# Muscular Dystrophy (MyoDys®)
· A gene therapy product candidate for muscular dystrophy.
· MyoDys® is comprised of plasmid DNA encoding the full-length human dystrophin gene.Pathway IV™ delivery technology is used to administer the pDNA to a patient’s limb skeletal muscles.
· Clinical objective: to delay or prevent loss of muscle function, initially in the hand and forearm, thereby enhancing the patient’s quality of life.
· Being developed with Transgene S.A. of Strasbourg, France as part of a strategic alliance to develop gene therapies for muscular dystrophy.
· Development status: Phase I study has been completed (data released June 5, 2003 by Transgene) which has shown safety and dystrophin expression in skeletal muscle in Duchenne and Becker patients using an alternative delivery approach. A Phase I/II study is now in preparation using Pathway IV™ that will provide regional delivery to forearm muscles.
# Peripheral Ischemia (PAD)
· A gene therapy opportunity that utilize Pathway IV™.
· Inducing revascularization of the limb by delivery of a gene(s) encoding an angiogenic factor to the affected limb.
· Clinical objective: reduce ischemia and to delay or prevent loss of limb, thereby enhancing the patient’s quality of life.
· Development status: still in research; currently evaluating various candidate angiogenic genes in model systems.
# Anemia (EPO)
· Utilize Pathway IV™ protocol.
· Once the DNA is inside target muscle cells, it is anticipated that the patient’s own body will naturally produce sufficient EPO (erythropoietin) to regulate and maintain a stable volume of red blood cells in the bloodstream.
· Development status: current studies are supported by a SBIR Phase II grant from the NIH. The initial target group for such a gene therapy approach will most likely be end-stage renal failure patients.
· A gene therapy product candidate for muscular dystrophy.
· MyoDys® is comprised of plasmid DNA encoding the full-length human dystrophin gene.Pathway IV™ delivery technology is used to administer the pDNA to a patient’s limb skeletal muscles.
· Clinical objective: to delay or prevent loss of muscle function, initially in the hand and forearm, thereby enhancing the patient’s quality of life.
· Being developed with Transgene S.A. of Strasbourg, France as part of a strategic alliance to develop gene therapies for muscular dystrophy.
· Development status: Phase I study has been completed (data released June 5, 2003 by Transgene) which has shown safety and dystrophin expression in skeletal muscle in Duchenne and Becker patients using an alternative delivery approach. A Phase I/II study is now in preparation using Pathway IV™ that will provide regional delivery to forearm muscles.
# Peripheral Ischemia (PAD)
· A gene therapy opportunity that utilize Pathway IV™.
· Inducing revascularization of the limb by delivery of a gene(s) encoding an angiogenic factor to the affected limb.
· Clinical objective: reduce ischemia and to delay or prevent loss of limb, thereby enhancing the patient’s quality of life.
· Development status: still in research; currently evaluating various candidate angiogenic genes in model systems.
# Anemia (EPO)
· Utilize Pathway IV™ protocol.
· Once the DNA is inside target muscle cells, it is anticipated that the patient’s own body will naturally produce sufficient EPO (erythropoietin) to regulate and maintain a stable volume of red blood cells in the bloodstream.
· Development status: current studies are supported by a SBIR Phase II grant from the NIH. The initial target group for such a gene therapy approach will most likely be end-stage renal failure patients.
# Cancer Vaccines
. One strategy to break tolerance and induce immunity involves genetic vaccination employing homologous TAA sequences from a different species.
. In proof-of-concept studies, Mirus Bio has demonstrated that Pathway IV ™ delivery of plasmid DNA encoding tumor antigens can:
.Generate a robust humoral response.
. Promote expansion of antigen-specific CD8+ T cells.
. Induce cytolytic T-lymphocyte (CTL) activity.
. Induce an effective anti-tumor response.
