Lexeo Licenses Rights to Potential Gene Therapy LX2006

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by Diana Campelo Delgado |

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LX2006 for Friedreich's ataxia

Lexeo Therapeutics has entered an agreement with Adverum Biotechnologies to license Adverum’s intellectual property rights and pre-clinical data of LX2006, an investigational gene therapy for Friedreich’s ataxia (FA).

Lexeo will continue to advance the gene therapy program through pre-clinical studies that support an investigational new drug (IND) application, which requests regulatory authorities to initiate clinical testing. A Phase 1 clinical trial is expected to begin this year.

“Our agreement with Adverum, adding to studies conducted by other academic partners, greatly strengthens our pre-clinical data package supporting LX2006,” R. Nolan Townsend, CEO of Lexeo Therapeutics, said in a press release.

Friedreich’s ataxia is caused by a mutation in the FXN gene that provides the instructions for making a protein called frataxin, which is essential for mitochondria — the cell’s energy-producing powerhouses — to work as they should.

Frataxin deficiency has a pronounced effect on heart muscle cells, which have the highest energy demands of all cells in the body, damaging heart muscles and impairing their function. Hence, heart failure, or cardiomyopathy, is the most common cause of death among FA patients.

“Our goal is to address this challenging component of Friedreich’s ataxia with the first potentially disease-modifying therapy for this underserved patient population,” Townsend said.

LX2006 is a gene therapy that is intended to address the heart problems associated with FA. It is administered intravenously (directly into a vein) and uses a modified harmless virus, called an adeno-associated virus (AAV), to deliver a functional copy of the FXN gene to cells in the heart.

A total of seven pre-clinical studies, conducted at research institutes in the U.S. and France, have helped optimize the current LX2006 program. These have investigated a number of vectors for delivering the gene to heart cells, multiple dose ranges, and routes of administration.

Also, in a new mouse model that more accurately mirrors the early cardiac abnormalities seen in FA patients, researchers found that LX2006 restored normal heart function.

In addition to this program for FA patients, Lexeo has two gene therapy programs in early clinical testing for Batten and Alzheimer’s diseases. LX1004, the Batten’s treatment, has completed a Phase 1/2 trial with promising safety and efficacy signs, while the Alzheimer’s LX1001 is in Phase 1 testing.