CANbridge Pharmaceuticals Inc. (HKEX:1228) is a China and U.S.-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 9 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases.

CANbridge is also building next-generation gene therapy development capability through a combination of collaboration with world-leading researchers and biotech companies and internal capacity.

CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School, the University of Washington School of Medicine, Scriptr Global and LogicBio.

We are led by a management team with significant industry experience in rare diseases, spanning R&D, clinical development, regulatory affairs, business development and commercialization. We are supported by a talent pool of 100 employees of which 13 have a Ph.D. and/or M.D. degree. And more than 70% of our employees have prior experience working at multinational biopharmaceutical

companies as of December 31, 2023. Our management team has a track record of successfully achieving approval and commercializing of rare disease therapies across the key markets, including China, the United States, Europe, Latin America and Southeast Asia. We leverage this expertise to play an active role in advancing the rare disease industry and shaping the rare disease ecosystem in

China. For example, our founder, Dr. James Qun Xue (“Dr. Xue”), Ph. D., is currently serving as the Deputy Director General of China’s Alliance for Rare Disease (CHARD).


Key pipelines and their milestones:

Livmarli® (maralixibat oral solution, formerly known as CAN108), an oral, minimally absorbed, reversible inhibitor of the ileal bile acid transporter (IBAT) that is under development to treat rare cholestatic liver diseases including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC)

In 2023, CANbridge received multiple marketing approvals for Livmarli in mainland China, Hong Kong, and Taiwan. These approvals make Livmarli the first and only approved product marketed for the treatment of cholestatic pruritus in patients with ALGS in these regions.


Hunterase® (idursulfase beta, formerly known as CAN101), an enzyme replacement therapy (ERT) for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome

In September 2020, we received marketing approval for Hunterase (CAN101) for MPS II in mainland China.


CAN106 (omoprubart), a novel, long-acting monoclonal antibody for the treatment of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis (MG) and other diseases that may benefit from treatment with an anti-C5 antibody

In June 2023, CANbridge Reported positive preliminary top-line data from the ongoing Phase 1b study of CAN106 being conducted in PNH patients in China.

In February 2022, a Phase I single ascending dose study of CAN106 in Singapore recorded positive topline results with complete complement blockade.


CAN103, an ERT for the treatment of Gaucher Disease (GD)

In October, 2023, CANbridge announced that the core part of the ongoing CAN103 Phase 2 trial in treatment-naïve patients aged 12 and above with Gaucher disease Types I and III reached full enrollment.

In January 2023, the first patient was dosed in phase 2 trial for the treatment of Gaucher disease in China.


Gene therapy

In addition to biologics and small molecules, we are investing in next-generation technology for gene therapies. Gene therapies provide a potentially one-time durable treatment for rare genetic diseases that have limited treatment options. As of December 31, 2022, we are developing gene therapies for the treatments of Fabry disease and Pompe disease, which we licensed from LogicBio Therapeutics. The license is for the development of two gene therapy products. In January 2023, we announced that we have exercised our option to secure the exclusive global rights to develop, manufacture and commercialize a novel second-generation gene therapy to treat spinal muscular atrophy (SMA) from UMass Chan Medical School. In addition, we are internally developing an adeno-associated virus (AAV) delivery platform targeting different tissues, such as the central nervous system (CNS) and muscle.

June 2012

CANbridge established.

July 2015

Acquires license for Apogenix's CAN008 for the treatment of glioblastoma in China

July 2016

Receives approval for CAN008 GBM Phase I/II trial in Taiwan

September 2016

Doses first patient in CAN008 GBM Phase I/II trial in Taiwan

July 2017

Submits IND application for CAN008 GBM Phase II/III trial in China

November 2017

Completes patient enrollment for CAN008 GBM Phase I trial in Taiwan

April 2018

Receives approval to commence CAN008 GBM Phase II/III trial in China

October 2018

Enters strategic partnership with WuXi Biologics for rare disease therapeutics development

July 2019

Submits NDA for CAN101 (Hunterase®) for Hunter syndrome in China

September 2019

Granted priority review for CAN101 (Hunterase®) in China

June 2020

Enters research agreement with UMass Medical School for rare disease gene therapy

September 2020

Enters into second research agreement with Umass Medical school for rare disease gene therapy

September 2020

First CANbridge rare disease treatment marketing approval in China:

Hunterase®, the first enzyme replacement therapy for Hunter syndrome in the region

April 2021

CANbridge Pharma announces strategic collaboration with LogicBio Therapeutics, securing licenses to gene delivery and editing platforms

April 2021

Mirum Pharma and CANbridge Pharma enter into exclusive licensing agreement to develop and commercialize Maralixibat in Greater China for rare liver disease

April 2021

Obtains CDE clearance from NMPA for an updated Phase II clinical first line trial application for CAN008 in China

