LEXEO Therapeutics, Inc., a clinical stage genetic medicine company, entered an in-license agreement with Cornell University to expedite development of the investigational gene therapy candidate LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy.

Under the license agreement, Lexeo has acquired certain rights, including rights to current and future data generated in an ongoing investigator-initiated Phase 1A trial of AAVrh.10hFXN to treat FA cardiomyopathy (NCT05302271). The agreement will support Lexeo’s efforts to develop a potentially life-changing therapy for this unmet need.

The investigator-initiated trial is being conducted by Weill Cornell Medicine, which has published preclinical data that supported the first ever gene therapy IND clearance for FA, and sponsored a natural history study for almost a decade to better characterize the condition and its progression.

Lexeo previously licensed know-how relating to AAVrh.10hFXN from Weill Cornell Medicine and collaborated with researchers there to further study the candidate, which Lexeo refers to as LX2006. Lexeo is studying LX2006 in the company-sponsored, open label, dose-ascending, multicenter SUNRISE-FA Phase 1/2 trial (NCT05445323).

“The larger aggregate data set, combined with Orphan Drug, Rare Pediatric Disease, and Fast Track designations from FDA, is anticipated to facilitate an accelerated path to regulatory engagements for LX2006,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “We are excited about the opportunity to advance research in FA cardiomyopathy, which is the leading cause of death in FA and has no approved treatment options today.”

The interim clinical data readout of LX2006 is expected mid-year 2024. With the newly-licensed data, the readout will now include participants across the two studies, approximately doubling the number of evaluable patients and including patients with a treatment duration out to 18-months. Lexeo also expects to provide an analysis of natural history data and baseline characteristics for study participants from both studies to characterize the cardiovascular disease phenotype seen in FA cardiomyopathy ahead of the interim readout.

Check out our AAV CDMO service to expedite your gene therapy research
About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Related News

Related Services

Plasmids GMP Services

Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.

AAV GMP Services

Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
aav icon

Technology Platforms

PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.