Spring Is Here – A Focus on Cultivating R&D Innovation

Spring Is Here – A Focus on Cultivating R&D Innovation

Spring is regarded as an exciting season of new growth and opportunity in many areas of life – certainly so in the farming regions in the upper Midwest where I grew up. After a long, cold winter, spring brings a sense of energy, excitement, vitality, and hope for the season to come. It is a season when we at Poseida share updates on some of the exciting R&D innovation that is happening at the Company.

To celebrate that progress and share our enthusiasm, we recently held a virtual R&D Day - the first such event focused on our genetic medicine platforms and programs. If you did not have an opportunity to join us live, a replay will be available on our website for a limited time and I encourage you to check out our progress [link].

For those who follow Poseida, you will know that at our core we are a genetic engineering platform technology company. We continue to cultivate and invest in novel technologies and approaches and believe that we are on the cutting edge of innovation with products already emerging for oncology, rare diseases and beyond. Our ongoing investment in these emerging areas requires perseverance and maintaining a long-term strategic view to unlock the promise of these new therapies for patients. It is core to our identity as a company.

As a side note, we also plan to have another R&D Day later in the Fall to highlight progress in the allogeneic cell therapy side of our business. Stay tuned.

The Season is Now for Our Non-Viral Approaches to Gene Editing and Gene Therapy

We are excited about our progress not just because of the science we are sharing, but because of the evolution and trajectory of the industry that are aligning beautifully with our progress and approach. The gene editing/gene therapy industry, and AAV-based gene therapies in particular, have been in a Winter of sorts – struggling with safety, efficacy and commercial adoption.? However, we are now seeing early signs of Spring and quickly growing interest in non-viral approaches to gene editing/gene therapy.

This new energy and interest in non-viral approaches is reinforced by regulatory recognition that non-viral approaches are desperately needed to bring new gene editing and gene therapy therapies to many patients. Candidly, viral approaches have simply failed to deliver on the promise for a variety of reasons, including significant safety concerns and other limitations – but the hope of better technological approaches is on the horizon, and we are leading the way forward. Perhaps the best sign in the U.S. is in the many recent comments by Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research at FDA, who has been very vocal about the agency’s support and flexibility and the high need for non-viral approaches for gene editing/gene therapy.

We are energized by the fact that we appear to be entering a season where our long-term vision and investments in innovative non-viral technologies and approaches are poised to breakthrough into mainstream recognition. The time is now to plant the seeds for growth.

Planting Seeds With A Few Highlights

Trying to highlight all the exciting science from R&D Day is beyond the scope of this posting, but a few highlights worth planting as a seed to encourage a deeper look:

  • KLKB1 and Factor VIII Programs: We shared updated data on two genomic medicine applications that demonstrate we are well on our way. Both these programs have significant potential in metabolic and hematology indications and with a profile that can enable future application of these approaches into broader disease areas.
  • Cas-CLOVER: We shared data confirming and validating the benefits and advantages of our proprietary Cas-CLOVER high fidelity nuclease for site-specific gene editing in multiple applications and disease areas. We continue to believe that Cas-CLOVER is the cleanest gene editing system available and our supporting evidence is growing daily.
  • Novel Lipids and DNA Delivery: We have achieved multiple breakthroughs in delivery technology, including novel lipids for in vivo delivery of our technology and also innovations specifically to enable better in vivo non-viral DNA delivery – which has been a challenge for many others.
  • Site-Specific Super piggyBac: We continue to make progress on our next generation gene editing/insertion technology, including data with unprecedented on-target efficiency. We believe this system represents the next exciting leap forward in genetic engineering.

Overall, our strong progress continues toward our vision of genetic medicine programs that can truly deliver on the promise of genomic therapies that are transformational for patients. We have started to demonstrate that we are on track to open up treatment options, including re-dosing, dose titration, and even an option to modulate, or dial down, expression, that have eluded other technologies and are out of reach for viral approaches.

Springing Forward

Once again, I encourage you to take a look at R&D Day and see for yourself the amazing work that is going on at Poseida to pursue the possibilities of what our technology can do for patients.

A special shout out to the leading academic experts who joined us for R&D Day, including Marc?Riedl, MD, MS, Professor of Medicine at?University of California, San Diego, and Steven W. Pipe, MD, Professor of Pediatrics and?Pathology, University of Michigan.? Support of these key academic luminaries is important and exemplifies how we like to work in collaboration with others who share our vision and passion.

I am continually amazed by the incredible scientific advancements made by our team and the commitment and diligence that they show in pursuing them. I am thankful daily for the opportunity to work with such a passionate and talented group of people and proud of my Poseida colleagues who show up every day to push the limits on creativity and innovation in the pursuit of better treatment options for patients with cancer, rare diseases and beyond.

Finally, a part of our strategy and culture is to seek collaborators and partners who share our vision of the future of genomic medicines – evidenced by our existing relationships with Roche and Astellas in cell therapy. In gene editing/gene therapy, we continue to evaluate conversations about partnership to find the right opportunities and the right partners to expand our impact. We know that there is broad and deep opportunity for our novel technologies. We also know that we cannot do it alone and so are motivated to find like-minded partners to come alongside us because patients are waiting.

Spring is here – time to grow.

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