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Lexeo Therapeutics announced that it has raised $ 100 million in a funding round to support the development of ongoing therapies, including LX1004, a potential gene therapy for CLN2 disease, also known as late childhood disease. from Batten.

Other potential treatments supported by this Series B funding 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 enter our next phase of growth, we are strongly encouraged by the support of this diverse range of long-term focused investors participating in our Series B funding,” said R. Nolan Townsend, CEO of Therapeutic Lexeo.

“With this infusion of capital, we plan to advance our pipeline from clinical stages to meaningful short-term data enablers,” he added.

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LX1004 is an adeno-associated virus (AAV) mediated gene therapy. It is designed to provide 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; brain and spinal cord).

When CLN2 is mutated, as in late childhood Batten disease, a lack of TTP1 causes cell death, especially neurons or nerve cells. This results in symptoms such as seizures and loss of motor skills and cognitive abilities.

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 administration of CLN2 using an AAV vector in the CNS of 10 patients with CLN2 disease. Reported results has shown a slowing of the known rate of neurological decline in children given gene therapy.

The company announced earlier this year that it plans to transition the therapy to a pivotal efficacy study in 2022.

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

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

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