In this interview with Dr. Ruby Yanru Chen-Tsai, CEO of Applied StemCell (ASC), we explore ASC’s recent acquisition by QHP Capital, new hires being added by the company, upcoming product launches, and the company’s plans for massive expansion in 2024 and beyond.
We also explore the critical importance of developing readily available, off-the-shelf cell and gene therapies (CGT) at scale and for affordable price points. Enjoy!
Interview with Ruby Chen-Tsai, CEO of Applied StemCell
Cade Hildreth: Applied StemCell (ASC) was recently bought by QHP Capital. What was the significance of this event?
Dr. Ruby Yanru Chen-Tsai: The significance of this event is that this investment will support ASC’s rapid future expansion. We are in a position to both leverage QHP’s strategic network and expand our presence as a CDMO that specializes in cell and gene therapies. In particular, we are committed to advancing regenerative medicine treatments within the biopharmaceutical and research sectors.
This partnership with QHP Capital is a significant step, because it combines ASC’s technology with QHP’s strategic network and industry expertise. Our collaboration will aim to expedite the realization of ASC’s vision to revolutionize the regenerative medicine market by developing iPSCs with superior efficacy, safety, and accelerated, scalable manufacturing capabilities.
As our partners, Matt Jenkins and Jeff Edwards of QHP Capital have shared, there are critical challenges facing the regenerative medicine market, such as accelerating pre-clinical research and achieving manufacturing economies of scale. They believe that Applied StemCell aligns with QHP’s investment strategy, because their approach is to provide strategic capital and support to companies with established offerings who are poised for industry-scale growth.
With QHP’s support, ASC is positioned for tremendous expansion in 2024 and beyond.
Cade Hildreth: How did you know that QHP was the right business partner to support ASC in its future growth trajectory?
Dr. Ruby Yanru Chen-Tsai: Today, one of the biggest challenges preventing the advancement of cell and gene therapies is the lack of an off-the-shelf product for patients. At this time, most cell therapy products rely on autologous treatments, meaning a patient’s own cells are used for each treatment. These therapies range from several hundred thousand dollars to $2 million or more per treatment and take months to produce, making the production of these therapies a very long and costly process.
Thankfully, researchers are actively innovating new ways to develop cell and gene products. Rather than sourcing the materials for these therapies from individual patients, researchers are looking to create a universal product that can be given to multiple patients.
Induced pluripotent stem cells (iPSCs) have the potential to help in this area. iPSCs are artificially derived cells with the capacity to turn into any cell in the human body. These new cells aid in basic research, drug screening, toxicological studies, disease modeling, cell therapy, and genomic medicine, among other applications.
QHP chose to invest in Applied StemCell, because we are a CDMO specializing in cell and gene therapy. Through ASC’s proprietary gene editing technologies and iPSC platforms, QHP is seeking to advance regenerative medicine while capitalizing on a global market that is estimated to be over $20 billion and growing at a rate of 15% per year over the next several years.
On our end, we knew QHP Capital was the right business partner for us because they have a proven track record of providing strategic capital and support to companies with established offerings who are poised for industry-scale growth. Additionally, they understand the importance of producing allogeneic, off-the-shelf cell and gene therapies at scale and for lower price points.
We spoke with more than 100 companies before deciding on QHP as our strategic partner, so the vetting process was very thorough and intensive.
Cade Hildreth: What is your five-year vision for ASC?
Dr. Ruby Yanru Chen-Tsai: Our vision is to revolutionize cell therapy space by providing high quality iPSCs and a cutting-edge gene editing platform that will enable new cell therapies to be unlocked. Using these revolutionary cell medicines, we will save human lives and offer cures at faster and more cost-effective way through the us of off-the shelf, on-demand treatments.
Cade Hildreth: What team members will be coming on board and what strengths will they bring to the company?
Dr. Ruby Yanru Chen-Tsai: We are excited to be adding two accomplished professionals to our C-level executive team, George Hong as our new Chief Commercial Officer (CCO) and Timothy J. Largen as our new Chief Operating Officer (COO).
George Hong (CCO) brings 20+ years of experience in academic and industry roles within the life science sector, serving as a sales, marketing, and commercial executive. George previously worked as CCO and President at Exellgene, Head of North America Sales at Advanced Instruments, Strategic Marketing Manager at MilliporeSigma, and Director of Sales and Marketing at Transgenomics.
Timothy J. Largen (COO) has a 25-year career marked by significant contributions to Advanced Therapy Medicinal Products (ATMPs). His experience spans GMP manufacturing, quality management, process engineering, business operations, and regulatory affairs. Previously, Tim held positions that include VP of Commercial Manufacturing at Adrian Biotech, VP of Manufacturing Science and Technology (MSAT) at RoslinCT, and 8+ years in commercial manufacturing and quality positions at Dendreon.
With these talented and experienced new C-level executives joining our team, we are ideally positioned to pursue our mission of making cell therapy affordable, scalable, and ready-to-use, so that we can save human lives.
we now offer unique, fully integrated solutions from research, process engineering, early clinical phases, all the way through commercial manufacturing.”
Cade Hildreth: How would you describe ASC’s proprietary TARGATT™ platform?
Dr. Ruby Yanru Chen-Tsai: ASC’s TARGATT technology revolutionizes gene editing with fast and precise integration of large DNA fragments (up to 20 kb) into a specific safe harbor locus. This method ensures stable integration of a single copy gene at a transcriptionally active site with high efficiency. ASC’s proprietary TARGATT platform offers versatility for various applications, including the development of cell lines/master cell banks (MCBs) with large fragment knock-ins, bioproduction, and library construction.
