CN Patent

CN118557756B — 一种基于aav的遗传性抗凝血酶缺乏的基因疗法

Assigned to Union Hospital Tongji Medical College Huazhong University of Science and Technology · Expires 2025-04-29 · 1y expired

What this patent protects

本发明公开了一种基于AAV的遗传性抗凝血酶缺乏的基因疗法，属于基因治疗技术领域。本发明首次探索了基于AAV载体介导的hSERPINC1基因递送在治疗遗传性AT缺乏症中的长效性和安全性，证明了其对恢复AT缺乏症小鼠血浆中AT抗原水平和活性，再平衡体内凝血和抗凝系统，及降低小鼠的血栓形成倾向的疗效。通过AAV基因疗法有望彻底治愈AT缺乏症，这一发现为AAV疗法用于治疗AT缺乏症的临床转化提供了临床前基础，为遗传性抗凝血酶缺乏等易栓症患者的长久根治提供了新的治疗方向。

USPTO Abstract

本发明公开了一种基于AAV的遗传性抗凝血酶缺乏的基因疗法，属于基因治疗技术领域。本发明首次探索了基于AAV载体介导的hSERPINC1基因递送在治疗遗传性AT缺乏症中的长效性和安全性，证明了其对恢复AT缺乏症小鼠血浆中AT抗原水平和活性，再平衡体内凝血和抗凝系统，及降低小鼠的血栓形成倾向的疗效。通过AAV基因疗法有望彻底治愈AT缺乏症，这一发现为AAV疗法用于治疗AT缺乏症的临床转化提供了临床前基础，为遗传性抗凝血酶缺乏等易栓症患者的长久根治提供了新的治疗方向。

Drugs covered by this patent

Patent Metadata

Patent number: CN118557756B
Jurisdiction: CN
Classification: —
Expires: 2025-04-29
Drug substance claim: No
Drug product claim: No
Assignee: Union Hospital Tongji Medical College Huazhong University of Science and Technology
Source: FDA Orange Book + USPTO grounding via Google Patents

Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.

Track this patent

Get a daily-checked alert when vulnerability score, expiry, classification, or assignee changes. Email, Slack, or Teams delivery. Pro: 50 watches, Free: 3.