Remove Immune Response Remove RNA Remove Therapies
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Cell and gene therapy development moves into cardiac indications

Drug Target Review

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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Second-Generation mRNA COVID-19 Vaccine Candidate, CV2CoV, Demonstrates Improved Immune Response and Protection in Preclinical Study

The Pharma Data

Better activation of innate and adaptive immune responses was achieved with CV2CoV, resulting in faster response onset, higher titers of antibodies, and stronger memory B and T cell activation as compared to the first-generation candidate, CVnCoV. “In Induction of innate immunity was investigated via specific cytokine markers.

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Developing an mRNA therapy for Duchenne muscular dystrophy

Drug Target Review

There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. AAV-based therapies often can’t be dosed more than once due to concerns about an immune response. Of note, mRNA is redosable as opposed to AAV.

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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. “As

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Decoding stem cells for personalised regenerative medicine

Drug Target Review

In research published in Scientific Reports , 1 investigators focused on mesenchymal stem cells (MSCs), known for their potential in treating cell defects and regulating immune responses. In the lab, they labelled RNA molecules with fluorescent markers, enabling them to easily locate them within individual cells.

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Study reveals how cancer immunotherapy may cause heart inflammation in some patients

Broad Institute

By Leah Eisenstadt November 6, 2024 Credit: Ricardo Job-Reese, Broad Communications Despite being one of the rarer complications from immune checkpoint inhibitors, myocarditis is the most deadly. This work provides a biological foundation for testing more targeted therapies for myocarditis due to an immune checkpoint inhibitor.

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Codon Digest: Seeing Colors After Gene Therapy

Codon

Their cone cells, which are responsible for color vision, don’t work like normal. But what happens if you restore these cone cells, using gene therapy? approved a gene therapy for hemophilia A for the first time. The therapy works by replacing beta-cells with “fresh” cells taken from a, err…dead person.