Advanced Therapies

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.

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Key features of great opportunities for us  are:

  • proprietary animal models
  • animal models predictive of for clinical 
  • early proof of concept already achieved
  • formulation adequate to state of development
  • disease clinically relatively well known
  • inventor on board with the company
  • management team’s strong network with key opinion leaders in relevant disease space
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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.

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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.