Actio Biosciences, Inc.

We are pleased to announce that the first participant has been dosed in our Phase 1 clinical trial of ABS-1230, an anticipated first-in-class selective small molecule #KCNT1 inhibitor in development for the treatment of KCNT1-related #epilepsy. KCNT1-related epilepsy is a rare, severe, and often fatal pediatric developmental epileptic encephalopathy that affects an estimated 2,500 individuals in the U.S. This milestone marks our second clinical initiation this year, underscoring our commitment to developing precision therapies for severe rare diseases as we work to evaluate the safety and potential of ABS-1230. We remain dedicated to advancing much-needed treatment options for patients and families impacted by this debilitating condition. Read more: https://lnkd.in/eZsZ8AeV

We're Hiring! We’re looking for an experienced Director / Senior Director of Regulatory Affairs to join our growing team. Reporting to the VP of Regulatory Affairs & Quality Assurance, you’ll lead global regulatory strategies, shape submissions to the FDA and international health authorities, and partner cross-functionally to bring life-changing therapies to patients. This is a high-visibility leadership role with impact across our pipeline. If you have 12+ years of Regulatory Affairs experience, deep knowledge of FDA/global requirements, and the drive to mentor and lead in a collaborative, mission-driven environment — we’d love to hear from you. Learn more and apply: https://lnkd.in/g2DRX4bz

Next week, Actio CEO, David B. Goldstein, will take part in the Epilepsy Private Company Showcase panel discussion at TD Cowen’s 5th Annual Novel Mechanisms in Neuropsychiatry Epilepsy Summit, offering the latest insights on our #KCNT1 related epilepsy program. Read more below and at: https://lnkd.in/eigmKbWf

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.

Sharing an enormous congratulations to this year’s Timmerman Report #TimmermanTraverse team for finishing their week in the Pacific Northwest in the North Cascades! These spectacular biotech leaders, including our own CEO, David B. Goldstein, tackled more than 20 miles and 7,000 ft of elevation gain in just two days, raising over $1M for our friends at Life Science Cares, Inc. What an incredible feat and even better cause. 

“What I think hasn’t been done is really focusing on what the rare disease itself teaches you about target biology. That’s the central insight we focus on.” This week on RARECast, Actio Founder and CEO David B. Goldstein shares why rare diseases offer powerful clues for uncovering therapeutic insights and how Actio is using that knowledge to advance genetics-led drug development. Tune in below to hear how we’re working to turn meaningful breakthroughs for one into transformative therapies for many.

We are pleased to share that U.S. FDA has cleared our investigational new drug application and granted Fast Track designation for ABS-1230, an expected first-in-class, orally administered small molecule #KCNT1 inhibitor, for the treatment of KCNT1-related #epilepsy. KCNT1-related epilepsy is a rare, severe and often fatal pediatric developmental epileptic encephalopathy that has no current disease-modifying therapy available. This is an encouraging milestone for both Actio and the children and families affected by this devastating condition. We are proud to take this important step toward advancing a potential therapy with the goal of transforming care and offering hope to the KCNT1 community. Learn more at https://lnkd.in/eATKuHEH

We’re pleased to be participating in this year’s Evercore Emerging Private Biotech Conference. Next Monday, Actio founder and CEO, David B. Goldstein, Ph.D., will present a company overview during a rare disease panel to share more about Actio and our recent progress. Looking forward to an insightful discussion with leaders driving innovation in rare disease. https://lnkd.in/eTmZ_YcM

Congratulations to Dr. John Link, a key scientific advisor to Actio, on being awarded the Warren Alpert prize for the discovery of lenacapavir, along with Tomas Cihlar and Wes Sundquist. Lenacapavir represents a revolutionary new tool in the fight against HIV, a long-acting antiviral dosed just twice a year, with a novel mechanism of action targeting the HIV capsid. Lenacapavir shows what can be accomplished when a deep understanding of complex biology is combined with exceptional chemistry, which is how we seek to work at Actio. Congratulations, John on this well-deserved recognition! Read more: https://lnkd.in/ePFie3bc

We are honored to support the important research underway in Matt Weston's lab at the Fralin Biomedical Research Institute at VTC showing the ability of our KCNT1 inhibitor, ABS-1230, to reduce seizures caused by KCNT1-related epilepsy. Thank you to the Fralin Biomedical team for your partnership and dedication. We truly believe in the power of rigorous, curiosity-driven science to unlock new possibilities in human health. We're proud to support researchers who share that vision and are working to translate bold ideas into impact. Read more below.