As part of WuXi AppTec’s ongoing efforts to collaboratively foster new thinking and actionable approaches in advancing breakthroughs for patients, we have launched a new interview series in 2022 – “Delivering on the Promise of New Modalities” – so leading voices of R&D can share how their approaches are addressing the barriers standing in the way of breakthroughs.
Up next in this interview series, Kunwoo Lee shares his insight as CEO of GenEdit. His company is focusing on developing targeted in vivo delivery platform technologies for genetic medicines, and recently secured $26 million in Series A financing. GenEdit is currently developing innovative therapeutics targeting the nervous system, treating a range of diseases with high unmet medical need. Earlier this year, GenEdit announced its collaboration with Sarepta Therapeutics to develop gene editing therapeutics for the treatment of neuromuscular diseases.
Congratulations on your recent progress, Kunwoo! What do you see as the major hurdles for genetic medicines?
Kunwoo: From our point of view, the top three challenges for genetic medicines are delivery, delivery and delivery. The potential to treat the underlying causes of genetic and sporadic diseases with genetic medicines is unprecedented – gene therapy, gene silencing, gene editing and others – but these technologies are only useful as therapeutics if they can reach the affected tissues and cells. Current delivery options are limited. We categorize the main challenges as tissue selectivity, payload flexibility, ability to re-dose and ease of manufacturing. In addition, safety has become a significant concern. A better delivery system would be truly enabling for genetic medicine to reach its potential.
How GenEdit plans to solve these challenges? What’s your technology and how it is differentiated?
Kunwoo: We believe that solving the delivery challenge requires new materials and a systematic approach. At GenEdit, we are overcoming this challenge with our proprietary technology, NanoGalaxy. This platform is composed of hydrophilic polymers, which can have diverse interactions compared to hydrophobic systems. By systematically screening a diverse library, we are able to analyze structure-activity relationships (SAR) which identifies structures and properties contributing to tissue selective delivery. Computational SAR and iterative screening accelerate the development of tissue-selective polymers.
NanoGalaxy is differentiated as it can deliver various genetic medicine payloads to tissues outside the liver from siRNA to mRNA beyond the size which adeno-associated virus (AAV) packing capacity has demonstrated. On top of that, the hydrophilic polymer can encapsulate various CRISPRs in ribonucleoprotein form, which is a unique feature.
Do you see any potential risks and challenges associated with your approach?
Kunwoo: Of course. We are developing a novel technology that has never been tested in humans and we understand that we have many challenges. One of our more interesting challenges is really an opportunity: our platform has the potential to be used to develop therapeutics for an enormous range of diseases. But we can’t do everything as a small company. Our solution is to focus on indications where the risk falls as much as possible onto our delivery technology and there is as little risk as possible for the target and payload. As our hydrophilic polymer nanoparticles are novel materials, we plan to engage with the FDA early in order to de-risk. Each validating milestone will enable new applications of the NanoGalaxy platform.
Gazing into our crystal ball, what’s the future for genetic medicines? Say 2030?
Kunwoo: We are focused on the future of genetic medicines and believe that they will be the mainstream of therapeutics in 2030. We are seeing an emergence of various genetic medicine candidates, including antisense oligonucleotide, siRNA, mRNA, new RNA systems, and CRISPRs. Eventually, in vivo genetic medicine will be generated from the combination of payload and delivery technology.
Industry wide, how genetic medicines would transform the current R&D landscape?
Kunwoo: The FDA had predicted 10-20 new approvals of gene therapies per year by 2025. The success will be dependent on platforms that are widely applicable and manufacturable. This is a goal which is not easy to achieve based on the current challenges that this industry is facing. Still, there is huge room for improvement in delivery technology regarding tissue selectivity, toxicity, immunogenicity, and manufacturing cost. At the same time, these new platforms are trying to address those challenges. In the end, a safer, cost-effective, and targeted delivery technology would overcome these limitations and could support 100+ approvals per year at half of today’s costs.
Thank you Kunwoo for sharing your insights! We with you great success in future endeavors.
Kunwoo: Thanks for inviting me. Enjoyed the discussion.
Kunwoo has been CEO of GenEdit since he founded the company in 2016. He saw that the potential of genetic medicine was held back by the limitations of delivery technology, and the company’s NanoGalaxy platform enables the systematic and iterative screening of polymer-based vectors. He published several high-profile papers in Nature journals that demonstrated delivery of mRNA, protein therapeutics, and CRISPR-Cas9 and CRISPR-Cas12a; and he has 10 patent filings, including the company’s core polymer nanoparticle technology. Prior to GenEdit, he completed graduate research at UC Berkeley and UCSF in the Department of Bioengineering, where he focused on novel delivery systems for macromolecular therapeutics. He was named as a Forbes 30 under 30 in 2017 and was a Siebel Scholar in 2016.