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.
Encouraging early results from first human CRISPR gene therapy trials
Posted on: 25 November 2019, source: NewAtlas
Promising preliminary data from one of the first human trials testing the safety and efficacy of a CRISPR gene therapy has just been revealed. Although it is too early to evaluate long-term effects, the initial reports are impressively successful for two patients with severe genetic blood diseases. Until February of this year, when pharmaceutical companies CRISPR Therapeutics and Vertex began a large global trial into a treatment called CTX001, no human outside of China had been officially treated with a CRISPR-based gene editing therapy. CTX001 was developed to treat two types of inherited blood disease, beta-thalassemia and sickle cell disease. Both conditions are caused by a mutation in a single gene and the treatment involves engineering a patient's stem cells with a single genetic change designed to raise levels of fetal hemoglobin in red blood cells.
Promising preliminary data from one of the first human trials testing the safety and efficacy of a CRISPR gene therapy has just been revealed. Although it is too early to evaluate long-term effects, the initial reports are impressively successful for two patients with severe genetic blood diseases. Until February of this year, when pharmaceutical companies CRISPR Therapeutics and Vertex began a large global trial into a treatment called CTX001, no human outside of China had been officially treated with a CRISPR-based gene editing therapy. CTX001 was developed to treat two types of inherited blood disease, beta-thalassemia and sickle cell disease. Both conditions are caused by a mutation in a single gene and the treatment involves engineering a patient's stem cells with a single genetic change designed to raise levels of fetal hemoglobin in red blood cells.
FDA Official Urges Wealthy Nations to Align Gene Therapy Rules
Posted on: 5 November 2019, source: BloombergLaw
Patients from low- and middle-income countries will have better access to gene therapies if the U.S. and other high-income countries align their regulations for these potentially curative treatments, the FDA’s biologics chief said. “We really want to see the delivery of safe and effective gene therapies, not just in high income countries but across the globe,” Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration, said Nov. 4 at the American Society for Gene and Cell Therapy’s policy summit.
Patients from low- and middle-income countries will have better access to gene therapies if the U.S. and other high-income countries align their regulations for these potentially curative treatments, the FDA’s biologics chief said. “We really want to see the delivery of safe and effective gene therapies, not just in high income countries but across the globe,” Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration, said Nov. 4 at the American Society for Gene and Cell Therapy’s policy summit.
Early Report: Baby Treated With Gene Therapy For Deadly Tay-Sachs Disease Appears To Stabilize
Posted on: 24 October 2019, source: wbur
It's a very early report, from just two patients, only a few months after treatment. But UMass Medical School Dean Terence Flotte this week shared at a conference what could be landmark news about a terrible genetic disease: Two young patients with Tay-Sachs disease showed no ill effects from a new gene therapy that aims to correct the defect at the heart of the disease. One of them, treated at just 7 months, has appeared to stabilize instead of following the typical quick slide toward death by age 4. "It seems right now that she's not degenerating," Flotte said. "But I would say it's too early to say that definitively."
It's a very early report, from just two patients, only a few months after treatment. But UMass Medical School Dean Terence Flotte this week shared at a conference what could be landmark news about a terrible genetic disease: Two young patients with Tay-Sachs disease showed no ill effects from a new gene therapy that aims to correct the defect at the heart of the disease. One of them, treated at just 7 months, has appeared to stabilize instead of following the typical quick slide toward death by age 4. "It seems right now that she's not degenerating," Flotte said. "But I would say it's too early to say that definitively."
World’s First Gene Therapy for Glycogen Storage Disease Produces Remarkable Results
Posted on: 20 September 2019, source: UConn Today
At the Association for Glycogen Storage Disease’s 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children’s presented his groundbreaking, one-year clinical trial results for the novel gene therapy treatment for glycogen storage disease (GSD). The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels and constant dependence on glucose consumption in the form of cornstarch every few hours for survival. If a cornstarch dose is missed, the disease can lead to seizures and even death. Weinstein, whose team first administered the investigational gene therapy at UConn John Dempsey Hospital in Farmington, Connecticut, on July 24, 2018, calls the results “remarkable.”
At the Association for Glycogen Storage Disease’s 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children’s presented his groundbreaking, one-year clinical trial results for the novel gene therapy treatment for glycogen storage disease (GSD). The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels and constant dependence on glucose consumption in the form of cornstarch every few hours for survival. If a cornstarch dose is missed, the disease can lead to seizures and even death. Weinstein, whose team first administered the investigational gene therapy at UConn John Dempsey Hospital in Farmington, Connecticut, on July 24, 2018, calls the results “remarkable.”
