Exegenesis Bio has received clearance from the US Food and Drug Administration (FDA) for its investigational new drug (IND) application for gene therapy EXG102-031.
The recombinant adeno-associated virus (rAAV)-based gene therapy is being studied to treat neovascular Age Related Macular Degeneration (nAMD), which causes severe loss of vision and irreversible blindness across the globe.
Exegenesis Bio CEO Zhenhua Wu said: “We are excited by the progress that our company has made and pleased to have reached this critical milestone; this is our second IND approval and the first in North America since the inception of Exegenesis Bio three years ago.
“This is a strong validation of the world-class R&D, CMC, quality and regulatory capabilities that we have built. We look forward to accelerating development of our innovative gene therapy pipeline in areas with high unmet medical needs and bringing these innovative treatments to patients worldwide.”
The EXG102-031 intraocular injection expresses a therapeutic fusion protein that can attach/neutralise all known Vascular Endothelial Growth Factor (VEGF) and Angiopoietin-2 (ANG2) subtypes.
These subtypes stimulate formation of abnormal blood vessel and vascular leakage in the retina.
A Phase I clinical trial of the gene therapy will assess the tolerability and safety along with visual acuity and central retinal thickness in nAMD patients. This trial is expected to be initiated in the first quarter of this year.
Damage to the macula in the center of the retina causes AMD, a progressive eye disease.
This damage can lead to blurred vision and blindness.
Globally, nAMD accounts for nearly 90% of all blindness related to AMD.