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Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC geneexpression.
Related: FDAApproves Rukobia for HIV Patients with Limited Treatment Options. Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins.
As a result, there is a high unmet need for an FDAapproved local injection therapy that is safe and effective. 1 and COX-2 geneexpression. Surgery can also have a significant recurrence rate. STP705 has also received Orphan Drug Designation for treatment of cholangiocarcinoma and primary sclerosing cholangitis.
Several saliva-based tests also received FDAapprovals and authorizations, making sample collection easier and less invasive compared with traditional nasopharyngeal swabbing. Traditionally, bulk samples are used for genomic profiling, which offers a broad overview of geneexpression profiles across a milieu of different cells.
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