Lario Therapeutics develops first-in-class precision medicines designed to be disease-modifying treatments for severe neurological disorders such as epilepsy and Parkinson's disease. The company focuses on a rapidly advancing pipeline built upon genetically validated targets for broad therapeutic potential. Their work aims to slow the progression of devastating early childhood syndromes and adult neurodegenerative conditions.
Funding
Funding not disclosed
Founders
Product
Problem
Severe neurological disorders, including epileptic conditions and Parkinson's disease, often lack effective, disease-modifying treatments, particularly for rare genetic syndromes presenting in early childhood. Current treatment options are limited, with no well-established standard of care for many of these conditions, resulting in treatment-resistant epilepsy, profound developmental delays, and movement difficulties.
Solution
Lario Therapeutics is developing first-in-class precision medicines targeting genetically validated targets to address the underlying causes of severe neurological disorders. Their approach focuses on modulating the activity of CaV2.3, a voltage-gated calcium channel in the brain, which plays a key role in neuronal excitation and has been implicated in various neurological conditions. By selectively inhibiting CaV2.3, Lario aims to reduce excessive neuronal excitation, control seizures, and potentially slow the progression of diseases like Parkinson's. Their pipeline includes programs targeting specific genetic epilepsies, drug-refractory epilepsy, and Parkinson's disease, with the goal of creating disease-modifying treatments.
Target Audience
The primary target audience includes patients suffering from severe neurological disorders such as genetic epilepsies, drug-refractory epilepsy, and Parkinson's disease, as well as the clinicians who treat these conditions.
Features
- Focus on precision medicine approaches targeting genetically validated targets.
- Development of first-in-class therapies for severe pediatric epilepsies with no established standard of care.
- Modulation of CaV2.3 calcium channels to reduce excessive neuronal excitation.
- Programs targeting Developmental Epileptic Encephalopathy Type 69 (DEE 69) and Developmental Epileptic Encephalopathy Type 2 (DEE2) or CDKL5-deficiency disorder.
- Research into CaV2.3 inhibition as a disease-modifying approach for Parkinson's disease.
- Pipeline includes programs in lead generation, lead optimization, and IND enabling studies.