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Related Concept Videos

FDA Approved Drugs: Changes to Approved Drugs01:26

FDA Approved Drugs: Changes to Approved Drugs

Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...
Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
Therapeutic Drug Monitoring: Overview and Classification01:16

Therapeutic Drug Monitoring: Overview and Classification

Therapeutic Drug Monitoring (TDM) is a clinical practice that measures specific drug levels in a patient's blood at designated intervals to ensure the drug concentration stays within a therapeutic range. This monitoring is crucial for optimizing individual dosage regimens, enhancing therapeutic efficacy, and minimizing drug-related toxicity. TDM is vital for drugs with narrow therapeutic windows, significant variability in pharmacokinetics, and a clear correlation between plasma levels and...

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Related Experiment Video

Updated: Jul 16, 2026

A Real-time Potency Assay for Chimeric Antigen Receptor T Cells Targeting Solid and Hematological Cancer Cells
08:46

A Real-time Potency Assay for Chimeric Antigen Receptor T Cells Targeting Solid and Hematological Cancer Cells

Published on: November 12, 2019

Marnetegragene Autotemcel: First Approval.

Simon Fung1

  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. mdt@adis.com.

Molecular Diagnosis & Therapy
|July 15, 2026
PubMed
Summary

Marnetegragene autotemcel is a novel gene therapy for treating severe leukocyte adhesion deficiency-I (LAD-I) in children. It received US approval for patients lacking an HLA-matched donor, marking a significant advancement in rare disease treatment.

Area of Science:

  • * Hematology
  • * Gene Therapy
  • * Immunology

Background:

  • * Leukocyte Adhesion Deficiency-I (LAD-I) is a severe primary immunodeficiency.
  • * Patients with severe LAD-I often lack HLA-matched donors for traditional stem cell transplants.
  • * Current treatment options for LAD-I are limited, especially for those without matched donors.

Purpose of the Study:

  • * To summarize the development milestones of marnetegragene autotemcel.
  • * To highlight the regulatory pathway leading to its first approval.
  • * To provide an overview of this novel gene therapy for LAD-I.

Main Methods:

  • * Development of an autologous stem cell-based gene therapy.
  • * Introduction of functional ITGB2 gene copies into patient hematopoietic stem cells.

Related Experiment Videos

Last Updated: Jul 16, 2026

A Real-time Potency Assay for Chimeric Antigen Receptor T Cells Targeting Solid and Hematological Cancer Cells
08:46

A Real-time Potency Assay for Chimeric Antigen Receptor T Cells Targeting Solid and Hematological Cancer Cells

Published on: November 12, 2019

  • * Clinical evaluation for efficacy and safety in pediatric patients with severe LAD-I.

    • Main Results:

    • * Marnetegragene autotemcel demonstrated successful gene addition to hematopoietic stem cells.
    • * The therapy showed promise in treating severe LAD-I in pediatric patients.
    • * Achieved first US approval in March 2026 for a specific patient population.

    Conclusions:

    • * Marnetegragene autotemcel represents a significant therapeutic advance for severe LAD-I.
    • * The gene therapy offers a new option for patients lacking HLA-matched sibling donors.
    • * This approval marks a milestone in the treatment of primary immunodeficiencies using gene therapy.