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FDA grants orphan drug designation for LHON gene therapy

Posted on: 26 September 2020, source: FDA
Neurophth Therapeutics has received orphan drug designation from the FDA for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a press release. Between 70% and 90% of LHON cases are caused by ND4 mutation, according to the release.
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New HIV Gene Therapy, CAR-T Treatments Could be on the Horizon for Patients

Posted on: 8 September 2020, source: BioSpace
The National Institutes of Health (NIH) has backed researchers at the University of Southern California and the Fred Hutchison Cancer Center with a five-year, $14.6 million grant to develop a gene therapy that could potentially control HIV without the need for daily medications. Most HIV patients take a well-regimented cocktail of medications each day to control the virus. This therapy could change that.
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Study Confirms Efficacy of Gene Therapy for Spinal Muscular Atrophy in Children

Posted on: 30 August 2020, source: Technology Networks
In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with SMA under the age of two years; however, the gene therapy had only been studied in children aged up to 8 months. Now, a new study discusses safety and early outcomes in a large cohort of SMA patients under the age of two years who were treated with gene therapy. The report is published in the journal Pediatrics.
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FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL

Posted on: 4 August 2020, source: FDA
The U.S. Food and Drug Administration approved Tecartus (brexucabtagene autoleucel), a cell-based gene therapy for treatment of adult patients diagnosed with mantle cell lymphoma (MCL) who have not responded to or who have relapsed following other kinds of treatment. Tecartus, a chimeric antigen receptor (CAR) T cell therapy, is the first cell-based gene therapy approved by the FDA for the treatment of MCL. “Tremendous progress has been made in the discovery of new therapies for debilitating diseases that are difficult to treat. This approval is yet another example of customized treatments that use a patient’s own immune system to help fight cancer, while using a scientific advance in this promising new area of medicine,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “We’re seeing continued advances in the field of gene therapy and remain committed to supporting innovation in this promising new area of medicine.”
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Two deaths in gene therapy trial for rare muscle disease

Posted on: 30 June 2020, source: ScienceMag
Two boys have died after receiving high doses of a gene therapy treatment for their rare muscle disease, Biopharma Dive reports. The patients, born with x-linked myotubular myopathy, developed liver problems that apparently led to sepsis, according to a 23 June letter to patient groups from trial sponsor Audentes Therapeutics. They were older patients and had existing liver disease; several younger patients who got lower doses of the treatment have done well and now breathe on their own without a ventilator.
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Preclinical Study of SLS-004, Potential Parkinson’s Gene Therapy, Starting

Posted on: 3 June 2020, source: Parkinson News
Seelos Therapeutics announced it is beginning, earlier than expected, a preclinical study into its investigational gene therapy candidate for Parkinson’s disease called SLS-004. Nerve cell damage in Parkinson’s is caused by the buildup of toxic forms of the alpha-synuclein protein, and resulting clumps of misfolded proteins known as Lewy bodies. These toxic aggregates damage and eventually kill nerve cells — called dopaminergic neurons — in a region of the brain that regulates muscle movement and coordination.
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FDA Gives Fabry Gene Therapy, FLT190, Orphan Drug Status

Posted on: 13 May 2020, source:
The U.S. Food and Drug Administration (FDA) has designated FLT190, an investigational gene therapy for Fabry disease, an orphan drug to support and speed its development and possible review, Freeline Therapeutics, its developer, announced. The therapy is being evaluated in a Phase 1/2 trial, called MARVEL1 (NCT04040049), that may be enrolling up to 15 adult male patients at three sites in Europe.
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Gene therapy for color blindness passes first phase of human testing

Posted on: 4 May 2020, source: NewAtlas
The results of a first human trial testing a gene therapy for complete color blindness have been published in the journal JAMA Ophthalmology. The research suggests the experimental gene therapy is safe, and potentially efficacious, opening the door to larger human trials in the future.
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Wyatt Technology Pioneers Multi-CQA Method for Gene Vectors

