The development of Orphan Drugs can have economic benefits. Rare diseases can be costly to manage, and the availability of effective treatments can help to reduce healthcare costs and improve productivity.

Product Pipeline,
Treasca™ – Treatment for Sickle Cell Anemia

Sickle Cell Anemia

Sickle cell anemia is a rare, inherited blood disorder caused by an HBB gene mutation. This gene provides instructions for making a protein called beta-globin, which is a component of hemoglobin. Hemoglobin is a protein found in red blood cells that carries oxygen from the lungs to the rest of the body.

In individuals with sickle cell anemia, the mutation in the HBB gene leads to the production of abnormal hemoglobin, which causes the red blood cells to become rigid and crescent-shaped, or “sickle-shaped.” These abnormal red blood cells can clog small blood vessels, which can lead to a range of complications, including anemia, infections, and organ damage.

Sickle cell anemia is a lifelong condition that can be managed with regular medical care and treatment. Treatment options for sickle cell anemia include medications to prevent and treat infections, blood transfusions to increase the number of red blood cells, and bone marrow or stem cell transplants to replace damaged bone marrow with healthy bone marrow.

There is currently no cure for sickle cell anemia, but research is ongoing to develop new treatments and therapies.

Treasca™ stages of development: Clinical Trial – Phase 3