Company Profile

The GENETIX Pharmaceuticals Mission is to develop and commercialize effective gene therapies for the treatment of serious, chronic human diseases. GENETIX has pioneered a curative therapy for hereditary blood disorders, currently in Phase I/II EU clinical trials.  As published in Science and PNAS, LentiGlobin™ hemoglobin gene therapy has demonstrated 100% correction in animal models of sickle cell disease and thalassemia major, the two most prevalent human genetic disorders, with large orphan markets. GENETIX’ proprietary LentiGlobin™ vector is based on ten years of research at Harvard and MIT. GENETIX holds exclusive patents for lentiviral gene transfer, a critical therapeutic gene, and the only gene regulator capable of high level gene expression. 

With encouraging preliminary clinical data, a clear path to commercialization, the potential to expand to additional high-value indications, an experienced team and committed US and European investors, GENETIX Pharmaceuticals is positioned to bring to market the first curative therapy for hereditary blood disorders.