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The original translational ideas and therapeutic targets of SineuGene come from their concrete basic research and the deep understanding of disease mechanisms underlying neurological disorders.

SineuGene is developing gene therapy, gene editing, and recombinant protein drugs for neurological diseases, especially ALS. The original intellectual property rights came from Tsinghua University, where innovative research has been carried out for 10 years in JiaLab. Currently, we have applied for more than 10 international/domestic patents for our pipelines including innovative therapies and advanced technologies.

SineuGene has developed the next generation disease models for neurological and neurodegenerative diseases, including ALS. We utilize CRISPR-Cas9-based approach to knock in the highly frequent and penetrant disease mutations into the corresponding gene loci of fly, mice, and miniature Bama pig. Compared to the traditional transgenic models that overexpress mutant gene for many times than their corresponding endogenous one, our unique disease models reflect the disease nature, which allows us to do better disease mechanism studies and to evaluate the potential therapeutic interventions in a more efficient way.

SineuGene respects basic research achievements and focuses on reliable animal model construction, which helps to develop their novel therapeutic targets. SineuGene aims at screening for new AAV serotypes with high ivity and permeability for CNS gene delivery, which provides new solutions for huge unmet medical needs for gene therapy for neurological diseases. SineuGene upgrades new gene editing tools and develops new and cell type-specific gene expression approaches, which helps to come up with novel gene therapies for brain disorders.

SineuGene actively maintains close collaboration and relationship with Tsinghua University, clinicians, patients, CRO companies, and regulatory agencies. SineuGene also is favored by venture capital. By gathering efforts and resources from the whole society, SineuGene aims to accelerate drug development for ALS and other devastating neurological diseases.