Actio Biosciences, Inc.

It’s an honor to welcome Dimitrios Arkilo, M.D., as our chief medical officer. Dr. Arkilo brings a unique combination of experience in clinical practice and the biotech industry, with a focus on neurology, epilepsy and rare disease, including a number of childhood conditions.   We look forward to Dr. Arkilo’s expert guidance in driving clinical strategy across our portfolio, including two lead programs heading to the clinic in #geneticepilepsy and #CMT2C. Learn more: https://lnkd.in/epJj9Bw7

Today, we’re pleased to share that we've received both orphan drug designation and rare pediatric disease designation from the FDA for ABS-0871, a TRPV4 inhibitor, for the treatment of Charcot-Marie-Tooth Disease 2C, or #CMT2C.   Receiving these designations emphasizes the urgent need for treatments for CMT2C patients, who often live with debilitating symptoms like muscle weakness, vocal cord paresis and respiratory complications. We’re proud to be pursuing solutions for CMT2C with support from our STAR Alliance Partner, the Charcot-Marie-Tooth Association through their Patients as Partners platform. We will continue to advance this program as quickly as possible.    Read more: https://lnkd.in/eaEKKVbV

This week, we hosted our first fireside chat moderated by Actio co-founder John McHutchison, AO, MD, with Sheila Gujrathi, MD, biotech entrepreneur and executive, healthcare investor, and deeply experienced drug developer, to discuss novel clinical trial design and some of the most important lessons learned along the way. Her insights were highly valued among the Actio team and it was a pleasure to hear her perspective. Extending a huge thank you to Dr. Gujarati for joining us!

We’re pleased to welcome renowned rare-disease drug developer Emil Kakkis, M.D., Ph.D., to our board of directors. Dr. Kakkis, a pioneer in rare disease drug development, founded Ultragenyx in 2010 to create a company centered on a novel development model that fundamentally changed the established paradigms in clinical protocols, endpoints and analyses, commercialization and access to therapies for patients with rare and ultra-rare genetic diseases. Additionally, Ultragenyx is recognized for its deep and meaningful engagement with patients and their caregivers to fully understand their needs. We look forward to his guidance and support in advancing our efforts to develop life-changing treatments that target underlying shared biology between rare and common diseases. Read more: https://lnkd.in/e2E-Nf98

Last month, we moved into our spectacular new office and lab space in the heart of San Diego's research community that features breathtaking views of the Pacific Ocean, a fitness center, a pickleball court, as well as indoor and outdoor collaboration areas. This move is a game-changer for us, enabling our expansion and boosting energy and efficiency with a beautiful 27,000 square-foot space and a lab facility over 4x the size of our previous. This is the perfect environment for the team to make a difference together and bring meaningful therapies from one to many. A huge thank you to our team and partners for making this possible. Here’s to the next steps on our journey!

At Actio, we believe the power of our team can make a real difference in genetics-led drug development. Between our diverse perspectives and deep expertise, we’ve worked hard to create an environment where collaboration and respect are woven into everything we do, from the lab and beyond. Jocelyn Ramirez is a Senior Associate of In Vitro Biology at Actio and an exemplary member of our team. To Jocelyn, Actio is a place where all voices are valued and heard, creating a collaborative path to achieving our goals. Learn more about Jocelyn and the respectful partnerships we’ve built to bring meaningful therapies from one to many.

We are delighted to welcome Ben Cravatt, Ph.D., to our board of directors. Dr. Cravatt brings to Actio genuine expertise across biology and chemistry having pioneered an approach to drug discovery and development that's at the heart of our mission.   Dr. Cravatt is currently professor and Norton B. Gilula Chair of Chemical Biology in the Department of Chemistry at The Scripps Research Institute, where his research group is interested in developing chemical proteomic technologies that enable protein and drug discovery on a global scale and applying these methods to characterize biochemical pathways that play important roles in human physiology and disease.   Read more here: https://lnkd.in/e3qWfSeX

Thank you to all who joined us for the Future of Biotechnology in Individualized Medicine conference this week. It was inspiring to engage in conversation as key opinion leaders explored the latest emerging genomic technologies that are reshaping drug target identification, precision biomarkers, and personalized therapies. Congratulations to our VP of genetics and bioinformatics, Ali Torkamani, for chairing such a successful event and to our co-founder and CEO, David B. Goldstein and board member Nina Kjellson for their participation!

There's still time to register and join us in La Jolla, CA for the Future of Biotechnology in Individualized Medicine conference on May 14th and 15th. "At Actio, we believe that advancing new treatments for diseases that affect one patient is fundamentally as important as advancing new treatments for diseases that affect many. We're honored to come together with other leaders in personalized medicine for the 2024 Future of Biotechnology in individualized medicine meeting to share ideas and insights that will drive the development of important new medicines for patients," said Actio co-founder and CEO, David B. Goldstein, who will be a panelist at the event and present the latest on Actio's approach and scientific rigor. We're proud to have had our VP of genetics and bioinformatics, Ali Torkamani, this year's conference chair, collaborate with Scripps Research Translational Institute and Advancing Precision Medicine to not only organize and plan this event, but also facilitate an opportunity to explore the latest in emerging genomic technologies at the forefront of drug target identification, precision biomarkers, and personalized therapies. Don't miss it. Register today: https://lnkd.in/ekZK8ecJ

We're looking forward to this year's Future of Biotechnology in Individualized Medicine conference to explore the latest in emerging genomic technologies that are reshaping drug target identification, precision biomarkers, and personalized therapies. Over the past year, our team, led by our VP of genetics and bioinformatics, Ali Torkamani, this year's conference chair, has been hard at work in collaboration with Scripps Research Translational Institute and Advancing Precision Medicine to bring this conference to life. The event, being held in La Jolla, CA, from Tuesday, May 14th through Wednesday, May 15th, will host many opportunities to engage with the precision medicine community through booths, sessions and networking. We look forward to seeing Ali and our CEO & co-founder, Dr. David B. Goldstein, Ph.D., take the stage during panel discussions at the event, and coming together with our industry colleagues to share ideas and recent innovations and trends in genomic medicine. Register today: https://lnkd.in/ekZK8ecJ