Gene Therapy for CLN2, LX1004, Supported by $100M in Financing

Yedida Y Bogachkov PhD avatar

by Yedida Y Bogachkov PhD |

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LX1004 gene therapy | Batten Disease News | illustration of a man poised over a microscope, with test tubes nearby

Lexeo Therapeutics announced that it raised $100 million in a financing round to help support the development of pipeline therapies, including LX1004, a potential gene therapy for CLN2 disease, also known as late infantile Batten disease.

Other potential treatments supported through this Series B financing include LX2006, a gene therapy for cardiomyopathy associated with Friedreich’s ataxia, and LX1001, a gene therapy for APOE4-associated Alzheimer’s disease, the company announced in a press release.

“As we embark on our next phase of growth, we are highly encouraged by the support of this diverse range of long-term focused investors participating in our Series B financing,” said R. Nolan Townsend, CEO of Lexeo Therapeutics.

“With this capital infusion, we plan to advance our clinical stage pipeline to meaningful near-term data catalysts,” he added.

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LX1004 is a gene therapy mediated by adeno-associated virus (AAV). It is designed to deliver a healthy copy of CLN2, the gene that provides instructions to make an enzyme called tripeptidyl peptidase 1 (TPP1), to the central nervous system (CNS; the brain and spinal cord).

When CLN2 is mutated, as is the case in late infantile Batten disease, a lack of TTP1 causes cells, in particular neurons or nerve cells, die. This results in symptoms such as seizures and the loss of motor skills and cognitive ability.

A previous Phase 1 clinical trial (NCT00151216) led by researchers at Weill Medical College of Cornell, where the therapy was developed, assessed the safety of delivering the CLN2 gene using an AAV vector into the CNS of 10 patients with CLN2 disease. Reported results showed a slowing in the known rate of neurological decline in children given the gene therapy.

The company earlier this year announced plans to move the therapy into a pivotal efficacy study in 2022.

“We believe in LEXEO’s mission and are proud to invest in a company led by experts in gene therapy. We are impressed by the company’s pipeline and are delighted to support its continued growth as it strives to develop meaningful treatments for patients,” said Dan Sundheim, founder of D1 Capital Partners, one of the financing leaders.

Recently, the U.S. Food and Drug Administration (FDA) granted both orphan drug and rare pediatric disease designations to LX1004. These designations offer financial and other incentives to support potential treatments for rare diseases.