The gene therapy landscape continues to accelerate in preclinical and clinical research, with programs constantly in development for targeted, personalized medicines. The goal is to safely incorporate genetic alterations to restore and repair the proteins of missing and/or faulty genes. Gene therapy requires DNA and/or RNA delivery and analysis, and while most ongoing research involves therapies being delivered in vivo via adeno-associated viral (AAV) vectors, other in vivo delivery methods are on the rise.

Quantitative, digital, and reverse transcription polymerase chain reactions (qPCR, dPCR, and RT-PCR, respectively) are fast and cost-effective techniques employed by Altasciences. These methods are invaluable for quantitative analysis of gene expression and for analyzing genetic variation in amplified DNA and RNA. This ability to analyze variation from limited samples has made genetic diagnosis easier than ever. But how are PCR analysis techniques applied? And how are they incorporated into studies?

Issue 37 of The Altascientist takes a closer look at these techniques, with in-depth information on:

• qPCR, dPCR, and RT-PCR and their utilities—including droplet digital PCR (ddPCR);
• regulatory considerations;
• PCR applications, advantages, and comparisons; 
• and case studies.

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