for people ages 18 years and up (full criteria)
at Irvine, California and other locations
study started
completion around



This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study.

Official Title

A Phase 1/2, Multicenter, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS).


AMT-162 is an investigational gene therapy that encodes an artificial microribonucleic acid (microRNA or miRNA) targeting the SOD1 gene. This clinical study will test the safety of AMT-162 and explore the hypothesis that it will silence expression of mutant cytosolic SOD1 and thereby ameliorate the course of ALS caused by this mutant gene.


Amyotrophic Lateral Sclerosis, Gene Therapy, AAV (adeno-associated virus), ALS, SOD1, Motor Neuron Disease, Sclerosis, AMT-162


You can join if…

Open to people ages 18 years and up

  1. Confirmed clinical and genetic diagnosis of SOD1-mediated ALS (SOD1-ALS) experiencing signs and/or symptoms of lower motor neuron dysfunction (weakness, atrophy, cramps, fasciculations), with or without upper motor neuron symptoms (weakness, bring reflexes, spasticity).
  2. ALSFRS-R score ≥ 25 at Screening.
  3. Slow vital capacity (SVC) ≥65% of predicted normal value.
  4. Capable of providing informed consent and complying with trial procedures, including: medically able to undergo lumbar puncture and has a responsible caregiver able to attend all clinic visit with the Participant.

You CAN'T join if...

  1. SOD1 pathogenic or likely pathogenic variants in amino acid regions 43-47.
  2. Pathogenic repeat expansion in the C9orf72 gene
  3. Any of the following prior or concomitant treatments:
    1. Any prior SOD1 suppression therapy with viral microRNA mediators
    2. Prior SOD suppression therapy with antisense oligonucleotide (ASO) mediators such as tofersen (QALSODY™). Exception: Patient who previously received tofersen may be enrolled if the last dose of tofersen was received at least 20 weeks prior to the first Screening assessment and if there were no previous tofersen-related serious adverse events or ongoing tofersen-related adverse events
    3. Other ALS medications riluzole (RILUTEK®, TIGLUTIK®), edaravone (RADICAVA®), and sodium phenylbutyrate and taururosdiol combination (RELYVRIO) or bioequivalents are allowed if dose is stable for 30 days prior to immunosuppression.
    4. Any prior administration of an AAV gene therapy.


  • University of California Irvine
    Irvine California 92697 United States
  • California Pacific Medical Center
    San Francisco California 94109 United States


not yet accepting patients
Start Date
Completion Date
UniQure Biopharma B.V.
Phase 1/2 Amyotropic Lateral Sclerosis (ALS) Research Study
Study Type
Expecting 12 study participants
Last Updated