LYSOSOMAL THERAPEUTICS

Developing CURES
For Neurodegenerative
diseases

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Lysosomal Therapeutics Inc. (LTI) discovers and develops new therapies for patients with severe neurological diseases affecting movement, cognition and general health.

 
 

Our innovative strategy leverages the clinically-validated link between common neurodegenerative diseases and rare lysosome-based genetic disorders to establish a unique and effective platform for novel drug discovery.

LTI-291, the company’s novel and first-in-class drug compound is aimed at treating patients with GBA-Associated parkinsonism (GBA-AP), a type of rapidly-progressing Parkinson’s disease caused by an underlying mutation in the GBA1 gene, originally discovered in Gaucher’s disease patients. 

OUR STORY

 
 
 

FOR PATIENTS

LTI is preparing to initiate human clinical studies with our drug candidate, LTI-291, for GBA-associated parkinsonism (GBA-AP).

 
 

RECENT RESEARCH INDICATES THAT

At least one million people in the United States, and more than five million worldwide, have Parkinson’s disease

 
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GBA1 MUTATION AND PARKINSON’S DISEASE

 

Approximately 10% of the overall patient population in the U.S. with clinically diagnosed Parkinson’s disease carry a GBA1 mutation.

Reports in 2004 were the first to describe that a mutated copy of the GBA1 gene is firmly associated with an increased lifetime risk of developing Parkinson's disease. We are committed to building on our understanding of the genetic and biological associations between lysosomal storage diseases and neurodegeneration.

 

OUR LEADERSHIP

 

Leveraging our world-class knowledge of lysosomal biology, enzymology and drug development

MANAGEMENT TEAM  |  BOARD OF DIRECTORS

 
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CONTACT US

LTI OFFICE

 

19 Blackstone Street
Cambridge, MA 02139

Phone: 617.714.9620
Fax: 617.945.1583

MEDIA: Kari Watson
MacDougall Biomedical Communications