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Using “search and replace” to address the fundamental causes of genetic disease.

Developed in the lab of Dr. David Liu at the Broad Institute, Prime Medicine was founded to deliver the promise of gene editing to patients with genetic diseases.  The ability to introduce nearly any desired edit to restore normal genetic function has long been viewed as the ‘holy grail’ in the gene editing space.  While there are approaches to accomplish this with first generation CRISPR-based technologies – which are based on introducing double strand breaks into DNA – efficiencies have historically been low (these technologies are very well suited to introducing gene disruptions). Base editing was a significant step forward in gene editing, but the technology only allows for select base-to-base changes.  Prime editing introduced, for the first time, the ability to efficiently introduce any base-to-base edit as well as to correct insertions and deletions, creating an opportunity to address more than 90 percent of known disease-causing genetic mutations.

First-generation CRISPR/Cas9 technologies can be likened to scissors, base editing to a pencil and eraser, and Prime Editing to a word processor, enabling search and replace genome editing and representing a significant improvement in precision and breadth of possibilities.

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“F-Prime was with us from the beginning and shared our vision for the promise of Prime’s technology. Together we can deliver hope to patients and families suffering from incurable genetic diseases.”

Keith Gottesdiener, CEO

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At F-Prime, we believe that Prime’s transformative technology is a major advancement towards their vision of creating a world where Prime Editing can potentially cure, halt and ultimately prevent genetic diseases.

It was an honor to support Prime through their Series A and Series B funding rounds alongside other leading healthcare investors. We share a common goal to improve the lives of patients with a wide range of genetic diseases.

Achieving broad distribution of RNAi therapeutics throughout the CNS has been a limitation in developing effective medicines for neurodegenerative diseases.

Treating neurological diseases is one of the most challenging existing medical problems we face today. Although gene silencing techniques such as RNAi or ASOs hold great promise, limited distribution throughout the entire brain, even with local injection, has significantly hampered their therapeutic potential. Atalanta Therapeutics founding scientists Anastasia Khvorova, PhD, Nobel laureate Craig Mello, PhD, and Neil Aronin, MD, from UMass Medical School’s RNA Therapeutics Institute, made a major breakthrough by identifying a novel RNAi structure called branched siRNA that enables broad distribution throughout the brain, including deep brain structures. Building on this initial discovery, scientists at Atalanta are developing a robust pipeline of RNAi therapeutics to address CNS diseases such as Huntington’s, Alzheimer’s and Parkinson’s.

At F-Prime, we immediately recognized the power of this novel technology and, in collaboration with the founding scientists who helped to spin the technology out into a newco. We quickly recruited Alicia Secor as the CEO and first employee who has, in turn, assembled a world-class management team of veteran biotech experts from the ground up.

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“F-Prime played a critical role in guiding the development of our strategy and execution of our launch. We remain confident in our partnership and can trust in their continued support as we progress as a company.”

Alicia Secor, CEO

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Following a Series A funding round, led exclusively by F-Prime, and fueled by strategic collaborations with Biogen and Genentech, Atalanta launched in 2021 with $110 million. F-Prime believes the company is positioned for success through our collaborative effort and aligned mission.

Our involvement with innovative companies like Atalanta strengthens our confidence that new treatment options for patients suffering from neurodegenerative diseases will one day be available and broadly accessible.

Many human proteins are intrinsically disordered and play a critical role in key biological processes — predicting their behaviour could unlock therapeutics acting at new high-value targets.

F-Prime Capital has been an active investor in computational approaches for drug discovery since 2016.  Evaluating many companies in the sector since that time, Peptone’s approach stood out as a potential breakthrough because of its unique position at the intersection of biology and physics and because of the hard problem being addressed: how to drug disordered proteins.

Disordered proteins span several biological classes, including hormones, enzymes, growth factors, cytokines and transcription factors and their impact on human physiology and disease has become increasingly apparent in recent years. Yet the fluidity of these proteins – they lack a consistent 3D structure – has historically made drug discovery challenging.

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“F-Prime has been a fantastic financing partner. Prior to investing they dug in deep and helped us develop our strategy.”

—Kamil Tamiola, PhD, CO-Founder, CEO

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Peptone was founded by Kamil Tamiola, PhD, and Matt Heberling, PhD with the goal of using experimentally derived biophysical data and molecular dynamics simulations to model the behaviour of disordered targets so enabling investigation of new drug candidates. The company has had multiple R&D collaborations with big pharmaceutical companies which have been highly productive.

In June 2022, F-Prime was delighted to co-lead a $40 million Series A financing of Peptone. The Series A funds are being used to advance the company’s tech platform, to establish a state-of-the-art research facility in Switzerland and to advance a proprietary drug discovery pipeline.