Lario Raises $2.4M for Precision Neuro Medicines
Lario, a biotech developing first-in-class precision medicines targeting voltage-gated neuronal calcium channels, has secured $2.4M in non-dilutive funding from the Michael J. Fox Foundation ($1.5M) and Wellcome ($0.9M). The company focuses on CaV2.3, CaV2.1, and CaV1.3 modulators as disease-modifying treatments for severe neurological disorders including genetic developmental epileptic encephalopathies (DEEs), drug-refractory epilepsies, Parkinson’s disease, PTSD, genetic hemiplegic migraine, and treatment-resistant hypertension. This capital supports platform expansion and program advancement.
Foundation Grants Validate Calcium Targets
The funding arrives amid heightened foundation interest in genetics-driven neurology. Lario recently received a $6M grant from MJFF to advance CaV2.3 inhibitors for Parkinson’s in collaboration with the Oxford Parkinson’s Disease Centre. Seed backing from Epidarex Capital and Golgi Neurosciences further bolsters its position.
Rare Epilepsies Demand New Modifiers
Ultra-rare genetic epilepsies like DEE69 (CACNA1E mutations) and DEE2 (CDKL5 deficiency) cause severe, treatment-resistant seizures in pediatric patients, with no approved disease-modifying therapies. Drug-refractory epilepsies and Parkinson’s affect large patient populations, but existing treatments fail to address underlying genetic drivers, leading to poor outcomes and high unmet need. Current antiepileptics and Parkinson’s drugs provide symptomatic relief at best, often with significant side effects.
Genetics-Guided Channel Modulators Differentiate
Lario links human genetics to selective inhibition of disease-implicated channels. Its CaV2.3 modulators target DEE69, CDKL5 deficiency, Parkinson’s, and PTSD; CaV2.1 for genetic hemiplegic migraine and neurodevelopmental disorders; CaV1.3 for Parkinson’s, hypertension, and autism syndromes. This precision contrasts with non-selective therapies that broadly suppress neuronal activity.
Unlike traditional approaches, Lario’s platform emerged from validation via MJFF’s Targets to Therapies initiative, emphasizing causal genetics over empirical screening.
MJFF Backing Signals Target Confidence
Repeated MJFF support—including Targets to Therapies selection and dual grants totaling $7.5M—highlights calcium channels as promising for Parkinson’s disease modification. Wellcome’s investment expands the platform’s scope. As an Epidarex Exeed spin-out in 2021, Lario leverages VC expertise in rare neuro from Epidarex and Golgi to translate genetics into therapies.
Precision Neuro Funding Momentum Builds
Foundation-led non-dilutive capital is fueling early-stage precision neurology, prioritizing human genetics-derived targets. Lario’s calcium channel focus fills a gap in disease-modifying options for epilepsies and Parkinson’s. Industry trends favor modality-agnostic platforms addressing multiple CNS indications.
IND-Enabling Studies Advance in 2026
Lario’s lead CaV2.3 program moves to IND-enabling studies in 2026, accelerated by MJFF collaborations and patient advocacy engagements like the CACNA1E International Family Conference presentation. Platform expansion targets additional indications, building on CDKL5 Forum recognition.