By Ratish Krishnan, Associate Director of Cell and Gene Therapy Bioprocessing Segment, MilliporeSigma
Gene therapies have come a very long way since the first clinical trials back in the early 2000s. Viral vectors and adeno-associated viruses (AAVs) have been studied for over 50 years, and their biology is far better understood than in the early days of gene therapy. As a result, gene therapy products with enhanced safety profiles have been approved, and initial long-term efficacy data has been collected. These first successes are driving more development and significant investment in the field. In fact, over 1,000 clinical trials are currently ongoing in the gene therapy space, which has become one of the most heavily funded biotech sectors. The urgency, uniqueness, and uncertainty surrounding gene therapy manufacturing creates many challenges. Let us take a look into the crystal ball of gene therapy to address unanswered questions and plan for uncertainty beyond 2020.