Gene Therapy
The goal of gene therapy is to treat or possibly prevent disease. Scientists have been investigating and evolving gene therapy for more than 50 years. One goal of gene therapy research is to determine whether a new or functional gene can be used to restore the function of, or inactivate, a mutated gene.
Our Science
At GeneLeap Biotech, we develop highly innovative drugs based on synthetic modified messenger RNA (mRNA). These mRNAs encode virtually any conceivable natural or engineered protein and are designed to be taken up by the cells in specific tissues and organs. We focus on diseases wherein enabling targeted cells to produce – or turn ‘on’ – one or more proteins helping the body to fight or prevent disease.
we are expanding the use of adeno-associated viral (AAV) vectors against strategically selected cell targets, including the retina, liver, and central nervous system. Additionally, we are actively advancing the development of oligonucleotide-based TLR9 agonists for immune-oncology applications allows reduced dosing frequency with improved efficacy for cancer patients with unmet medical needs distinguishing from the current treatments in the clinic.