Loucst Walk RNA Innovation Conference

May 9, 2023, Conference in Review

Locust Walk’s second annual Innovation Conference was a huge success with over 350 attendees. This year’s theme was focused on next generation RNA medicine in partnership with Dr. Frank Slack, Director of the Harvard Medical School Initiative for RNA Medicine, and Dr. Phil Zamore, Chair of the UMass Chan RNA Therapeutics Institute. We brought together private companies and full-time researchers on the cutting edge of RNA therapeutics to feature their work for investors, strategic partners, and other academics interested in the latest innovations in RNA as well as their translation to the clinic. Presenting companies represented 6 major pillars of innovation in RNA: 

• Protein Expression 
• RNA Editing 
• RNA-Mediated Protein & Transcriptome Regulation 
• RNA Targeting SMOLs 
• Translation Inhibition 
• Novel Delivery Approaches  

We believe there is great potential and breadth of therapeutic applications of RNA therapeutics. As well, targeting RNA in vivo will maintain the momentum that began with the regulatory and commercial successes of mRNA vaccines. 

For this year’s conference, Locust Walk invited 27 companies and 6 academic groups to present their innovations in RNA. Academics who presented their research have developed innovations that are either in the process of becoming a new company or have the potential to become one within 12 months of the conference date. Of the 350 attendees were investors, strategic partners, and academics with more who were unable to join live watching our recorded sessions. Our goal was to stimulate curated deal flow for biopharma investors and strategic partners interested in RNA by connecting them with the foremost innovators in the space. To this end, Locust Walk also assembled a world-class list of experts for our fireside chat, a lunchtime scientific panel about small molecules targeting RNA, and afternoon business market conditions panel. 

Keynote with Dr. Frank Slack and Dr. Phil Zamore
Watch recording here

Two pioneers in the field, Dr. Frank Slack and Dr. Phil Zamore, each of whom has founded an academic research institute dedicated to the progress of RNA, reviewed the history and idiosyncrasies of each of their institutes and how they execute on the common vision of moving the field of RNA forward. Both institutes complete this aim by encouraging research collaborations between academics with diverse backgrounds and expertise, then providing opportunities to share their research projects with industry via symposia and company spinouts. Dr. Zamore noted that researchers from the UMass Chan RNA Therapeutics Institute have co-founded well known public biotech companies as Voyager Therapeutics and Intellia Therapeutics. Dr. Slack shared that discoveries out of the Harvard Medical School Initiative for RNA Medicine have produced at least one spinout company per year since its founding in 2014. Drs. Slack and Zamore highlighted several specific areas of research within RNA whose recent progress elicits their excitement for their clinical potential: RNA-targeting enzymes, new RNA modifications, ADARs, and RNA-based bacterial tools that can be leveraged for therapeutic use in humans, like CRISPR-Cas9. They also discussed RNA therapeutics manufacturing as an area for further investment, and the complexity and synergy differences between different therapeutic formats of RNA, e.g., viral vector manufacturing, synthetic RNA manufacturing, and mRNA vaccine manufacturing.

Scientific Panel: The Future of RNA-Targeting Small Molecules
Watch recording here  

Daniel Brog, a Senior Vice President at Locust Walk, led a discussion with 4 biotech industry leaders in RNA on the innovations and future directions of small molecules targeting RNA, one of the conference’s 6 pillars of RNA innovation. The panelists included Paul August, PhD, Chief Scientific Officer of ReviR Therapeutics; Michael Gilman, PhD, CEO of Arrakis Therapeutics; Dom Reynolds, PhD, SVP of Drug Discovery at Remix Therapeutics; and Dominique Verhelle, PhD, co-founder, President, and CEO of NextRNA Therapeutics. The group noted the progress – and limitations – of therapeutic RNA-based antisense oligonucleotides and siRNAs as a catalyst for the rapid growth of interest in small molecules targeting RNA. They discussed Evrysdi (risdiplam), a small molecule splicing modifier approved in 2020 for spinal muscular atrophy, as a specific trigger event for the growth in interest in RNA-targeting small molecules. Developments in sequencing technologies and scientific understanding of the structural biology and functions of non-coding RNAs were also highlighted as catalysts. The panelists explained the selectivity advantages of RNA targets over protein targets in context of specific programs in development at each company. They discussed drug development challenges of RNA-targeted small molecules, including recapitulating in vivo RNA structures during in vitro screening assays and discovering small molecule binder-degraders with detectable functional activity, as well as solutions – for example, resolving small molecule screening volume bottlenecks by developing cell-free assays, in the case of one panelist’s company. Tune into the recording for more details on the topics above and the panelists’ perspectives on future directions of RNA-targeted small molecule development in biopharma and future directions of RNA-targeted small molecule development in biopharma.

Business Panel: RNA Partnerships, M&A, & Financings: What We’ve Learned & Where We’re Headed
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Geoff Meyerson, Locust Walk’s CEO and Co-founder, moderated a panel of 3 seasoned RNA biotech CEOs to discuss recent trends, lessons, and future directions of the transaction landscape for companies developing RNA therapeutics. The panel featured Josh Mandel-Brehm, CEO of CAMP4 Therapeutics; Jerry McMahon, PhD, CEO of STORM Therapeutics; and Leslie Williams, CEO, co-founder, and President of hC Bioscience. The group discussed trends that have come forth as a function of current market conditions, such as the shift away from broad platform strategies towards asset-based, indication-specific drug development with a clear line of sight to the clinic. Williams shared her observations on the growth of interest from investors and partners for truly differentiated RNA programs despite the market’s challenges. McMahon added color from his point of view at the helm of STORM and Mandel-Brehm followed with his perspective on the pace of fundraising for RNA biotech companies – in summary, there have always been challenges, but the market environment has shifted the composition of investor syndicates behind RNA companies and the speed with which they commit to investments. The panelists discussed the tradeoffs for innovative RNA companies of pursuing business development for non-dilutive funding in a capital-constrained market, illustrating their views with specific examples from recent experience. Tune into the recording for specific details on each panelist’s experience in the recent fundraising environment as well as their perspective on where investment and partnerships in RNA are headed next.