Current AD, PD drugs aren’t working for those who need them most. We are committed to changing the treatment landscape for these patients. Powered by CLAIRIgene’s epigenome editing platform, our pipeline includes therapeutics that have demonstrated promising activity our preclinical studies.
PLATFORM OPPORTUNITY: CNS Gene Therapy Portfolio
- * CLRI-001 Parkinson’s SNCA
- CLRI-002 Alzheimer’s APOE e4-
- CLRI-003 Alzheimer’s APOE e3-/e4-
- CLRI-004 Lewy Body Dementia SNCA
- CLRI-005 Familial Parkinson’s SNCA
Additional potential: Rare neurodegenerative diseases caused by dominant mutations and imprinting diseases
*Licensed from Duke by Seelos Therapeutics. Sponsored Research Agreement established between CLAIRIgene/Seelos Therapeutics
CLRI-002: Gene therapy targeting APOE4
Preliminary experimental evidence clearly supports further development of CRLI-002, CLAIRIgene’s therapeutic for clinical use in APOE4 subpopulations of LOAD.
- Optimized delivery of CLRI-002 through our proprietary AAV packaging system
- Reduces APOE4 without impacting other APOE alleles
- CLRI-002 mediated reduction in ApoE4 resulted in significant reduction in the level of the pathological p-Tau, a neuropathological characteristic of AD.
- Little to no toxicity observed in ongoing studies
CLRI-001: Gene therapy targeting SNCA
Licensed to Seelos Therapeutics https://seelostherapeutics.com/sls-004/
- All-in-one lentiviral vector, for targeted DNA-methylation editing within intron 1
- The system is based on CRISPR-dCas9 fused with the catalytic domain of DNA methyltransferase 3A (DNMT3A), an enzyme that is responsible for DNA methylation
- The system was delivered to dopaminergic neurons derived from human induced pluripotent stem cells (hiPSCs) from a PD patient
- As a result, the expression of SNCA was modified and disease-related cellular-phenotypes characteristics of the neurons were reversed