Date: 2021-03-01
Type of information: Licensing agreement
Compound: LX2006
Company: Lexeo Therapeutics (USA-NY) Adverum Biotechnologies (USA-CA)
Therapeutic area: Rare diseases - Neurodegenerative diseases
Type agreement: licensing
Action mechanism: gene therapy. adeno-associated virus (AAV)-mediated treatment delivered through intravenous administration to address the Friedreich's ataxia cardiac disease pathology
Disease: Friedreich's ataxia
Details: • On March 1, 2021, Lexeo Therapeutics, a clinical-stage gene therapy company, announced it has acquired worldwide intellectual property rights and pre-clinical data from Adverum Biotechnologies to its Friedreich's ataxia gene therapy program. With exclusive rights
to data from seven pre-clinical studies now combined, LEXEO will advance LX2006 through final IND-enabling studies and into a planned Phase 1 clinical trial in 2021.
LX2006 is an adeno-associated virus (AAV)-mediated treatment delivered through intravenous
administration to address the Friedreich's ataxia cardiac disease pathology. This program is supported by seven distinct pre-clinical studies conducted at preeminent scientific institutions in France and the United
States examining multiple vector constructs, dose ranges and routes of administration, all contributing to the optimization of the current LX2006 program construct that will be taken forward into the clinic in 2021.
In several pre-clinical studies, LX2006 has demonstrated
efficacy in reversing the abnormal cardiac phenotype in Friedreich's ataxia disease models by transferring a normal frataxin gene to the heart.
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