With the IND clearance, the biotechnology company is set to launch a phase 1 trial of the recombinant adeno-associated virus-based gene therapy candidate in the first quarter of this year to assess its safety and tolerability

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EXG102-031 is an investigational gene therapy candidate of Exegenesis Bio for nAMD. (Credit: Mahmoud Ahmed from Pixabay)

Exegenesis Bio has been granted approval from the US Food and Drug Administration (FDA) for the company’s investigational new drug (IND) application for EXG102-031.

According to the biotechnology company, EXG102-031 is a recombinant adeno-associated virus (rAAV) based gene therapy candidate. It is intended to be used for the treatment of neovascular age related macular degeneration (nAMD), which is considered to be a major cause of severe vision loss and irreversible blindness around the world.

With the IND clearance, Exegenesis Bio is set to launch a phase 1 trial of the investigational gene therapy in the first quarter of this year.

The early-stage clinical study will assess the safety and tolerability of the rAAV-based gene therapy candidate along with its visual acuity and central retinal thickness in patients suffering from neovascular age related macular degeneration.

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 3 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.”

Exegenesis designed the EXG102-031 intraocular injection to express a therapeutic fusion protein that will bind or neutralise all known subtypes of Vascular Endothelial Growth Factor (VEGF) and Angiopoietin-2 (ANG2).

Both VEGF and ANG2 are known to stimulate the formation of abnormal blood vessels and vascular leakage in the retina.