Hunterase^®

Description: Hunterase^®, (iduronidase-2-sulfatase) is a recombinant human enzyme replacement therapy (ERT).

Indication: Mucopolysaccharidosis type II (MPS II) or Hunter syndrome

Status: Hunterase is designated an orphan drug in the United States and Korea and is marketed in 12 countries. Hunterase was approved for marketing in China in September 2020.

Ownership: Exclusive license to develop and commercialize in Greater China from South Korea’s GC Pharma

CAN108 (Maralixibat)

Description: CAN108 is an orally administered small molecule ASBT inhibitor.

Indication: multiple rare cholestatic liver diseases, including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA)

Status: USA FDA Approved, China NDA filed (ALGS) and EMA filed in EU (PFIC); Phase IIb (BA)

Ownership: Exclusive Greater China license from Mirum Pharmaceuticals

Note: For BA, we are supporting the patient recruitment and clinical site management in China for a global Phase II clinical trial initiated by our license partner in the U.S. and Europe. IND approval was obtained from the NMPA in May 2021 and we plan to begin patient enrolment in China as part of the global Phase II trial.

CAN106

Description: CAN106 is a recombinant human monoclonal antibody that specifically binds to and neutralizes C5 in the complement system.

Indication: PNH and other complement-mediated disorders

Status: Phase 1b/2 in China

Trial registration: ClinicalTrials.gov NCT05539248  

Ownership: Greater China right from Wuxi Biologics；Exclusive worldwide right from Privus

CAN103

Description: CAN103 is a recombinant human enzyme replacement therapy (ERT).

Indication: Gaucher disease, one of the most common lysosomal storage disorders (LSDs)

Status: IND Approved

Ownership: Exclusive global license from WuXi Biologics

CAN107

Description: CAN107 is a fibroblast growth factor 23 (FGF23) blocking antibody.

Indication: X-linked hypophosphatemia (XLH), usually diagnosed in children and an inherited disorder characterized by low levels of phosphate in the blood due abnormal kidney processing in the kidneys, leading to soft, weak bones 

Status: Preclinical

Ownership: Exclusive global license from WuXi Biologics and Privus

CAN104

Description: CAN104 is a recombinant human enzyme replacement therapy (ERT).

Indication: Fabry Disease

Status: Preclinical

Ownership: Exclusive global license from WuXi Biologics

CAN105

Description: CAN105 is a bispecific antibody.

Indication: Hemophilia A, a genetic deficiency in clotting factor VIII which causes increased bleeding and usually affects males

Status: Preclinical

Ownership: Exclusive Greater China license from WuXi Biologics

CAN201:

Description: CAN201 is a gene therapy program utilizing adeno-associated virus (AAV) sL65 capsid.

Indication: Fabry disease, also called Anderson-Fabry disease, a rare inherited lysosomal storage disorder (LSD) of glycosphingolipid (fat) metabolism resulting from the absent or markedly deficient activity of the lysosomal enzyme, α-galactosidase A (α-Gal A)

Status: Preclinical

Ownership: Exclusive global license to sL65 capsid from LogicBio Therapeutics

CAN202:

Description: CAN202 is a gene therapy program utilizing adeno-associated virus (AAV) sL65 capsid.

Indication: Pompe disease, also called Glycogen storage disease type II, an autosomal recessive metabolic disorder which damages muscle and nerve cells throughout the body, caused by an accumulation of glycogen in the lysosome due to deficiency of the lysosomal acid alpha-glucosidase enzyme

Status: Preclinical

Ownership: Exclusive global license to sL65 capsid from LogicBio Therapeutics