Target ALS

A New York based ALS research organization that's leading the fight for a cure.

Amy Easton

Senior Director, Scientific Programs at Target ALS

Amy Easton is the Sr. Director of Scientific Programs at Target ALS. She has over 20 years of experience developing therapies for neurodegenerative diseases. Her background includes significant experience working with genetic models of disease,  developing novel gene-based therapeutics, and generating pk/pd data to support IND packages. 

Amy received her Ph.D. in Neuroscience from the University of Northwestern, where she studied the genetics of circadian and sleep biology. Following a post-doctoral fellowship at Rockefeller University, she chose to take on a research position in industry with Bristol-Myers Squibb in Connecticut.  For more than 10 years, her lab conducted preclinical translational studies for schizophrenia, Alzheimer’s disease, and Progressive Supranuclear Palsy. She also led a large in vivo screening effort to support a collaboration between Bristol Myers Squibb and Lexicon Genetics, designed to identify new therapeutic targets by knocking out the druggable genome.  

In 2016, Amy relocated to San Francisco to assume the role of Head of Translational Neuroscience at Genentech. Her team was responsible for preclinical efficacy, pk/pd and biomarker research across neurodegenerative disease areas, including Alzheimer’s disease, Parkinson’s disease, Amyotrophic Lateral Sclerosis, Multiple Sclerosis, and neuropathic pain. Amy became a part of the senior leadership team, contributing to the neuroscience strategy for the department.

In 2022, Amy chose to move to Target ALS to fully dedicate her time and efforts to ALS. Here, along with an amazing team of scientists, she leads the growth of the Grants portfolio, Core Resources, and Clinical research. 

Career Highlights

  • Generated in vivo data to support entry of multiple NMEs into Phase I Clinical trials for schizophrenia,  Alzheimer’s disease, and neuropathic pain
  • Led multiple programs from early-phase drug discovery screening to IND 
  • Led Genentech’s strategy to develop new modalities targeting RNA, including antisense oligonucleotide therapeutics
  • Led translational research to support the clinical development plan for GDC-0134, a dual leucine kinase inhibitor for the treatment of ALS
  • Launched the Target ALS Data Engine, a large internet-based database for public sharing of large multi-omic datasets supporting global research for ALS
  • Scientific leadership of Target ALS global clinical research studies

Publications & Contributions:

Select Publications

  • The Novel Nicotinic Alpha7 Receptor Partial Agonist, BMS-933043, Improves Cognition and Sensory Processing in Preclinical Models of Schizophrenia, PLoS One. 2016. Bristow LJ, Easton AE et al.
  • Muscle Specific Kinase (MuSK) activation preserves neuromuscular junctions in the diaphragm but is not sufficient to provide a functional benefit in the SOD1G93A mouse model of ALS. Neurobiol. Dis. 2019 Sengupta-Ghosh et al.
  • Genetic inactivation of RIP1 kinase does not ameliorate disease in a mouse model of ALS. Cell Death Differ. 2021 Dominguez S, et al.
  • Acute neurotoxicity of antisense oligonucleotides after intracerebroventricular injection into mouse brain can be predicted from sequence features. Nucleic Acid Therapeutics. 2022 Hagedorn, P.*, Brown, J.*, Easton, A.* et al.
  • Identification and characterization of a MAPT- targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies. Mol Ther Nucleic Acids. 2022 Easton, A*. Jensen, M., Wang, C. et al.
  • Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection. Mol Ther Nucleic Acids. 2023 Byrnes, A. et al.
  • TMEM106B reduction does not rescue GRN deficiency in iPSC-derived human microglia and mouse models. iScience. 2023 Dominguez et al.
  • Neutral or Detrimental Effects of TREM2 Agonist Antibodies in Preclinical Models of Alzheimer’s Disease and Multiple Sclerosis. J. Neurosci. 2024 Exteberria, A. et al.
  • Primate cerebrospinal fluid CHI3L1 reflects brain TREM2 agonism. Alzheimer’s Dement. 2024 Schauer et al.

Speaking Engagements/Conference Presentations

  • Genentech Research Off-site, Asilomar Conference Center, Monterey, CA. 2019
  • Genentech-Roche RNA Therapeutics Workshop, Basel, Switzerland, 2019
  • Target ALS Annual Meeting, Boston, 2023-2025
  • UCSF Innovation Ventures, Livermore, CA, 2024
  • ALS Drug Development Summit, Boston 2025

Awards & Recognitions

Awards or Honors

  • GNE Key Contributor Award for leadership of program achieving “Dev-go” milestone. (2021)
  • GNE Devi Sci Innovation Award for developing a novel NHP model of Alzheimer’s Disease. (2019)
  • GNE Key Contributor Award for championing the ASO technology platform. (2018)
  • BMS Innovation Award for collaboration with the University of Maryland. (2010)
  • AASR Honorable mention for Young Investigator Award (2002)
  • NIH National Research Service Award (1999-2001)
  • NIMH Summer Intramural Research Training Award (1997)
  • Beneficial Hodson Scholarship & Ionic Society Membership (1991-1992)

Professional Affiliations

Membership in Scientific Organizations

  • International Alliance of ALS/MND Directors Forum
  • International Alliance Genetics Taskforce

Leadership Positions

  • Co-chair ALL ALS NHS Working Group
  • Member of NINDS Biorepository Committee for ALS/FTD samples

Latest Posts by Amy Easton