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In vivo CAR T: Faster, cheaper, and more effective cancer care

Drug Target Review

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.

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

Broad Institute

Merkin Prize in Biomedical Technology for developing chimeric antigen receptor (CAR) T-cell therapy, a groundbreaking form of personalized cancer immunotherapy that turns T cells into tumor killers and has led to durable remissions in tens of thousands of patients with previously incurable blood cancers.

Therapies 119
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The Role of the FDA’s Oncology Center of Excellence in Advancing Cancer Care

The Premier Consulting Blog

Established in 2017 under the 21st Century Cures Act, the OCE brings together multidisciplinary scientific expertise to accelerate the review and approval of drugs, biologics, and medical devices for cancer care. These reviews have provided life-saving cancer therapies a median of 3.1

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Pirtobrutinib

New Drug Approvals

Compounds useful as kinase inhibitors (WO 2017/103611 A1). MORE US10918622 US10695323 US10464905 US10342780 US12109193 Medical uses In the United States, pirtobrutinib is indicated to treat relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. [1]

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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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Leniolisib

New Drug Approvals

5] [7] [8] It is the first approved medication for the treatment of activated PI3K delta syndrome. [5] 5] [7] [8] It is the first approved medication for the treatment of activated PI3K delta syndrome. [5] World Health Organization (2017). New Drug Therapy Approvals 2023 (PDF). mL) and the mixture stirred at rt for 1 h.

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Leveraging agonist antibodies to address immunological diseases

Drug Target Review

This targeted enhancement not only boosts the body’s natural mechanisms but also holds promise for developing novel treatments in immunology, where modulating immune responses can lead to more effective and durable therapeutic outcomes. In these conditions, the goal of therapy is typically to suppress or mitigate immune activity.

Disease 52