What is KH631?

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.

Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)

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.

Condition or disease:

Neovascular Age-related Macular Degeneration

Arms and Interventions:

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