Lysosomal Therapeutics Secures $4.8M in Financial Funding.


Lysosomal Therapeutics Secures $4.8M in Financial Funding.

A company leveraging its expertise in lysosomal biology to develop novel small molecules for use in treatment of neurodegenerative diseases, Lysosomal Therapeutics, has recently announced that it has managed to raise a total $4.8 million in financial funding. Atlas Venture was the lead investor, with additional participation from Hatteras Ventures Partners, Lilly Ventures, Sanofi-Genzyme BioVentures, Roche Venture Fund, Partner  Innovation Fund and several angel investors, including Orion Equity Partners, LLC. LTI was founded by Dimitri Krainc, M.D., Ph.D., and former Genzyme executives Henri Termeer, Bob Carpenter and Peter Wirth. Kees Been is the founding president and chief executive officer of the company.


LTI’s unique approach to discovering new drugs for neurodegenerative diseases is based on research performed in Krainc’s lab at Massachusetts General Hospital(MGM), a founder member Partners Healtch Care and an affiliate of Harvard Medical School. The work was completed at the MGH by Kranic and Joseph Mazzulli, Ph.D , LIT’s Scientific co-founder and former postdoctoral fellow in the labs, builds on the association between Gaucher disease, a rare lysosomal storage disorder caused by mutations in the gene for glucocerebrosidase , and a predisposition to Parkinson’s disease.

They have demonstrated that increasing the Gcase activity in human neurons of GD and PD patients can normalize lysosomal function and improve neuronal survival. This research continues at Northwestern University Where Kranic Is currently Ward Professor, and chairman of the department neurology, and Mazzulli is an assistant professor of neurology.


Compounds that enhance GCase activity represent a potential breakthrough opportunity to develop an entirely new class of agents to treat GD and PD. The risk of developing PD in the broader population has also been firmly linked to GCase mutations. This illustrates how rare diseases can be used as model systems for developing therapeutics for common neurodegenerative disorders.

“This inventive approach to treating Parkinson’s disease was really facilitated by the success of current treatment strategies, such as enzyme replacement therapy, that have enabled the Gaucher disease patient population to live longer,” said Been. “However, an unfortunate consequence of this longevity is the finding that Gaucher patients experience a 20-fold increased risk of developing Parkinson’s disease. At LTI, our lead development program is a completely novel approach to treating Parkinson’s disease by developing small molecules that can cross the blood-brain barrier to increase the activity of the GCase enzyme in the lysosome and potentially reduce Parkinson’s disease risk.”

“This seed funding will be instrumental in enabling LTI to progress its GCase program through the lead-generation process and in setting us up for preclinical development,” said Carpenter, who chairs LTI’s board of directors. “I look forward to working with Kees and his team to refine LTI’s GCase modulator platform over the coming months, with an eye toward advancing breakthrough therapeutics for Parkinson’s disease.”

“We are pleased to support LTI through this seed round of funding,” said Bruce Booth, Ph.D., partner at Atlas Venture. “Atlas believes that LTI’s GCase modulator platform holds great promise as a foundation for discovering therapeutics for neurodegenerative diseases like Parkinson’s, through exploring the implications of known relationships between lysosomal storage and neurodegenerative disorders.”

“Our long-term mission is to develop targeted therapies for children and adults with neurodegenerative disorders that will be based on in-depth, mechanistic understanding of these disorders,” added Krainc.

With this recent funding, new board members shall be included, such as Booth, Clay Thorp, general partner at Hatteras Venture Partners, Steve Hall, Ph.D. Venture partner at Lilly Ventures, Bernard Davitian, vice president and managing director of Sanofi-Genzyme BioVentures, and Carole Nuechterlein, head of Roche Venture Fund. CEO Been also serves as a member of the board of directors. Nessan Bermingham, Ph.D., venture partner at Atlas Venture, and Reza Halse, Ph.D., partner at Partners Innovation Fund, have been appointed as board observers.

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