Tiny capsules packed with gene-editing tools offer alternative to viral delivery of gene therapy
Posted on: 10 September 2019, source: University of Wisconsin
New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even stubborn viral infections. But the typical method for delivering gene therapies to specific tissues in the body can be complicated and may cause troubling side effects. Researchers at the University of Wisconsin–Madison have addressed many of those problems by packing a gene-editing payload into a tiny, customizable, synthetic nanocapsule. They described the delivery system and its cargo today (Sept. 9, 2019) in the journal Nature Nanotechnology.
New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even stubborn viral infections. But the typical method for delivering gene therapies to specific tissues in the body can be complicated and may cause troubling side effects. Researchers at the University of Wisconsin–Madison have addressed many of those problems by packing a gene-editing payload into a tiny, customizable, synthetic nanocapsule. They described the delivery system and its cargo today (Sept. 9, 2019) in the journal Nature Nanotechnology.
Gene Therapy Developers Issue Principles for Human Genome Editing
Posted on: 28 August 2019, source: raps.org
The Alliance for Regenerative Medicine’s (ARM) Gene Editing Task Force on Tuesday released a set of principles for human genome editing endorsed by thirteen of its members who are involved in the development of gene therapies or gene-editing technology. While the principles endorse somatic cell gene editing and the development of regulatory standards for gene editing, the document asserts that it is too early to support any form of human germline gene editing due to unanswered ethical, legal and safety questions. “As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles,” said ARM CEO Janet Lambert.
The Alliance for Regenerative Medicine’s (ARM) Gene Editing Task Force on Tuesday released a set of principles for human genome editing endorsed by thirteen of its members who are involved in the development of gene therapies or gene-editing technology. While the principles endorse somatic cell gene editing and the development of regulatory standards for gene editing, the document asserts that it is too early to support any form of human germline gene editing due to unanswered ethical, legal and safety questions. “As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles,” said ARM CEO Janet Lambert.
Novartis ousted top scientists over manipulation of data for gene therapy
Posted on: 14 August 2019, source: statnews.com
Novartis dismissed the top two scientists at its gene therapy division shortly after CEO Vas Narasimhan learned of internal data falsification that has since snowballed into a damaging scandal, a person familiar with the situation said Wednesday. The company previously said it was “in the process of exiting” scientists who were responsible for the scandal but did not identify them. In a statement on Wednesday, Novartis (NVS) said that it had appointed a new chief scientific officer for AveXis and that other scientists, Brian and Allan Kaspar, “have not been not been involved in any operations at AveXis since early May 2019,” without elaborating. The person familiar with the situation, who spoke on condition of anonymity, confirmed that the departure of the Kaspars, who are brothers, was connected to the disclosure of data manipulation related to the gene therapy Zolgensma.
Novartis dismissed the top two scientists at its gene therapy division shortly after CEO Vas Narasimhan learned of internal data falsification that has since snowballed into a damaging scandal, a person familiar with the situation said Wednesday. The company previously said it was “in the process of exiting” scientists who were responsible for the scandal but did not identify them. In a statement on Wednesday, Novartis (NVS) said that it had appointed a new chief scientific officer for AveXis and that other scientists, Brian and Allan Kaspar, “have not been not been involved in any operations at AveXis since early May 2019,” without elaborating. The person familiar with the situation, who spoke on condition of anonymity, confirmed that the departure of the Kaspars, who are brothers, was connected to the disclosure of data manipulation related to the gene therapy Zolgensma.
Gene Therapy News
- Gene therapy is facing its biggest challenge yet - Nature.com
Wed, 04 Dec 2019 10:05 GMT - Hopkins team invents non-viral system for getting gene therapy into cells - FierceBiotech
Fri, 06 Dec 2019 19:00 GMT - Bluebird Sickle Cell Gene Therapy Ends Painful Attacks for Some - Bloomberg
Sat, 07 Dec 2019 19:00 GMT - Buyer beware of this $1 million gene therapy for aging - MIT Technology Review
Fri, 06 Dec 2019 17:38 GMT - Amid Multi-Billion-Dollar Deals, Panels Highlight Gene Therapy Challenges - Genetic Engineering & Biotechnology News
Wed, 04 Dec 2019 14:40 GMT
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