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U.S. FDA Grants Orphan Drug Designation to Riliprubart for Treating Antibody-Mediated Rejection in Solid Organ Transplant Recipients

The Pharma Data

This designation represents a significant regulatory milestone for Sanofi and further underscores the company’s expanding efforts to address rare and serious immune-mediated disorders. This form of rejection can occur early or late after transplantation and is notoriously difficult to treat.

FDA
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2025 Merkin Prize in Biomedical Technology awarded to pioneers of CAR T-cell therapy

Broad Institute

He showed that these synthetic CAR T cells could launch an immune response when they encountered the corresponding tumor protein, called CD19. That ability to stick around, which is critical for attacking cancer over time, was another turning point. In some cases, patients treated with the new therapy remained cancer-free for years.

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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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A Visual Guide to Gene Delivery

Codon

Casgevy , the first FDA-approved CRISPR-based therapy, is also an ex vivo gene therapy; it aims to cure sickle cell disease by editing a patient’s blood-producing stem cells so they generate functional fetal hemoglobin, then returning these corrected cells to the bloodstream. This changed with the discovery of AAVs.

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The Long Road to End Tuberculosis

Codon

If present before vaccination, memory immune cells primed to destroy the environmental relatives might cross-react with and neutralize the BCG strain before it has a chance to set up an immune response against itself and M. Food and Drug Administration (FDA)-approved drug library, some of which inhibit E.

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A Visual Guide to Genome Editors

Codon

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Collectively, these repeat-protospacer regions are known as CRISPR arrays.

DNA
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Kala Gets the Greenlight from FDA for Dry Eye Disease Treatment

The Pharma Data

EYSUVIS is the first FDA-approved corticosteroid specifically for dry eye disease treatment. The corticosteroid then works to relieve swelling, redness and itching by targeting the immune response that drives dry eye disease flares. . Over-the-counter treatments fail due to the body’s natural protective system.