A Phase I/II exploratory clinical trial to assess the safety and efficacy of dose escalation of hemophilia B gene therapy is being conducted by St. Jude’s Children Research Hospital. Based on the outcome of this trial, uniQure is now preparing additional clinical development work to establish safety, tolerability and proof-of-concept with a Factor IX gene therapy produced using uniQure’s proprietary production system.
This gene therapy serves a large market as it could replace Factor IX replacement therapy entirely. uniQure intends to partner it in the short-term.
Acute intermittent porphyria
uniQure has demonstrated that its product results in normalization of the PBGD protein in an animal model of AIP. It completely prevented the occurrence of attacks and significantly ameliorated the neuropathy that develops in untreated mice. uniQure’s partner at the Centro de Investigación Médica Aplicada (CIMA) in Pamplona (Spain) has shown persistence of expression of genes in the liver for longer than a year. Patient enrolment in a pre-treatment observational study has commenced and a clinical study with a patient treatment is expected to begin in 2nd half of 2012.
PD symptoms are caused by degeneration and death of nerve cells in the specific part of the brain that produces dopamine, a chemical, which sends messages in the brain to control movement.
uniQure’s aim is to inject rAAVs (recombinant Adeno Assosiated Viruses) that carry the gene for GDNF into the brain. uniQure is currently assembling all the necessary licenses and expertise to administer GDNF directly into the brain. The company is also collaborating with various academic institutes in establishing in vivo proof-of concept in diseases such as Multiple Systems Atrophy, Huntington’s and hearing loss.
With its partner Institut Pasteur, Paris, France, uniQure is developing a gene therapy for Sanfilippo B through the introduction of a functional gene into the patients’ brain cells. Institut Pasteur have shown proof-of concept for this product and uniQure has established a cGMP compliant process. An explorative clinical phase I/II study is expected to be initiated in 2012. uniQure receives payments for manufacturing and has the option to the commercial rights for the therapy after a successful Phase I/II trial has been finalized.
uniQure research is focused on the development of cures for rare and most of the time congenital diseases (orphan diseases). Its research efforts will fill an unmet medical need for the treatment in the areas of metabolic disorders, ocular diseases and disorders of the central and peripheral nervous system. In addition, several pre-clinical studies are in progress, which could allow us to target selected non-orphan diseases associated with serious morbidity or high mortality caused by a genetic defect.