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Neuro Disease Biotech Raises $20M in Early Funds

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3 February 2015. Lysosomal Therapeutics Inc., a biotechnology company developing treatments for inherited neurodegenerative disorders, raised $20 million in its first venture funding round. The Cambridge, Massachusetts enterprise, begun last year, is backed by several venture capital funds and pharmaceutical companies.

Lysosomal Therapeutics is based on the research of Dimitri Krainc, a neurologist now at Northwestern University, but earlier at Harvard Medical School and Massachusetts General Hospital in Boston when he and colleagues founded the company. Krainc’s research investigates accumulation of mutated proteins that occur in several neurodegenerative diseases, such as Parkinson’s disease, as well as processes that degrade the functioning of lysosomes, the parts of cells that digest and clear these aberrant proteins.

Parkinson’s disease is a progressive disease of the central nervous system and affects more than 1 million in North America and some 4 million worldwide. Symptoms often begin as tremors or shaking in limbs, but can advance into psychiatric conditions, including depression and hallucinations.

Studies done by Krainc and colleagues trace lysosomal storage disorders to mutations in the gene for glucocerebrosidase, also known as GBA or GCase, which are linked to Parkinson’s and Gaucher disease. Their findings indicate increased activity of GCase in neurons of Parkinson’s disease patients can help normalize their lysosomal storage functions. Lysosomal Therapeutics is developing a molecular therapy that stimulates GCase, as a treatment for Parkinson’s disease. The company is also investigating other lysosomal enzyme deficiencies as potential therapy targets.

First-round investors in Lysosomal Therapeutics are most of the same seed fund providers in May 2014: Atlas Venture, Hatteras Venture Partners, Lilly Ventures, Sanofi-Genzyme BioVentures, Roche Venture Fund, Partners Innovation Fund and several original angel investors. Atlas Venture led the round, raising $20 million.

In addition to gaining nearly $5 million in seed funds, Lysosomal Therapeutics also received a $230,000 Michael J. Fox Foundation grant in December 2014 to study GCase as a target for Parkinson’s disease therapies, as well as a biomarker for diagnostics. The biomarker can also help predict Parkinson’s disease patients most likely to respond to GCase treatments.

Proceeds from the new financing are expected to advance Lysosomal Therapeutics Inc.’s (LTI’s)  development of a Parkinson’s disease candidate, but also related conditions. Bruce Booth, a partner at Atlas Venture noted in a company statement that investors “are not only supporting further development of LTI’s breakthrough therapeutic mechanism for the treatment of Parkinson’s disease, but we are also enabling LTI to expand beyond Parkinson’s disease and build out a platform around additional novel targets implicated in underserved orphan and neurodegenerative disorders.”

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