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Sarepta Strengthens ELEVIDYS Safety Measures for Non-Ambulatory Duchenne Patients

The Pharma Data

Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). Sarepta’s team is exploring adding sirolimus — a well-established immunosuppressive medication — to the regimen in a way that might enable a greater degree of control over the patient’s immune response.

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Roche shares Elevidys safety update in non-ambulatory Duchenne muscular dystrophy patients

The Pharma Data

This update comes after two cases of fatal acute liver failure (ALF) were reported in non-ambulatory DMD patients who received Elevidys, a phenomenon that underscores the ongoing complexities and safety signals related to adeno-associated virus (AAV)-mediated gene therapy. Chief Medical Officer and Head of Global Product Development at Roche.

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A Visual Guide to Genome Editors

Codon

.” Four years earlier, in 2019, Gray had become the first patient with sickle cell anemia — a genetic disorder that causes red blood cells to become sticky and rigid — to receive an experimental treatment using CRISPR genome editing. Collectively, these repeat-protospacer regions are known as CRISPR arrays.

DNA 81
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Researchers design gene therapy that can effectively target glioblastoma

Science Daily: Pharmacology News

Glioblastoma (GBM), an aggressive brain cancer, is notoriously resistant to treatment, with recurrent GBM associated with survival of less than 10 months.

Therapies 212
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mRNA Therapeutics: Revolutionizing Treatment Beyond Vaccines

DrugBank

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. This process triggers a robust immune response, enabling the immune system to recognize and attack cancer cells.

Vaccine 97
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Targeted drug treatment leads tumor cells to imitate viral infection

Broad Institute

Targeted drug treatment leads tumor cells to imitate viral infection By Ari Navetta July 11, 2024 Breadcrumb Home Targeted drug treatment leads tumor cells to imitate viral infection Exploiting "viral mimicry," mIDH1 inhibitors trick tumors into thinking they are infected with a virus.

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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.