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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.

Switching Gene Therapy On and Off with a Pill

Posted on: 2 July 2024, source: GEN
Gene therapy has a reputation as a one-time intervention that has a lifelong effect. It’s an all-or-nothing proposition. But what if it wasn’t? What if gene therapy could be dialed up or down, on a daily basis, with a simple pill? The possibility has inspired MeiraGTx to develop a riboswitch technology that is designed to allow for the precise, dose-responsive control of gene expression by oral small molecules. The riboswitch technology is just part of MeiraGTx’s work in gene therapy. The company has technologies for the optimization of adeno-associated virus (AAV) vectors and for the design of promoter sequences. Also, the company has an internally developed manufacturing platform process and several production facilities. Finally, the company has several gene therapy programs in late-stage clinical trials.
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Toddler Born Deaf Can Hear After Gene Therapy Trial Breakthrough Her Parents Call "Mind-Blowing"

Posted on: 20 May 2024, source: CBS News
One of the youngest children in the world to receive a new type of gene therapy to treat genetic deafness can now hear for the first time in her life. The family of the toddler taking part in a medical trial has called the change in their daughter "mind-blowing." Opal Sandy, now 18 months old, was born with total deafness due to a fault in the OTOF gene, which makes a protein called Otoferlin. Otoferlin enables communication between cells of the inner ear, or cochlea, and the brain. As part of a trial run by Cambridge University, Opal received an infusion of a working copy of the OTOF gene in her right ear. The surgical procedure took only 16 minutes and was carried out just before she reached her first birthday.
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First Patient Begins Newly Approved Sickle Cell Gene Therapy

Posted on: 6 May 2024, source: The New York Times
A 12-year-old boy in the Washington, D.C., area faces months of procedures to remedy his disease. “I want to be cured,” he said. On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. But it also signals the difficulties patients face as they seek a pair of new sickle cell treatments.
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FDA approves Orchard’s Lenmeldy gene therapy for MLD

Posted on: 19 March 2024, source: pharmaceutical-technology.com
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: medicalxpress.com
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: healthnews.com
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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