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List Of Gene Therapies
This article contains a list of commercially available gene therapies. Gene therapies * Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available) * Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma * Betibeglogene autotemcel (Zynteglo): treatment for beta thalassemia * Brexucabtagene autoleucel (Tecartus): treatment for mantle cell lymphoma and acute lymphoblastic leukemia * Cambiogenplasmid (Neovasculgen): treatment for vascular endothelial growth factor peripheral artery disease * Ciltacabtagene autoleucel (Carvykti): treatment for multiple myeloma * Elivaldogene autotemcel (Skysona): treatment for cerebral adrenoleukodystrophy * Etranacogene dezaparvovec (Hemgenix): treatment for hemophilia B * Gendicine: treatment for head and neck squamous cell carcinoma * Idecabtagene vicleucel (Abecma): treatment for multiple myeloma * Nadofaragene firadenovec (Adstiladrin): treatment for bladder c ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Gene Therapy
Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. It is thought to be able to cure many genetic disorders or treat them over time. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in phase I.Gene Therapy Cli ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Etranacogene Dezaparvovec
Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes. Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding. The most common adverse reactions include liver enzyme elevations, headache, mild infusion-related reactions and flu-like symptoms. Etranacogene dezaparvovec was approved for medical use in the United States in November 2022. Medical uses Etranacogene dezaparvovec is indicated for the treatment of adults with hemophilia B (congenital Factor IX deficiency) who use Factor IX prophylaxis therapy, ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is a cancer of the lymphoid line of blood cells characterized by the development of large numbers of immature lymphocytes. Symptoms may include feeling tired, pale skin color, fever, easy bleeding or bruising, enlarged lymph nodes, or bone pain. As an acute leukemia, ALL progresses rapidly and is typically fatal within weeks or months if left untreated. In most cases, the cause is unknown. Genetic risk factors may include Down syndrome, Li-Fraumeni syndrome, or neurofibromatosis type 1. Environmental risk factors may include significant radiation exposure or prior chemotherapy. Evidence regarding electromagnetic fields or pesticides is unclear. Some hypothesize that an abnormal immune response to a common infection may be a trigger. The underlying mechanism involves multiple genetic mutations that results in rapid cell division. The excessive immature lymphocytes in the bone marrow interfere with the production of new red blood cells, white ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Tisagenlecleucel
Tisagenlecleucel, sold under the brand name Kymriah, is a CAR T cells medication for the treatment of B-cell acute lymphoblastic leukemia (ALL) which uses the body's own T cells to fight cancer (adoptive cell transfer). Serious side effects occur in most patients. The most common serious side effects are cytokine release syndrome (a potentially life-threatening condition that can cause fever, vomiting, shortness of breath, pain and low blood pressure) and decreases in platelets (components that help the blood to clot), hemoglobin (the protein found in red blood cells that carries oxygen around the body) or white blood cells including neutrophils and lymphocytes. Serious infections occur in around three in ten diffuse large B-cell lymphoma (DLBCL) patients. T cells from a person with cancer are removed, genetically engineered to make a specific chimeric cell surface receptor with components from both a T-cell receptor and an antibody specific to a protein on the cancer cell, and t ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Melanoma
Melanoma, also redundantly known as malignant melanoma, is a type of skin cancer that develops from the pigment-producing cells known as melanocytes. Melanomas typically occur in the skin, but may rarely occur in the mouth, intestines, or eye (uveal melanoma). In women, they most commonly occur on the legs, while in men, they most commonly occur on the back. About 25% of melanomas develop from moles. Changes in a mole that can indicate melanoma include an increase in size, irregular edges, change in color, itchiness, or skin breakdown. The primary cause of melanoma is ultraviolet light (UV) exposure in those with low levels of the skin pigment melanin. The UV light may be from the sun or other sources, such as tanning devices. Those with many moles, a history of affected family members, and poor immune function are at greater risk. A number of rare genetic conditions, such as xeroderma pigmentosum, also increase the risk. Diagnosis is by biopsy and analysis of any skin lesion ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Talimogene Laherparepvec
Talimogene laherparepvec, sold under the brand name Imlygic, is a biopharmaceutical medication used to treat melanoma that cannot be operated on; it is injected directly into a subset of lesions which generates a systemic immune response against the recipient's cancer. The final four year analysis from the pivotal phase 3 study upon which TVEC was approved by the FDA showed a 31.5% response rate with a 16.9% complete response (CR) rate. There was also a substantial and statistically significant survival benefit in patients with earlier metastatic disease (stages IIIb-IVM1a) and in patients who hadn't received prior systemic treatment for melanoma. The earlier stage group had a reduction in the risk of death of approximately 50% with one in four patients appearing to have met, or be close to be reaching, the medical definition of cure. Real world use of talimogene laherparepvec have shown response rates of up to 88.5% with CR rates of up to 61.5%. Around half of people treated wi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Adenosine Deaminase Deficiency
