# NIH Reporter #1R41NS150094-01

Epigenetic Silencing of VCP via an ASO-Conjugate Based Induced Proximity System for the Treatment of FTD-ALS in VCP Disease

**Recipient:** JUNIPERO THERAPEUTICS, INC.

**Award Amount:** $306,872.00
**Ceiling:** $306,872.00

**Awarded:** September 08, 2026

**Identifier:** 1R41NS150094-01

The award funds a Phase I study to develop and test an epigenetic ASO-conjugate therapy targeting VCP to treat neurodegenerative diseases FTD and ALS, aiming for durable gene silencing and therapeutic benefit.

### Description

This project aims to develop a novel epigenetic ASO-conjugate platform to achieve durable gene silencing of Valosin-containing protein (VCP) for treating frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) associated with VCP disease. The study includes engineering and screening ASO-conjugates, dose-response screening in HEK293 cells, and demonstrating in vivo efficacy in a mouse model. The approach combines ASO precision with epigenetic persistence to potentially enable single-dose therapeutic benefit.

[View original record](https://reporter.nih.gov/search/0C83gI3MXki4-tEhFD4UKQ/project-details/11409656)