Actio Biosciences – a biotechnology company utilizing a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases – recently announced the completion of a $55 million Series A financing. The funding round was led by Canaan and DROIA Ventures, with participation from existing investors Deerfield Management and EcoR1, and new investor Euclidean Capital.
Even though it is well known that a single gene can lead to rare diseases, the function of mutations in those single genes and how they might be modulated for therapeutic intervention is often not well understood. And to overcome this and better utilize the breadth of genetic information now available for drug discovery and development, Actio is leveraging its human genetics platform to identify relevant disease targets, understand their function in disease, and create medicines that modulate them.
The company’s platform comprises a vast proprietary target database – called the Rare Disease Target Atlas – and unparalleled expertise in bioinformatics, biology, and chemistry. And Actio will initially focus on identifying targets and advancing programs for rare disease indications and plans to utilize insights throughout the development process to inform the potential relevance of those targets in more common diseases.
The company established a partnership with the Rare Disease Translational Center at The Jackson Laboratory (JAX), a leading nonprofit biomedical research institution dedicated to understanding the molecular underpinning of rare diseases and their treatments. And Actio is partnering with JAX to evaluate targets and phenotypes of interest in high-quality genetically engineered mouse models at scale, enabling high confidence in the outcomes observed. Plus, Actio is also collaborating with prominent academic centers to provide further validation of the role of certain genes in disease and insights into disease pathology to inform better the design of preclinical studies, rare patient natural history studies, and future clinical trials.
Actio’s pipeline is being led by a program targeting TRPV4, an ion channel target identified as high value through the company’s platform. And Actio plans to initially evaluate it for the treatment of rare diseases caused by TRPV4 mutations, including Charcot-Marie-Tooth disease type 2C (CMT2C), characterized by severe muscle weakness, vocal cord paresis and respiratory complications, and serious bone diseases like metatropic dysplasia.
The evaluation of this program in novel construct valid preclinical TRPV4 rare disease models has demonstrated marked improvements in motor function, mobility, and bone morphology and increased lifespan compared to untreated controls.
Distinguished Founders and Leadership
Actio was founded by human genetics expert David Goldstein, Ph.D. – who serves as the company’s CEO and a board member, and pioneering drug developer John McHutchison, AO, M.D., who also serves as a board member. And the Actio leadership team and board include the following:
– David Goldstein, Ph.D., co-founder and CEO
– David Breckenridge, Ph.D., chief scientific officer
– John Link, Ph.D., senior advisor, drug discovery
– Sunil Sahdeo, Ph.D., vice president, biology
– Nick Stock, Ph.D., senior vice president, Chemistry
– Ali Torkamani, Ph.D., vice president, bioinformatics and genetics
– Anne Sandan, CPA, vice president, human resources and finance operations
Board of Directors
– George Golumbeski, Ph.D., board chairman and partner, DROIA Ventures
– Jason Fuller, Ph.D., board member and partner, Deerfield Management
– David Goldstein, Ph.D., board member and CEO, Actio
– Nina Kjellson, board member and general partner, Canaan
– John McHutchison, AO, M.D., board member and co-founder, Actio
– Alex Tkachenko, Ph.D., board member and executive in residence, EcoR1
KEY QUOTES:
“Defining a high-impact drug target for a common disease with a heterogenous population has a very low probability of success and has long been a major challenge for drug discovery. By starting with a focus on rare diseases, we gain invaluable insights that not only define new therapeutic benefits in high-need indications but also de-risk and guide therapeutic expansion to more common conditions, benefiting even more patients. We are thrilled to have the backing of this esteemed group of investors, along with our world-class team, whose collective support will enable the advancement of our programs to bring life-changing medicines to those that need them most.”
– David Goldstein, Ph.D., co-founder and CEO of Actio
“In today’s financing environment, investors can be quite selective where to place our bets. At Canaan, we had a thesis in precision genetics, and Actio stood out for scientific excellence, a seasoned team and very impressive pipeline progress in the short time since inception. We are passionate about investing in teams that have deep, relevant expertise, yet aren’t afraid to tackle the hard problems from new angles and with multidisciplinary approaches.”
– Nina Kjellson, board member and general partner at Canaan
“Since our founding, we’ve built a proprietary platform designed to de-risk targets of interest and establish biological connections between rare and common disease states that is unlike anything else in the industry today. That work has led to the prioritization of several high-value targets, including our lead TRPV4, program for the treatment of CMT2C and other bone diseases with high unmet need. Beyond this program, we are working expeditiously to expand our pipeline and have identified two additional programs that we plan to evaluate for genetic epilepsies and genetic skin conditions.”
– John McHutchison, AO, M.D., co-founder and board member of Actio
“The Actio team is comprised of proven geneticists and drug hunters that have both the expertise and drive to advance a company rooted in the science of precision medicine, with a big-picture vision of translating that science into medicines that could ultimately address the most common diseases. DROIA is honored to partner with this team to advance a platform and pipeline that could change the trajectory of how we leverage the complexities of biology and human genetics to create promising new therapies.”
– Dr. Golumbeski, board chairman and partner at DROIA Ventures
