Purecodon (hong Kong) Biopharma Ltd.

Purecell Biomedical Technology Company Limited

Zhenhua Sun

Chijian Zuo

Yinjia Gao

Yuping Liu

Yang Zhao

Qiangbo Hou

Yingwen Li

Zonghao Qiu

Ke Huang

Hui Cao

Jiafeng Zhu

Chaoxuan Jing

Binxi Ge

The present disclosure relates to a recombinant nucleic acid molecule and an application thereof in preparation of a circular RNA, and in particular, to a recombinant nucleic acid molecule for preparing a circular RNA, a recombinant expression vector, a circular RNA, a composition, a method for preparing a circular RNA, a method for expressing a target polypeptide in a cell, a method for screening a target coding region sequence, a system for screening a target coding region sequence, and a method for screening a ribozyme recognition site sequence. The recombinant nucleic acid molecule provided by the present disclosure provides a Clean PIE system with a novel structure for preparing a circRNA in vitro, which can avoid introducing additional exon sequences into the circular RNA, improve the sequence accuracy of circular RNA molecules, reduce changes in a secondary structure of the circular RNA, and further reduce the immunogenicity of the circular RNA, and has a good application prospects in the fields of nucleic acid vaccines, expression of therapeutic proteins, gene therapy, and the like.

Recombinant nucleic acid molecule and application thereof in preparation of circular RNA

The present disclosure relates to a recombinant nucleic acid molecule based on point mutation of translation initiation element and use thereof in the preparation of circular RNA, and in particular to a recombinant nucleic acid molecule for preparing circular RNA, a recombinant expression vector, circular RNA, a composition, a method for preparing circular RNA, a method for expressing a target polypeptide in a cell, a method for preventing or treating a disease, a method for editing a translation initiation element sequence, and a system for editing a translation initiation element sequence. The recombinant nucleic acid molecule provided by the present disclosure provides a novel Clean PIE system for the in vitro preparation of circular RNA, which can avoid introducing additional exon sequences into circular RNA, improve sequence accuracy of circular RNA molecules, reduce changes in a secondary structure of circular RNA, and then reduce immunogenicity of circular RNA, and has a good application prospect in the fields of nucleic acid vaccines, expression of therapeutic proteins, gene therapy, etc.

Recombinant nucleic acid molecule based on point mutation of translation initiation element and use thereof in preparation of circular RNA

The present disclosure belongs to the technical field of gene therapy, and specifically relates to a series of lipid compounds as well as lipid vectors, nucleic acid lipid nanoparticle compositions, and pharmaceutical preparations containing the same. The compound having the structure of formula (I) provided by the present disclosure may be used in combination with other lipid compounds to prepare a lipid vector, which exhibits pH responsiveness, has high encapsulation efficiency for nucleic acid drugs, and greatly enhances the in-vivo delivery efficiency of nucleic acid drugs. Moreover, it is possible to select lipid compounds with different structures as lipid vectors to adjust the enrichment of nucleic acid drugs in different organs, thereby having good market application prospect.

Lipid compound as well as lipid vector, nucleic acid lipid nanoparticle composition, and pharmaceutical preparation comprising the same

The present invention belongs to the technical field of gene therapy, and particularly relates to a series of lipid compounds as well as a lipid carrier, nucleic acid lipid nanoparticle composition and pharmaceutical preparation containing the same. A compound having a structure of a formula (I) provided by the present invention can be used for preparing a lipid carrier together with other lipid compounds, and exhibits pH response, and the entrapment efficiency to a nucleic acid drug is high, which greatly improves in-vivo delivery efficiency of the nucleic acid drug; and furthermore, a lipid compound with a specific structure can be chosen as a lipid carrier based on an organ in which the nucleic acid drug needs to be enriched, having a good market application prospect.

Lipid compound as well as lipid carrier, nucleic acid lipid nanoparticle composition and pharmaceutical preparation containing same

The present disclosure relates to a recombinant nucleic acid molecule of the transcriptional circular RNA and its application in protein expression. Specifically, the present disclosure relates to a recombinant nucleic acid molecule of the transcriptional circular RNA, recombinant expression vector, pre-circularized RNA, circular RNA, recombinant host cell, pharmaceutical composition and protein preparing method. The transcription product of the recombinant nucleic acid molecule in this present disclosure is a circular RNA which containing specific IRES element. IRES element can increase the protein expression level of circular RNA in eukaryotic cells, achieve efficient and persistent expression of protein. It has important application value in many fields like: Preparation of mRNA infectious disease vaccines, therapeutic mRNA tumor vaccines, mRNA-based dendritic cell tumor vaccines, mRNA-based gene therapy, mRNA-based chimeric antigen receptor T cell therapy, and protein supplement therapy.

Recombinant nucleic acid molecule of transcriptional circular RNA and its application in protein expression

This invention relates to a medium system and a method for ex vivo expansion of natural killer (NK) cells. This invention directly cultures Ficoll-separated PBMC by using immobilized anti-CD137 and RetroNectin, and uses OK-432 as a biological effector under the co-existence of GM-CSF, IL-4, IL-2, IL-15, and IL-21 for ex vivo activation and proliferation of NK cells, creating an efficient method for ex vivo expansion of NK cells. The expression rate of NK cells CD3-CD16+/CD56+ prepared by the method is as high as 92.5% or more. After 14 days of culture, NK cells can be expanded 1000 to 2000 times and have strong in vitro cytotoxic activity.

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Suzhou, China

Zhenhua Sun