Drug intelligence / Profile preview

AAVshNPY

Development stage
Preclinical
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies, Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
Administration
Intracerebral, Local (potential For Organ/tissue Targeted Injection), Other Parenteral Routes If Validated
01

Overview

AAVshNPY is an investigational adeno-associated virus (AAV) gene therapy designed to deliver a short hairpin RNA (shRNA) targeting the **neuropeptide Y (NPY) gene** in vivo. The intended mechanism is gene silencing of NPY via RNA interference. NPY is a peptide neurotransmitter involved in regulating appetite, energy balance, and other physiological processes. The goal of AAVshNPY is to reduce NPY expression in target tissues, potentially addressing diseases involving NPY overactivity, such as obesity or certain metabolic disorders. The therapy utilizes an engineered viral vector (AAV) to introduce shRNA directed against NPY into target cells, leading to post-transcriptional knockdown of NPY mRNA. Delivery is generally performed via direct injection (e.g. intracerebral or targeted tissue delivery), depending on the development protocol.

Other names
AAV-shNPY
02

Targets

NPYR (Neuropeptide Y receptors)NPY (Neuropeptide Y)

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