Form Type:  SC 13G/A
Filing Date:  2/14/2020 
CIK:  0001628738 
Address:  600 CALIFORNIA ST., 17TH FLOOR 
City, State, Zip:  SAN FRANCISCO, California 94108 
Telephone:  415-638-6556 
Fiscal Year:  12/31 
Description of Business
We are a clinical stage biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases caused by single gene defects. We believe that gene therapy has powerful potential to treat these diseases through delivery of a functional copy of the gene to affected cells, resulting in production of the normal protein. We have built a compelling portfolio of product candidates, including AT132 for the treatment of X-Linked Myotubular Myopathy, or XLMTM, AT845 for the treatment of Pompe disease, AT342 for the treatment of Crigler-Najjar Syndrome, or Crigler-Najjar, and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia, or CASQ2-CPVT. We have ongoing Phase 1/2 clinical trials of AT132 for the treatment of XLMTM and AT342 for the treatment of Crigler-Najjar.
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