# US 11,077,206 B2 · Composition for gene therapy or transfection, and preparation method and use thereof

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- Number: US 11,077,206 B2
- Document ID: US11077206B2
- Status: Granted
- Kind: B2
- Application number: 16/465,573
- Filed: 2018-09-06
- Published: 2019-09-26
- Granted: 2021-08-03
- Earliest claimed: 2017-10-09
- Assignees: Shanghai Changzheng Hospital
- Applicants: Shanghai Changzheng Hospital
- Inventors: Jianru Xiao; Wanwan Shen; Xiaopan Cai; Yiyun Cheng
- Web page: https://patentgrep.com/patent/US11077206B2

## CPC

- A61K 48/0041: {the non-active part being polymeric}
- A61K 31/353: 3,4-Dihydrobenzopyrans, e.g. chroman, catechin
- A61K 47/22: Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
- A61K 47/30: Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K 47/34: Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers (A61K47/10 takes precedence)
- A61K 48/00: Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61P 5/00: Drugs for disorders of the endocrine system
- A61P 9/00: Drugs for disorders of the cardiovascular system
- A61P 17/00: Drugs for dermatological disorders
- A61P 25/18: Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
- A61P 29/00: Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
- A61P 35/00: Antineoplastic agents
- A61P 37/02: Immunomodulators
- C12N 15/1137: {against enzymes (viral enzymes C12N15/1131; receptors C12N15/1138)}
- C12N 15/87: Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N 15/88: using microencapsulation, e.g. using {amphiphile} liposome vesicle
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2320/32: Special delivery means, e.g. tissue-specific
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

A composition for gene therapy or transfection includes nucleic acid, natural polyphenol, and cationic molecules. There are several phenolic hydroxyl groups in the natural polyphenol, so as to eliminate free radicals, prevent cancer, fight inflammation, and provide antioxidants feature. Compared with the delivery of cationic molecules alone, the composition can significantly improve the transfection efficiency by about 60-70%, reduce the dose of effective transfection of cationic molecules by about 20 times, thereby reducing the toxicity of transfection and showing better organism compatibility. The composition of the present invention does not introduce other chemical reagents, does not require synthesis, has high biosafety, and can efficiently and safely deliver a plurality of nucleic acid molecules into cells. The composition can be used as a highly effective and low-toxicity nucleic acid drug in the treatment of gene-related diseases, and has good medicinal value.

## Claims

### Claim 1

A composition for gene therapy or transfection, comprising: siRNA or modified siRNA, epigallocatechin gallate, and polylysine cationic polymers.