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. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.
Gene therapy to cure leukemia at one-tenth global costs in the works
Posted on: 13 June 2022, source: Business Standard
ImmunoACT, an IIT-Bombay spin-off backed by Hyderabad-based Laurus Labs, is testing a new gene-therapy treatment for cancer, which they claim would not only cure the disease, but also costs one-tenth of the global price. “We are working with CD19 CAR T Cell for leukemia and lymphoma. What we do is we take out some blood and plasma from the cancer patient, and then isolate the T-cells (which are immune cells) from this sample. These cells are then genetically modified so that they eliminate cancer cells. Then this is transfused back to the patient,” explains Rahul Purwar, founder and chairman of ImmunoACT. The product is named H-CAR T-19.
ImmunoACT, an IIT-Bombay spin-off backed by Hyderabad-based Laurus Labs, is testing a new gene-therapy treatment for cancer, which they claim would not only cure the disease, but also costs one-tenth of the global price. “We are working with CD19 CAR T Cell for leukemia and lymphoma. What we do is we take out some blood and plasma from the cancer patient, and then isolate the T-cells (which are immune cells) from this sample. These cells are then genetically modified so that they eliminate cancer cells. Then this is transfused back to the patient,” explains Rahul Purwar, founder and chairman of ImmunoACT. The product is named H-CAR T-19.
Gene Therapy Successfully Treats Spinal Cord Injuries Without Side Effects
Posted on: 31 May 2022, source: SciTechDaily
In mouse studies, pain-blocking neurotransmitters produced long-lasting benefits without detectable side effects. An international team of researchers led by scientists at the University of California San Diego School of Medicine reported that a gene therapy that inhibits targeted nerve cell signaling effectively reduced neuropathic pain in mice with spinal cord or peripheral nerve injuries with no detectable side effects.
In mouse studies, pain-blocking neurotransmitters produced long-lasting benefits without detectable side effects. An international team of researchers led by scientists at the University of California San Diego School of Medicine reported that a gene therapy that inhibits targeted nerve cell signaling effectively reduced neuropathic pain in mice with spinal cord or peripheral nerve injuries with no detectable side effects.
Updates on gene therapy for ‘bubble boy’ disease and cellular immunotherapy at ASGCT
Posted on: 15 May 2022, source: ASGCT
St. Jude Children’s Research Hospital scientists will discuss research on cellular therapies for pediatric cancer and more at the annual gathering of the professional society for gene and cellular therapy researchers.
St. Jude Children’s Research Hospital scientists will discuss research on cellular therapies for pediatric cancer and more at the annual gathering of the professional society for gene and cellular therapy researchers.
AAV Durability: The Road to a One-Shot Cure
Posted on: 15 April 2022, source: Hansonwade
Inconsistent long-term clinical data has highlighted the need to better understand the durability of AAV gene therapy. At Hanson Wade Intelligence, our Market Research division, we have classified 121 AAV gene therapy trials into 4 distinct groups based on the target tissue type – liver, muscle, CNS and ocular tissues. This allowed us to summarise available data on clinical durability as well as the key findings we observed across tissues, trials, doses and serotypes. We hope that this report will provide gene therapy developers with valuable insights on the durability of the current generation of AAV gene therapies.
Inconsistent long-term clinical data has highlighted the need to better understand the durability of AAV gene therapy. At Hanson Wade Intelligence, our Market Research division, we have classified 121 AAV gene therapy trials into 4 distinct groups based on the target tissue type – liver, muscle, CNS and ocular tissues. This allowed us to summarise available data on clinical durability as well as the key findings we observed across tissues, trials, doses and serotypes. We hope that this report will provide gene therapy developers with valuable insights on the durability of the current generation of AAV gene therapies.
Gene Therapy Sector Needs Better Cell Lines for Vector Production
Posted on: 4 April 2022, source: Genengnews
Gene therapy developers targeting diseases requiring high dose therapies face a challenge. Current vector production cell lines and genetic manipulation techniques are too costly for large-scale manufacturing, according to new research. The study, by scientists at Portugal’s Instituto de Biologia Experimental e Tecnológica (iBET), looked at areas of gene therapy production where innovation is needed.
Gene therapy developers targeting diseases requiring high dose therapies face a challenge. Current vector production cell lines and genetic manipulation techniques are too costly for large-scale manufacturing, according to new research. The study, by scientists at Portugal’s Instituto de Biologia Experimental e Tecnológica (iBET), looked at areas of gene therapy production where innovation is needed.
Gene Therapy Turns to the Use of Synthetic Biology Principles
Posted on: 4 March 2022, source: Genengnews
A new university spinout company is hoping to use synthetic biology to transform gene therapy manufacture. SynGenSys, from the University of Sheffield, plans to tap into engineering design principles to create synthetic promoters to target therapeutic genes to the correct patient tissues. “Typically, people use viral promoters, such as cytomegalovirus as their go-to promoter, but it’s a really complex piece of DNA active in many human tissues,” says David C. James, PhD, professor of bioprocess engineering and co-founder of SynGenSys.
A new university spinout company is hoping to use synthetic biology to transform gene therapy manufacture. SynGenSys, from the University of Sheffield, plans to tap into engineering design principles to create synthetic promoters to target therapeutic genes to the correct patient tissues. “Typically, people use viral promoters, such as cytomegalovirus as their go-to promoter, but it’s a really complex piece of DNA active in many human tissues,” says David C. James, PhD, professor of bioprocess engineering and co-founder of SynGenSys.
Lentiviral vector-based gene therapy demonstrates long-term safety and efficacy for Wiskott-Aldrich Syndrome
Posted on: 11 February 2022, source: MedicalXpress
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.
Gene Therapy News
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Sarepta Therapeutics' Investigational Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy Demonstrates Significant Functional Improvements Across Multiple Studies - GlobeNewswire
Jul 6, 2022 | 14:15 pm
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Updates from uniQure about their gene therapy for Huntington's disease - HDBuzz
Jul 6, 2022 | 01:28 am
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New gene therapy improvement has massive potential - Drug Target Review
Jun 20, 2022 | 09:00 am
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New way forward for gene therapy - Medical Xpress
Jun 15, 2022 | 09:00 am
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Drug Target Review Cell & Gene Therapy ebook - Drug Target Review
Jun 15, 2022 | 09:00 am
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