New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases
Posted on: 13 September 2021, source: Broad Institute of MIT and Harvard
Genetic muscle diseases lead to progressive muscle wasting and often early death, with few treatment options and no cure. Some gene therapies that use a harmless virus to deliver a functioning copy of a disease-causing gene to cells have shown promise in clinical trials for a subset of muscular dystrophies, but have faced challenges. High doses of the gene-carrying virus are needed to reach the muscles throughout the body and the viruses used in these trials often end up in the liver more than in the muscle. This has led to high levels of the virus in the liver, severe adverse side effects, and even death in some trial participants. Researchers at the Broad Institute of MIT and Harvard and Harvard University have developed a new family of adeno-associated viruses (AAVs) - the gene-delivering workhorse of gene therapy - that improve targeting of the muscle tissue, which could be safer and more effective for patients with muscle disease.
Genetic muscle diseases lead to progressive muscle wasting and often early death, with few treatment options and no cure. Some gene therapies that use a harmless virus to deliver a functioning copy of a disease-causing gene to cells have shown promise in clinical trials for a subset of muscular dystrophies, but have faced challenges. High doses of the gene-carrying virus are needed to reach the muscles throughout the body and the viruses used in these trials often end up in the liver more than in the muscle. This has led to high levels of the virus in the liver, severe adverse side effects, and even death in some trial participants. Researchers at the Broad Institute of MIT and Harvard and Harvard University have developed a new family of adeno-associated viruses (AAVs) - the gene-delivering workhorse of gene therapy - that improve targeting of the muscle tissue, which could be safer and more effective for patients with muscle disease.
Lysogene Announces First Patient in the United States Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis
Posted on: 30 August 2021, source: BioPharma Dive
Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced dosing of the first patient in the United States with LYS-GM101 investigational gene therapy at CHOC Hospital (CHOC) in a global adaptative-design clinical trial (NCT04273269) for the treatment of GM1 gangliosidosis. This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene (GBL1). The clinical trial includes a safety phase and a confirmatory efficacy phase. The trial will enroll 16 patients with a diagnosis of early or late infantile GM1 gangliosidosis at sites in the US and Europe.
Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced dosing of the first patient in the United States with LYS-GM101 investigational gene therapy at CHOC Hospital (CHOC) in a global adaptative-design clinical trial (NCT04273269) for the treatment of GM1 gangliosidosis. This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene (GBL1). The clinical trial includes a safety phase and a confirmatory efficacy phase. The trial will enroll 16 patients with a diagnosis of early or late infantile GM1 gangliosidosis at sites in the US and Europe.
FDA allows Novartis gene therapy trials to resume after nearly 2 year pause
Posted on: 10 August 2021, source: BioPharma Dive
Novartis on Tuesday said it will begin a new study of its gene therapy Zolgensma in older patients with spinal muscular atrophy following the Food and Drug Administration's clearance of administration via a spinal infusion. The regulator had suspended testing of that dosing in October 2019 due to safety concerns.
Novartis on Tuesday said it will begin a new study of its gene therapy Zolgensma in older patients with spinal muscular atrophy following the Food and Drug Administration's clearance of administration via a spinal infusion. The regulator had suspended testing of that dosing in October 2019 due to safety concerns.
Gene therapy delivered to the brain shows promise in children with rare neurodegenerative disease
Posted on: 16 July 2021, source: FierceBiotech
Scientists from Ohio State University have developed a novel method for delivering gene therapy to specific regions of the brain. Now, they have evidence from a small clinical trial in children that the treatment could address a rare, inherited neurodegenerative disease. And they believe their technique could eventually be used to treat more common brain diseases, like Alzheimer’s and Parkinson’s. The Ohio State team developed the gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, which hampers the body’s ability to make dopamine and serotonin and results in developmental delays and a range of motor and behavioral symptoms. The gene therapy uses a viral vector to carry DNA-expressing AADC to the brain.
Scientists from Ohio State University have developed a novel method for delivering gene therapy to specific regions of the brain. Now, they have evidence from a small clinical trial in children that the treatment could address a rare, inherited neurodegenerative disease. And they believe their technique could eventually be used to treat more common brain diseases, like Alzheimer’s and Parkinson’s. The Ohio State team developed the gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, which hampers the body’s ability to make dopamine and serotonin and results in developmental delays and a range of motor and behavioral symptoms. The gene therapy uses a viral vector to carry DNA-expressing AADC to the brain.
