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 11 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, as well as glioblastoma multiforme. 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 117 employees of which 15 have a Ph.D. and/or M.D. degree. And more than 80% of our employees have prior experience working at multinational biopharmaceutical

companies as of December 31, 2022. 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).


We received marketing approval for Hunterase® (CAN101) for MPS II in mainland China in September 2020.

We initiated a Phase 1 clinical trial in healthy volunteers for CAN106 in Singapore in February 2021; obtained the IND approval from the China’s National Medical Products Administration (NMPA) for a CAN106 study in paroxysmal nocturnal hemoglobinuria (PNH) in July 2021; and reported positive top-line

CAN106 Phase 1 data for the single ascending dose study in Singapore in February 2022. Results suggest complete blockade of complement function. CAN106 was shown to be safe and well-tolerated.


In addition, the Livmarli NDA for ALGS was accepted and granted priority review by NMPA in January 2022. The first patient was dosed in Livmarli Phase 2 Study in biliary atresia in China in July 2022. In addition, the first patient was dosed in CAN103 phase 1/2 trial for the treatment of Gaucher disease in China in July 2022 and the first patient was dosed in phase 2 trial for the treatment of Gaucher disease in China in January 2023.


In the rare oncology area, we are developing CAN008 for the treatment of glioblastoma multiforme (GBM). In 2018, we completed a Phase 1 clinical trial for CAN008 in Taiwan in newly diagnosed patients. We received IND approval from the NMPA to commence first-line Phase 2 clinical trial of CAN008, dosed the first patient in a Phase 2 clinical trial of CAN008 for the first-line treatment of GBM patients in mainland China, in October 2021, and completed phase 2 clinical trial patient enrolment in March 2023.


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

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
Marcelo Cheresky
Chief Commercial Officer
Chris Chen
Vice President of Human Resources
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
Ms. Qian Ma
Director of Legal and Compliance, Board Secretary, Joint Company Secretary
Stella Mao
Senior Director, Public Affairs
Jeff Kou, Ph.D.
Director, Head of Program Management & Clinical Operations
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. Kan Chen
Non-executive Director
Dr. Derek Paul Di Rocco, Ph.D
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
Dr. Lan Hu
Independent non-executive Director
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