HOW DOES A GENE THERAPY GET DEVELOPED?

Stages of Development for LX2006 Gene Therapy

The drug development process can be thought of as a series of stages, and successful drugs must pass through each stage to become available to patients.

Link to Lexeo Therapeutics

Dr Ron Crystal, Professor and Chairman of the Genetic Medicine Department at Weill Cornell Medicine, studied adeno associated virus serotypes in cellular and animal model systems.

Dr. Crystal published several papers on AAVrh.10 serotype showing good CNS expression of the transgene payload.

2018: Dr. Crystal founded Lexeo with National Institutes of Health small business grants.

January 2021: Lexeo raised a $85 million Series A to develop 18 preclinical and clinical programs, including a lead program for cardiomyopathy in FA.

March 2021: LEXEO announced completion of a license of all intellectual property rights and pre-clinical data associated with Adverum Biotechnologies’ investigational Friedreich’s ataxia program.

AAVrh.10hFXN is a nonhuman primate-derived serotype rh.10 capsid with a constitutive promoter driving the normal human frataxin cDNA. AAVrh.10hFXN is administered intravenously, a vector and route which in experimental animal models effectively delivers genes to the heart. Based on preclinical efficacy data in two murine models, intravenous AAVrh.10hFXN reverses the consequences of FA cardiomyopathy.

2022: Dr. Crystal opened an IND to study AAVrh.10hFXN gene therapy for the cardiomyopathy of Friedreich’s Ataxia.

Lexeo announced approval of their Investigational New Drug (IND) application for LX2006, an AAV-based gene therapy candidate for Friedreich’s Ataxia cardiomyopathy.

 

July 2022: LEXEO initiated an open-label, dose-escalation Phase 1/II clinical trial of LX2006 in patients with FA cardiomyopathy.

June 2023: Lexeo announced the completion of the first dose cohort and the dosing of the first patient in the second dose cohort in SUNRISE-FA, a Phase 1/II clinical trial of LX2006 in patients with Friedreich’s ataxia (FA) cardiomyopathy.

Initial data from the first two cohorts, including myocardial protein expression and biomarkers measuring serum and cardiac structure and function is expected in the first half of 2024.

April 2024: Lexeo and Weill Cornell agreed to grant Lexeo a license to current and future clinical data from the Dr. Ron Crystal’s ongoing investigator-initiated Phase 1A trial of AAVrh.10hFXN to treat FA cardiomyopathy.

HOW DOES THIS TREATMENT WORK?

Gene Therapy for Friedreich’s Ataxia

This brief video serves as an introduction to gene therapy for FA. The aim of this video and accompanying educational resources is to provide information to help individuals and families make informed decisions regarding participation in clinical trials exploring investigational drugs or gene therapy.

Image of Video title - Gene Therapy

News and Press Releases

News
| Jul 15, 2024

LEXEO Therapeutics Announces Positive Interim Phase 1/2 Clinical Data of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy

Industry News
News
| Apr 22, 2024

Lexeo Therapeutics Announces License Agreement to Accelerate Development of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy

Industry News
News
| Apr 16, 2024

Lexeo Therapeutics Granted FDA Fast Track Designation for LX2006, an AAV-Based Gene Therapy Candidate for the Treatment of Friedreich's Ataxia Cardiomyopathy

Industry News
News
| Jun 13, 2023

Lexeo Therapeutics Announces Completion of First Cohort and Dosing in Second Cohort in SUNRISE-FA, a Phase 1/2 Clinical Trial of LX2006 for the Treatment of Friedreich's Ataxia Cardiomyopathy

Industry News
News
| Feb 16, 2022

Lexeo Announces FDA Clearance of IND Application for LX2006 for Friedreich's Ataxia Cardiomyopathy

Industry News
News
| Jun 30, 2021

Lexeo Therapeutics Receives Rare Pediatric Disease & Orphan Drug Designations for LX2006

Industry News
News
| Mar 1, 2021

Lexeo Therapeutics Announces License Agreement and Consolidation of Pre-Clinical Data Package

Industry News
News
| Jan 6, 2021

Lexeo Therapeutics Launches with $85 Million Series A Financing to Develop Gene Therapies

Industry News

LX2006 Lexeo Therapeutics Gene Therapy Clinical Trials

Gene Replacement
Clinical Trial | Ages 18-50

Gene Therapy for Cardiomyopathy Associated with Friedreich’s Ataxia – LX2006

Lexeo Therapeutics
Phase I/II | In-person
Gene Replacement
Clinical Trial | Ages 18-50

Phase 1 Clinical Trial of an Investigational Gene Therapy for Cardiac Disease Associated with Friedreich’s Ataxia – AAVrh.10hFXN

Weill Medical College of Cornell University
Phase I | In-person