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Spinal Muscular Atrophy Neonatal Screening Program
15 March 2024
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Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
15 March 2024
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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.
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.
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.
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.
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.
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.
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.
FDA Signals Imminent Approval for Sarepta’s Duchenne Gene Therapy: A Major Breakthrough for Patients
Posted on: 12 January 2024, source: medriva.com
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.
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.
Revolutionary gene therapy shows promise in treating autism
Posted on: 6 December 2023, source: Earth.com
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.
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.
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.
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.
Gene therapy restores hearing in children with rare form of deafness
Posted on: 28 October 2023, source: Science
Several deaf children can hear after receiving gene therapy—a first for the approach—a team at Fudan University reported today at a meeting in Belgium. The children were born deaf because they inherited two defective copies of the gene for a protein called otoferlin that helps the inner ear’s hair cells transmit sound to the brain. In an attempt to restore this function, researchers injected harmless viruses carrying DNA for a working copy of the otoferlin gene into the children’s ears. Four of five patients treated now have some hearing, the MIT Technology Review reports.
Several deaf children can hear after receiving gene therapy—a first for the approach—a team at Fudan University reported today at a meeting in Belgium. The children were born deaf because they inherited two defective copies of the gene for a protein called otoferlin that helps the inner ear’s hair cells transmit sound to the brain. In an attempt to restore this function, researchers injected harmless viruses carrying DNA for a working copy of the otoferlin gene into the children’s ears. Four of five patients treated now have some hearing, the MIT Technology Review reports.
Gene Therapy News
- FDA Approves First Gene Therapy For Kids With Early-onset MLD - RTTNews
Tue, 19 Mar 2024 07:54:1 GMT - Gene Therapy Is Now First FDA-Approved Treatment for Rare & Fatal CNS Disorder - MedCity News
Mon, 18 Mar 2024 23:04:5 GMT - Orchard-Kyowa scores US approval for gene therapy Lenmeldy - BioWorld Online
Mon, 18 Mar 2024 22:35:1 GMT - Pediatric Neuron Disease Gets Its First Gene Therapy - Medpage Today
Mon, 18 Mar 2024 22:30:0 GMT - Orchard's gene therapy for MLD finally gets US approval - pharmaphorum
Mon, 18 Mar 2024 21:43:0 GMT
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Recent Modified Items
- Gene Therapy Conferences 2024
- FDA Signals Imminent Approval for Sarepta’s Duchenne Gene Therapy: A Major Breakthrough for Patients
- FDA Approves CRISPR Gene Therapy to Treat Blood Disorder
- Gene Therapy Success in Hereditary Angioedema Patients
- Team performs the first gene therapy tests in a whole human liver