About us
Genetix Pharmaceuticals is committed to advancing the field of gene therapy through the development and commercialization of innovative treatments for chronic human diseases and cancers. Our core focus is the transfer and expression of therapeutic genes to a diverse range of target cells, with the goal of providing long-term, effective gene therapy for conditions such as genetic blood disorders, macular degeneration, and neurodegenerative diseases.
At the heart of our platform is a proprietary, next-generation lentiviral gene therapy vector. This vector has been extensively engineered for safety and efficacy in human applications, leveraging the unique characteristics of lentiviral systems to achieve stable transduction in both dividing and non-dividing cells across multiple preclinical in-vitro and in-vivo models. This breakthrough technology enables the development of both in-vivo and ex-vivo therapies for a range of chronic, inherited, and degenerative diseases.
To support our therapeutic development, we are advancing functional genomics research aimed at identifying new, therapeutically relevant genes. Through our exclusive licenses in the areas of leukemias and lymphomas, we are expanding our proprietary gene library. Additionally, our cardiovascular-focused research is centered on identifying gene vectors and biologics that promote angiogenesis, particularly for the treatment of coronary artery disease, with ongoing pre-clinical investigations showing promising results.
Genetix Pharmaceuticals is pioneering a new era in gene therapy, with the goal of providing transformative, precision medicine to treat complex diseases at their genetic root. Our ongoing research and technology development are paving the way for future breakthroughs in the treatment of serious, chronic conditions.