On October 25, 2021 CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, reported 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 (Press release, CANbridge Life Sciences, OCT 25, 2021, View Source [SID1234591931]). 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.
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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.