Actio Biosciences, Inc.

We’re pleased to share our participation in two upcoming investor conferences in October! → Our chief executive officer, David B. Goldstein, Ph.D., will take part in a fireside chat and 1x1 meetings at the #RBC2024 RBC Capital Markets Biotechnology Private Company Virtual Conference on Tuesday, October 1st. → Additionally, our team will participate in 1x1 meetings Leerink Partners Biopharma Private Company Connect on Wednesday, October 23rd. We look forward to these opportunities to connect with the industry and discuss our efforts to develop new therapeutics that target shared underlying biology in both rare and common diseases. Learn more: https://lnkd.in/eyM8X3jC

Actio's Co-Founder and CEO, David B. Goldstein, will take part in the "Data-for-purpose: Go big, go deep, or go long?" panel on BioCentury Inc.'s third day of Grand Rounds in Nashville next week, to discuss optimal ways to make data fit for purpose among other leading industry drug developers, academics and investors.    Sunil Sahdeo Ph.D., Actio Vice President, Head of Operations, will also be there to present a poster on practicing inclusive precision medicine at Actio.   Don't miss the insights. Learn more and join us: https://lnkd.in/gakuvuEn 

We’re overjoyed to have a few adorable new additions to our Actio family. There are some proud new parents in our mix and we couldn’t be happier for them - so we hosted celebrations to welcome their little ones. Wishing the proud, new parents endless moments of joy, cuddles, and sweet baby smiles!

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