Startup Lexeo Raises $85M to Pursue 3 New Gene Therapies

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Lexeo Therapeutics recently launched with an initial funding of $85 million to develop gene therapies for Friedreich’s ataxia (FA) and other disorders.

The proceeds will support the clinical development of three gene therapy programs, including an anticipated Phase 1 trial this year to test LX2006 for FA-associated heart disease (cardiomyopathy).

Clinical development of gene therapies for forms of Batten and Alzheimer’s disease caused by single gene mutations (monogenic conditions) will also advance with this initial funding.

“We are thrilled to launch today with a mission to advance Lexeo’s promising clinical-stage pipeline of gene therapy treatments for patients diagnosed with some of society’s most challenging diseases,” R. Nolan Townsend, chief executive officer of Lexeo, said in a press release.

FA is a rare genetic disease caused by mutations in the FXN gene, and a consequent deficiency in the frataxin protein. Frataxin is essential for mitochondria, the energy-producing compartments of cells, to work as they should.

Frataxin deficiency has a pronounced effect on heart muscle cells, which have the highest mitochondria density of all cells in the body, damaging heart muscles and impairing their function. Thus, severe heart disease is the most frequent cause of death among FA patients.

LX2006 is a gene therapy designed to treat and prevent heart problems associated with FA, namely a form of heart disease called cardiomyopathy in which heart muscles become enlarged, thick, or rigid, causing weakness of the heart muscles.

To achieve that, the intravenously (into-the-vein) administered treatment uses an attenuated virus, called adeno-associated virus, to deliver a functional copy of the FXN gene to cells in the heart. Lexeo is completing preclinical studies of LX2006 to support an investigational new drug application requesting that the treatment advance to clinical trials.

The gene therapies for Batten and Alzheimer’s — dubbed LX1004 and LX1001 — are already in early stages of clinical testing. LX1004, which corrects mutations in the CLN2 gene leading to Batten, completed a Phase 1/2 trial with promising safety and efficacy signs. An ongoing Phase 1 trial is testing LX1001 in people with Alzheimer’s who carry two copies of the APOE4 gene.

Lexeo has 15 additional AAV gene therapy in its pipeline for the treatment of single gene or inherited conditions. All were initially developed at Weill Cornell Medicine.

The team at Lexeo includes industry veterans that have extensive expertise in the field of rare diseases and gene therapies.

Ronald G. Crystal, MD, founder and chief scientific advisor of Lexeo, is a gene therapy pioneer, with more than 30 years of experience with viral vectors for gene therapy, who also has a vast knowledge about diseases of the heart and central nervous system (brain and spinal cord).

“I am excited to work with Lexeo Therapeutics to move our extensive academic portfolio into clinical development and ultimately bring it to patients,” said Crystal, who’s also professor and chairman of Weill Cornell’s department of genetic medicine, and director of the Belfer Gene Therapy Core Facility.

“Lexeo’s AAV-mediated gene therapy programs have the potential for broad applicability across a range of therapeutic indications, and in a single company pipeline present an opportunity for the natural evolution of gene therapy from rare genetic conditions to more common diseases,” he added.

Crystal is joined at Lexeo by Townsend, who served for years as a leader of the rare disease program at Pfizer.

“We have the pleasure of collaborating with Dr. Ronald Crystal — one of the industry’s most accomplished pioneers in the gene therapy space — and, drawing on my years of rare disease leadership experience at Pfizer, will together build a world-class gene therapy organization driven by premier science that has the potential to address a range of therapeutic indications,” said Townsend.

The chairman of Lexeo is Steven Altschuler, MD, who was chairman of gene therapy biotech pioneer Spark Therapeutics and involved in the first gene therapy, called Luxturna, that was approved by the U.S. Food and Drug Administration for people with inherited eye disease.

“Lexeo’s impressive management team, with Nolan’s years of rare disease leadership experience at Pfizer, as well as its pioneering scientific founder and high-quality investor syndicate, will propel the development of the company’s pipeline of promising and innovative programs,” said Altschuler. “I am honored to have the opportunity to partner with Nolan and his team to build a leading gene therapy company.”

The $85-million Series A financing was led by Longitude Capital and Omega Funds, along with Lundbeckfonden Ventures, PBM Capital, Janus Henderson Investors, Invus, Woodline Partners LP, the Alzheimer’s Drug Discovery Foundation, and Alexandria Venture Investments.