A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Inclusion Criteria:

• Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant

• Male and female participants aged 4 years and older at the time of informed consent

• Onset of neurological symptoms from 1 to 10 years

• Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline

• Females of childbearing potential who are sexually active willing to follow thecontraceptive guidance

• Male participants with a female partner of childbearing potential willing to followthe contraceptive guidance

Exclusion Criteria:

• A history of medical conditions other than GM1 or GM2 gangliosidosis that, in theopinion of the Principal Investigator, would confound scientific rigor or theinterpretation of results

• Body weight of <10 kg

• The presence of another neurologic disease

• The presence of moderate or severe hepatic impairment

• The presence of moderate or severe renal impairment

• Platelet count of <100x10^9/L

• The dose of any anti-epileptic treatment(s) was not stable (required a change indose within the previous 3 months) and/or a new anti-epileptic treatment (drug orprocedure) was prescribed in the month before Baseline

• Prior use of an investigational drug within the 3 months before Screening; or priorparticipation in a clinical study involving gene therapy or stem celltransplantation within 2 years prior to Screening

• A positive serum pregnancy test (for women of childbearing potential)