeutic approaches to address challenging and life-threatening health conditions. Scriptr Global is developing several unique and proprietary platforms created by Douglas Anderson, Ph.D., from the University of Rochester to modify DNA, RNA and protein expression in highly targeted approaches. For more information, please email: info@scriptrglobal.com

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. is a China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies.

CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with significant market potential, targeting some of the most prevalent rare diseases and rare oncology.

These include Hunter syndrome (MPS II) and other lysosomal storage disorders (LSDs), complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases as well as glioblastoma multiforme (GBM).

CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments. CANbridge global partners include, but are not limited to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Medical School (UMass) and LogicBio.

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

View source version on businesswire.com: https://www.businesswire.com/news/home/20211025005278/en/