Today, I’d like to touch on the topic of manufacturing, as I believe it is the most critical issue facing cell and gene therapy developers in our quest to deliver safe and effective products to patients.
For many years, product developers have placed nearly exclusive emphasis on proving clinical efficacy of a drug or device, and left manufacturing scale-up as a concern for a later date until after clinical data were gathered.
However, as the complexity of therapies being developed today increases, so too does the complexity of manufacturing these products. There are many issues that need to be taken into consideration early on in the development phase, such as: establishing robust processes that result in consistent cell/gene outputs, ensuring process consistency and product potency, as well as establishing controls for safe and consistent raw materials.
The billion-dollar questions become: How early on in the development phase, and what comes first? Gathering clinical efficacy data or manufacturing scale-up efforts?
From my perspective at DiscGenics, investing in the manufacturing process well into the clinical phase is no longer going to be an option for cell and gene therapy developers. Our industry must address the issues related to product manufacturing and quality early in clinical development if cell and gene therapies are truly going to be successful bringing life-altering and, in many cases, life-saving, therapies to patients in the future.
At DiscGenics, we are currently conducting a Phase I/II trial of IDCT in patients with degenerative disc disease (DDD). In preparation for commercialization, we decided to embark on the scale-up and scale-out of our manufacturing processes before we have clinical data in our hands. We’ve done so based on compelling preclinical data for our cell therapy, as well as a desire to ensure optimization of our in-house manufacturing capabilities and compliance with cGMP regulations in preparation for commercialization.
We recognize that while addressing manufacturing scale-up in parallel to clinical development is critical, it is a significant undertaking, both in terms of resources and capabilities. To support our scale-up initiatives, we recently announced an engagement with CCRM and GE Healthcare. Through this collaboration we are learning from the remarkable scale-up know-how and process development expertise of our colleagues at CCRM and GE Healthcare. We’re looking forward to a productive partnership and to sharing our progress as we continue down the road to commercial-scale manufacturing.