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SineuGene screens for new AAV capsid variances specifically targeting CNS, in order to accelerate gene therapy in CNS.

SineuGene and PackGene collaborate on screening for AAV capsid variances for CNS-specific gene delivery. We created the AAV capsid variance library by randomly replacing amino acids with or inserting that into the corresponding sequences located on the AAV capsid surface, in accordance with the newly-published AAV capsid protein structure. We screen for the new capsids on BBB on chip in vitro or by injections of the capsid libraries intrathecally into macaque or intravenously into marmoset in vivo. In combination with high-throughput sequencing and AI, the validated new capsid with high permeability for the non-human primate CNS may help to end up with new AAV serotypes for future clinical applications in brain disorders.

Focusing on the development of oligonucleotide for the treatment of neurological diseases, and offering a SineuGene-specific disease-allele treatment for the diseases.

SineuGene focuses on therapeutic interventions for neurological disorders through a robust oligonucleotide drug development platform encompassing modules, including?sequence design, chemical modification,?efficacy screening?and?CNS delivery. By designing customized sequence libraries based on precision medicine, the company conducts comprehensive screening and evaluations using in vitro?and in vivo?disease models, including disease mutant cell lines, patient-derived iPSCs (induced pluripotent stem cells), and patient-derived animal models. These assessments aim to evaluate drug efficacy and minimize off-target effects, ultimately aiding in the development of highly specific oligonucleotide therapeutics to halt disease progression.