RARE Daily

CANbridge and Stitchr Global Collaborate to Develop Gene Therapy for Dystrophinopathies

October 25, 2021

China-based global rare disease-focused CANbridge Pharmaceuticals has entered into a research collaboration and license agreement with Scriptr Global for the development of a gene therapy treatment targeting dystrophinopathies.

Photo: James Xue, founder, chairman and CEO of CANbridge Pharmaceuticals

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.

Scriptr Global is developing several proprietary platforms created by Douglas Anderson from the University of Rochester to modify DNA, RNA and protein expression in highly targeted approaches.

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, founder, chairman and CEO of CANbridge Pharmaceuticals.

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.

CANbridge’s pipeline of 13 drug assets target some of the most prevalent rare diseases and rare oncology including Hunter syndrome (MPS II) and other lysosomal storage disorders, complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases as well as glioblastoma multiforme (GBM).

Author: Rare Daily Staff

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