Danaher, a United States-headquartered life sciences conglomerate, announced a strategic partnership in Shanghai on Monday between its operating company Cytiva and CorrectSequence Therapeutics, a Shanghai-based biotech company.

The aim of the partnership is to jointly develop innovative gene therapy solutions based on lipid nanoparticle delivery systems to produce breakthroughs in genetic editing technology for the treatment of hereditary diseases.

Recent advancements in medical science, particularly in gene editing technology, have engendered new hopes for the treatment of genetic disorders. The single-base gene editing technology by CorrectSequence Therapeutics boasts high precision and low off-target effects, enabling precise and permanent alterations to individual base pairs in the vast genome. This offers a new possibility for curing hereditary diseases caused by genetic mutations.

However, overcoming major challenges, including tissue delivery, scalable production and quality control, is crucial for the clinical application of base editing technology in gene therapy.

Mou Xiaodun, CEO of CorrectSequence Therapeutics, said the company's original base editing technology has cured almost 20 patients with beta-thalassemia and sickle cell anemia, and this collaboration is expected to accelerate the development of an in vivo delivery platform. Cytiva's GMP-compliant production platform will also complement the Shanghai company's innovative gene editing platform, reducing development and time-to-market cycles, she said.

"Both parties will focus on chronic metabolic diseases, aiming to achieve long-term control with a single injection, and providing a more disruptive treatment option for patients in China and globally," said Mou.

Fang Zhuo, chief scientific officer of Danaher China, said the collaboration will leverage Cytiva's gene editing therapy process development platform to drive gene therapy from technical research and development to scaled production and clinical application. In addition, Danaher's mass spectrometry, chromatography and flow cytometry technologies will be introduced for quality control throughout the synthesis and delivery processes.

"Looking ahead, we aim to collaborate with top local scholars, academic and medical institutions, and innovative technology companies in a broader range of research and innovation transformation fields," Fang said.

"By leveraging Danaher's mature technologies and processes, we will accelerate clinical applications, benefiting more patients in China and globally."

Li Lei, president of Cytiva in China, said, "Through this collaboration with CorrectSequence Therapeutics, we hope to establish a replicable success model for the future, supporting the construction of the gene therapy industry ecosystem in China and enhancing the overall innovation capabilities and international competitiveness of the domestic biopharmaceutical industry."