Novel second-generation gene therapy is more efficient than the benchmark vector in a mouse model with spinal muscular atrophy (SMA), according to initial data presented at the American Society of Gene and Cell Therapy (ASCGT) annual meeting.

More precisely, the new gene therapy extended the lifespan of the animals in a dose-dependent manner, restored their muscle function as demonstrated by the righting and grid tests, and showed better neuromuscular junction innervation close to wild-type animals. It also did not have the liver toxicity caused by the benchmark vector.

Importantly, animals treated with the second-generation gene therapy had higher expression of the SMN1 gene in the brain and spinal cord as well as lower peripheral tissue compared to the mice treated with the benchmark vector.

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“These data encourage us to support the continued development of this second-generation vector as a potential best-in-class gene therapy for SMA,” Yunxiang Zhu, PhD, vice president and head of global research at CANbridge Pharmaceuticals, said.

Read more about gene therapy for SMA

“We are seeking to develop a next-generation gene therapy for SMA that leverages the advances in gene therapy that have occurred since the first gene therapy was developed, over a decade ago,” said Guangping Gao, PhD, president of the ASCGT from UMass Chan Medical School in Worcester.

The novel gene therapy carries a codon-optimized human SMN1 gene under the control of the endogenous SMN1 promoter. This ensures that the expression of the gene is highly efficient and regulated across different tissues. Safety and efficacy can also be improved even at a lower dose.

The novel gene therapy is being codeveloped by CANbridge Pharmaceuticals and the Horae Gene Therapy Center at the UMass Chan Medical School in Worcester.


CANbridge-UMass Chan Medical School gene therapy research presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. News release. CANbridge Pharmaceuticals; May 17, 2022.