Viral vector-based gene therapies in the clinic

Area of Science:

• Gene Therapy
• Molecular Medicine
• Biotechnology

Background:

• Gene therapies offer a promising approach to treat diseases previously incurable by conventional methods.
• Viral vectors are widely utilized for gene delivery due to their high transduction efficiency.
• Decades of research have led to approved viral vector-based gene therapies for various diseases.

Purpose of the Study:

• To review the diversity of viral vectors used in gene therapy.
• To analyze approved viral vector-based gene therapy products and their applications.
• To discuss the current clinical landscape and translational challenges of in vivo viral vector gene therapies.

Main Methods:

• Systematic review of approved gene therapy products.
• Analysis of over 200 active clinical trials utilizing viral vectors.
• Critical assessment of translational challenges and potential solutions.

Main Results:

• 13 approved viral vector-based gene therapy products and their clinical applications were reviewed.
• Analysis of 200+ active trials revealed diverse therapeutic applications of various viral vectors.
• Key translational challenges for in vivo gene therapy were identified.

Conclusions:

• Viral vector-based gene therapies have demonstrated significant clinical success and hold vast therapeutic potential.
• Ongoing clinical trials continue to expand the applications of gene therapy.
• Addressing translational challenges is crucial for the advancement of in vivo viral vector gene therapies.