Regulatory Considerations for Gene Therapy Products in the US, EU, and Japan
نویسندگان
چکیده
Developers of gene therapy products (GTPs) must adhere to additional regulation beyond that of traditional small-molecule therapeutics, due to the unique mechanism-of-action of GTPs and the subsequent novel risks arisen. We have provided herein a summary of the regulatory structure under which GTPs fall in the United States, the European Union, and Japan, and a comprehensive overview of the regulatory guidance applicable to the developer of GTP. Understanding the regulatory requirements for seeking GTP market approval in these major jurisdictions is crucial for an effective and expedient path to market. The novel challenges facing GTP developers is highlighted by a case study of alipogene tiparvovec (Glybera).
منابع مشابه
The development of regulatory standards for gene therapy in the European Union.
This note examines the EU’s efforts to regulate gene therapy, considering the Union’s resolve to establish scientifically, economically, and morally sound parameters acceptable to its varied constituency. This includes discussion of legal and ethical considerations, biotechnology goals in the EU, and EU-wide uniform regulations. THE DEVELOPMENT OF REGULATORY STANDARDS FOR GENE THERAPY IN THE EU...
متن کاملDNA Vaccines: Regulatory Considerations and Safety Aspects.
DNA vaccines have great potential as preventive or therapeutic vaccines against viral, bacterial, or parasitic diseases as well as cancer, and may also be used as gene therapy products. Although many human and veterinary DNA vaccines have been investigated in laboratory trials, only four of these have been approved for commercial use. In this paper an overview of the regulatory requirements for...
متن کاملH∞ Sampled-Data Controller Design for Stochastic Genetic Regulatory Networks
Artificially regulating gene expression is an important step in developing new treatment for system-level disease such as cancer. In this paper, we propose a method to regulate gene expression based on sampled-data measurements of gene products concentrations. Inherent noisy behaviour of Gene regulatory networks are modeled with stochastic nonlinear differential equation. To synthesize feed...
متن کاملفناوری ویرایش ژن کریسپر ـ کَس 9 از منظر حقوق مالکیت فکری و ایمنی زیستی
In recent years, inexpensive and fruitful gene editing techniques such as CRISPR-Cas9 and NaAgo have revolutionized the biotechnology industry. Genetically edited organisms, gene therapy, treatment of diseases such as AIDS and editing human cells are some of the marvelous applications of such technologies. Using such technologies in large scale or granting exclusive rights on their products or ...
متن کاملThe Role of Gene Therapy in Cartilage Repair
The key principle of gene delivery to articulations by direct intra-articular injection is to release complementary DNA(cDNA)-encoding medical products that will lead to maintained, endogenous production of the gene products withinthe articulation. In fact, this has been accomplished for both in vivo and ex vivo gene delivery, using several vectors,genes, and cells in some animal models. Some c...
متن کامل