加拿大细胞和基因治疗的商业化：现状、挑战和机遇。

IF 7.5 2区医学 Q1 PHARMACOLOGY & PHARMACY

Drug Discovery Today Pub Date : 2025-08-13 DOI:10.1016/j.drudis.2025.104453

Logan S. Germain , Louise M. Winn

{"title":"加拿大细胞和基因治疗的商业化：现状、挑战和机遇。","authors":"Logan S. Germain , Louise M. Winn","doi":"10.1016/j.drudis.2025.104453","DOIUrl":null,"url":null,"abstract":"<div><div>Cell and gene therapies (CGTs) offer transformative treatments for certain genetic diseases and cancers, with a growing number of global approvals. Yet Canadian commercialization lags behind the USA and Europe. This review identifies key barriers and proposes policy solutions informed by international examples. A policy-oriented environmental scan was conducted using regulatory documents, peer-reviewed literature, government reports, and industry publications. Barriers and solutions are organized into four domains: regulation, manufacturing, pricing and reimbursement, and access and equity. Despite recent investment downturns in this therapeutic area, Canada’s approval of its first clustered regularly interspaced short palindromic repeats (CRISPR)-based therapy and chimeric antigen receptor (CAR) T-cell expansion suggest future growth. Strategic reforms could improve domestic CGT innovation, affordability, and equitable access.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 10","pages":"Article 104453"},"PeriodicalIF":7.5000,"publicationDate":"2025-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Commercialization of cell and gene therapy in Canada: Current landscape, challenges and opportunities\",\"authors\":\"Logan S. Germain , Louise M. Winn\",\"doi\":\"10.1016/j.drudis.2025.104453\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Cell and gene therapies (CGTs) offer transformative treatments for certain genetic diseases and cancers, with a growing number of global approvals. Yet Canadian commercialization lags behind the USA and Europe. This review identifies key barriers and proposes policy solutions informed by international examples. A policy-oriented environmental scan was conducted using regulatory documents, peer-reviewed literature, government reports, and industry publications. Barriers and solutions are organized into four domains: regulation, manufacturing, pricing and reimbursement, and access and equity. Despite recent investment downturns in this therapeutic area, Canada’s approval of its first clustered regularly interspaced short palindromic repeats (CRISPR)-based therapy and chimeric antigen receptor (CAR) T-cell expansion suggest future growth. Strategic reforms could improve domestic CGT innovation, affordability, and equitable access.</div></div>\",\"PeriodicalId\":301,\"journal\":{\"name\":\"Drug Discovery Today\",\"volume\":\"30 10\",\"pages\":\"Article 104453\"},\"PeriodicalIF\":7.5000,\"publicationDate\":\"2025-08-13\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Drug Discovery Today\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S1359644625001667\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Drug Discovery Today","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1359644625001667","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}

引用次数: 0

摘要

细胞和基因疗法（cgt）为某些遗传疾病和癌症提供变革性治疗，全球批准的数量越来越多。然而，加拿大的商业化落后于美国和欧洲。本次审查确定了主要障碍，并根据国际范例提出了政策解决方案。政策导向的环境扫描使用监管文件、同行评审文献、政府报告和行业出版物进行。障碍和解决方案分为四个领域：监管、制造、定价和报销，以及获取和公平。尽管最近在该治疗领域的投资有所下降，但加拿大批准了首个基于聚集规律间隔短回文重复序列（CRISPR）的治疗和嵌合抗原受体（CAR） t细胞扩增，这预示着未来的增长。战略改革可以改善国内CGT创新、可负担性和公平获取。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Commercialization of cell and gene therapy in Canada: Current landscape, challenges and opportunities

Cell and gene therapies (CGTs) offer transformative treatments for certain genetic diseases and cancers, with a growing number of global approvals. Yet Canadian commercialization lags behind the USA and Europe. This review identifies key barriers and proposes policy solutions informed by international examples. A policy-oriented environmental scan was conducted using regulatory documents, peer-reviewed literature, government reports, and industry publications. Barriers and solutions are organized into four domains: regulation, manufacturing, pricing and reimbursement, and access and equity. Despite recent investment downturns in this therapeutic area, Canada’s approval of its first clustered regularly interspaced short palindromic repeats (CRISPR)-based therapy and chimeric antigen receptor (CAR) T-cell expansion suggest future growth. Strategic reforms could improve domestic CGT innovation, affordability, and equitable access.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Drug Discovery Today 医学-药学

CiteScore

14.80

自引率

2.70%

发文量

293

审稿时长

6 months

期刊介绍： Drug Discovery Today delivers informed and highly current reviews for the discovery community. The magazine addresses not only the rapid scientific developments in drug discovery associated technologies but also the management, commercial and regulatory issues that increasingly play a part in how R&D is planned, structured and executed. Features include comment by international experts, news and analysis of important developments, reviews of key scientific and strategic issues, overviews of recent progress in specific therapeutic areas and conference reports.