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.

FDA Lifts Clinical Hold; Green-Lights Vertex and CRISPR’s Sickle Cell Gene Therapy Trial

Posted on: 14 October 2018, source: BioSpace
The U.S. Food and Drug Administration (FDA) has lifted a clinical hold and accepted an Investigational New Drug Application (IND) for an experimental sickle cell disease treatment being co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics. In May the FDA placed a hold on the trial and IND for CTX001, an investigational gene editing treatment, citing concerns over questions that had not been addressed in the IND. The two companies initially submitted the IND in April in support of a planned Phase I/II trial.

Screening Clinical Cell Products for Replication Competent Retrovirus: The National Gene Vector Biorepository Experience

Posted on: 17 September 2018, source: Mol Ther Methods Clin Dev
Replication-competent retrovirus (RCR) is a safety concern for individuals treated with retroviral gene therapy. RCR detection assays are used to detect RCR in manufactured vector, transduced cell products infused into research subjects, and in the research subjects after treatment. In this study, we reviewed 286 control (n = 4) and transduced cell products (n = 282) screened for RCR in the National Gene Vector Biorepository. The transduced cell samples were submitted from 14 clinical trials. All vector products were previously shown to be negative for RCR prior to use in cell transduction. After transduction, all 282 transduced cell products were negative for RCR. In addition, 241 of the clinical trial participants were also screened for RCR by analyzing peripheral blood at least 1 month after infusion, all of which were also negative for evidence of RCR infection. The majority of vector products used in the clinical trials were generated in the PG13 packaging cell line. The findings suggest that screening of the retroviral vector product generated in PG13 cell line may be sufficient and that further screening of transduced cells does not provide added value.

European Commission Approves Novartis’ Kymriah CAR-T Cell Therapy

Posted on: 29 August 2018, source: Novartis
Novartis announced that the European Commission (EC) approved its chimeric antigen receptor T cell (CAR-T) cell therapy Kymriah (tisagenlecleucel) for the treatment of pediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) and for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.

Health Officials Remove Special NIH Oversite Rules Governing Gene Therapy Experiments

Posted on: 20 August 2018, source: BioSpace
As gene therapies have become reality, the U.S. government is removing some special regulations that had been set up long ago over concerns of exotic safety risks. The Associated Press reported that a National Institutes of Health oversight panel will no longer be called upon to review all gene therapy applications. That panel will now take a broader advisory role, the AP said. The U.S. Food and Drug Administration (FDA) will now look at gene therapy treatments as it down all other types of medications.

FDA Seeks to Enhance Manufacturing of Cell and Gene Therapies

Posted on: 3 August 2018, source: Biopharma International
More consistent and reliable production processes are critical for advancing innovative treatments. While cell- and gene-therapy products hold the promise of transforming the treatment of many diseases, difficulties in achieving consistent process control could stymie advancement in this field, cautions Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER). It’s “not simple” to bring a clinical production process to commercial scale, he says, voicing fears that important manufacturing issues could hinder efforts to advance new discoveries able to transform medical care.

New EU initiatives to optimize the interplay between the Pharma and the GMO framework

Posted on: 18 July 2018, source: European Commission
The EU's Regulation on advanced therapies, is designed to ensure the free movement of advanced therapy products within Europe, to facilitate access to the EU market, and to foster the competitiveness of European companies in the field, while guaranteeing the highest level of health protection for patients. A Good Practice document on the assessment of GMO-related aspects in the context of clinical trials with human cells genetically modified has been developed by the national competent authorities and the Commission services.

Caution required for using CRISPR/Cas9 in potential gene therapies

Posted on: 18 July 2018, source: Innovation Toronto
Scientists at the Wellcome Sanger Institute have discovered that CRISPR/Cas9 gene editing can cause greater genetic damage in cells than was previously thought. These results create safety implications for gene therapies using CRISPR/Cas9 in the future as the unexpected damage could lead to dangerous changes in some cells. Reported in the journal Nature Biotechnology, the study also revealed that standard tests for detecting DNA changes miss finding this genetic damage, and that caution and specific testing will be required for any potential gene therapies.