• Hemophilia

This page is made to present regional, national and international collaborative project currently involving Defymed.

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.