# Therapeutics for Huntington's Disease

> **NIH NIH R43** · SYNTHETIC BIODESIGN, LLC · 2020 · $214,977

## Abstract

Huntington’s disease is a devastating neurodegenerative disease with no current therapeutic options.
The proposed work will identify novel therapeutic leads for development as badly needed new drugs in phase II
studies. This will be achieved using a novel natural products-based approach.
 Natural products or their derivatives have accounted for most of our current drugs, demonstrating their
unique status as “privileged” molecules that are well adapted for therapeutic use. However, natural products
have previously been unsuitable for modern, high-throughput (HT) drug discovery screening approaches, and
as such, were long ago abandoned in drug discovery programs of large pharma. The substitute has been high-
throughput screening of combinatorial chemical libraries. These unnatural chemical libraries have yielded few
new pharmaceutics leading to a significant decline in pharmaceutical drug discovery productivity.
 Recent research in the field of synthetic biology has identified numerous biosynthetic pathways, largely
from microbial sources, that have the capacity to produce tremendous numbers of novel natural products that
are suitable for high-throughput screening. In this work, Synthetic Biodesign will combine a novel biosynthetic
approach with a novel whole-animal high-throughput screen to demonstrate that natural products can be
reintegrated into the drug discovery pipeline and this can be done using modern HT methodologies.

## Key facts

- **NIH application ID:** 10009015
- **Project number:** 1R43NS113676-01A1
- **Recipient organization:** SYNTHETIC BIODESIGN, LLC
- **Principal Investigator:** Duane Ruffner
- **Activity code:** R43 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $214,977
- **Award type:** 1
- **Project period:** 2020-09-15 → 2022-09-14

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10009015

## Citation

> US National Institutes of Health, RePORTER application 10009015, Therapeutics for Huntington's Disease (1R43NS113676-01A1). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10009015. Licensed CC0.

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