2023 Gene Therapy Landscape Review

The review highlights the growing importance of understanding immunogenicity in gene therapy, considering over 80 withdrawn or suspended trials in the field. The focus on immunogenicity aids in informed vector identification for gene therapy. The use of proprietary vectors, like STRIVE™ and AIM™, is emphasized for efficient downstream processes. In the context of neurological disorders, specifically Duchenne muscular dystrophy, the gene therapy space is rapidly expanding, with a notable increase in assets. A substantial portion of these assets, particularly 78.3%, is in the preclinical stage, primarily targeting neurological, eye, and blood disorders.

AAV vectors remain the dominant delivery system, constituting 53.7% of the gene therapy space, but interest in non-viral vectors, especially LNPs, is on the rise. The past year witnessed significant drug approvals for various disorders, reflecting the field’s progress. Most trials are in phase 1/2, indicating a focus on safety and dosing, but the expectation is for more assets to progress to higher phases in the coming years. Despite a dip in investment between 2020 and 2022, 2023 recorded a notable increase to $85 billion, signaling renewed interest and potential growth in the gene therapy space, driven by recent drug approvals and expanding indications.