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 take a deep dive into 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