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.”
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.”
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.
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.
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 Making Waves for Lung Cancer Patients
Posted on: 23 July 2019, source: mesothelioma.com
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Should gene therapy patients be asked to pay?
Posted on: 28 March 2019, source: evaluate.com
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.
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.
New Gene Therapy Shows Promise For Patients With Sickle Cell Disease
Posted on: 9 March 2019, source: CBS News
An experimental and innovative type of gene therapy is being used to attempt to cure sickle cell anemia. The director of the National Institutes of Health says the results of an NIH clinical trial on a gene therapy for sickle cell anemia "looks like a cure" for the painful and often deadly genetic disease that affects 100,000 Americans, with nearly all being African Americans. 60 Minutes follows the trial for more than a year, focusing on a Florida woman who now appears to be rid of sickle cell anemia. Dr. Jon LaPook's report will be broadcast on 60 Minutes, Sunday, March 10 at 7:00 p.m. ET/PT on CBS.
An experimental and innovative type of gene therapy is being used to attempt to cure sickle cell anemia. The director of the National Institutes of Health says the results of an NIH clinical trial on a gene therapy for sickle cell anemia "looks like a cure" for the painful and often deadly genetic disease that affects 100,000 Americans, with nearly all being African Americans. 60 Minutes follows the trial for more than a year, focusing on a Florida woman who now appears to be rid of sickle cell anemia. Dr. Jon LaPook's report will be broadcast on 60 Minutes, Sunday, March 10 at 7:00 p.m. ET/PT on CBS.
Gene Therapy Drug Approval to Skyrocket by 2025
Posted on: 16 January 2019, source: FDA
The FDA expects to approve 10 to 20 cell and gene therapy products a year within the next six years, and is hiring 50 more clinical reviewers to make it happen, the agency announced Jan. 15: "The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced by a large upswing in the number of investigational new drug (IND) applications. Based on this activity, we anticipate that the number of product approvals for cell and gene therapies will grow in the coming years, reflecting significant scientific advancement and the clinical promise of these new innovations."
The FDA expects to approve 10 to 20 cell and gene therapy products a year within the next six years, and is hiring 50 more clinical reviewers to make it happen, the agency announced Jan. 15: "The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced by a large upswing in the number of investigational new drug (IND) applications. Based on this activity, we anticipate that the number of product approvals for cell and gene therapies will grow in the coming years, reflecting significant scientific advancement and the clinical promise of these new innovations."
Gene therapy for Crigler-Najjar syndrome - First patient treated in the European clinical trial
Posted on: 25 December 2018, source: Eurekalerts
Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon, the AFM-Telethon laboratory, is sponsoring this multicenter international phase I/II trial, injecting intravenously an AAV vector that is able to transfer the UGT1A1 gene (coding for the production of bilirubin GT) into liver cells. A total of 17 patients will be treated over the next few months.
Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon, the AFM-Telethon laboratory, is sponsoring this multicenter international phase I/II trial, injecting intravenously an AAV vector that is able to transfer the UGT1A1 gene (coding for the production of bilirubin GT) into liver cells. A total of 17 patients will be treated over the next few months.
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.
Health Officials Remove Special NIH Oversite Rules Governing Gene Therapy Experiments
Posted on: 20 August 2018, source: BioSpace
As gene therapies have become reality, the U.S. government is removing some special regulations that had been set up long ago over concerns of exotic safety risks. The Associated Press reported that a National Institutes of Health oversight panel will no longer be called upon to review all gene therapy applications. That panel will now take a broader advisory role, the AP said. The U.S. Food and Drug Administration (FDA) will now look at gene therapy treatments as it down all other types of medications.
As gene therapies have become reality, the U.S. government is removing some special regulations that had been set up long ago over concerns of exotic safety risks. The Associated Press reported that a National Institutes of Health oversight panel will no longer be called upon to review all gene therapy applications. That panel will now take a broader advisory role, the AP said. The U.S. Food and Drug Administration (FDA) will now look at gene therapy treatments as it down all other types of medications.
FDA Seeks to Enhance Manufacturing of Cell and Gene Therapies
Posted on: 3 August 2018, source: Biopharma International
More consistent and reliable production processes are critical for advancing innovative treatments. While cell- and gene-therapy products hold the promise of transforming the treatment of many diseases, difficulties in achieving consistent process control could stymie advancement in this field, cautions Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER). It’s “not simple” to bring a clinical production process to commercial scale, he says, voicing fears that important manufacturing issues could hinder efforts to advance new discoveries able to transform medical care.
More consistent and reliable production processes are critical for advancing innovative treatments. While cell- and gene-therapy products hold the promise of transforming the treatment of many diseases, difficulties in achieving consistent process control could stymie advancement in this field, cautions Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER). It’s “not simple” to bring a clinical production process to commercial scale, he says, voicing fears that important manufacturing issues could hinder efforts to advance new discoveries able to transform medical care.
New EU initiatives to optimize the interplay between the Pharma and the GMO framework
Posted on: 18 July 2018, source: European Commission
The EU's Regulation on advanced therapies, is designed to ensure the free movement of advanced therapy products within Europe, to facilitate access to the EU market, and to foster the competitiveness of European companies in the field, while guaranteeing the highest level of health protection for patients. A Good Practice document on the assessment of GMO-related aspects in the context of clinical trials with human cells genetically modified has been developed by the national competent authorities and the Commission services.
The EU's Regulation on advanced therapies, is designed to ensure the free movement of advanced therapy products within Europe, to facilitate access to the EU market, and to foster the competitiveness of European companies in the field, while guaranteeing the highest level of health protection for patients. A Good Practice document on the assessment of GMO-related aspects in the context of clinical trials with human cells genetically modified has been developed by the national competent authorities and the Commission services.
Caution required for using CRISPR/Cas9 in potential gene therapies
Posted on: 18 July 2018, source: Innovation Toronto
Scientists at the Wellcome Sanger Institute have discovered that CRISPR/Cas9 gene editing can cause greater genetic damage in cells than was previously thought. These results create safety implications for gene therapies using CRISPR/Cas9 in the future as the unexpected damage could lead to dangerous changes in some cells. Reported in the journal Nature Biotechnology, the study also revealed that standard tests for detecting DNA changes miss finding this genetic damage, and that caution and specific testing will be required for any potential gene therapies.
Scientists at the Wellcome Sanger Institute have discovered that CRISPR/Cas9 gene editing can cause greater genetic damage in cells than was previously thought. These results create safety implications for gene therapies using CRISPR/Cas9 in the future as the unexpected damage could lead to dangerous changes in some cells. Reported in the journal Nature Biotechnology, the study also revealed that standard tests for detecting DNA changes miss finding this genetic damage, and that caution and specific testing will be required for any potential gene therapies.