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

Highlights

  • 20 years of drug discovery experience in biotech/pharma for neuropsychiatric and neurodegenerative disease
  • Focus on preclinical translational research including pk/pd, in vivo efficacy, and biomarker studies
  • Expertise in developing small molecule, antibody, and antisense oligonucleotide therapeutics
  • Successfully advanced several drugs from initial portfolio entry through various stages of development, including the Phase I clinical trial of GDC-0134 in ALS

Education

  • PhD in Neuroscience from Northwestern University
  • Post-doctoral fellowship at Rockefeller University

Experience

  • Senior Director, Scientific Programs, Target ALS (Nov 2022–Present)
    • Set scientific strategy and manage scientific portfolio including grants, state-of-the art core resources, and clinical research studies
  • Principal Scientific Manager, Head of Translational Neuroscience Genentech, Inc. 2016-2022
    • Led translational research for neuroscience department, led project teams working to develop novel therapeutics for neurodegenerative disease
  • Principal Scientist, Neuroscience Bristol-Myers Squibb 2005-2016
    • Led in vivo screening and characterization of drugs to treat neurological and neurodegenerative disorders

Research Focus

  • Schizophrenia, Alzheimer’s disease,  Parkinson’s and FTD/ALS

Publications & Contributions

  • 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.
  • 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.

Research Highlights

  • Schizophrenia Drug Development at Bristol Myers Squibb (BMS):

Contributed to a cross-functional team that screened and identified a drug with potential for treating cognitive impairment in schizophrenia. The team spent five years optimizing a potent, selective, safe, and efficacious molecule, successfully advancing it into Phase I clinical trials. While the molecule did not proceed into Phase II, the preclinical program and Phase I study were exceptionally well-executed.

  • Gene Targeting for Pain Treatment at BMS & Lexicon Genetics:
    Led behavioral phenotyping in a collaboration between BMS and Lexicon Genetics to screen the “druggable” genome for novel therapeutic targets. The deletion of the AAK1 gene reduced pain sensitivity in animal models, leading to the development of LX9211, a drug currently in Phase IIb trials for diabetic neuropathic pain.
  • Antisense Oligonucleotides (ASOs) for Tau Pathology at BMS:
    Spearheaded the development of ASOs targeting the MAPT (Tau) gene to treat neurodegenerative diseases such as Alzheimer’s and PSP. The team successfully reduced Tau levels in mouse models, and further testing in monkeys demonstrated the potential for ASOs to lower Tau in the CSF, providing a promising biomarker for clinical use. Although clinical trials were not pursued due to organizational changes, the work was published and remains influential in the field.
  • Advancing ASOs at Genentech:
    Led efforts to establish ASOs as a therapeutic modality at Genentech, generating proof-of-concept for in vivo utility and helping develop a framework for preclinical ASO programs. Genentech’s RNA-directed therapies portfolio, which now includes several ASOs, stems from these foundational efforts.
  • Preclinical Research on GDC-0134 for ALS:
    Worked on a cross-functional team to characterize GDC-0134, a dual leucine kinase inhibitor for ALS. The team demonstrated neuroprotective effects on motor neurons in mice, contributing important data for human dose projections and exploring NfL as a potential biomarker for ALS. Despite the clinical development of GDC-0134 being halted for safety reasons, the preclinical data remains a significant accomplishment.

Awards & Recognitions

  • 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)

Speaking Engagements/Conference Presentations

Selected to present on antisense oligonucleotide therapies at Genentech’s biannual off-site held at Asilomar Conference Center, Monterey, CA. 2019.

Amy will always remember this presentation given the historical significance of the venue for Genentech as a site to share inspiration and innovative ideas (described well in Mukherjee’s The Gene) and also given Genentech’s roots in genetic engineering.

Professional Affiliations

International ALS Directors Forum

Leadership Positions

Co-chair ALL ALS NHS Working Group

On this page

Latest Posts by Amy Easton