Lario Therapeutics

Lario Therapeutics is a biopharmaceutical company developing first-in-class, disease-modifying precision medicines for severe neurological disorders, with a focus on genetically validated targets. The company was founded in 2021 by CEO Dr. Henning Steinhagen and Head of Project Management Daniel Gill. It was established as a spin-out from Epidarex Exeed, the therapeutic discovery engine of Epidarex Capital, with initial seed investment from Epidarex Capital and Axxam.

Lario Therapeutics' primary mission is to create novel treatments for patients with conditions such as severe pediatric epilepsies, including CDKL5 Deficiency Disorder (CDD), and neurodegenerative disorders like Parkinson's disease. The company's therapeutic pipeline centers on modulating ion channels, such as their lead programs on CaV2.3 inhibitors. These orally active, CNS-penetrant compounds are being investigated for their potential as anti-seizure therapeutics and as a disease-modifying approach for Parkinson's. The company's approach is based on the discovery that CaV2.3 is a key target for the CDKL5 protein, which is deregulated in CDD, a prevalent form of genetic Developmental and Epileptic Encephalopathy (DEE).

The company's business model involves advancing its pipeline of genetically validated programs through preclinical development towards Investigational New Drug (IND) applications and clinical trials. Lario Therapeutics has secured significant funding to support its research, including a $6 million grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF) in July 2024 to support its preclinical research on CaV2.3 calcium channel inhibitors. This grant also supports a collaboration with the Oxford Parkinson's Disease Centre. In November 2023, the company received the “Company Making a Difference Award” from the Loulou Foundation at the CDKL5 Forum, recognizing its commitment to preclinical therapeutic development for CDD.

Keywords: biopharmaceutical, precision medicine, neurological disorders, neurodegenerative diseases, genetic epilepsy, Parkinson's disease, drug discovery, ion channel inhibitors, CaV2.3 modulators, CDKL5 Deficiency Disorder, disease-modifying treatments, preclinical research, CNS-penetrant, anti-seizure therapeutics, therapeutic pipeline, venture capital, biotechnology, Edinburgh, clinical trials, orphan diseases