The first-generation gene therapy product KH631 is for unmet medical needs of ocular diseases by utilizing the naturally occurring adeno-associated vector (AAV) library platform, which has huge market potential.
Brief Summary:
VAN-2201 is Phase I clinical trial to assess the safety and tolerability of KH631 in subjects with neovascular AMD. KH631 is gene therapy designed to deliver a protein which targets and blocks VEGF via an adeno-associated viral vector. The standard of care for patients with neovascular AMD are anti-VEGF treaments, which have demonstrated improvement in vision and reduction in fluid. A one time placement of a product which inhibits VEGF has the potential to reduce the patient burden of regular intraocular injections.
Neovascular Age-related Macular Degeneration
Arms | Intervention/treatment |
Experimental: KH631 Dose 1 KH631 One-Time Intraocular Injection Dose Level 1 |
Drug: KH631 KH631: AAV vector containing a coding sequence for an anti-VEGF protein |
Experimental: KH631 Dose 2 KH631 One-Time Intraocular Injection Dose Level 2 |
Drug: KH631 KH631: AAV vector containing a coding sequence for an anti-VEGF protein |
Experimental: KH631 Dose 3 KH631 One-Time Intraocular Injection Dose Level 3 |
Drug: KH631 KH631: AAV vector containing a coding sequence for an anti-VEGF protein |
Experimental: KH631 Dose 4 KH631 One-Time Intraocular Injection Dose Level 4 |
Drug: KH631 KH631: AAV vector containing a coding sequence for an anti-VEGF protein |