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Welcome to Gene Therapy Net

Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.

FDA approves Orchard’s Lenmeldy gene therapy for MLD

Posted on: 19 March 2024, source:
The US Food and Drug Administration (FDA) has granted approval to Orchard Therapeutics‘ Lenmeldy (atidarsagene autotemcel) gene therapy for use in children with specific forms of metachromatic leukodystrophy (MLD). This is the first FDA approval for a gene therapy addressing pre-symptomatic late infantile, early juvenile or early symptomatic early juvenile MLD. Lenmeldy is a single-use, personalised infusion, utilising the patient’s genetically modified hematopoietic stem cells (HSCs) to halt disease progression. The gene therapy involves collecting HSCs from the patient and modifying them to include functional copies of the ARSA gene. The modified cells are re-introduced into the patient’s bone marrow where they engraft and produce immune cells that can break down sulfatides, the substances that accumulate harmfully in MLD.
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Team performs the first gene therapy tests in a whole human liver

Posted on: 14 March 2024, source:
In a worldwide first-of-its-kind study published in the journal Nature Communications this week, a team of scientists from Children's Medical Research Institute (CMRI) have tested novel gene therapies in a whole human liver, with the goal of developing more effective treatments for life-threatening inherited diseases.
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Gene Therapy Success in Hereditary Angioedema Patients

Posted on: 2 February 2024, source: Technologynetworks
A single dose of a CRISPR/Cas9 therapy has been successful in treating hereditary angioedema. A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher. The patients from New Zealand, the Netherlands and the UK have hereditary angioedema, a genetic disorder characterised by severe, painful and unpredictable swelling attacks. These interfere with daily life and can affect airways and prove fatal.
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FDA Approves CRISPR Gene Therapy to Treat Blood Disorder

Posted on: 24 January 2024, source:
The U.S. Food and Drug Administration (FDA) has approved CRISPR gene editing cell therapy to treat a rare inherited blood disorder called transfusion-dependent beta-thalassemia (TDT). CRISPR Therapeutics and Vertex Pharmaceuticals’ gene editing cell therapy, branded as Casgevy, has officially been approved to treat TDT — a rare inherited blood disorder that requires regular blood transfusions — in patients 12 and over. Vertex announced Tuesday that it had been given FDA approval more than two months ahead of its original expected action date, and just over a month after the same treatment to treat sickle cell disease was approved.
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FDA Signals Imminent Approval for Sarepta’s Duchenne Gene Therapy: A Major Breakthrough for Patients

Posted on: 12 January 2024, source:
The world of gene therapy is on the cusp of a significant milestone. Peter Marks, the Food and Drug Administration’s (FDA) top regulator of gene therapies, has indicated the full approval of Sarepta’s Duchenne gene therapy, Elevidys, is imminent. This development has the potential to positively impact patients, caregivers, and the healthcare industry as a whole.
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Revolutionary gene therapy shows promise in treating autism

Posted on: 6 December 2023, source:
Chinese scientists have achieved a major advancement in gene therapy for autism, as reported in the journal Nature Neuroscience. This innovative therapy, developed by a research team in Shanghai, has been successfully tested on mice. The research marks a significant step in the treatment of autism spectrum disorder (ASD) through genetic base editing within the brain.
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Cell & Gene Therapy Space Gears Up for First CRISPR Approval

Posted on: 13 November 2023, source: Biospace
Halloween brings to mind pumpkins and trick or treat, but for the 100,000 Americans suffering from sickle cell disease, this past October 31 had a different meaning when an FDA advisory panel deemed the first potentially curative SCD therapy safe enough for clinical use, bolstering hopes of an FDA approval in December. It would also be the first-ever approval for CRISPR, the genetic modification technique that won its discoverers the Nobel Prize in 2020 for their 2012 breakthrough.
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