ISSUE NO. 36 — Nonclinical Studies in Cell and Gene Therapy

Recent advances in gene therapy have allowed us to approach diseases differently than medicines and surgery—they are “living drugs” that provide cures for a number of conditions by terminating the disease process at the cellular or genetic level. It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure.

However, there are significant challenges associated with the successful development of these complex, leading-edge therapies. The in vivo nonclinical study of cell and gene therapies includes a thorough understanding of on-and-off-target activity, immune responses, and other adverse events, just to name a few. All of which require careful monitoring, and rigorous assessments. 

In Issue 36 of The Altascientist, we examine the essential factors in the development of nonclinical cell and gene therapy, including insights into mitigating complex challenges and maximizing translational opportunities to first-in-human trials.

This comprehensive publication covers the following: 

• ICH S-12 guideline on biodistribution studies
• species selection
• choosing the right vector for your cell and gene therapy studies
• the importance of germline mitigation
• exaggerated on-target effects
• immunogenicity
• nonclinical study planning
• case studies from Altasciences

GENE THERAPY VS. CELL THERAPY: WHAT IS THE DIFFERENCE? 

Gene and cell therapies are often discussed together, but they are not always interchangeable. Some therapies are considered both cell and gene therapies, as they alter genes in specific types of cells. 

Gene therapy involves introducing or substituting a faulty gene. There are three primary vectors employed in gene therapy: adeno-associated virus (AAV), adenovirus, or lentivirus vectors. 

Cell therapy, on the other hand, involves the injection of living cells (either autologous or allogeneic) into a subject. This encompasses immunotherapy, oncology, and regenerative medicine. Examples of cell therapies include CAR-T and natural killer (NK) cell therapies.

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