close

Location

345 Park Avenue South, 6th Floor
New York, NY 10010
(212) 547-9879

Friedreich Ataxia

LX2006

Friedreich ataxia (FA) is a genetic, progressive, degenerative multi-system disorder with a prevalence of one in 50,000 in the United States. It is estimated that approximately 80% of those with FA will develop cardiomyopathy.

  • Breakthrough Therapy Designation (FDA)
  • Regenerative Medicine Advanced Therapy (RMAT) Designation (FDA)
  • Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Participant (FDA)
  • Rare Pediatric Disease Designation (FDA)
  • Orphan Drug Designation (FDA)
  • Orphan Medicinal Product Designation (EC)
Programs: Indication: Target
Pre-clinical: Discovery Preclinical
Clinical: Phase I/II Phase II/III
Cardiac programs
Pre-clinical: Discovery Preclinical
Clinical: Phase I/II Phase II/III
LX2006 Cardiac Friedreich Ataxia FXN

Disease Overview

Friedreich ataxia (FA) is a genetic, progressive, degenerative multi-system disorder with a prevalence of 1:50,000 in the United States. It is estimated that approximately 80% of people with FA will develop cardiomyopathy. Friedreich ataxia is caused by a mutation in the FXN gene that disrupts the normal production of the protein frataxin, which is critical to the function of mitochondria in a cell and to the maintenance of cardiac function.

Friedreich ataxia is inherited in an autosomal recessive manner, where both inherited genes are abnormal, and symptoms usually begin in childhood. Absence of fully functional frataxin leads to damage to peripheral nerves and the parts of the brain that controls movement and balance, leading to neurological symptoms that include impaired muscle coordination, or ataxia, that worsen over time.

As the disease progresses, patients typically experience various heart conditions, including thickening of the heart muscle, or hypertrophic cardiomyopathy, and arrhythmias. Hypertrophic cardiomyopathy, fibrosis, heart failure and arrhythmias are the cause of death in approximately two-thirds of people with FA.

There is no approved treatment for FA cardiomyopathy. As a result, people with cardiomyopathy associated with Friedreich ataxia have significant unmet need.

LX2006 mechanism

We are developing LX2006 as an AAV-based gene therapy delivered intravenously for the treatment of FA cardiomyopathy.

LX2006 is designed to transfer the FXN gene to myocardial cells and increase frataxin levels in the mitochondria.
The increase in frataxin levels in the mitochondria restores mitochondrial function and energy production in cardiac myocytes.

Interim data from Phase I/II clinical trials of LX2006 demonstrate clinically meaningful improvements in cardiac biomarkers as well as functional cardiac and neurologic measures.