Market Analysis and Prospect of Gene Therapy Industry

doi:10.13523/j.cb.2304027

China Biotechnology

2023, Vol. 43

Issue (6): 102-112 DOI: 10.13523/j.cb.2304027

Market Analysis and Prospect of Gene Therapy Industry

FENG Xue-jiao^1,^**(

),HENG Chao¹,YU Xin-yu²,WANG Jun-shu¹

1 Institute of Science and Technology Strategy, Jiangxi Academy of Sciences, Nanchang 330096,China
2 School of Public Policy and Administration, Nanchang University, Nanchang 330096, China

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Abstract

Gene therapy provides a new strategy for the treatment of major refractory diseases with a promising market outlook. By of the end of 2022, a total of 45 gene therapy drugs around the world were approved to be listed, and more than 3 000 drugs were in clinical trials. There are also 4 types of domestic gene therapy products available in China. Currently, the top three leading enterprises in the world account for more than 50% of the market share. As there is an increasing number of patients with rare diseases and tumors in China year by year, the domestic gene therapy market continues to grow. Driven by industry development policies, a number of enterprises such as Yao Ming Ju Nuo, Legend Biology, and Keji Biology have emerged and achieved certain progress. With the gradual improvement of policies, the deepening of scientific research, and the lowering of treatment costs, gene therapy is expected to become a conventional and safe treatment.

Key words： Gene therapy Gene editing Market analysis Rare disease

Received: 14 April 2023 Published: 04 July 2023

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	Xue-jiao FENG
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Cite this article:

FENG Xue-jiao, HENG Chao, YU Xin-yu, WANG Jun-shu. Market Analysis and Prospect of Gene Therapy Industry. China Biotechnology, 2023, 43(6): 102-112.

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https://manu60.magtech.com.cn/biotech/10.13523/j.cb.2304027 OR https://manu60.magtech.com.cn/biotech/Y2023/V43/I6/102

Fig.1 Market size of global gene therapy from 2016 to 2025E E indicates the data of this year is a predicted value Data Source: Frost Sullivan

Fig.2 Distribution of technology source countries in the global gene industry Data Source: Smart Bud Patent Database (PatSnap)

Fig.3 Annual distribution of the number of gene therapy drugs in the market Data Source: Smart Bud Patent Database (PatSnap)

