Inotersen

An article about the medication Inotersen

Engineered Monoclonal Antibodies

Diagram of engineered monoclonal antibodies

Engineered monoclonal antibodies are a class of biological therapies that are designed to target specific antigens on the surface of cells. These antibodies are produced using recombinant DNA technologies and are used in the treatment of various diseases, including cancer, autoimmune disorders, and infectious diseases.

Structure and Function

Monoclonal antibodies are composed of two identical heavy chains and two identical light chains, forming a Y-shaped molecule. The tips of the "Y" contain the antigen-binding sites, which are highly specific to the target antigen. This specificity allows monoclonal antibodies to bind to their target with high affinity, blocking or modulating the function of the antigen.

Types of Engineered Monoclonal Antibodies

There are several types of engineered monoclonal antibodies, each designed for specific therapeutic purposes:

• Chimeric antibodies: These antibodies are composed of murine (mouse) variable regions and human constant regions. They are less immunogenic than fully murine antibodies.

• Humanized antibodies: These antibodies are mostly human, with only the antigen-binding sites derived from murine sources. This reduces the risk of immune reactions.

• Fully human antibodies: These are entirely human in origin, produced using transgenic mice or phage display technologies.

• Bispecific antibodies: These antibodies are engineered to bind two different antigens simultaneously, offering unique therapeutic mechanisms.

Applications in Medicine

Engineered monoclonal antibodies have revolutionized the treatment of many diseases:

• Cancer therapy: Monoclonal antibodies can target specific tumor antigens, leading to direct tumor cell killing or recruitment of immune cells to attack the tumor.

• Autoimmune diseases: By targeting specific components of the immune system, monoclonal antibodies can reduce inflammation and tissue damage in diseases such as rheumatoid arthritis and multiple sclerosis.

• Infectious diseases: Monoclonal antibodies can neutralize pathogens or their toxins, providing passive immunity or enhancing the host's immune response.

Production

The production of engineered monoclonal antibodies involves several steps:

1. Antigen identification: The target antigen is identified and characterized. 2. Hybridoma technology: B cells from immunized animals are fused with myeloma cells to create hybridomas that produce the desired antibody. 3. Recombinant DNA technology: Genes encoding the antibody are cloned and expressed in suitable host cells, such as Chinese hamster ovary cells. 4. Purification and formulation: The antibodies are purified and formulated for clinical use.

Challenges and Future Directions

While engineered monoclonal antibodies have shown great promise, there are challenges such as high production costs, potential for immune reactions, and the development of resistance. Ongoing research aims to improve antibody design, reduce immunogenicity, and enhance therapeutic efficacy.

Related Pages

• Monoclonal antibody therapy
• Biopharmaceutical
• Immunotherapy
• Hybridoma technology

Inotersen is a medication used in the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. It is an antisense oligonucleotide that targets the transthyretin (TTR) protein, reducing its production and thereby alleviating the symptoms associated with the disease.

Mechanism of Action

Inotersen works by binding to the messenger RNA (mRNA) of the transthyretin protein, which is primarily produced in the liver. By binding to the mRNA, inotersen prevents the translation of the TTR protein, leading to a decrease in its levels in the bloodstream. This reduction in TTR protein helps to prevent the formation of amyloid deposits in tissues, which are responsible for the symptoms of hATTR.

Clinical Use

Inotersen is indicated for the treatment of adults with hereditary transthyretin-mediated amyloidosis. This condition is characterized by the accumulation of amyloid fibrils in various organs and tissues, leading to a range of symptoms including peripheral neuropathy, autonomic dysfunction, and cardiomyopathy.

Administration

Inotersen is administered via subcutaneous injection. The typical dosing regimen involves weekly injections, which patients can self-administer after receiving appropriate training.

Side Effects

Common side effects of inotersen include injection site reactions, nausea, headache, and fatigue. More serious side effects can include thrombocytopenia (low platelet count) and glomerulonephritis (inflammation of the kidneys). Regular monitoring of platelet counts and renal function is recommended during treatment.

Monitoring

Patients receiving inotersen require regular monitoring to ensure safety and efficacy. This includes:

• Regular platelet count checks to monitor for thrombocytopenia.
• Renal function tests to detect any signs of kidney damage.
• Liver function tests, as the liver is the primary site of TTR production.

Related pages

• Transthyretin
• Amyloidosis
• Antisense oligonucleotide

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  Inotersen