Posted on: 13 April 2020, source: Wyatt
Wyatt Technology, the world leader in instrumentation for absolute macromolecular and nanoparticle characterization, announces the release of a novel method suitable for determining multiple critical quality attributes of adeno-associated virus and similar small gene vectors, in a single chromatographic run. The method is based on SEC-MALS, which combines size-exclusion chromatography for separation with multi-angle light scattering for characterization. SEC-MALS was introduced by Wyatt in 1985 and has been a mainstay of biophysical characterization of biotherapeutics such as monoclonal antibodies and virus-like particles. It utilizes Wyatt’s DAWN MALS instrument coupled to an industry-standard SEC system and offers full support for FDA 21CFR(11) compliance.
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New gene therapy product Zolgensma to treat spinal muscular atrophy

Posted on: 30 March 2020, source: EMA
The EMA has recommended granting a conditional marketing authorisation in the European Union for the gene therapy Zolgensma(onasemnogene abeparvovec)to treat babies and young children with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. There are currently limited treatment options for children with SMAin the EU. Patients also receive physical aids to support muscular functions and help them and their families cope with the symptoms of the disease.
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First patient undergoes Luxturna gene therapy on NHS

Posted on: 20 February 2020, source:
The NHS has reported treating its first patient with Novartis’ Luxturna (voretigene neparvovec) a “revolutionary” new gene therapy that can restore eyesight, as part of its NHS Long Term Plan. The therapy is for those born with an inherited retinal disorder - Leber’s Congenital Amaurosis (LCA) - who have poor sight which swiftly deteriorates, with many ultimately losing their vision completely in childhood.
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Conference cancellations and postponements

Posted on: 11 March 2020, source: Gene Therapy Net
Please note that due to the evolving COVID-19 situation, several conferences and meetings will be cancelled or postponed. Gene Therapy Net is continually monitoring COVID-19 updated guidelines from WHO and CDC. Check the individual conference websites regularly to be informed well. At this time, there is no plan to cancel or postpone ASGCT’s 23rd Annual Meeting in May, the ESGCT Spring School in April, or the ESGCT meeting in October. The following meetings have been postponed: NVGCT, DG-GT Symposium, SFTCG annual meeting, Advanced Therapies 2020.
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FDA Continues Strong Support of Innovation in Development of Gene Therapy Products

Posted on: 1 February 2020, source: FDA
This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices. In that spirit, today, the FDA is announcing the release of a number of important policies: six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.
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Dog Study Revives Concerns About Virus Used for Gene Therapy

Posted on: 7 January 2020, source: Science
Adeno-associated viruses are popular vectors for delivering gene therapies to patients’ cells because, researchers believed, the DNA these viruses carry rarely inserts into the host genome. But according to results presented last month (December 9) at the American Society of Hematology (ASH) meeting in Orlando, Florida, this assumption may be wrong. In six dogs that were treated with an AAV-based gene therapy for the blood-clotting disorder hemophilia B, all of them carried the therapeutic DNA within their genomes, and in some cases, the genetic material had inserted near genes known to play a role in cell growth, Science reports.
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NJIT to Launch New Programs in Cell and Gene Therapy

Posted on: 23 December 2019, source: NJIT
New Jersey Institute of Technology (NJIT) in collaboration with New Jersey Innovation Institute (NJII), an NJIT corporation, has announced it will soon offer both a Professional Science Masters (PSM) degree program and professional graduate certificate in the rapidly expanding field of cell and gene therapy. The 30-credit M.Sc. degree — available in spring 2020 as part of NJIT’s Department of Chemistry and Environmental Science’s pharmaceutical chemistry master's program — aims to "train the next generation of scientists and engineers in modern aspects of cell and gene therapy, emphasizing cell biology and cellular therapeutic development, regenerative medicine, process development and biomanufacturing, analytical technologies, quality and regulatory compliance.”
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Hopkins team invents non-viral system for getting gene therapy into cells