序号	商品名	生产厂家	适应证	疗法分类	相关病毒载体	获批时间 /地区
1	Vitravene	Ionis、Pharma、Novartis	巨细胞病毒性视网膜炎	其他	逆转录病毒	1998年/ 美国、欧盟
2	今又生 (Gendicine)	深圳市赛百诺基因技术有限公司	头颈部鳞状细胞癌	基因修饰腺相关病毒	腺相关病毒	2003年/ 中国
3	Macugen	Pfizer、Eyetech	HIV阳性患者的巨细胞病毒性视网膜炎	其他		2004年/ 美国
4	Rigvir	Latima	黑色素瘤	基因修饰的ECHO-7 肠道病毒	肠道病毒	2004年/ 拉脱维亚
5	安科瑞(Oncorine)	上海三维生物技术有限公司	头颈部肿瘤、肝癌、胰腺癌、宫颈癌等	基因修饰溶瘤病毒	溶瘤病毒	2005年/ 中国
6	Rexin-G	Epeius Biotechnologies (USA)	实体瘤	其他	逆转录病毒	2005年/ 菲律宾、美国
7	Neovasculgen	Human stem cells institute	周边血管动脉疾病	DNA质粒的基因疗法		2011年/ 俄罗斯
8	Glybera	UniQure	治疗严格限制脂肪饮食却仍然发生严重或反复胰腺炎发作的脂蛋白脂酶缺乏症	AAV	AAV	2012年/ 欧盟
9	Kynamro	Ionis Pharma、Kastle	纯合子家族性高胆固醇血症	其他	反义寡核苷酸	2013年/ 美国
10	Defitelio	Jazz	肝小静脉闭塞症伴随造血干细胞移植后肾或肺功能障碍			2013年/ 欧盟
11	Imlygic	Angen Inc.	不能通过手术完全切除的晚期黑色素瘤	溶瘤病毒	单纯疱疹病毒	2015年/ 美国、欧盟
12	Strimvelis	GSK	由于腺苷脱氨酶缺乏症而导致的严重联合免疫缺陷	基因修饰的自体干细胞	逆转录病毒	2016年/ 欧盟
13	Zalmoxis	MolMed	用于造血干细胞移植后患者免疫系统的辅助治疗	基因修饰的异体干细胞	逆转录病毒	2016年/ 欧盟
14	Spinraza	Ionis Pharmaceuticals	脊髓性肌萎缩症	其他	反义寡核苷酸	2016年/ 美国
15	Exondys 51	Sarepta Therapeutics	DMD基因发生51外显子跳跃基因突变的进行性假肥大性肌营养不良(DMD)	其他	反义寡核苷酸	2016年/ 美国
16	Kynriah	Novartis Pharmaceuticals Corporatlon	难治或复发性B淋巴细胞白血病	CAR-T	慢病毒	2017年/ 美国
17	Luxturna	Spark Therapeutics, Inc.	RE65基因突变相关的视网膜萎缩	病毒载体疗法	腺相关病毒	2017年/ 美国
18	Yescarta	Kite Pharma,Inc.	复发或难治性成人大B细胞淋巴瘤	CAR-T	逆转录病毒	2017年/ 美国
19	Invossa-K	TissueGene	退行性膝关节炎	同种异体细胞基因疗法	逆转录病毒	2017年/ 韩国
20	Tegsedi	Ionis Pharmaceuticals	遗传性转甲状腺素蛋白淀粉样变性	其他	反义寡核苷酸	2018年/ 欧盟
21	Onpattro	Alnylam,Sanofi	遗传性转甲状腺素蛋白淀粉样变性	siRNA		2018年/ 美国
22	Givlaari	Alnylam	成人急性肝卟啉症	siRNA		2019年/ 美国
23	Vyondys53	Sarepta Therapeutics	抗肌萎缩蛋白基因外显子53剪切突变的DMD		反义寡核苷酸	2019年 / 美国
24	Waylivra	Ionis Pharmaceuticals, Akcea Therapeutics	家族性乳糜微粒血症综合征成年患者控制饮食之外的辅助疗法		反义寡核苷酸	2019年/ 欧盟
25	Zolgensma	Novartis Pharnaceuticals Corporation	脊髓性肌肉萎缩症	病毒载体疗法	腺相关病毒	2019年/ 美国
26	Zynteglo	Bluebird Bio	β-地中海	基因修饰的自体造血干细胞	慢病毒	2019年/ 欧盟
27	Collategene	大阪大学、风投公司	重症下肢缺血	质粒的基因疗法		2019年/ 日本
28	Libmedly	Orchard Therapeutics (Netherlands) BV	异色性白细胞营养不良	基因修饰的自体造血干细胞	慢病毒	2020年/ 欧盟
29	Tecartus	Kite Pharma, Inc.	成人复发或难治性大B细胞淋巴瘤	CAR-T	逆转录病毒	2020年/ 美国
30	Leqvio	Novartis	成人原发性高胆固醇血症或混合型血脂异常	siRNA		2020年/ 欧盟
31	Oxlumo	Alnylam	原发性高草酸尿症1型(PH1)	siRNA		2020年/ 欧盟
32	Viltepso	NS Pharma	DMD基因发生53外显子跳跃基因突变的进行性假肥大性肌营养不良	mRNA		2020年/ 美国
33	Skysona	Bluebird	早期脑肾上腺脑白质营养不良	基因修饰的自体造血干细胞	慢病毒	2021年/ 欧盟
34	Abecma	Celgene Corporation	成人复发或难治性多发性骨髓瘤	CAR-T	慢病毒	2021年/ 美国
35	Breyanzi	Juno Therapeutics, Inc	成人复发或难治性大B细胞淋巴瘤	CAR-T	慢病毒	2021年/ 美国
36	Delytact	Daiichi Sankyo	恶性胶质瘤	溶瘤病毒	单纯疱疹病毒	2021年/ 日本
37	倍诺达	药明巨诺	二线或以上系统性治疗后成人患者的复发或难治性大B细胞淋巴瘤	CAR-T		2021年/ 中国
38	Amondys 45	Sarepta Therapeutics	进行性假肥大性肌营养不良	mRNA		2021年/ 美国
39	CARVYKTI	传奇生物	复发或难治性多发性骨髓瘤	CAR-T		2022年/ 美国
40	Upstaza	PTC Therapeutics	芳香族 L-氨基酸脱羧酶缺乏症	病毒载体疗法	腺相关病毒	2022年/ 欧盟
41	Roctavian	BioMarin	成人无FVIII 因子抑制史且AAV5抗体阴性的严重血友病A	病毒载体疗法	AAV5	2022年/ 欧盟
42	Amvuttra (vutrisiran)	Alnylam	成人遗传性转甲状腺素蛋白淀粉样变性伴多发性神经病	siRNA		2022年/ 美国
43	Ebvallo	Atara Biotherapeutics	淋巴增殖性疾病	通用型T细胞基因疗法		2022年/ 欧盟
44	Hemgenix	UniQure	成人血友病B	病毒载体疗法	AAV5	2022年/ 美国
45	Adstiladrin	Ferring Pharmaceuticals	高风险非肌层浸润性膀胱癌	病毒载体疗法	腺相关病毒	2022年/ 美国

Table 1 Status of gene therapy drugs on the market

Fig.4 Phases of gene therapy clinical trials Data Source: American Society for Gene and Cell Therapy

Fig.5 Geographical distribution of gene therapy clinical trials Data Source: American Society for Gene and Cell Therapy, ClinicalTrials.gov

Fig.6 Indications addressed by of gene therapy clinical trials Data Source: Evaluate Pharma, RSM US

Table 2 Major gene therapy companies worldwide

Fig.7 Number of new cancer cases in China from 2016 to 2020 Data Source: National Cancer Center

Table 3 Approved gene therapy drugs in China

Table 4 Gene therapy drugs clinical trials in China (excluding CAR-T)

Table 5 Major gene therapy enterprises in China


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