ADVANCED THERAPIES

From care to cure

 

After decades of research and great uncertainty, in 2013 EMA approved the first gene therapy medicinal product, Glybera, with the indication of lipoprotein lipase deficiency.

 

In the past few years, various stem cell therapies have been approved by many regulatory bodies around the world, such as Prochymal (acute graft-vs-host disease) and ChondroCelect (repair damage to the cartilage in the knee).

 

We believe that a great time awaits gene therapies and regenerative therapies and think that many patients can finally find relief from their rare disorders.

      

OPPORTUNITY PROFILE

Key features of great opportunities:

 

  • safety of vectors to transport genes or new cultures to breed stem cells 

  • unencumbered intellectual property

  • a reasonable clinical plan minimizing time to regulatory submission and compliant to the new FDA and EMA guidelines on advanced medicinal products

  • a credible and efficient plan to scale production to GMP standards while reducing manufacturing costs

  • management has a documented expertise in the space

 

STAGE OF DEVELOPMENT

We invest in companies developing early stage treatments in

 

  • preclinical

  • early clinical stage

 

We do not have any preference for any of these stages, but understand that risks and returns vary according to the stage of development reached.

  

INVESTMENT THESIS

Stem cell therapies and gene replacement therapies represent the new frontier of the pharmaceutical scientific research.

 

While still controversial from a technical and a regulatory standpoint, advanced therapies hold the promise of shifting the paradigm from “cure” to “care”.

 

Clinical experience with the advanced therapy, particularly if the number of patients is relevant considering disease prevalence and some midterm results are available.