Study of ALN-GO1 in Healthy Adult Subjects and Patients With Primary Hyperoxaluria Type 1

Inclusion Criteria for Parts A and B:

- Women of child bearing potential must have a negative pregnancy test, cannot be
breastfeeding, and must be willing to use contraception.

- Willing to provide written informed consent and to comply with study requirements.

Additional Inclusion Criteria for Part B:

- Confirmation of PH1 disease

- Meet 24 hour urine oxalate excretion requirements

- Estimated GFR of >45 mL/min/1.73m2.

- If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90
days

Exclusion Criteria for Parts A and B:

- Clinically significant health concerns (with the exception of PH1 for patients in Part
B)

- Clinically significant ECG abnormalities

- Abnormal for AST/ALT and any other clinical safety laboratory result considered
clinically significant

- Received an investigational agent within 3 months before the first dose of study drug
or are in follow-up of another clinical study

- Known history of allergic reaction to an oligonucleotide or GalNAc

- History of intolerance to subcutaneous injection