F-Prime has long been interested in precision medicine approaches that tackle the underlying cause of diseases, ranging from investments in small molecules (e.g., Blueprint Medicines) to gene editing technologies (e.g., Beam Therapeutics).

In 2020, we developed an interest in the epigenetic modulation of oncogenic pathways and came across a small molecule inhibitor of MMSET, known as KTX-1001. MMSET is a histone methyltransferase that is overexpressed due to the t(4;14) translocation that is present in up to 20% of multiple myeloma patients.  This target has been highly evasive, with several companies trying and failing to develop a direct inhibitor. When we learned that there was a first-in-class, selective MMSET inhibitor available for licensing, we saw a unique opportunity to build a company dedicated to advancing drugs against MMSET, and potentially other similarly compelling epigenetic targets in cancer.

We found partners early on that were similarly eager to help build and ensure the long-term success of the company, including Atlas Venture and academic thought leaders in methyltransferase biology such as Or Gozani, MD, PhD, Professor of Biology at Stanford University. Our sister fund, Eight Roads Ventures, also joined the syndicate. K36 recently emerged from stealth with experienced biotech executive Terry Connolly leading the company as CEO, and industry veteran Lori Kunkel, MD, joining the Board of Directors. With the $30M Series A financing, co-led by F-Prime, K36 is well positioned to progress KTX-1001 through the clinic and plans to submit an IND for KTX-1001 in the first half of 2022.

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“From the beginning, F-Prime championed/advocated the founding of our company. F-Prime partner and K36 board member, Chong Xu, PhD, helped negotiate the license and assembled the Scientific Advisory Board. Together, we work to put precision medicine on the forefront to help patients.”

Terry Connolly, PhD, CEO

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While we are fortunate to have many more available therapeutic options for multiple myeloma patients today, the t(4;14) translocation is classified as a high risk genetic alteration and is associated with a poorer prognosis. We believe that direct MMSET inhibition represents a promising new modality that can benefit this population of multiple myeloma patients. This is a great opportunity and we are very proud to support K36 on its mission to develop small molecule epigenetic modulators and breakthrough precision medicines that address the unmet medical needs of cancer patients worldwide.

Neurological disease remains the largest cause of disability and second leading cause of mortality, worldwide, but life-changing therapies could be on the horizon.

Back in December 2018, we had the pleasure of sitting down with leading neurologist and neuroscientist Professor Christopher Shaw, molecular neurobiologist Dr. Youn Bok Lee and vector biologist Dr. Do Young Lee, the driving forces behind AviadoBio’s science, to learn more about their vision to develop transformative gene therapies for neurological disorders like frontotemporal dementia and ALS. Dr. Shaw described the remarkable genetic discoveries his team had uncovered in his lab at King’s College London and the journey that led them to develop the technology that inspired us to build a company around the promise we saw. The improved understanding of the genetic and pathological underpinnings of these neurodegenerative diseases and emerging clinical efficacy from use of targeted therapies in neurology have driven an increased interest in genetic medicines for these diseases – as well as a deeper understanding of key challenges in CNS gene therapy such as distribution and delivery.

Company building and incubation is some of the most rewarding work we engage in and, in fact, AviadoBio’s early home was our London office. In its earliest stages, we were delighted to work with Jonathan Jones, part of the founding team of CRISPR Therapeutics, and Graeme Fielder, former Head of Business Development at Audentes, as Entrepreneurs-in-Residence alongside a team of pioneers and experts in neurodegenerative disease, neurosurgery and genetic medicine. In 2021, the company emerged from stealth with former Novartis executive, Lisa Deschamps, joining as CEO and an $80 million Series A financing.

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“F-Prime believed in AviadoBio’s promise and supported us by providing a management team, a generous workspace and the finances needed to advance. Their commitment led us from a scientific concept and personal passion to a potential reality of hope for patients in less than 3 years!”

Lisa Deschamps, CEO

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Although we have seen an unparalleled pace of development for a wide range of diseases and a record number of drugs being approved by the FDA over the last several years, there have been some disease areas that have shown little progress. One of those is the challenging disease space of neurodegenerative disorders where there are limited treatment options for patients and major obstacles during the drug development process.

At F-Prime, we remain optimistic that we could be at the forefront of a breakthrough because of companies like AviadoBio.