Drug intelligence / Profile preview

synHSV

Development stage
Preclinical
Lead developer
Replay
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
Administration
Ophthalmic, Intrathecal, Topical
01

Overview

synHSV is a high-payload capacity, gene-deleted herpes simplex virus type 1 (HSV-1) viral vector platform developed by Replay Therapeutics. It is designed to deliver large genetic payloads that exceed the capacity of traditional adeno-associated virus (AAV) vectors. While AAV is limited to approximately 4.7kb, synHSV can accommodate payloads up to 8 times larger (30-40kb), with next-generation iterations targeting up to 150kb. The platform leverages the natural neurotropism of HSV-1 and its ability to remain episomal in the nucleus without integrating into the host genome. It is being utilized across several of Replay's product companies, including Eudora for retinal diseases (e.g., Stargardt disease, Usher syndrome) and Kaleibe for CNS disorders (e.g., Parkinson's disease, Friedreich's ataxia), as well as for skin conditions like dystrophic epidermolysis bullosa.

Other names
synthetic HSV-1 vectorsynHSV platform
02

Targets

MYH9 (Non-muscle myosin IIA)

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