SSelect is our next-generation gene editing technology. It stands for “Site-specific, efficient, large–cargo targeting”. For SSelect, we evolved the TARGATT enzyme, which is a bacterial enzyme, so that it can recognize human genomic sequences. As a result, SSelect can do gene knock-in in one-step both in vivo (gene therapy) or ex vivo (cell therapy).
This expands the application of TARGATT from allogeneic cell therapy to autologous cell therapy, as well as to gene therapy. We have two patents covering the SSelect technology, one that is granted and another one that is pending.
To summarize, SSelect fills in all of the technology gaps here, plus it expands the application from allogeneic cell therapy to all cell and gene therapy modalities. It really pushes the boundaries of existing gene editing tools that are currently on the market.
Cade Hildreth: What key advantages are experienced by clients who use ASC’s GMP-grade allogeneic iPSC platform and your TARGATT™ technology?
Dr. Ruby Yanru Chen-Tsai: ASC’s technology stands out in several aspects. One is efficiency for large cargo knock-in, where TARGATT and SSelect have the highest efficiency.
Simplicity and ease of use is another important feature of our technology. This directly translates to into cost savings and product consistency.
Even more importantly is the reduction in off-target mutations, because this dramatically enhances product safety. This is one of the most important things that FDA looks at when doing product safety evaluations.
In conclusion, our TARGATT technology address three critical pain points in gene editing and offers safe and efficacious solutions for clinical development. These are:
- Safety: Our approach addresses issues linked to random integration, including position effects, gene silencing, and genomic instability arising from multiple transgene copies.
- Cost Reduction: Clients can expect approximately one-tenth of the cost per dose, coupled with significantly accelerated timelines.
- FTO: Applied StemCell holds the Freedom to Operate (FTO) for the utilization of gene editing technology.
Cade Hildreth: What upcoming product and service releases does Applied StemCell have planned for 2024 (and beyond)?
Dr. Ruby Yanru Chen-Tsai: I’m excited to share that we have three important offering that are soon to be launched. These include:
- GMP-grade TARGATT Master iPSCs for allogeneic cell product development and manufacturing
- Next generation genome editing SSelectTM for all cell gene therapy modality application (large cargo knock-in system)
- End-to-end integrated solution provider from development to commercial manufacturing in cell medicine
Cade Hildreth: As a leading CRO/CDMO who supports the manufacture of cell and gene products, what capacity do you have to support preclinical and clinical-level manufacturing?
Dr. Ruby Yanru Chen-Tsai: Our involvement spans across R&D projects, fully equipped facilities, process development, establishment of cell banks (MCB, WCB), and GMP manufacturing (clinical Phase I, II, III), all within the regulated scope of the US FDA cGMP regulations.
We have a footprint of 5 GMP Manufacturing clean rooms, 5 cell culture rooms and 3 molecular biology rooms. We also have additional space in the same building that we can expand into to accommodate future growth needs. We are planning to do more expansion in 2025 so that we can accommodate more phase 3 and commercial manufacturing projects.
For our manufacturing scope of work (SOW), we can support:
- iPSCs, iPSC-derived cells
- Gene-edited cells
- CAR-T, Blood Cells, and NK Cells
Cade Hildreth: Strategically, why did ASC choose to specialize in iPSC and gene editing technologies over other emerging technologies?
First, iPSCs are self renewable, meaning that these cells provide unlimited source of starting materials for cell products. Secondly, iPSC can differentiate and become virtually any type of cells in a human body, so it provides a diverse spectrum of cell products. One iPSC cell line can be developed into specific cell types such as neurons, cardiomyocytes, blood cells, immune cells, pancreatic cells and so on.
Furthermore, there is no ethical concerns because iPSC is reprogrammed or generated from adult cells. No embryos are needed to make iPSC.
These unique properties make iPSC a diverse, unlimited starting cell for developing innovative cell therapies, for disease modeling-in -dish, and for cell-based drug screening.
iPSCs are the center and basis for regenerative medicine or cell replacement therapy. For example, if you have age-related macular degeneration, meaning your retinal cells are not functioning, you can replace them with functioning iPSC derived retinal cells. That’s regenerative medicine, and that’s what the future medicine is all about. That’s also why we started Applied StemCell.
We have been doing CRISPR for over 10 years, probably the longest compared to other similar companies. In fact, we were the first commercial organization to license the CRISPR technology from MIT Broad Institute. Our proprietary TARGATT gene knock-in technology has several features that are superior to CRISPR including large cargo KI, high efficiency, up to 10x better than CRISPR. On top of that, we have clear FTO and license terms for our partners and customer, both for research and therapeutic purposes.
I am even more excited about our next generation gene editing technologies, which includes SSelect gene insertion technology. SSelect is going to open an unprecedented opportunity for Applied StemCell. More importantly, this disruptive technology could overcome some of the most challenging bottlenecks in cell and gene therapy. For example, SSelect offers higher efficacy and safety to cell products by its feature of safe genomic insertion site and no off-target mutations. SSelect also provides manufacturing efficiency and product consistency by its feature of non-viral cell transfection and proprietary validated genomic site.
Technology is really the core of Applied StemCell and it is also what makes Applied StemCell a better company.
Cade Hildreth: What types of customers does ASC typically serve?
We have a diverse customer base that includes global biopharma companies, biotech companies, therapeutic companies, other CRO/CDMO companies, and academic researchers. Our customers are leaders in iPSC innovation and well funded.
We have established strong and long-term relationships with our customers. Some of them stay with us for many years.
Cade Hildreth: How can people get in touch with you or learn more about ASC?
People can get in touch with us by email [email protected] or by visiting us at AppliedStemCell.com. Thank you!