Lysosomal Therapeutics

Lysosomal Therapeutics Inc. (LTI) was a biopharmaceutical company established in 2011, headquartered in Cambridge, Massachusetts. The company focused on developing small-molecule therapies for severe neurodegenerative diseases, particularly those with a genetic link to lysosomal dysfunction. In October 2020, LTI was acquired by BIAL, a Portuguese pharmaceutical company, which then established BIAL Biotech in the United States to continue the development of LTI's programs.

LTI’s scientific foundation was built upon the research of its founders, which includes Dr. Dimitri Krainc, Dr. Peter Lansbury, Henri Termeer, and Bob Carpenter. Dr. Krainc's work at Massachusetts General Hospital and Harvard Medical School was instrumental in launching the company. His research focused on defining the molecular pathways in neurodegeneration, especially by uncovering pathogenic mechanisms related to mitochondria and lysosomes. Dr. Lansbury, a Professor of Neurology at Harvard Medical School and Chief Scientific Officer at LTI, has a deep background in the biochemistry of protein aggregation in diseases like Alzheimer's and Parkinson's. He previously founded Link Medicine, which was sold to AstraZeneca. The company's founding President and CEO was Kees Been, a biotech industry veteran with experience at companies like Biogen and a background in molecular biology and business administration.

The company's primary business was the research and development of drug candidates to treat the root cause of neurodegenerative disorders like Parkinson's disease. LTI's approach was centered on the clinically validated connection between lysosomal storage disorders, such as Gaucher disease, and a predisposition to Parkinson's. Specifically, the company worked on developing molecules that could activate the glucocerebrosidase (GCase) enzyme. Mutations in the GBA1 gene lead to reduced GCase activity, which is a significant risk factor for Parkinson's disease, contributing to the toxic aggregation of the alpha-synuclein protein. LTI developed small molecules capable of crossing the blood-brain barrier to increase GCase activity, aiming to restore lysosomal function and normalize alpha-synuclein levels. Its lead drug candidate, LTI-291, was designed to stimulate GCase activity in the brain and was tested in human clinical trials for safety and tolerability in both healthy volunteers and individuals with Parkinson's carrying a GBA1 mutation.

LTI's business model relied on venture capital funding and strategic partnerships with larger pharmaceutical companies. The company successfully raised significant capital through multiple funding rounds, including a $4.8 million seed round in 2014 and a $20 million Series A round in 2015. Investors included prominent venture funds such as Atlas Venture, Hatteras Venture Partners, and the corporate venture arms of Roche, Eli Lilly, and Sanofi. The company also received grant funding from The Michael J. Fox Foundation for Parkinson's Research to support a biomarker initiative aimed at patient selection for clinical trials. In 2017, Allergan purchased an exclusive option to acquire LTI, though the deal did not proceed, leading to the eventual acquisition by BIAL in 2020.

Keywords: Lysosomal Therapeutics, BIAL Biotech, neurodegenerative disease treatment, Parkinson's disease, GBA1 gene mutation, glucocerebrosidase, GCase activator, LTI-291, small-molecule therapy, lysosomal storage disorders, alpha-synuclein, drug discovery, Dimitri Krainc, Peter Lansbury, Kees Been, neurodegeneration research, Gaucher disease, clinical trials, biopharmaceutical, venture capital, pharmaceutical acquisition, personalized medicine, neurogenetics, blood-brain barrier, enzyme activation