Homology Medicines is focused on advancing our genetic medicines platform as one-time treatments and potential cures for patients with rare diseases
Clinical trials are an important part of the drug development process and are designed to gain an understanding of the safety and efficacy of a therapy in a specific population. Clinical trials are required for a potential therapy to be reviewed and approved by a regulatory agency before potentially becoming available to patients.
Early laboratory research to discover/design potential treatments for a particular disease
Laboratory work continues, including extensive testing of potential treatment in disease models
Regulatory authorities review preclinical data and trial plans and provide clearance to begin human testing
Clinical trials usually involve multiple phases and are designed to evaluate safety and efficacy of a potential treatment in a particular population of people
Regulatory authorities review data from clinical trials and determine whether the treatment should be approved for patient use outside of the clinical trial setting