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.

 

Posted on: 4 May 2021, source: News-Medical.net
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.

fShare

Tweet

Posted on: 5 April 2021, source: News-Medical.net
A UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule. This new approach to CAR T gene therapy -- a type of immunotherapy that involves genetically engineering the body's own blood-forming stem cells to create HIV-fighting T cells -- has the potential to not only destroy HIV-infected cells but to create "memory cells" that could provide lifelong protection from infection with the virus that causes AIDS.

fShare

Tweet

Posted on: 13 March 2021, source: BioSpace
The European Medicines Agency (EMA) has launched a safety review of bluebird bio’s thalassaemia drug Zynteglo, a conditionally licensed gene therapy in Europe. This review comes after researchers identified an acute myeloid leukaemia (AML) case in a patient who received bb1111 (LentiGlobin), an investigational gene therapy for sickle cell disease (SCD), in a Phase I/II study. This therapy relies on the same viral vector found in Zynteglo which delivers a gene into cells.

fShare

Tweet

Posted on: 22 February 2021, source: Science
A company has stopped its clinical studies of a promising gene therapy for the blood disorder sickle cell disease after two people who participated developed leukemia-like cancer. Bluebird bio is now investigating whether a virus it uses to deliver a therapeutic gene caused the cancers, reviving old concerns about the risks of this approach. It’s also possible the cancers stemmed from chemotherapy the patients received to prepare their bodies for the gene’s delivery. “This is really a sad development whatever the cause,” says Donald Kohn of the University of California, Los Angeles, who has led gene therapy trials for sickle cell and other diseases.

fShare

Tweet

Posted on: 24 January 2021, source: FDA
FDA is issuing this guidance to provide manufacturers of licensed and investigational cellular therapy and gene therapy (CGT) products with risk-based recommendations to minimize potential transmission of the novel coronavirus, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). This guidance is intended to supplement the recommendations to drug and biological product manufacturers provided in FDA’s “Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing; Guidance for Industry” issued in June 2020 (Ref. 1) (June 2020 GMP Guidance). The recommendations in this guidance specifically consider the source material (cells and/or tissues) recovered from donors and how the CGT product will be manufactured (e.g., cell expansion in culture, viral reduction steps, formulation).

fShare

Tweet

Posted on: 23 December 2020, source: Science
It’s troubling news that gene therapy researchers have long anticipated: A hemophilia patient injected with a virus carrying a therapeutic gene in a clinical trial has developed a liver tumor. The U.S. Food and Drug Administration (FDA) has halted the associated clinical trials, and uniQure, the Dutch firm behind the studies, is now investigating whether the virus itself caused the cancer. Gene therapy experts say that’s unlikely. The patient had underlying conditions that predisposed him to liver cancer. Still, scientists say it’s crucial to rule out any role for adeno-associated virus (AAV), the viral delivery system, or vector, that is used in hundreds of other gene therapy trials. “Everyone will want to know what happened,” says physician-scientist David Lillicrap of Queen’s University, a hemophilia researcher who was not involved with the uniQure study.

fShare

Tweet

Posted on: 12 December 2020, source: The Scientist
It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye. In a Phase 3 gene therapy trial intended to improve vision among patients with Leber hereditary optic neuropathy, recipients gained somewhat better sight in both eyes even though only one was treated. The results and an investigation into possible explanations for the findings were published December 9 in Science Translational Medicine.

fShare

Tweet