Actio Biosciences raises US$66M to accelerate rare disease therapies

USA—Actio Biosciences, a biotechnology company based in the USA, has successfully closed a $66 million Series B financing round to advance its pipeline of genetics-driven small molecule therapeutics targeting both rare and common diseases.

This significant funding round was co-led by new investor Regeneron Ventures and existing investor Deerfield Management, with additional participation from current investors Canaan, Euclidean Capital, and Droia Ventures.

The capital raised will primarily support the development of Actio’s two lead oral drug candidates: ABS-1230 and ABS-0871.

 ABS-1230 is a selective inhibitor of the potassium channel subfamily T member 1 (KCNT1) and is being developed to treat KCNT1-related epilepsy, a severe and rare pediatric epileptic encephalopathy.

This condition affects approximately 2,500 individuals in the US and is characterized by frequent, difficult-to-control seizures.

Encouragingly, ABS-1230 has demonstrated the ability in preclinical studies to inhibit all known pathogenic mutations in the KCNT1 gene, suggesting broad applicability for patients with this genetic form of epilepsy.

Actio plans to initiate the healthy volunteer phase of a Phase I clinical trial for ABS-1230 in the second half of 2025.

Following this, the company aims to expand into a Phase Ib proof-of-concept study involving patients with KCNT1-related epilepsy in early 2026.

The US Food and Drug Administration (FDA) has recognized the potential of ABS-1230 by granting it both orphan drug and rare pediatric disease designations, which can facilitate a faster and more efficient development and review process.

Meanwhile, ABS-0871 is another promising small molecule in Actio’s pipeline.

It functions as an inhibitor of transient receptor potential vanilloid 4 (TRPV4) and is being developed to treat Charcot-Marie-Tooth disease type 2C (CMT2C), a rare inherited neurological disorder that affects motor and sensory nerves.

ABS-0871 is currently undergoing a Phase I clinical trial with healthy volunteers, with plans to move into a Phase Ib trial in patients with TRPV4-positive CMT2C in 2026.

This therapy has also received orphan drug, fast track, and rare pediatric disease designations from the FDA, highlighting its potential to address significant unmet medical needs.

David Goldstein, Ph.D., co-founder and CEO of Actio Biosciences, expressed confidence in the company’s progress, stating that their precision medicine strategy targets the root causes of disease through genetically informed drug development.

He emphasized that both ABS-1230 and ABS-0871 have the potential to be transformative, disease-modifying therapies for their respective rare conditions, with growing evidence supporting their expansion into broader patient populations.

The recent funding from leading industry investors underscores the value of Actio’s approach and equips the company with essential resources to continue advancing its innovative programs.

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