Expectations On The Pathway To GMP For Gene-Modified Cell Therapies

The global gene and cell therapy industry has experienced rapid growth and a number of treatments are advancing toward commercialization. The ribonucleoprotein (RNP) modality has the potential to improve the treatment of infectious diseases, cancers, and a wide range of rare diseases including neuro-degenerative diseases. This growth is increasing demand for GMP-compliant CDMOs capable of providing CRISPR components, viral vectors, guide RNAs (gRNA), and therapeutic complexes such as RNPs.

However, gene and cell therapy developers face significant challenges when initiating clinical programs that rely on CRISPR technology. Increasing regulatory scrutiny, GMP manufacturing lead times, and complex production processes complicate the task of moving a program into the clinic and scaling to serve larger patient populations. Learn more about the manufacturing obstacles developers face and how a robust understanding of the RNP’s critical process parameters and critical quality attributes (CQAs) is crucial for ensuring product consistency, enhancing patient safety, and maintaining high product quality and regulatory compliance.