A year after getting UniQure's gene therapy, hemophilia patients are still doing better
Posted on: 23 June 2021, source: BioPharma Dive
One year after receiving an experimental gene therapy developed by the Dutch drugmaker UniQure, patients with hemophilia B aren't having nearly as many bleeding issues as they used to have. In hemophilia, genetic mutations prevent the body from making proteins needed to clot blood. People with the less common, "B" form of the disorder are missing a protein known as Factor IX. UniQure's medicine is meant to provide a working version of the gene so patients can generate their own clotting protein, and rely less — or ideally, not at all — on so-called replacement factor treatments.
One year after receiving an experimental gene therapy developed by the Dutch drugmaker UniQure, patients with hemophilia B aren't having nearly as many bleeding issues as they used to have. In hemophilia, genetic mutations prevent the body from making proteins needed to clot blood. People with the less common, "B" form of the disorder are missing a protein known as Factor IX. UniQure's medicine is meant to provide a working version of the gene so patients can generate their own clotting protein, and rely less — or ideally, not at all — on so-called replacement factor treatments.
Decades after a tragic failure, gene therapy successfully treats a rare liver disease
Posted on: 19 May 2021, source: Science
Twenty-two years ago, one of scientists’ first attempts at gene therapy ended in tragedy when a young man died. The story of Jesse Gelsinger, who had a rare liver disorder, became a textbook example of irresponsible medical research. For years, the case hobbled efforts to treat diseases by adding new DNA to a sick person’s cells. Now, a fresh effort to cure Gelsinger’s disease is bearing fruit, in the latest sign of the field’s resurgence.
Twenty-two years ago, one of scientists’ first attempts at gene therapy ended in tragedy when a young man died. The story of Jesse Gelsinger, who had a rare liver disorder, became a textbook example of irresponsible medical research. For years, the case hobbled efforts to treat diseases by adding new DNA to a sick person’s cells. Now, a fresh effort to cure Gelsinger’s disease is bearing fruit, in the latest sign of the field’s resurgence.
New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy
Posted on: 4 May 2021, source: News-Medical.net
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.
New approach to CAR T gene therapy yields efficient and longer-lasting response against HIV
Posted on: 5 April 2021, source: News-Medical.net
A UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule. This new approach to CAR T gene therapy -- a type of immunotherapy that involves genetically engineering the body's own blood-forming stem cells to create HIV-fighting T cells -- has the potential to not only destroy HIV-infected cells but to create "memory cells" that could provide lifelong protection from infection with the virus that causes AIDS.
A UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule. This new approach to CAR T gene therapy -- a type of immunotherapy that involves genetically engineering the body's own blood-forming stem cells to create HIV-fighting T cells -- has the potential to not only destroy HIV-infected cells but to create "memory cells" that could provide lifelong protection from infection with the virus that causes AIDS.
EMA Launches Safety Review After Gene Therapy Trial Patient Develops Blood Cancer
Posted on: 13 March 2021, source: BioSpace
The European Medicines Agency (EMA) has launched a safety review of bluebird bio’s thalassaemia drug Zynteglo, a conditionally licensed gene therapy in Europe. This review comes after researchers identified an acute myeloid leukaemia (AML) case in a patient who received bb1111 (LentiGlobin), an investigational gene therapy for sickle cell disease (SCD), in a Phase I/II study. This therapy relies on the same viral vector found in Zynteglo which delivers a gene into cells.
The European Medicines Agency (EMA) has launched a safety review of bluebird bio’s thalassaemia drug Zynteglo, a conditionally licensed gene therapy in Europe. This review comes after researchers identified an acute myeloid leukaemia (AML) case in a patient who received bb1111 (LentiGlobin), an investigational gene therapy for sickle cell disease (SCD), in a Phase I/II study. This therapy relies on the same viral vector found in Zynteglo which delivers a gene into cells.