Adenosine deaminase deficiency (ADA deficiency) is a metabolic disorder that causes immunodeficiency. It is caused by mutations in the ADA gene. It accounts for about 10–15% of all cases of autosomal recessive forms of severe combined immunodeficiency (SCID) among non-inbred populations. ADA deficiency can present in infancy, childhood, adolescence, or adulthood. Age of onset and severity is related to some 29 known genotypes associated with the disorder. It occurs in fewer than one in 100,000 live births worldwide. Signs and symptoms The main symptoms of ADA deficiency are pneumonia, chronic diarrhea, and widespread skin rashes. Affected children also grow much more slowly than healthy children and some have developmental delay. Most individuals with ADA deficiency are diagnosed with SCID in the first 6 months of life. An association with polyarteritis nodosa has been reported.Liebowitz J, Hellmann DB1, Schnappauf O (2019) Thirty years of followup in 3 patients with fami ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Strimvelis
Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence, sold under the brand name Strimvelis, is a medication used to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The most common side effect is pyrexia (fever). Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. ADA-SCID is a rare inherited condition in which there is a change (mutation) in the gene needed to make an enzyme called adenosine deaminase (ADA). As a result, people lack the ADA enzyme. Because ADA is essential for maintaining healthy lymphocytes (white blood cells that fight off infections), the immune system of people with ADA-SCID does not work properly and without effective treatment they rarely survive more than two years. Strimvelis is the first ''ex vivo'' autologous gene therapy approved by the E ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle wasting. It is usually diagnosed in infancy or early childhood and if left untreated it is the most common genetic cause of infant death. It may also appear later in life and then have a milder course of the disease. The common feature is progressive weakness of voluntary muscles, with arm, leg and respiratory muscles being affected first. Associated problems may include poor head control, difficulties swallowing, scoliosis, and joint contractures. The age of onset and the severity of symptoms form the basis of the traditional classification of spinal muscular atrophy into a number of types. Spinal muscular atrophy is due to an abnormality (mutation) in the ''SMN1'' gene which encodes SMN, a protein necessary for survival of motor neurons. Loss of these neurons in the spinal cord prevents signalling between the brain and skeletal muscles. Another g ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Onasemnogene Abeparvovec
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time intravenous injection, infusion into a vein. Onasemnogene abeparvovec works by providing a new copy of the gene that makes the SMN1, human SMN protein. The treatment must be accompanied by a course of corticosteroids of at least two months. Common side effects include vomiting and increased liver enzymes. Onasemnogene abeparvovec was first approved for medical use in the United States in 2019 as a treatment for children less than two years old. It was later approved in other jurisdictions with similar scope. The approval scope in certain jurisdictions, including the European Union and Canada, is somewhat different. Medical uses Onasemnogene abeparvovec has been developed to treat spinal muscular atrophy, a disease linked to a mutation in the survival motor neuron 1, ''SMN1'' gene on chromosome 5q and diagnosed pr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Nadofaragene Firadenovec
Nadofaragene firadenovec, sold under the brand name Adstiladrin, is a gene therapy for the treatment of bladder cancer. It is a non-replicating (cannot multiply in human cells) adenoviral vector-based gene therapy. The most common adverse events including laboratory abnormalities, include increased glucose, instillation site discharge, increased triglycerides, fatigue, bladder spasm, micturition urgency, increased creatinine, hematuria, decreased phosphate, chills, dysuria, and pyrexia. Nadofaragene firadenovec was approved for medical use in the United States in December 2022. Medical uses Nadofaragene firadenovec is indicated for the treatment of adults with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. History The safety and effectiveness of nadofaragene firadenovec was evaluated in a multicenter clinical study (Study CS-003 (NCT02773849)) that included ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Idecabtagene Vicleucel
Idecabtagene vicleucel, sold under the brand name Abecma, is a cell-based gene therapy to treat multiple myeloma. The most common side effects include cytokine release syndrome (CRS), infections, fatigue, musculoskeletal pain, and a weakened immune system (hypogammaglobulinemia). Idecabtagene vicleucel is a B-cell maturation antigen (BCMA)-directed genetically modified Autologous immune enhancement therapy, autologous Chimeric antigen receptor T cell, chimeric antigen receptor (CAR) T-cell therapy. Each dose is customized using a patient's own T-cells, which are a type of white blood cell, that are collected and genetically modified to include a new gene that facilitates targeting and killing myeloma cells, and infused back into the patient. Idecabtagene vicleucel was approved for medical use in the United States in March 2021. It is the first cell-based gene therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma. It was approved ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
