Gene Therapy as a Treatment Modality in Cardiac and CNS Diseases: Jay Barth, MD


Lexeo recently announced FDA clearance of its investigational new drug application for LX2006 for Friedreich's ataxia cardiomyopathy.

"We view gene therapy as a treatment modality, a means toward an end, to treat diseases to improve patients' lives, not an end in and of itself." —Jay Barth, MD, chief medical officer, Lexeo Therapeutics

Gene therapy is often thought of for its application within the rare disease landscape, but its applicability is much broader in more prevalent diseases, such as Alzheimer, as well as cardiovascular and central nervous system (CNS) diseases.

Jay Barth, MD, chief medical officer at Lexeo Therapeutics, discussed the potential of utilizing adeno-associated virus (AAV) gene therapy in some of those therapeutic areas in a recent interview with CGTL.

Lexeo recently announced FDA approval of its investigational new drug application (IND) for LX2006 for Friedreich ataxia cardiomyopathy, as well as plan to initiative a phase 1/2 clinical trial mid-year.

“The potential of LX2006 is supported by our robust preclinical studies showing that LX2006 was able to significantly reverse the cardiac manifestations of the disease,” Barth said in a statement announcing the IND clearance.1

Lexeo’s pipeline also includes 3 other cardiac-targeted gene therapy candidates that are in the discovery phase, as well as 4 additional gene therapy candidates targeted towards central nervous system disorders, 2 of which are in clinical-stage development: LX1001 for the treatment of APOE2+ Alzheimer disease, which has received fast track designation,2 and LX1004 for the treatment of CLN2 Batten disease, which has received rare pediatric disease and orphan drug designations from the FDA.3

1. LEXEO Announces FDA Clearance of IND Application for LX2006 for Friedreich’s Ataxia Cardiomyopathy. News release. LEXEO Therapeutics. February 16, 2022.
2. LEXEO Therapeutics Announces FDA Fast Track Designation Granted to LX1001. News release. LEXEO Therapeutics. April 20, 2021.
3. LEXEO Therapeutics Receives Rare Pediatric Disease & Orphan Drug Designations for LX1004. News release. LEXEO Therapeutics. July 28, 2021.
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