Consensus-driven target product profiles for curative sickle cell disease gene therapies

IF 4.6 2区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL

Molecular Therapy-Methods & Clinical Development Pub Date : 2024-06-22 DOI:10.1016/j.omtm.2024.101287

Daima Bukini, Julie Makani, Joseph McCune, Dennis Lee, Cathy Bansbach, Serena De Vita, Dominic Kemps, Elianna Amin, Jonathan Spector, John Tisdale

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Abstract

Therapeutic innovation to address sickle cell disease (SCD) is at an historical apex, characterized by a drug discovery, development, and commercialization landscape that includes potentially curative gene therapies. Given the wide geographic distribution of SCD, with a major presence in Africa, it is imperative that new medicines are designed to meet the specific needs of persons with SCD everywhere. Target product profiles (TPPs) detail the desired attributes of new medicines and serve as a guide for drug developers. To support research efforts for curative treatments for SCD, we mobilized a large multi-disciplinary expert group to generate consensus-driven TPPs for and SCD gene therapies, utilizing a modified Delphi methodology supplemented with virtual workshops. The main findings are TPPs that describe 20 minimal and optimal criteria for novel gene therapy products in categories of scope (3 criteria), performance/safety (11 criteria), manufacturing (4 criteria), and administration (2 criteria). TPPs for and products differed in some performance/safety criteria and all criteria pertaining to manufacturing and administration. These outputs will ideally support development of durable treatments that are safe, efficacious, and practical for persons with SCD in global settings.

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治疗镰状细胞病基因疗法的共识驱动目标产品图谱

针对镰状细胞病（SCD）的治疗创新正处于历史的顶峰，其特点是药物发现、开发和商业化的格局包括可能治愈疾病的基因疗法。鉴于 SCD 的地理分布广泛，主要分布在非洲，当务之急是设计新药以满足各地 SCD 患者的特殊需求。目标产品简介 (TPP) 详细描述了新药的理想属性，可作为药物开发人员的指南。为了支持 SCD 治疗方法的研究工作，我们动员了一个大型多学科专家组，利用改良的德尔菲方法并辅以虚拟研讨会，为 SCD 基因疗法制定了共识驱动的 TPPs。主要研究结果是TPPs，描述了新型基因治疗产品的20项最低和最优标准，包括范围（3项标准）、性能/安全性（11项标准）、生产（4项标准）和管理（2项标准）。在某些性能/安全标准以及与生产和管理有关的所有标准方面，针对不同产品的技术选择方案存在差异。这些成果将为开发安全、有效且实用的持久治疗方法提供理想支持，以造福全球 SCD 患者。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Molecular Therapy-Methods & Clinical Development Biochemistry, Genetics and Molecular Biology-Molecular Biology

CiteScore

9.90

自引率

4.30%

发文量

163

审稿时长

12 weeks

期刊介绍： The aim of Molecular Therapy—Methods & Clinical Development is to build upon the success of Molecular Therapy in publishing important peer-reviewed methods and procedures, as well as translational advances in the broad array of fields under the molecular therapy umbrella. Topics of particular interest within the journal''s scope include: Gene vector engineering and production, Methods for targeted genome editing and engineering, Methods and technology development for cell reprogramming and directed differentiation of pluripotent cells, Methods for gene and cell vector delivery, Development of biomaterials and nanoparticles for applications in gene and cell therapy and regenerative medicine, Analysis of gene and cell vector biodistribution and tracking, Pharmacology/toxicology studies of new and next-generation vectors, Methods for cell isolation, engineering, culture, expansion, and transplantation, Cell processing, storage, and banking for therapeutic application, Preclinical and QC/QA assay development, Translational and clinical scale-up and Good Manufacturing procedures and process development, Clinical protocol development, Computational and bioinformatic methods for analysis, modeling, or visualization of biological data, Negotiating the regulatory approval process and obtaining such approval for clinical trials.

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