Gene therapy is the process of creating a gene, usually a carbon copy of the original, in a laboratory in order to introduce it to the human body to cure or prevent a specific illness. Some of the new genes are a mutation or a combination of one or more genes.
Each year, new gene therapies are discovered and put to the test before introducing them to patients who can use them to recover from a number of diseases and ailments. Scientists, researchers, chemists, and doctors work long hours to come up with new therapies in gene therapies and mutation that will help hundreds of thousands of individuals live longer and healthier lives.
For more information, go to poseida.com today. In the meantime, here are 13 modern therapies that are breaking medical boundaries:
1. Instiladrin® (nadofaragene firadenovec/Sny3; rAd-IFN/Syn3)
This gene therapy is indicated for BCG (Bacillus Camette-Guérin) which is an unresponsive non-muscle invasive bladder cancer (NMIBC).
The new modified gene will take hold of nearby cells containing the tumor and pollutes and activates the translation and transcription of these genes. They are antiproliferative, immune-modulating, antiviral and antitumor.
This gene therapy is indicated for the persistent epithelial defects (PED) which do not respond favorably to the standard care treatment currently indicated for these types of defects.
This is a natural, unmodified gene that regulates, downward, the gap in hemichannel protein Cx43 (Connexin43). This gel is placed under the contact lens, or on the amniotic membrane to ensure the gene therapy makes contact with the diseased cornea.
3. OXB-301 (TroVax®; MVA-5T4)
This gene therapy is indicated for colorectal and ovarian cancers. The active ingredient is made up of an MVA (modified vaccinia virus Ankara). It is intended to destroy the cells containing cancer and at the same time stimulate the immune system.
4. RT-100 (AC6 Gene Transfer)
This particular gene treatment is designed for heart failure and reduced ejection fraction (HFrEF). HfrEF affects the left side of the heart, this side doesn’t pump any blood out to the body due to insufficient squeezing of the ventricle.
It is injected directly into the arteries during a cardiac catheterization procedure. This gene therapy is able to enter cells but cannot reproduce itself.
5. Toca 511 (vocimagene amiretrorepvec) and Toca FC
This gene therapy is indicated to help a number of Cancers including melanoma, recently diagnosed high-grade preclinical glioma, colorectal Cancer, phase I breast Cancer, lung cancer, and renal cell cancer along with metastatic solid tumors.
This injectable is two-part immunotherapy for Cancer. The Toca FC is constructed as an extended-release oral medication.
6. VB-111 (ofranergene obadenovec)
This is a gene therapy used in phase III Ovarian Cancer that is recurrent platinum-resistant and other solid tumors. This is one of the therapies that target the anticancer biologic cells as a blockade to the tumor growth rather than eradicating it once it is formed.
This DNA-based medication is designed to help with diabetic peripheral neuropathy as well as non-healing chronic ischemic ulcers on the feet from diabetes. Other indications include acute myocardial infarction, or heart attack and amyotrophic lateral sclerosis (ALS).
ALS is a disease that attacks only certain types of cells in the spinal cord and the brain. These cells are crucial to keeping the muscles in the body moving. Some of the early signs of this illness include slurred speech, muscle twitching, and muscle weakness.
8. Zolgensma® (formerly AVXS-101)
This therapy is specifically designed for SMA (spinal muscular atrophy) and contains an adeno-associated virus 9, which is nonreplicating. This AAV9 capsule is used to administer a functional copy of the SMN gene directly into the nucleus of the cells in the patient. This medication is given to help the cell’s production of the protein SMN to a tolerable level.
9. OTL-103 (formerly GSK2696275)
This therapy is for WAS or Wiskott Aldrich Syndrome. WAS patients have the reduced ability to form blood clots due to abnormal immune deficiency in their system. Their platelets are decreased in size and number of cell fragments used for clotting purposes.
Collected from the bone marrow, the transduced lentiviral vector encoding or the mobilized peripheral blood, this is a genetically modified hematopoietic autologous stem cell.
This is for the treatment of Choroideremia, a progressive loss of vision mostly in males. The beginning of the condition has been often the onset of night blindness and can occur in early childhood.
This gene therapy contains a recombinant human complementary DNA or cDNA that can be found in the eye under the REP1. This is a surgically injected sub-retinal style treatment.
For the treatment of Sanfilippo Type A Syndrome or Mucopolysaccharidosis IIIA (MPS IIIA). This syndrome appears when the body needs to break down the enzymes Hepara Sulfate (HS) and they are defective or absent altogether.
This gene therapy contains a healthy copy of the SGSH or N-sulfoglucosamine sulfohydrolase and is administered, through an injection, directly into the brain. This is to stimulate the production of the missing or insufficient enzyme. The goal is to halt or slow down the progression of the illness.
Cerebral Adrenoleukodystrophy or CALD is the most severe form of Adrenoleukodystrophy or ALD. It mostly affects those with X chromosomes, and therefore is mostly found in boys. It is considered as the breakdown of the myelin or protective stealth covering cells. These cells are responsible for muscle control and thinking.
This therapy uses the patient’s own stem cells as a transplant to modify the functional copy of the ABCD1 gene. This gene will result in the production of adrenoleukodystrophy proteins.
13. Axalimogene filolisbac (AXAL; ADXS11-001) and ADXS-DUAL
This gene therapy is indicated for the treatment of Metastatic Cervical Cancer in women. The immunotherapy is targeted from a platform of technology using live attenuated Listeria Monocytoenes or Lm. This is a beginner gene to secrete antigen and adjunct proteins. This is the second-generation of axalimogene filolisbac.
If you have any of these illnesses or diseases, talk to your doctor about gene therapy. There may be a trail or a new therapy available through your health care provider that is newer and maybe looking for patients for testing.
“If you are unsure if you have a health condition, consider an initial round of blood tests. HealthLabs.com is taking the next steps into more readily accessible lab testing for patients. With fast, private, and affordable lab testing, you are able to monitor your health on your own terms.”