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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

Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation.

FDA 40
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2025 Merkin Prize in Biomedical Technology awarded to pioneers of CAR T-cell therapy

Broad Institute

The work of June, Levine, Rivire, and Sadelain laid the foundation for a whole new class of treatments. The power of a patients immune system The idea of redirecting a persons own immune system to recognize and kill cancer cells had been around for decades, but how to do it effectively and safely remained a mystery.

Therapies 119
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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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The Long Road to End Tuberculosis

Codon

In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. Over time, the frequency of resistance gene variants in the population will continue increasing because only the bacteria lacking those variants will die with treatment.

Vaccine 114
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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. Therapeutics The first Cas13-based clinical trial was approved in 2024.

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Kala Gets the Greenlight from FDA for Dry Eye Disease Treatment

The Pharma Data

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . EYSUVIS is the first FDA-approved corticosteroid specifically for dry eye disease treatment. Over-the-counter treatments fail due to the body’s natural protective system. Roughly 16.4

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Pfizer and BioNTech Initiate Rolling Submission of Biologics License Application for U.S. FDA Approval of Their COVID 19 Vaccine

The Pharma Data

FDA EMERGENCY USE AUTHORIZATION PRESCRIBING INFORMATION: Do not administer Pfizer-BioNTech COVID-19 Vaccine to individuals with known history of a severe allergic reaction (e.g., Immunocompromised persons, including individuals receiving immunosuppressant therapy, may have a diminished immune response to the Pfizer-BioNTech COVID-19 Vaccine.