Viral vector-based gene therapies in the clinic: An update

Area of Science:

• Biotechnology
• Molecular Biology
• Medical Science

Background:

• Gene therapy has seen significant progress due to innovations in vector engineering and virology.
• The U.S. Food and Drug Administration (FDA) has approved seven new viral vector-based gene therapies since 2021, with five utilizing adeno-associated virus (AAV) vectors.

Purpose of the Study:

• To provide an updated analysis of the clinical landscape of viral vector-based gene therapies.
• To highlight new FDA-approved products and ongoing clinical trials by vector type, disease indication, and clinical phase since 2021.

Main Methods:

• Review of FDA-approved gene therapies and ongoing clinical trials.
• Analysis of viral vector types, including adeno-associated virus (AAV), herpes simplex virus (HSV), adenovirus, and lentiviral vectors.
• Categorization of therapies by disease indication (hematologic, neuromuscular, dermatologic, neurogenetic, oncology, immune-related disorders) and clinical phase.

Main Results:

• Five of the seven new FDA-approved gene therapies since 2021 employ AAV vectors, confirming their leading role in in vivo gene delivery.
• Approved therapies target a range of diseases, including hematologic, neuromuscular, dermatologic, and neurogenetic disorders.
• Non-AAV vectors like HSV and adenovirus are being explored in cancer trials, while lentiviral vectors are used in oncology and immune disorders.

Conclusions:

• Viral vector gene therapy is a maturing field, moving towards precision platforms capable of treating both rare monogenic and complex diseases.
• Advances in capsid engineering allow for tailored vector selection based on specific anatomical targeting needs.
• The expanding clinical applications and regulatory approvals underscore the growing potential of gene therapy in modern medicine.