Technology
“Full Stack” iPSC Therapeutics
Development of complex iPSC-based therapeutics benefits from an engineering mindset. Each layer of the development and manufacturing process is considered part of the ‘Full Stack’ that leads to finished product. An integrated stepwise approach improves the probability of arriving at the right therapeutic candidate and reduces downstream risks to manufacturing and delivery to the patient.
iPSC as the Material Input
Induced pluripotent stem cells (iPSC) are the subject of The 2012 Nobel Prize in Physiology or Medicine. This discovery unlocked the potential to generate unlimited quantities of authentic human cells for use in research and the development of advanced medicines. Like any new technology, there is know-how and discipline to utilize it effectively.
Engineering to Vasculogenic Cells
We engineer from iPSC to vasculogenic cells, meaning those cells capable of forming healthy blood vessels on their own when delivered into an oxygen-starved tissue. Proprietary methods are identified that yield the vasculogenic cell, and then the cell is tested in a relevant animal model. Arriving at a lead candidate and follow-on candidates is an iterative process.
Accurate Measures of the Product
Development and manufacture from iPSC of an authentic human cell as a therapeutic candidate requires exceptional understanding of the product. Assay development is critical to characterize the product and its function, understand the critical quality attributes (CQAs) relevant for manufacturing, and establish release criteria.
Capacity to Functionalize the Product
Gene modification technology, and specifically the discovery of CRISPR/CAS9, is the subject of The Nobel Prize in Chemistry 2020. This technology is revolutionizing basic research and medicine with precise tools to edit gene sequences. We are integrating this expertise into our platform in order to broaden future applications of our iPSC-derived vasculogenic cell.
Seamless from Cryo to Injection
Cryopreservation, cold-chain logistics, thawing, and mechanics for injection of biologic therapeutics — particularly a cell therapeutic, or “living medicine” — each bring their own challenges and risks. We are utilizing the ProFusion™ Therapeutic Infusion Needle & Compass® CT technologies (Cook Regentec) for efficient intramuscular cell delivery.