Actio Biosciences
@actiobiosci
Bringing meaningful therapies from one to many.
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 honored to support the @FralinBiomed 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.
With a grant from @actiobiosci, the Weston lab is researching whether an Actio KCNT1 inhibitor can reduce seizures caused by a rare genetic mutation. Read more: news.vt.edu/articles/2025/…
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 proud to share our CEO David Goldstein was named one of the Leaders of Influence in Life Sciences by @SDbusiness. This honor reflects his exceptional leadership & commitment to innovation in #precisionmedicine. Congrats, David! sdbj.com/issues/leaders…
It was an honor to welcome members of the @cmtausa 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 were proud to lace up our sneakers to walk again this year for the @cmtausa & the #CMT2C community. From Actio teammates to families, friends, & advocates, this community continues to inspire us with its strength, resilience, and determination.
We’re proud to sponsor the 2025 @CMTNeuropathy 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’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 excited to follow Actio co-founder & CEO, David B. Goldstein's journey on this year's Timmerman Traverse! Every year, @ldtimmerman @timmermanreport & a team hike to raise money for a network of nonprofits through @LS_Cares. Congrats to the team! timmermanreport.com/2025/04/meet-t…
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…
This week, we’re opening our San Diego lab doors to welcome the @cmtausa 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
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.
We’re thrilled to share that the first participant has been dosed in the Phase 1 clinical trial of our lead program, ABS-0871, a TRPV4 inhibitor, for the treatment of TRPV4+CMT2C, a severe peripheral nerve disorder. Read more about the trial: actiobiosciences.com/actio-bioscien…
It’s #RareDiseaseDay, but rare is not all that rare. 300 mil people worldwide live w/ a rare disease. At Actio, we’re making a difference w/ advances in genetics & precision medicine to develop life-changing treatments. More @ rarediseaseday.org & actiobiosciences.com
The Actio management team will attend 2 upcoming investor meetings, TD Cowen's 45th Annual Health Care Conference & the Leerink Partners Global Healthcare Conference. We're looking forward to one-on-one meetings & connecting w/ the industry. actiobiosciences.com/actio-bioscien…
It's an honor to be part of @KCNT1_Epilepsy's #PathwaytoTrials webinar series. Actio CEO, David Goldstein will join to discuss #KCNT1 epilepsy & the potential impact new precision medicines could make for those living with the disease. Register: us02web.zoom.us/meeting/regist…
It's #InternationalEpilepsyDay. At Actio, we recognize the critical importance of advancing research, improving treatment options, & fostering understanding to help create a better future for all those impacted. Learn more at epilepsy.com. #EpilepsyAwareness
Addressing underlying causes of rare diseases resonates w/ many. This was evident at JPM, where we connected w/ Actio board member & @ultragenyx CEO, Emil Kakkis, whose enthusiasm reinforced our drive. We’re excited to continue working toward making this vision a reality.
What a week! The Actio team closed out #JPM25 with a great sense of accomplishment from days of productive meetings and making valuable industry connections. We're energized by the opportunities ahead.
Actio co-founder and CEO, David B. Goldstein, Ph.D., took the stage Monday at #JPM25! The team was excited to see him share more on our pipeline progress and what’s ahead for Actio in 2025.
