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Gene therapy for long-term treatment of GSDIa
Unmet Need Glycogen storage disease type Ia (GSDIa), or von Gierke disease, is a rare heritable metabolic disorder that impacts the liver and kidneys due to an inability to break down glycogen. An estimated 1…
A method for treating critical limb ischemia and other ischemic injuries
Unmet Need The most severe stage of peripheral artery disease, a chronic vascular disease characterized by impaired circulation to patients’ lower extremities, is critical limb ischemia (CLI), and it affects approximately two million people in…
An injectable hydrogel scaffold that delivers gene therapies locally
Unmet Need Gene therapy has applications ranging from tissue engineering to vaccine development to infectious disease. Most approaches to deliver nucleic material involve the systemic intravenous delivery of condensed nucleic acid (e.g., within a virus…