Related Experiment Video

Updated: May 4, 2026

Real-time Imaging of Axonal Transport of Quantum Dot-labeled BDNF in Primary Neurons
10:53

Real-time Imaging of Axonal Transport of Quantum Dot-labeled BDNF in Primary Neurons

Published on: September 15, 2014

14.5K

Foundation-directed therapeutic development in Huntington's disease

Celia Dominguez1, Ignacio Munoz-Sanjuan

  • 1CHDI Management, Inc., Advisors to CHDI Foundation, Inc., 6080 Center Drive, Los Angeles, California 90045, United States.

Journal of Medicinal Chemistry
|January 18, 2014
PubMed

Insights

Huntington's disease (HD), a neurodegenerative disorder caused by a gene mutation, currently lacks disease-modifying treatments. CHDI Foundation is advancing therapeutic development through collaborations and internal programs to combat this devastating condition.

Area of Science:

  • Neuroscience
  • Genetics
  • Pharmacology

Background:

  • Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder.
  • It stems from CAG repeat expansion in the huntingtin gene (HTT).
  • Pathologically, HD involves neuronal loss in cortical, striatal, and hypothalamic areas, leading to chorea, cognitive decline, and psychiatric issues.

Purpose of the Study:

  • To outline the therapeutic development efforts of the CHDI Foundation for Huntington's disease.
  • To highlight collaborations and internal programs aimed at finding treatments for HD.

Main Methods:

  • Review of foundation-directed therapeutic development strategies.
  • Description of collaborative and internal research programs in various stages of development.

Main Results:

  • CHDI Foundation is actively pursuing multiple therapeutic avenues for HD.
  • These efforts encompass both collaborative projects and internal research initiatives.

Conclusions:

  • There are currently no effective disease-modifying treatments for Huntington's disease.
  • CHDI Foundation is dedicated to accelerating the development of therapies to treat HD.
  • The foundation's approach involves strategic collaborations and internal programs to address this unmet medical need.

More Related Videos

Generation of Native, Untagged Huntingtin Exon1 Monomer and Fibrils Using a SUMO Fusion Strategy11:22

Generation of Native, Untagged Huntingtin Exon1 Monomer and Fibrils Using a SUMO Fusion Strategy

Published on: June 27, 2018

12.0K
Ole Isacson: Development of New Therapies for Parkinson's Disease23:53

Ole Isacson: Development of New Therapies for Parkinson's Disease

Published on: April 29, 2007

7.5K

Related Concept Videos

Huntington Disease l: Introduction01:21

Huntington Disease l: Introduction

Huntington disease or HD is a progressive, fatal neurodegenerative disorder inherited in an autosomal dominant pattern.PathophysiologyIt is caused by expansion of the CAG trinucleotide repeat in the HTT gene on chromosome 4 (4p16.3), producing an abnormal huntingtin protein with an expanded polyglutamine tract. This misfolded protein disrupts cellular function, leading to neuronal death. Normal alleles have ≤26 repeats, 27–35 are intermediate (risk of expansion), 36–39 show...
110
Drug Discovery: Overview01:26

Drug Discovery: Overview

Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
10.4K
Alzheimer's Disease: Treatment01:22

Alzheimer's Disease: Treatment

Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...
1.3K
Parkinson's Disease: Treatment01:24

Parkinson's Disease: Treatment

Neurodegenerative disorders, such as Parkinson's Disease (PD), involve the gradual and irreversible destruction of neurons in particular brain areas. These disorders exhibit standard features like proteinopathies, selective vulnerability of some neurons, and an interaction of intrinsic properties, genetics, and environmental influences in neural injury.
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of...
1.4K
Preclinical Development: Overview01:28

Preclinical Development: Overview

Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
4.8K
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
121