CLAIRIgene is pioneering a novel approach to gene-targeted epigenome therapy, specifically aimed at addressing unmet medical needs in central nervous system (CNS) disorders such as Alzheimer's disease (AD) and Parkinson's disease (PD). The company's primary technology leverages an innovative AAV-CRISPR/deactivated (d)Cas9-repressor platform, which allows for precise modulation of gene expression without altering the underlying DNA sequence. This epigenome editing technique utilizes deactivated nucleases to modify epigenetic markers, enabling the activation or repression of disease-associated genes. By employing a proprietary all-in-one AAV system, CLAIRIgene aims to enhance the efficiency of gene delivery, overcoming challenges associated with traditional dual AAV vectors and bulky CRISPR/Cas systems.

The company's lead program, CLRI-002, targets the APOE4 allele, which is linked to late-onset Alzheimer's disease, and has shown promising results in preclinical studies. CLAIRIgene's platform also includes additional candidates targeting various neurodegenerative conditions, such as CLRI-001 for Parkinson's disease and CLRI-004 for Lewy Body Dementia. The technology is backed by significant funding from reputable sources, including the National Institute of Aging and the Michael J. Fox Foundation, and is supported by partnerships with established organizations like Seelos Therapeutics and Duke University. This collaborative approach, combined with the expertise of its founding scientists, positions CLAIRIgene at the forefront of developing next-generation therapeutics for CNS disorders.

United States

CLAIRIgene is a preclinical stage biopharmaceutical company focused on development gene therapies for neurodegenerative diseases. Founded by innovative scientists with decades of experience, CLAIRIgene is committed to rapidly advancing their therapeutics to the clinic.

CLAIRIgene

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