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Research and development in the area is currently growing at a fast rate, and the National Institute of Health reports hundreds of clinical trials to test gene therapies for different geneticdiseases, immune system disorders, oncology treatments, neurogenerative diseases, infectious diseases, and more.
Immune systems can be weakened in individuals with HIV/AIDS; cancer and transplant patients taking certain immunosuppressive drugs; and those with geneticdiseases affecting the immune system like congenital agammaglobulinemia and congenital IgA deficiency.
Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat geneticdiseases. Researchers will compare the two-time points to recognize the optimum timing of peak immuneresponse following nasal vaccination.
Verily collaborates with Janssen to launch COVID-19 immuneresponse study. Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immuneresponses to a coronavirus infection, with people participating in the research from within their homes. .
AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited geneticdisease that causes cardiac and central nervous system dysfunction.
GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative geneticdiseases that includes Tay-Sachs and Sandhoff diseases. aTyr Pharma completed target enrollment in its Phase Ib/IIa trial of ATYR1923 in pulmonary sarcoidosis, a form of interstitial lung disease.
Patients with this disease have a very high level of LDL-C, which increases their risk of coronary artery disease, including heart attack, stroke and atherosclerosis. Chemotherapy, immunosuppressant drugs and prophylactic antibiotic treatments may be used, along with a hematopoietic stem cell transplant.
With early nanoparticle studies, researchers often found the human body’s innate immuneresponse to drugs and the short-lasting effects of drugs challenging, as they can also reduce a drug’s efficacy. However, after a period of slow movement in the field, the FDA approved the first nanodrug, Doxil, for Kaposi sarcoma in 1995.
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