Michael Mendicino, Owner, Chief Consultant & Advisor at Hybrid Concepts International, is excited to ‘be part of the emerging technology revolution that is our cell and gene therapy space’. We caught up with him at Cell Therapy Manufacturing & Gene Therapy Congress to discuss the current trends in gene therapies.
For Mendicino, the improvement in gene editing technologies is one of the most important recent developments in the industry. Although not necessarily a new or unfamiliar concept, he emphasises that the new technologies that are continuously emerging, such as Zinc Finger, TALENs and CRISPR, are vital to the future of gene therapies.
Mendicino sees a number of potential breakthroughs that are currently in the process of development that will allow for these new technologies to become viable treatment options:
‘For 2017 everyone is looking to see a licenser for marketing authorisation for the CAR-T product either coming from Novartis or from Kite Pharma. I think that will be tremendous for the field and will legitimise the field, not only for the companies that apply for the BLA (Biologics License Application) and get approval , but also for other companies that are not that far behind them, specifically in the CAR-T space, but also in cell and gene related product spaces.’
When building a BLA strategy that meets all the requirements, Mendicino breaks it down into three disciplines that need to be considered separately, as well as in conjunction with each other. Firstly, there is the ‘chemistry, manufacturing or controls’, or the ‘quality’ of the product. The second is ‘pharmacology and toxicology’, also known as the ‘pre-clinical or the non-clinical’, and the third is the clinical. Often these three considerations are handled in parallel, but this may need revising throughout the process. This is due to factors such as the product itself, as well as ‘the clinical indications sought, the patient population, the recruitment rates and whether or not the company has gone through some major CMC change that required scale up for commercial inventory.’
Watch the full interview as Michael Mendicino explores the methods of helping gene therapies progress to market above or here.