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Actio Biosciences will advance its genetics-driven drugs. (Credit: tilialucida\/Shutterstock)<\/p>\n
US-based clinical-stage biotechnology company Actio Biosciences has closed a $66m Series B financing round, co-led by Regeneron Ventures and Deerfield Management.<\/p>\n
Other participants include existing investors Canaan, Droia Ventures, and Euclidean Capital.<\/p>\n
The funding aims to advance Actio\u2019s genetics-driven pipeline of small-molecule therapeutics targeting both rare and common diseases.<\/p>\n
Deerfield Management partner Jason Fuller said: \u201cActio has done an exceptional job of identifying serious unmet needs in rare disease and advancing solutions that may also be applicable to patients with more common diseases.<\/p>\n
\u201cWe\u2019re pleased to support the company, encouraged by its progress, and excited by the team\u2019s potential to make a difference for patients.\u201d<\/p>\n
Actio will use the funds to develop its lead programmes, ABS-1230 and ABS-0871, which received the US Food and Drug Administration (FDA) rare paediatric and orphan drug designations.<\/p>\n
ABS-1230, a KCNT1 inhibitor, is designed to treat KCNT1-related epilepsy, a rare and often fatal paediatric epileptic encephalopathy affecting approximately 2,500 individuals in the US.<\/p>\n
The drug has shown promise in preclinical studies by inhibiting pathogenic mutations in the KCNT1 gene.<\/p>\n
Actio plans to initiate a Phase 1 clinical trial for ABS-1230 in the second half of 2025, with a Phase 1b study in early 2026.<\/p>\n
ABS-0871, a TRPV4 inhibitor that has been granted FDA fast-track designation, is being evaluated for Charcot-Marie-Tooth disease type 2C (CMT2C) and overactive bladder<\/a>.<\/p>\n CMT2C is a rare inherited disorder affecting motor and sensory functions, with a US prevalence of approximately 2,500 individuals.<\/p>\n Preclinical evaluations have shown significant improvements in motor function. ABS-0871 is currently in a Phase 1 trial, with plans for a Phase 1b study in 2026.<\/p>\n Actio co-founder and CEO David Goldstein said: \u201cWe have made tremendous progress across our pipeline \u2013 executing a precision medicine strategy that targets the root causes of disease through genetically informed drug development.<\/p>\n \u201cABS-1230 and ABS-0871 have the potential to be transformative disease-modifying therapies in their respective rare indications, and growing evidence supports expansion into broader indications.<\/p>\n \u201cThis new funding from industry-leading investors speaks to the value of our approach and provides us with important resources to continue advancing our programmes.\u201d<\/p>\n Sign up for our weekly news round-up!<\/p>\n![\"Email](\"https:\/\/www.worldpharmaceuticals.net\/wp-content\/themes\/goodlife-wp-B2B\/assets\/images\/newsletter-new.svg\")
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