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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). These initiatives reflect a careful and comprehensive approach to patient safety — and a strong understanding of the delicate balance between delivering a potentially life-changing therapy and mitigating its associated risks.

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ISSUE 44 — The Importance of Cytokine Release Assays to Derisk the Development of Immunomodulatory Drugs

Alta Sciences

ISSUE 44 — The Importance of Cytokine Release Assays to Derisk the Development of Immunomodulatory Drugs pmjackson Mon, 07/07/2025 - 21:31 New immunomodulatory drugs have the potential to cause serious immune-related adverse events, including cytokine release syndrome (CRS).

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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.

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FDA takes major step to ease access to CAR-T therapy

BioPharma Drive: Drug Pricing

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. BCMA and CD19 are the protein targets of the seven approved CAR-T therapies. You can unsubscribe at anytime.

Therapies 176
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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. We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun.

RNA 128
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4-1BB and the critical importance of local control

SugarCone Biotech

Targeting 4-1BB remained of interest to the immuno-oncology field as cell culture experiments and tumor models in mice suggested that robust anti-tumor immune responses could be triggered by anti-4-1BB antibody therapies. This is the T cell type most closely associated with anti-tumor immune responses.

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The Altascientist Issue 36: Navigating the Key Considerations of Your Nonclinical Cell and Gene Therapy Studies

Alta Sciences

It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure. However, there are significant challenges associated with the successful development of these complex, leading-edge therapies. what is the altascientist?