From carefully considering each step of the patient’s journey, to our effort to deliver transformative medicines, we’re taking an informed approach to bringing meaningful therapies from one to many. Learn more from Judith Sosa, our VP of clinical operations below.

This week, we’re opening our San Diego lab doors to welcome the Charcot-Marie-Tooth Association community for tours & a look at our team’s research & development in Charcot-Marie-Tooth disease Type 2C, (#CMT2C), a neuropathy caused by mutations in the TRPV4 gene. Learn more at cmtausa.org/actiobio

Next week, the Actio team is excited to participate in 1x1 meetings with investors at the Piper Sandler Spring Biopharma Symposium on April 16th in Boston See you there! actiobiosciences.com/actio-bioscien…

We're excited to follow Actio co-founder & CEO, David B. Goldstein's journey on this year's Timmerman Traverse! Every year, Luke Timmerman Timmerman Report & a team hike to raise money for a network of nonprofits through Life Science Cares. Congrats to the team! timmermanreport.com/2025/04/meet-t…

We’re excited to welcome two seasoned industry professionals to Actio’s leadership team: Viet Nguyen, MD, joins as VP of Clinical Development to lead our KCNT1 epilepsy program, and Margie Holmes steps in as VP, Regulatory Affairs & QA.

We’re proud to sponsor the 2025 HereditaryNeuropathy Summit Research + Retreat, April 24–26 in Nashville! The Actio team looks forward to connecting with the CMT community—patients, researchers, industry leaders, and more—to support collaboration and innovation.

We were proud to lace up our sneakers to walk again this year for the Charcot-Marie-Tooth Association & the #CMT2C community. From Actio teammates to families, friends, & advocates, this community continues to inspire us with its strength, resilience, and determination.

It was an honor to welcome members of the Charcot-Marie-Tooth Association community to our lab for a behind-the-scenes look at our research focused on Charcot-Marie-Tooth disease Type 2C, #CMT2C. It was a privilege to hear patients' stories & connect w/ the community that motivates our work every day.

We're proud to share our CEO David Goldstein was named one of the Leaders of Influence in Life Sciences by San Diego Business Journal. This honor reflects his exceptional leadership & commitment to innovation in #precisionmedicine. Congrats, David! sdbj.com/issues/leaders…

Today, we announced the successful completion of a $66 million Series B financing that will propel the advancement of our pipeline of small-molecule therapeutics targeting both rare & common diseases. Thank you to our investors, partners & team. Read more: businesswire.com/news/home/2025…

We’re honored to support the Fralin Biomedical Research Institute as Dr. Matt Weston’s lab explores our KCNT1 inhibitor as a potential way to reduce seizures. We're grateful for this partnership and inspired by science like this that pushes boundaries. Read more below.

We’ll be at this year’s Evercore Emerging Private Biotech Conference next week. Actio founder & CEO, David Goldstein, Ph.D., will present during a rare disease panel to share recent company and program progress. Looking forward to an insightful discussion. actiobiosciences.com/actio-bioscien…

We’re pleased to share that the FDA has cleared our IND and granted Fast Track for ABS-1230, an expected first-in-class oral #KCNT1 inhibitor for KCNT1-related #epilepsy, a severe pediatric condition with no disease-modifying therapies. Learn more: actiobiosciences.com/actio-bioscien…

On #RARECast, Actio Founder & CEO David Goldstein shares why rare diseases offer powerful clues for uncovering therapeutic insights & how Actio is using that knowledge to advance genetics-led drug development. Tune in to hear more below.

Congratulations to this year’s Timmerman Report #TimmermanTraverse team for finishing their week in the North Cascades! Biotech leaders, including our CEO, David Goldstein, tackled >20 miles & 7k ft of elevation gain in 2 days, raising >$1M, for our friends Life Science Cares.

It’s a pleasure to be in New York City this week for Cantor's 2025 Global Healthcare Conference! We’re eager to engage with investors in 1x1 conversations and highlight the recent milestones driving our momentum.

Next week, Actio CEO David B. Goldstein will join the Epilepsy Private Company Showcase at TD Cowen’s 5th Annual Neuropsychiatry Epilepsy Summit, sharing insights on our #KCNT1 epilepsy program. Learn more below and at: actiobiosciences.com/actio-bioscien…

Today, we shared that the first participant has been dosed in the Phase 1 clinical trial of ABS-1230 in development for the treatment of KCNT1-related #epilepsy, our second clinical initiation this year. Read more: actiobiosciences.com/actio-bioscien…

Actio CFO, Cayce Denton, will join fellow CFOs & local investors to share insights from both sides of the funding table at Biocom California series, "Private Capital in a Tight Market: Funding Strategies for Life Science CFOs."  biocom.org/events/event/?…

We are honored to welcome KCNT1 Epilepsy Foundation Foundation leadership, Justin West, MD, Co-Founder, President, Director of Clinical Medicine and Sarah Drislane, Executive Director, at Actio to celebrate the initiation of our Phase 1 trial of ABS-1230 for #KCNT1 -related epilepsy.