May 2021

Completes $98 million series D and $58 million series E financing

May 2021

Begins commercialization of Hunterase® (CAN101) in mainland China

February 2022

Reported Positive Top-Line CAN106 Phase 1 Data

May 2022

First Patient Dosed in CAN106 Phase 1b/2 Trial for Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

May 2022

LIVMARLI® New Drug Application/Orphan Drug Registration (NDA/ORD) for Alagille Syndrome Accepted by the Taiwan Food and Drug Administration

May 2022

CANbridge-UMass Chan Medical School Gene Therapy Research Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

June 2022

LIVMARLI® New Drug Application (NDA), for Alagille Syndrome, Accepted by China’s National Medical Products Administration

July 2022

Forms Scientific Advisory Board to Guide Global Development of CAN106 in Complement-mediated Diseases

July 2022

First Patient Dosed in LIVMARLI® (Maralixibat) EMBARK Phase 2 Study in Biliary Atresia in China

July 2022

First Patient Dosed in CANbridge Pharmaceuticals CAN103 Phase 1/2   Trial for the Treatment of Gaucher Disease in China

October 2022

CANbridge-UMass Chan Medical School Gene Therapy Research in Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual Congress

November 2022

Orphan Drug Designation Granted to CAN 106 for the Treatment of Myasthenia Gravis

January 2023

First Patient Dosed in CAN103 Phase 2 Trial for the Treatment of Gaucher Disease in China

May 2023

CANbridge and UMass Chan Medical School reported at the 26th ASGCT Annual Meeting that Spinal Muscular Atrophy gene therapy (CAN203) extends life and improves motor function in mice via intracerebroventricular injection

May 2023

Livmarli® was approved for marketing in mainland China

June 2023

Positive preliminary data were obtained from the Phase 1b clinical trial of omoprubart (CAN106) injection for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China

September 2023

Livmarli® was approved for marketing in Hong Kong

October 2023

Livmarli® was approved for marketing in Taiwan

October 2023

CAN103 for the treatment of Gaucher disease completed patient enrollment in the core part of the Phase 2 clinical trial in China

October 2023

Abstract of gene therapy for Fabry disease presented at the 30th Annual Meeting of the European Society for Gene and Cell Therapy

Our Team
James Xue, Ph.D.
Founder, Chairman and CEO
Glenn Hassan
Chief Financial Officer
Gerry Cox, M.D., Ph.D.
Chief Development Strategist & Interim Chief Medical Officer
Chris Chen
Vice President of Human Resources
Max Hung
General Manager of Taiwan
Jeff Kou, Ph.D.
Director, Head of Program Management & Clinical Operations
Bettie Li
Senior Director, Head of Finance Operation and Controller
Dan Li
Vice President of Business Development
Dr. Pauline Li
Senior Vice President of Clinical Development and Operations
Fannie Man
Mainland China Commercial Head, General Manager of Hong Kong and Macau
Stella Mao
Senior Director, Public Affairs
Ms. Qian Ma
Director of Legal and Compliance, Board Secretary, Joint Company Secretary
Joe Shen
Senior Vice President
Jason West
Vice President, Head of Gene Therapy Research
Shirley Yue
Senior Director of Procurement and Supply Chain
Rebecca Zhang
Senior Vice President of Regulatory Affairs
Wei Zhang
Senior Director, CMC
James Xue, Ph.D.
Founder, Chairman and CEO
Guangping Gao, Ph.D.
Advisory Board
Dr. Mark Goldberg
Advisory Board
James Xue, Ph.D.
Founder, Chairman and CEO
Dr. Lan Hu
Independent non-executive Director
Dr. Kan Chen
Non-executive Director
Mr. Edward Hu
Non-Executive Director
Mr. James Arthur Geraghty
Independent non-executive Director
Dr. Richard James Gregory, Ph.D.
Independent non-executive Director
Mr. Peng Kuan Chan
Independent non-executive Director
Contact Us
Room1501, 15/F(16), No. 10, Futong East Street, Chaoyang District, Beijing.
Tel: 010 6478 0016
Fax: 010 6478 0019
Adverse Events Reporting:
Suite 708, Huawen International Mansion, No.999, West Zhongshan Road, Changning District, Shanghai
Tel: 021 52956319
Fax: 021 52665300
Adverse Events Reporting:
Hong Kong
Hong Kong
Unit A131, 16/F, Tower 5, The Gateway, Harbour City, 15 Canton Road, Tsim Sha Tsui, Hong Kong
Adverse Events Reporting:
Room209, 2F, No. 141, Sec. 1, Keelung Rd., Xinyi Dist., Taipei, Taiwan
Adverse Events Reporting:
4 Burlington Woods Drive, Burlington, MA 01803, USA