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Gene Therapy and Pharmacokinetics

Camargo

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 genetic diseases, immune system disorders, oncology treatments, neurogenerative diseases, infectious diseases, and more.

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FDA Authorizes COVID-19 Vaccine Boosters for Immunocompromised Individuals

XTalks

Immune systems can be weakened in individuals with HIV/AIDS; cancer and transplant patients taking certain immunosuppressive drugs; and those with genetic diseases affecting the immune system like congenital agammaglobulinemia and congenital IgA deficiency.

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Shape Therapeutics-Roche’s Deal; AllStripes Raises $50M; Datavant-Real Chemistry’s Partnership; BlueWillow’s Nasal Vaccine

Delveinsight

Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases. Researchers will compare the two-time points to recognize the optimum timing of peak immune response following nasal vaccination.

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Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

Delveinsight

Verily collaborates with Janssen to launch COVID-19 immune response study. Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immune responses to a coronavirus infection, with people participating in the research from within their homes. .

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Biopharma Money on the Move: October 21-27

The Pharma Data

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 genetic disease that causes cardiac and central nervous system dysfunction.

Gene 52
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Clinical Catch-Up: December 21-25 | BioSpace

The Pharma Data

GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases 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.

Trials 52
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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

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.

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