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.
Chinese Researchers Claim to Have Genetically Engineered the First HIV-Immune Babies
Posted on: 28 November 2018, source: fortune.com
Researchers in China (Clinical project 'Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos') used genetic engineering tools (CRISPR) to create twins theoretically immune to HIV, smallpox, and cholera, MIT Technology Review reported. The medical breakthrough is controversial, as many worry about eugenics and designer babies for the wealthy. The twins, named Lulu and Nana, according to lead scientist He Jiankui of Shenzhen in a YouTube video, were the result of in vitro fertilization (IVF). A few weeks old, they appear to be healthy. When they were a single cell, genetic surgery using a popular tool, CRISPR, “removed the doorway through which HIV enters to infect people.”
Researchers in China (Clinical project 'Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos') used genetic engineering tools (CRISPR) to create twins theoretically immune to HIV, smallpox, and cholera, MIT Technology Review reported. The medical breakthrough is controversial, as many worry about eugenics and designer babies for the wealthy. The twins, named Lulu and Nana, according to lead scientist He Jiankui of Shenzhen in a YouTube video, were the result of in vitro fertilization (IVF). A few weeks old, they appear to be healthy. When they were a single cell, genetic surgery using a popular tool, CRISPR, “removed the doorway through which HIV enters to infect people.”
Watch the presentation of He Jiankui and discussion during Second International Summit on Human Genome Editing in Hongkong, Wednesday November 28th, 2018.
Spark Therapeutics' Luxturna Gets EU Approval for Vision Loss
Posted on: 26 November 2018, source: Nasdaq
Spark Therapeutics, Inc. announced that the European Commission has approved the marketing authorization application ("MAA") for its gene therapy, Luxturna (voretigene neparvovec). The MAA sought approval for the therapy in adult as well as pediatric patients as a one-time treatment for vision loss due to inherited retinal dystrophy caused by a genetic mutation in both copies of the RPE65-gene and who have enough viable retinal cells. The RPE65 mutations, a progressive disease, can lead to total blindness.
Spark Therapeutics, Inc. announced that the European Commission has approved the marketing authorization application ("MAA") for its gene therapy, Luxturna (voretigene neparvovec). The MAA sought approval for the therapy in adult as well as pediatric patients as a one-time treatment for vision loss due to inherited retinal dystrophy caused by a genetic mutation in both copies of the RPE65-gene and who have enough viable retinal cells. The RPE65 mutations, a progressive disease, can lead to total blindness.
Gene therapy drugs step toward approval in Japan
Posted on: 14 November 2018, source: Nikkei Asia
Novartis has applied with Japanese authorities to have a gene therapy drug approved, a move that could give Japan at least two such drugs in the coming year, Nikkei has learned. The Switzerland-based pharmaceutical hopes to receive the all-clear in Japan for a neurological disorder treatment sometime next year. It has also applied for approval for the same drug in the U.S. and Europe. In the U.S., the drug is said to be prohibitively expensive -- as much as $4 million to $5 million per patient. The drug treats spinal muscular atrophy, and Novartis expects to be able to market the treatment as early as next year.
Novartis has applied with Japanese authorities to have a gene therapy drug approved, a move that could give Japan at least two such drugs in the coming year, Nikkei has learned. The Switzerland-based pharmaceutical hopes to receive the all-clear in Japan for a neurological disorder treatment sometime next year. It has also applied for approval for the same drug in the U.S. and Europe. In the U.S., the drug is said to be prohibitively expensive -- as much as $4 million to $5 million per patient. The drug treats spinal muscular atrophy, and Novartis expects to be able to market the treatment as early as next year.
Infectivity of adeno-associated virus serotypes in mouse testis
Posted on: 7 November 2018, source: BMC Biotechnology
In a recent study, researchers from the Institute for Stem Cell Biology and Regenerative Medicine (InStem), Bengaluru, National Centre for Biological Sciences (NCBS), Bengaluru, Christian Medical College, Vellore and the Indian Institute of Technology, Kanpur, have described how a type of virus, called adeno-associated virus, behaves when injected into the mouse testis. The findings of the study, supported by the Department of Biotechnology (DBT) and InStem were published in the journal BMC Biotechnology.
In a recent study, researchers from the Institute for Stem Cell Biology and Regenerative Medicine (InStem), Bengaluru, National Centre for Biological Sciences (NCBS), Bengaluru, Christian Medical College, Vellore and the Indian Institute of Technology, Kanpur, have described how a type of virus, called adeno-associated virus, behaves when injected into the mouse testis. The findings of the study, supported by the Department of Biotechnology (DBT) and InStem were published in the journal BMC Biotechnology.
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.
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.
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.
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.
Gene Therapy News
- Orchard's Gene Therapy for Rare Immune Disease Works for Over a Year - Labiotech.eu
Fri, 07 Dec 2018 16:27 GMT - Early clinical trial data show gene therapy reversing sickle cell anemia: Treatment uses reduced-intensity preconditioning to enhance its global transportability - Science Daily
Tue, 04 Dec 2018 02:26 GMT - Exclusive: Orchard CEO Touts Transformative Power of Gene Therapy Following ASH Data Presentation - BioSpace
Thu, 06 Dec 2018 20:15 GMT - XyloCor closes $17M Series A round to develop gene therapy for refractory angina - MedCity News
Thu, 06 Dec 2018 18:09 GMT - Parkinson's Gene Therapy Wires New Brain Circuits - WebMD
Wed, 28 Nov 2018 08:00 GMT
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