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 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.
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.
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.
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.
First cohort enrolled in trial with innovative gene therapy for arthritis
Posted on: 18 July 2018, source: Arthrogen
Arthrogen, a clinical stage gene therapy company together with the Centre for Human Drug Research (CHDR), a Leiden based independent clinical research organization, announced that the first three arthritis patients have been enrolled in a phase Ib gene therapy trial for treatment of arthritis with ART-I02. ART-I02 is an adeno-associated virus (AAV5) vector encoding the human IFN-β gene under control of an inflammation-responsive promoter. ART-I02 is designed to produce the anti-inflammatory protein IFN-β in the synovial cells in the joint. The aim is to achieve a sustained clinical remission with a single treatment. This current clinical phase Ib trial, evaluates the safety, tolerability, pharmacokinetics, immunogenicity and anti-inflammatory activity of ART-I02 treatment in patients with rheumatoid arthritis (RA) or osteoarthritis (OA). In total 12 patients with RA or OA in their hand joints will be included. Parallel to this study, a similar phase Ib trial has started in Canada treating 15 patients with RA in their wrist.
Arthrogen, a clinical stage gene therapy company together with the Centre for Human Drug Research (CHDR), a Leiden based independent clinical research organization, announced that the first three arthritis patients have been enrolled in a phase Ib gene therapy trial for treatment of arthritis with ART-I02. ART-I02 is an adeno-associated virus (AAV5) vector encoding the human IFN-β gene under control of an inflammation-responsive promoter. ART-I02 is designed to produce the anti-inflammatory protein IFN-β in the synovial cells in the joint. The aim is to achieve a sustained clinical remission with a single treatment. This current clinical phase Ib trial, evaluates the safety, tolerability, pharmacokinetics, immunogenicity and anti-inflammatory activity of ART-I02 treatment in patients with rheumatoid arthritis (RA) or osteoarthritis (OA). In total 12 patients with RA or OA in their hand joints will be included. Parallel to this study, a similar phase Ib trial has started in Canada treating 15 patients with RA in their wrist.
FDA Aims to Facilitate Gene Therapy Development for Hemophilia, Rare Diseases
Posted on: 13 July 2018, source: Pharmacy & Healthcare
Significant progress has been made in gene therapy research and development over the years, with promise to treat and cure some of the most complex diseases. In a statement, FDA Commissioner Scott Gottlieb, MD, announced the agency’s latest efforts to speed up the path to approval for gene therapies, specifically for certain specialty and rare diseases. To help expedite advancement in the field, the FDA has released a new policy framework for the development, review, and approval of gene therapies to address the challenges of bringing these drugs to market.
Significant progress has been made in gene therapy research and development over the years, with promise to treat and cure some of the most complex diseases. In a statement, FDA Commissioner Scott Gottlieb, MD, announced the agency’s latest efforts to speed up the path to approval for gene therapies, specifically for certain specialty and rare diseases. To help expedite advancement in the field, the FDA has released a new policy framework for the development, review, and approval of gene therapies to address the challenges of bringing these drugs to market.
New UK consortium looks to make breakthrough in gene therapy manufacture
Posted on: 3 July 2018, source: Biopharma-reporter
Plasticell, alongside Great Ormond Street Hospital and the UCL Institute of Child Health, have announced they will collaborate to develop technology to better develop and manufacture gene therapies. Full story.
Plasticell, alongside Great Ormond Street Hospital and the UCL Institute of Child Health, have announced they will collaborate to develop technology to better develop and manufacture gene therapies. Full story.
Gene-Therapy Company Crispr Drops as FDA Puts Trial on Hold
Posted on: 10 June 2018, source: Bloomberg
Crispr Therapeutics AG plunged after the gene-editing company said a planned trial of its treatment to help people with sickle-cell disease was being put on hold by U.S. regulators. Crispr is developing the therapy with Vertex Pharmaceuticals Inc., and the trial had yet to begin. The companies said in a statement Wednesday that the Food and Drug Administration placed a halt on the experiment until the agency could get more information from the companies.
Crispr Therapeutics AG plunged after the gene-editing company said a planned trial of its treatment to help people with sickle-cell disease was being put on hold by U.S. regulators. Crispr is developing the therapy with Vertex Pharmaceuticals Inc., and the trial had yet to begin. The companies said in a statement Wednesday that the Food and Drug Administration placed a halt on the experiment until the agency could get more information from the companies.
Gene Therapy News
- Gene therapy: Gene therapy before the cradle
Fri, 17 Aug 2018 05:15 GMT - Hemophilia has been lifelong affliction, but gene therapy may change that timeline
Sun, 19 Aug 2018 11:09 GMT - Officials remove special rules for gene therapy experiments
Sat, 18 Aug 2018 15:34 GMT - Health Officials Remove Special NIH Oversite Rules Governing Gene Therapy Experiments: Report
Thu, 16 Aug 2018 13:41 GMT - A cure for blindness? Scientists manage to RESTORE sight in mice using GENE THERAPY
Thu, 16 Aug 2018 07:08 GMT
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Gene Therapy Literature
Scroll through the latest articles in gene therapy scientific journals.
Recent early view article in Journal of Gene Medicine:
You can buy a unique research paper online on gene therapy topics here.
Recent early view article in Journal of Gene Medicine:
You can buy a unique research paper online on gene therapy topics here.