Gene Therapy: An Overview of Approved and Pipeline Technologies

Excerpt

1. Gene therapy is an area of therapeutics aimed at curing, or significantly improving the management of, diseases with few or no treatment alternatives.

2. A large proportion of the candidates for gene therapy include advanced-stage cancer or hematological conditions. In addition, rare or inherited disorders are also frequent targets of gene therapy.

3. While gene therapy developments are still largely in the research stage, companies are increasingly investing in these technologies. Recently, a number of products have been approved outside of Canada or are in the advanced stage of clinical research.

4. The upfront current cost of gene therapy is generally very high. Multi-stakeholder dialogues around management of cost and reimbursement of these products are necessary.

5. Specialized manufacturing facilities, care centres, and clinicians trained to conduct customized procedures for such therapies are vital to ensure accessibility and quality of care.