Posted on: 12 December 2019, source: FierceBiotech
One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune responses. A team from Johns Hopkins Medicine is proposing an alternative method for transporting large therapies into cells—including genes and even the gene-editing system CRISPR. It’s a nano-container made of a polymer that biodegrades once it’s inside the cell, unleashing the therapy. The researchers described the invention in the journal Science Advances.
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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.
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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.
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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."
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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.”
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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.
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Gene Therapy Developers Issue Principles for Human Genome Editing

Posted on: 28 August 2019, source:
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.
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Novartis ousted top scientists over manipulation of data for gene therapy

Posted on: 14 August 2019, source:
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.
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Gene Therapy Making Waves for Lung Cancer Patients

Posted on: 23 July 2019, source:
The development of lung cancer treatment have come a long way within the last decade. The progression of gene therapy through clinical trials is contributing to millions of lives extended as well as more patients going into remission. The success gene therapy is playing towards these common cancers are making waves in the rare space as well. An uncommon cancer affecting the lungs- mesothelioma, will hopefully start to leverage these types of treatments in the near future.
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The Prickly Debate on Germline Gene Therapy, and Moving It Forward

Posted on: 26 June 2019, source: SingularityHub
In 2016, a healthy baby boy came screaming into the world in a Mexican clinic. Harboring DNA from three parents, the baby had had his genes dramatically altered while still an embryo. Without the treatment, an inheritable neurological disorder would’ve killed him before the age of three. Two years later across the world in China, two girls, also as embryos, had their genes edited using CRISPR to make them (theoretically) resistant to HIV infection. The edits didn’t hit their intended target—instead, the girls may be more prone to infections, have altered brain functions, and even experience earlier deaths.
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Avexis Receives FDA Approval For Zolgensma, First And Only Gene Therapy For Pediatric Patients With Spinal Muscular Atrophy

Posted on: 25 May 2019, source: Novartis
AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.
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NIH Streamlines Gene Therapy Oversight and Charts a Course for Considering Emerging Technology

Posted on: 26 April 2019, source: NIH
In August 2018, the National Institutes of Health and the Food and Drug Administration (FDA) announced a joint proposal to streamline duplicative and burdensome oversight over gene therapy. As part of this effort and following public input, NIH today released the amended NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules. The amendments remove the requirement to register and report on human gene therapy protocols under the NIH Guidelines to the NIH. Robust oversight continues under the FDA, which has regulatory oversight of all human gene therapy clinical trials. In addition, NIH-funded human gene therapy research remains subject to the usual NIH oversight that applies to all NIH-funded research, and oversight by local authorities such as Institutional Review Boards and Institutional Biosafety Committees.
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Novartis Says Baby's Death May Be Tied to Gene Therapy

Posted on: 24 April 2019, source: Bloomberg
Novartis AG said the death of a six-month-old baby in a study evaluating its experimental gene therapy Zolgensma was considered by the clinical trial investigator to be potentially related to the treatment. The death of the patient with a devastating disease known as spinal muscular atrophy occurred in a late-stage trial in Europe that’s still enrolling patients, the Swiss drugmaker said in an emailed statement. Initial findings show it involved a severe respiratory infection followed by neurological complications. The results of an autopsy are pending, and details have been reported to regulators, the company said.
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First gene therapy to treat rare blood disease nears European approval

Posted on: 2 April 2019, source: STATnews
The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months.
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Should gene therapy patients be asked to pay?

Posted on: 28 March 2019, source:
The price in value-based payment deals should by definition be justified, so why is patient cost-sharing part of the calculation? Spark covers this cost, but there is no guarantee that all gene therapy companies will. As Novartis’s gene therapy Zolgensma nears a US FDA decision, the sector has only a vague idea of how much the Swiss group will charge for the spinal muscular atrophy (SMA) treatment, other than it will be a number followed by six zeroes. Just as importantly, the families with children suffering from this fatal condition do not know what, if anything, they will be asked to pay. However, there are some clues from the way in which the first US-approved gene therapy, Spark Therapeutics’ Luxturna, was financed.
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