Lysosomal Therapeutics (LTI) develops small molecules that cross the blood–brain barrier to increase glucocerebrosidase (GCase) activity and address lysosome-linked neurodegeneration. Its lead program targets Gaucher-related neurodegeneration, Parkinson’s disease and other synucleinopathies. The company plans to advance its GCase activator candidate toward a clinical start while expanding its platform to explore other lysosomal enzyme deficiencies and genetic links to common neurodegenerative disorders. Recent funding will support compound optimization, preclinical development and a biomarker initiative intended to help select patients for trials and predict drug response. LTI has previously received grant funding from The Michael J. Fox Foundation for Parkinson’s Research to support the biomarker effort. To support platform expansion and partnering, LTI has hired Darren Braccia as vice president of business development. Lysosomal Therapeutics (LTI) leverages lysosomal biology to discover small molecules that increase glucocerebrosidase (GCase) activity and normalize lysosomal function in neurons. Its lead program targets Gaucher-related neurodegeneration, Parkinson’s disease and other synucleinopathies with compounds designed to cross the blood‑brain barrier. The company’s discovery work originated in Dimitri Krainc’s lab at Massachusetts General Hospital and continues with collaborators at Northwestern University. LTI plans to advance its GCase modulator platform through lead generation and into preclinical development. The firm is also investigating other lysosomal enzyme deficiencies and their links to common neurodegenerative diseases. LTI was founded by Krainc and former Genzyme executives Henri Termeer, Bob Carpenter and Peter Wirth; Kees Been is CEO. The company recently secured seed funding to support near‑term R&D and program advancement.

Total raised

$25M

Funding rounds

2

Latest round

Series A

Latest activity

Feb 2015