China – based biopharma CANbridge Pharmaceuticals Inc. (HKG: 1228) has unveiled a license deal to secure exclusive global rights to a novel, second – generation gene therapy for spinal muscular atrophy (SMA) from UMass Chan Medical School in the United States. The deal includes all development, manufacturing, and commercialization rights for scAAV9 – SMN1p – co – hSMN1, a self – complementary AAV9 gene therapy expressing a codon – optimized human SMN1 gene under the control of an endogenous promoter. Financial details of the agreement were not disclosed.
About SMA and the Gene Therapy
Spinal muscular atrophy (SMA) is a rare genetic disease caused by the lack of a functional survival motor neuron 1 (SMN1) gene, which codes for a protein essential to motor neuron survival. The loss of motor neurons leads to progressive muscle weakness and death at an early age. The most common form of the disease appears between birth and 6 months, often leading to death by the age of 2 if untreated. Gene therapies, such as the one licensed by CANbridge, are designed to deliver a fully functional human SMN1 gene via a viral vector that enters the spinal cord, halting the degeneration of motor neurons.
Development and Preclinical Data
CANbridge has been collaborating with UMass Chan Medical School on the development of this gene therapy, sponsoring preclinical development to assess its potential to outperform the currently approved first – generation gene therapy, Novartis’s Zolgensma (onasemnogene abeparvovec). Animal data presented at various meetings in 2022, including the American Society of Gene and Cell Therapy (ASGCT), showed that UMass’s candidate therapy outperformed a benchmark similar to Zolgensma in several key endpoints, including reduced liver toxicity. The key difference lies in the choice of vector, with UMass’s second – generation vector utilizing the endogenous SMN1 promoter to control gene expression in different tissues.
Expansion of Gene Therapy Portfolio
CANbridge is building a specialist gene therapy portfolio and also announced an amendment to an earlier April 2021 deal with US – headquartered LogicBio Therapeutics Inc. (LogicBio, NASDQ: LOGC). The original deal granted exclusive global rights to develop, manufacture, and commercialize LogicBio’s adeno – associated virus (AAV) sL65 for Fabry and Pompe gene therapies. Under this week’s amendment, CANbridge is also granted non – exclusive global rights to LogicBio’s proprietary manufacturing process for Fabry/Pompe gene therapies. Option rights to sL65 – based therapies for two additional indications and to a second therapy, LB – 001, have been removed from the agreement.-Fineline Info & Tech
