PeriphaGen is a biopharmaceutical company focused on peripheral nerve gene therapy and committed to the development of innovative products for difficult-to-treat neurological disorders.
Periphagen’s wide range of HSV-1 vector technology has unique capabilities for focal gene delivery to treat human disease
WHY HERPES SIMPLEX VIRUS TYPE-1?
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Efficient neuronal retrograde transport from skin inoculation.
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Though it naturally infects sensory neurons, HSV-1 is capable of efficiently transducing almost any cell type.
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It's large size and ability to remove numerous nonessential accessory genes allows for a large inserted DNA capacity (20 kB).
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Removal of immediate early genes creates a replication defective vector with minimal residual viral gene expression.
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Able to express multiple transgenes from single vector.
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It has natural long term expression of transgenes.
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Cost effective production, purification, and release capabilities are in place.
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Favorable immunologic response and distribution data are available from human clinical trials.
Illustration of Herpes Simplex Virus Type-1 showing its basic components. The HSV-1 virus contains unique long and unique short sequences bounded by inverted repeat sequences and contains several genes that are essential for its biological function and several accessory genes that can be removed without affecting its ability to unction as a DNA delivery vehicle.
PERIPHAGEN'S NET PLATFORM
Periphagen has developed its NET platform based on replication-defective HSV-1. Using this gene delivery vector it is possible to specifically target neurons and treat neurological diseases with more potent molecules without the side effects of current treatments.
Following a simple intradermal skin injection the NET drug candidate is taken up by resident nerve endings (1) then, via retrograde transport, moves to the dorsal root ganglion where it transduces neurons with high efficiency (2).
PeriphaGen's NET platform can express a variety of gene-based products that can be used for therapeutic effect including: peptide neurotransmitters, trophic factors, channels, receptors, immune modulators, second messengers, and siRNAs.
Over 100 publications from PeriphaGen team members and collaborators have documented the use of NET-type vectors for the treatment of various disorders. Selected references pertaining to particular fields of interest are listed in the attached document.
