Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

May 19 2010

Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery technologies, announced today that it has received a National Institutes of Health (NIH) small business technology transfer (STTR) grant for a program on "Lentiviral Gene Therapy for Mucopolysaccharidosis." In this program, Lentigen will collaborate with Dr. R. Scott McIvor Professor, Department of Genetics, Cell Biology and Development, and Dr. Walter Low, Professor, Departments of Neurosurgery and Physiology at the University of Minnesota, Minneapolis, MN.

Hunter syndrome (Mucopolysaccharidosis type II, MPS II) is a rare X-linked recessive, inherited lysosomal storage disorder with an onset at 2-4 years of age.  It has been estimated to affect approximately 1 in 155,000 live male births.  It is caused by the absence of iduronate-2-sulfatase, resulting in systemic accumulation of glycosaminoglycans. Affected individuals suffer from skeletal abnormalities, organomegaly, life-threatening obstructive airway disease, and, in the severely enzyme deficient form, neurologic degeneration and death by age 15. The University of Minnesota is a world-leading center for the treatment of lysosomal storage diseases by hematopoietic stem cell transplantation.

The goal of this program is to evaluate the feasibility of using lentiviral vectors to restore the missing gene in patient's cells and return the cells back to the patient.  The specific research that will be conducted under this grant will use a mouse model of MPS II created in the laboratory of Dr. Joseph Muenzer, University of North Carolina, to establish and test conditions for introduction of the correcting gene and its effectiveness when reintroduced into the animals.

"The demonstrated safety and effectiveness of lentiviral vectors to mediate gene transfer into hematopoietic stem cells and provide clinical benefit in patients with Adrenoleukodystrophy (Lorenzo Oil's disease), as reported in the journal Science in November 2009, makes them the vector of choice for this application" stated Dr. Boro Dropulic, Lentigen's President and Chief Scientific Officer.  "This project is consistent with our strategy of applying Lentigen's technology in diseases of high unmet therapeutic need."