Gene therapy trials for sickle cell disease halted after two patients develop cancer
Posted on: 22 February 2021, source: Science
A company has stopped its clinical studies of a promising gene therapy for the blood disorder sickle cell disease after two people who participated developed leukemia-like cancer. Bluebird bio is now investigating whether a virus it uses to deliver a therapeutic gene caused the cancers, reviving old concerns about the risks of this approach. It’s also possible the cancers stemmed from chemotherapy the patients received to prepare their bodies for the gene’s delivery. “This is really a sad development whatever the cause,” says Donald Kohn of the University of California, Los Angeles, who has led gene therapy trials for sickle cell and other diseases.
A company has stopped its clinical studies of a promising gene therapy for the blood disorder sickle cell disease after two people who participated developed leukemia-like cancer. Bluebird bio is now investigating whether a virus it uses to deliver a therapeutic gene caused the cancers, reviving old concerns about the risks of this approach. It’s also possible the cancers stemmed from chemotherapy the patients received to prepare their bodies for the gene’s delivery. “This is really a sad development whatever the cause,” says Donald Kohn of the University of California, Los Angeles, who has led gene therapy trials for sickle cell and other diseases.
Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During COVID-19 Public Health Emergency
Posted on: 24 January 2021, source: FDA
FDA is issuing this guidance to provide manufacturers of licensed and investigational cellular therapy and gene therapy (CGT) products with risk-based recommendations to minimize potential transmission of the novel coronavirus, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). This guidance is intended to supplement the recommendations to drug and biological product manufacturers provided in FDA’s “Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing; Guidance for Industry” issued in June 2020 (Ref. 1) (June 2020 GMP Guidance). The recommendations in this guidance specifically consider the source material (cells and/or tissues) recovered from donors and how the CGT product will be manufactured (e.g., cell expansion in culture, viral reduction steps, formulation).
FDA is issuing this guidance to provide manufacturers of licensed and investigational cellular therapy and gene therapy (CGT) products with risk-based recommendations to minimize potential transmission of the novel coronavirus, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). This guidance is intended to supplement the recommendations to drug and biological product manufacturers provided in FDA’s “Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing; Guidance for Industry” issued in June 2020 (Ref. 1) (June 2020 GMP Guidance). The recommendations in this guidance specifically consider the source material (cells and/or tissues) recovered from donors and how the CGT product will be manufactured (e.g., cell expansion in culture, viral reduction steps, formulation).
Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment
Posted on: 23 December 2020, source: Science
It’s troubling news that gene therapy researchers have long anticipated: A hemophilia patient injected with a virus carrying a therapeutic gene in a clinical trial has developed a liver tumor. The U.S. Food and Drug Administration (FDA) has halted the associated clinical trials, and uniQure, the Dutch firm behind the studies, is now investigating whether the virus itself caused the cancer. Gene therapy experts say that’s unlikely. The patient had underlying conditions that predisposed him to liver cancer. Still, scientists say it’s crucial to rule out any role for adeno-associated virus (AAV), the viral delivery system, or vector, that is used in hundreds of other gene therapy trials. “Everyone will want to know what happened,” says physician-scientist David Lillicrap of Queen’s University, a hemophilia researcher who was not involved with the uniQure study.
It’s troubling news that gene therapy researchers have long anticipated: A hemophilia patient injected with a virus carrying a therapeutic gene in a clinical trial has developed a liver tumor. The U.S. Food and Drug Administration (FDA) has halted the associated clinical trials, and uniQure, the Dutch firm behind the studies, is now investigating whether the virus itself caused the cancer. Gene therapy experts say that’s unlikely. The patient had underlying conditions that predisposed him to liver cancer. Still, scientists say it’s crucial to rule out any role for adeno-associated virus (AAV), the viral delivery system, or vector, that is used in hundreds of other gene therapy trials. “Everyone will want to know what happened,” says physician-scientist David Lillicrap of Queen’s University, a hemophilia researcher who was not involved with the uniQure study.
Gene Therapy in One Eye Improves Vision in Both Eyes
Posted on: 12 December 2020, source: The Scientist
It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye. In a Phase 3 gene therapy trial intended to improve vision among patients with Leber hereditary optic neuropathy, recipients gained somewhat better sight in both eyes even though only one was treated. The results and an investigation into possible explanations for the findings were published December 9 in Science Translational Medicine.
