Grant Award Details

Development of a SYF2 antisense oligonucleotide (ASO) treatment for ALS
Grant Number: 
Project Objective: 
  • To complete studies supporting the declaration of a lead a lead SYF2 ASO candidate for the treatment of ALS that is ready for translational stage activities.
Disease Focus: 
Amyotrophic Lateral Sclerosis
Neurological Disorders
Human Stem Cell Use: 
iPS Cell
Award Value: 

Progress Reports

Reporting Period: 
Year 1

Grant Application Details

Application Title: 
  • Development of a SYF2 antisense oligonucleotide (ASO) treatment for ALS
Public Abstract: 

Research Objective

We will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS).


ALS is fatal and incurable, and if successful, we will develop a treatment that slows or stops ALS progression across a broad range of patients.

Major Proposed Activities

  • Selection of the lead drug by testing several candidates for efficacy and safety on ALS patient-derived nerve cells.
  • Confirmation that the lead drug is effective and stable in mice.
  • Confirmation that the lead drug is safe in mice.
Statement of Benefit to California: 

ALS is a fatal, incurable disease and California has one of the highest number of ALS patients of any state. By testing our drug on stem cell-derived nerve cells from Californian ALS patients, we will increase the chances that it will be effective on the types of ALS patients found in California. If successful, our drug will substantially slow or stop ALS disease progression.