Pioneering One-and-Done Ex-vivo Gene Therapy Through our GMAC Platform

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Overview of Each Indication

LCAT Deficiency

・LCAT deficiency is a rare genetic disorder that affects the production and function of an enzyme called lecithin-cholesterol acyltransferase (LCAT). LCAT transfers fatty acids to cholesterol to form a type of cholesterol called cholesteryl esters, which are then transported into the bloodstream. In LCAT deficiency, this process is impaired, accumulating unesterified cholesterol and phospholipids in the blood and decreasing levels of cholesteryl esters.

・The symptoms of LCAT deficiency can vary widely but may include kidney damage, anemia, vision problems, and an increased risk of atherosclerosis and cardiovascular disease. Treatment generally focuses on managing symptoms and preventing complications like kidney failure or heart disease.

Hemophilia A

・Hemophilia A is a rare genetic disorder in which the blood cannot clot normally due to a deficiency or absence of clotting factor VIII (FVIII). FVIII is a protein that helps blood to clot and prevent excessive bleeding. In Hemophilia A, this clotting factor is either missing or present in reduced amounts, making it difficult for the body to stop bleeding after an injury.

・The severity of Hemophilia A can vary widely depending on the amount of clotting factor in the blood. Mild cases may only cause prolonged bleeding after injury, while severe cases may lead to spontaneous bleeding, joint damage, and life-threatening bleeding episodes. Treatment typically involves regular infusions of FVIII to replace the missing or deficient clotting factor. In addition, preventive measures such as avoiding contact sports and avoiding injury can help reduce the risk of bleeding episodes.

Hemophilia B

・Hemophilia B, also known as Christmas disease, is a rare genetic disorder in which the blood cannot clot normally due to a deficiency or absence of clotting factor IX (FIX). FIX is a protein that helps blood to clot and prevent excessive bleeding. In Hemophilia B, this clotting factor is either missing or present in reduced amounts, making it difficult for the body to stop bleeding after an injury.

・The severity of Hemophilia B can vary widely depending on the amount of clotting factor in the blood. Mild cases may only cause prolonged bleeding after injury, while severe cases may lead to spontaneous bleeding, joint damage, and life-threatening bleeding episodes. Treatment typically involves regular infusions of FIX to replace the missing or deficient clotting factor. In addition, preventive measures such as avoiding contact sports and avoiding injury can help reduce the risk of bleeding episodes.

Fabry disease

・Fabry disease is a rare genetic disorder in which the body cannot properly break down a type of fat called globotriaosylceramide (GB3). This leads to a buildup of GB3 in various organs and tissues, particularly the kidneys, heart, and nervous system.

・Symptoms of Fabry disease can include chronic pain, skin rashes, gastrointestinal problems, hearing and vision loss, and an increased risk of heart and kidney disease. Treatment may include enzyme replacement therapy to help break down GB3 and medications to manage pain and gastrointestinal problems. Early diagnosis and treatment can help improve outcomes and prevent complications.

Retinitis pigmentosa

・Retinitis pigmentosa is a rare genetic disorder that affects the retina, the part of the eye responsible for detecting light and transmitting visual signals to the brain. The disease causes the gradual degeneration of the light-sensitive cells in the retina, leading to vision loss and, eventually, blindness.

・Symptoms of retinitis pigmentosa may include night blindness, difficulty seeing in low light, decreased peripheral vision, and loss of central vision. Treatment options are currently limited and may consist of assistive devices such as magnifiers and visual aids, as well as gene therapy in some cases. Early diagnosis and regular eye exams can help with early intervention and management of the disorder.