It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye. In a Phase 3 gene therapy trial intended to improve vision among patients with Leber hereditary optic neuropathy, recipients gained somewhat better sight in both eyes even though only one was treated. The results and an investigation into possible explanations for the findings were published December 9 in Science Translational Medicine.
NIH project aims to maximize efficiency of AAV platform for gene therapy
Posted on: 19 November 2020, source: News-Medical.Net
Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH). The NIH Platform Vector Gene Therapy (PaVe-GT) project is reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free through December 16, 2020. "PaVe-GT is an experimental translational science initiative that aims to leverage the power of platform vectors and disease relatedness to help deliver on the promise of gene therapy to patients with rare diseases," states the NIH PaVe-GT team.
Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH). The NIH Platform Vector Gene Therapy (PaVe-GT) project is reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free through December 16, 2020. "PaVe-GT is an experimental translational science initiative that aims to leverage the power of platform vectors and disease relatedness to help deliver on the promise of gene therapy to patients with rare diseases," states the NIH PaVe-GT team.
COVID-19, manufacturing challenges limit cell and gene therapy progress, FDA official says
Posted on: 30 October 2020, source: FDA/Healio.com
Progress in the field of gene and cell therapy continues with rapid development despite the negative impact of COVID-19 on research into novel treatments, according to the director of FDA’s Center for Biologics Evaluation and Research. Peter Marks, MD, PhD, delivered this assessment during the virtual Cell & Gene Meeting on the Mesa.
Progress in the field of gene and cell therapy continues with rapid development despite the negative impact of COVID-19 on research into novel treatments, according to the director of FDA’s Center for Biologics Evaluation and Research. Peter Marks, MD, PhD, delivered this assessment during the virtual Cell & Gene Meeting on the Mesa.
Gene Therapy in Mesothelioma Patients
Posted on: 28 September 2020, source: mesothelioma.com
Around 1 in 16 people will be diagnosed with lung cancer in their lifetime; this accounts for about 13% of all new cancer diagnoses. However, even with a relatively lower diagnosis rate, lung cancer currently has the highest number of cancer deaths at nearly 22%. Among all the various subtypes of cancer categorized under the umbrella term, “lung cancer,” mesothelioma is one of the most lethal forms.
Despite accounting for around .2% of all cancer diagnoses in the United States, mesothelioma’s lethality is among the highest of all forms of cancer. Pleural mesothelioma, the most common form of this cancer, has a 10-year survival rate of only about 5%. Currently, the only known cause of mesothelioma is asbestos exposure, and although no amount of exposure is safe, it is important to note that your risk of developing mesothelioma drastically increases the longer you are exposed. In addition, due to a prolonged latency period, many patients are diagnosed far too late for proper treatment — usually around the age of 65-74.
Around 1 in 16 people will be diagnosed with lung cancer in their lifetime; this accounts for about 13% of all new cancer diagnoses. However, even with a relatively lower diagnosis rate, lung cancer currently has the highest number of cancer deaths at nearly 22%. Among all the various subtypes of cancer categorized under the umbrella term, “lung cancer,” mesothelioma is one of the most lethal forms.
Despite accounting for around .2% of all cancer diagnoses in the United States, mesothelioma’s lethality is among the highest of all forms of cancer. Pleural mesothelioma, the most common form of this cancer, has a 10-year survival rate of only about 5%. Currently, the only known cause of mesothelioma is asbestos exposure, and although no amount of exposure is safe, it is important to note that your risk of developing mesothelioma drastically increases the longer you are exposed. In addition, due to a prolonged latency period, many patients are diagnosed far too late for proper treatment — usually around the age of 65-74.
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.
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.
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.
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.
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.
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.
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.”
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.”
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.
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.
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.
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.
FDA Gives Fabry Gene Therapy, FLT190, Orphan Drug Status
Posted on: 13 May 2020, source: fabrydiseasenews.com
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.
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.
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.
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.
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.
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.
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.
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.
First patient undergoes Luxturna gene therapy on NHS
Posted on: 20 February 2020, source: pharmatimes.com
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.
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.
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.
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.
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.
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.
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.
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.