Hemophilia Challenge

Hemophilia is an inherited disorder caused by low or deficient level of clotting factor/s leading to the inability to form proper blood clot. This deficiency is caused by genetic mutations on the X chromosome encoding both clotting factors VIII (FVIII) and IX (FIX).

There are two main kinds of Hemophilia that depend on the type of missing factor:

  • FVIII deficiency causes Hemophilia A which is the most common form (85%) with approximately 150,000 patients worldwide
  • FIX deficiency causes Hemophilia B (HemB) which is the rarest form (15%) with approximately 27,000 patients worldwide

Unmet medical needs

Approximately % 50

of people with Hemophilia around the world still receive inadequate treatment or have no access to treatment

Costs

More than $ 199950

treatment costs encompasses per patient per year, including indirect costs

Resistance

Approximately % 1

of patients with severe form of Hemophilia will develop inhibitors against injected factors

Hemophilia

Rare inherited disorder (genetic mutations on the X chromosome)

2 mains types : hemophilia A (factor VIII) and B (factor IX)

Low or deficient levels of clotting factors

3 forms depending on clotting factor level : severe, moderate, mild

One injection of factors every 2-3 days= prophylactic treatment

Spontaneous bleeding is the most dangerous complication

Our solution to treat Hemophilia

The goal of Defymed is to use MailPan® technology as a platform for cell encapsulation. In the case of Hemophilia A, the aim is to macro-encapsulate factor VIII secreting cells in immuno-protective membrane.

The combined solution implies and offers:

  • No need for immunosuppressive drugs/treatments thanks to a semi-permeable membrane allowing the passage of small molecules (such as oxygen or nutrients) while preventing the passage of molecules/cells of the immune system.
  • Physiological site of implantation to optimize efficacy and time of action of the secreted molecule.
  • Cell therapy for long term, autonomous and near-physiological treatment of the disease.
  • Confinement of secreting-cells inside an encapsulation device (bio-artificial organ) with a controlled 3D environment.