Head of Corporate Development & Portfolio Manager Ascend Gene and Cell Therapies
Q: What are the biggest challenges in the advanced therapy industry?
This is difficult to answer as there are multiple challenges some of which are common to gene therapies, others common to autologous cell therapies and others common to allogeneic/PSC derived cell therapies. Manufacturing and product characterisation are key challenges that the whole field faces to varying degrees with impact on scalability and safety. We need better methods to characterise our products which will help us develop more consistent and scalable processes. This will ultimately improve safety, efficacy and patient access so everyone wins.
Q: What advanced therapy innovations are you most excited about?
The allogeneic cell therapy field is quite exciting and the data that has been reported in the early clinical trials are encouraging. Having off-the-shelf cell therapies is a critical step to widespread adoption. Of course, these products are very difficult to make and are often using a number of cutting-edge technologies. The use of pluripotent stem cells as starting materials may also enable tens of thousands of doses to be produced in a single batch which will really bring down cost-of-goods but for this to be realised there is a need for significant advances in cell selection and fill and finish technologies.
Q: What are your predictions for the next 5 years for the Advanced Therapy Industry?
I expect we will see an increasing number of advanced therapies aimed at treating more common and complex diseases reach late-stage clinical trials. We are already seeing this happening. Take the example of RGX-314, a gene therapy which has entered Phase 3 clinical studies to treat wet-AMD, a common cause of blindness. This is very encouraging and helps validate the notion that advanced therapies are a third target engagement modality that should be considered alongside small molecules and biopharmaceuticals and not a last resort. There are approved biopharmaceuticals that can work very effectively in treating wet-AMD yet there are advantages to using a gene therapy approach that justifies the investment in the discovery and development of RGX-314.