DEPARTMENT OF BIOCHEMICAL ENGINEERING

MANUFACTURING & COMMERCIALISATION OF STEM CELL & GENE THERAPIES

ATMP Development Workshop

Mechanism of action, toxicity & efficacy

作用机制、毒性和疗效

Dr Bernice Wright Office hours: Mon. 3-4pm, Tues. 4-5pm,

Email: b.wright@ucl.ac.uk Thu. 11am-12pm
 Overview of Conventional Gene Therapy

AAV – adeno-
associated virus

注射进入患者细胞 - 找回？

非病毒载体

治疗性核酸

病毒载体
Yu et al. (2021) Adv Sci (Weinh), 8(14):2100540
用治疗性核酸转导细胞
Approved Gene Therapies

https://alliancerm.org/available-products/
 CAR-T Cell Therapy
分离病人的T细胞 CAR-encoding 编码基因
chimeric 嵌合
antigen 抗原
receptor 受体

病毒类载体将编码基因传递到T细胞中

嵌合抗原受体（CAR）- T细胞疗法

T细胞在其表面表达CAR，被称
为CAR-T细胞

CAR-T细胞扩增后被放回患者的血液中

https://rsscience.com/gene-therapy/?utm_content=cmp-true
Approved CAR-T Therapies

https://alliancerm.org/available-products/
 Stem Cells

https://www.novusbio.com/research-areas/stem-cells
Approved Stem Cell Therapies

Spherox

https://alliancerm.org/available-products/
 Tissue Engineering

支架

生物因素

https://www.weforum.org/agenda/2019/06/30-years-since-the-mouse-with-a-human-ear-what-has-been-achieved/.
Approved Tissue Engineered Products

https://alliancerm.org/available-products/
 Workshop Activity

Describe the preclinical and clinical development of an approved ATMP.

1. Describe the mechanism of action of the drug using an annotated

diagram.

2. Outline the toxicity of the drug, explaining the use of preclinical models
to show this.

3. Outline the efficacy of the drug at preclinical and clinical stages.

• Present those workshop tasks in a PowerPoint presentation with your

group.
• Presentation – 7 min, Q&A – 3 min
• ALL members of the group must contribute to the workshop activity.
 Team B

Kymriah
Team A Team C

Omidubicel
Luxturna

Team D

Stratagraft