Experts Provide Recommendations for Regulation of Medicinal Products Based on Gene Editing

Researchers from the Scientific Centre for Expert Evaluation of Medicinal Products of the Russian Federation analyzed the existing international experience and regulatory requirements relating to the development of medicinal products based in genome editing of postnatal somatic cells.

Somatic cell genome-editing systems are one of the last technologies in gene therapy for treatment of monogenic hereditary cancer or HIV. Gene editing can be used to change or remove a defective gene using transcription activator-like effector nuclease (TALEN), clustered regularly interspaced short palindromic repeat (CRISPR), or zinc-finger nuclease (ZFN).

According to the authors, regulatory and legislative authorities must take a special approach to the development, manufacture, and assessment of medicinal products based on genome editing, while also considering the ethical aspects of their use. Since most of the requirements and recommendations for the development of such products are limited to risk evaluation and off target effects, the authors recommended consideration of marketing authorization in the regulatory approach.

Read the full article (Russian) in Bulletin of the Scientific Centre for Expert Evaluation of Medicinal Products.