Lario Therapeutics wins $2.4m grants to advance calcium channel drugs for Parkinson’s and PTSD

The funding is split between two programmes. The larger award, $1.5 million (€1.27m) from MJFF, will support Lario’s work on CaV1.3, a calcium channel encoded by the gene CACNA1D that has been implicated in the biology of Parkinson’s disease progression. CaV1.3 was recently highlighted by MJFF’s Targets to Therapies (T2T) initiative – an expert-led programme that evaluated more than 290 potential drug targets and prioritised 21 for further validation – as one of the most promising disease-modifying targets in Parkinson’s.

A separate $900,000 (€763,000) grant from Wellcome will fund work on a second channel, CaV2.3, as a potential target in PTSD, building on large-scale human genetics research linking variation in its encoding gene, CACNA1E, to increased risk of the condition.

“Lario was founded to translate strong human genetics and neuronal biology into precision medicines for patients with severe neurological disease,” said Lario’s CEO Henning Steinhagen in a press release. “We are grateful for the continued support of The Michael J. Fox Foundation, and the funding from Wellcome which support us to advance these unique programmes towards the clinic, taking us one step closer to providing meaningful treatments for patients with high unmet need.”

A genetics-led approach in a field short on disease-modifying drugs

The rationale behind Lario’s platform is that dysfunctional calcium channels, which regulate when and how neurons fire electrical signals, are directly involved in the pathology of several neurological diseases. The company is developing selective small-molecule inhibitors designed to target specific channels that human genetics have linked to disease, rather than the broader symptoms.

In PD, this distinction matters especially. The condition affects an estimated six to 11 million people worldwide, and despite a robust clinical pipeline targeting well-known mechanisms such as alpha-synuclein, LRRK2 and GBA1, there are still no approved therapies that slow or halt disease progression. Most current treatments manage motor symptoms but do not address the underlying neurodegeneration.

The Michael J. Fox Foundation’s T2T initiative was designed to address this gap and systematically identify and de-risk targets that could attract drug development investment in treatments that could halt the progression of the disease. CaV1.3’s inclusion among the final 21 targets reflects what the T2T team considered strong mechanistic evidence linking the channel to neuronal vulnerability in PD.

Building a multi-indication platform from a single target class

The new grants expand what is already a substantial portfolio. Lario was founded in 2021 as a spin-out from Epidarex Exeed, with seed investment from Epidarex and Axxam. In 2024, it received a separate $6 million (€5.08m) grant from the Michael J. Fox Foundation to develop selective CaV2.3 inhibitors for Parkinson’s disease and to explore the channel’s therapeutic potential across other CNS conditions. The Wellcome grant now extends CaV2.3 work into PTSD, building on large-scale human genetics research linking variation in CACNA1E to increased risk of the condition.

Lario’s lead programme, however, remains focused on CaV2.3 in severe developmental and epileptic encephalopathies (DEEs), a condition that genetic and functional studies have also linked to the CACNA1E gene. The company plans to initiate IND-enabling studies this year, with first-in-human clinical trials to follow.

“These awards recognise the growing body of evidence linking neuronal calcium channel dysfunction to the core biology of neurological and psychiatric diseases,” said Tom Otis, Lario’s CSO, who also serves as an adviser to the T2T initiative. “By combining selective small-molecule chemistry with rigorous target biology, we are building a unique platform designed to deliver precision therapies for patients suffering from epilepsy, Parkinson’s disease and post-traumatic stress disorder.”