Interview with Daniel Leonard, Director, Global Patient Advocacy, UniQure
Posted on: 11 April 2017, source: Gene Therapy for Rare Disorders 2017
Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company that was known as a leader in patient centricity. In his current role Dan works primarily with hemophilia organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Daniel recently sat down with David Snowdon, Program Director of Gene Therapy for Rare Disorders to discuss his work in this space and how he sees the field evolving. Full interview.
Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company that was known as a leader in patient centricity. In his current role Dan works primarily with hemophilia organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Daniel recently sat down with David Snowdon, Program Director of Gene Therapy for Rare Disorders to discuss his work in this space and how he sees the field evolving. Full interview.
AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
Posted on: 23 March 2017, source: press release
AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Gaucher disease. This late-stage preclinical program becomes AVROBIO’s second therapy for lysosomal storage disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program. The program for Gaucher disease was licensed from Dr. Stefan Karlsson at Lund University in Sweden, where preclinical proof-of-concept has been demonstrated. Terms of the license agreement were not disclosed.
AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Gaucher disease. This late-stage preclinical program becomes AVROBIO’s second therapy for lysosomal storage disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program. The program for Gaucher disease was licensed from Dr. Stefan Karlsson at Lund University in Sweden, where preclinical proof-of-concept has been demonstrated. Terms of the license agreement were not disclosed.
Doctors reverse teen's sickle cell disease with innovative gene therapy
Posted on: 5 March 2017, source: normangeestar.net
A 16-year-old boy in France has become the first person ever to be successfully treated with a new first-of-its-kind gene therapy for sickle cell disease. Doctors removed the teen's bone marrow and genetically altered it in the laboratory to correct the defect in his DNA that causes sickle cell disease. The study stated 15 months after the very first treatment that the boy who once had significant internal damage - so much so that it led to a removed spleen and a hip replacement - now has no sign of the disease.
A 16-year-old boy in France has become the first person ever to be successfully treated with a new first-of-its-kind gene therapy for sickle cell disease. Doctors removed the teen's bone marrow and genetically altered it in the laboratory to correct the defect in his DNA that causes sickle cell disease. The study stated 15 months after the very first treatment that the boy who once had significant internal damage - so much so that it led to a removed spleen and a hip replacement - now has no sign of the disease.
Researchers Use Gene Therapy To Restore Hearing In Deaf Mice
Posted on: 13 February 2017, source: Medical News
A team of researchers at Harvard Medical School and Boston Children's Hospital reported back in 2015 that they were able to restore rudimentary hearing in genetically deaf mice using gene therapy. The Boston Children's hospital research team recently reports that they have restored a much higher level of hearing - down to 25 decibels, the equivalent of a whisper. They used an improved gene therapy vector developed at Massachusetts Eye and Ear. Although previous vectors have only been able to penetrate the cochlea's inner hair cells, the first Nature Biotechnology study reveals that a new synthetic vector, "Anc80," is capable of transferring genes to the inaccessible outer hair cells when introduced into the cochlea.
A team of researchers at Harvard Medical School and Boston Children's Hospital reported back in 2015 that they were able to restore rudimentary hearing in genetically deaf mice using gene therapy. The Boston Children's hospital research team recently reports that they have restored a much higher level of hearing - down to 25 decibels, the equivalent of a whisper. They used an improved gene therapy vector developed at Massachusetts Eye and Ear. Although previous vectors have only been able to penetrate the cochlea's inner hair cells, the first Nature Biotechnology study reveals that a new synthetic vector, "Anc80," is capable of transferring genes to the inaccessible outer hair cells when introduced into the cochlea.
Sarepta eyes a different approach to Duchenne: Gene Therapy
Posted on: 10 January 2017, source: Business Journal
After winning approval in September for the first-ever Duchenne muscular dystrophy drug, Cambridge-based Sarepta Therapeutics is broadening its approach to treating the disease through a pair of gene therapy programs. Sarepta (Nasdaq: SRPT) announced Tuesday that it had reached an agreement with Ohio-based Nationwide Children’s Hospital to exclusively license a pre-clinical program that seeks to treat DMD through gene therapy. The company also said it had entered into a research agreement with the hospital to develop a second pre-clinical gene therapy program, with an exclusive option to license it later.
After winning approval in September for the first-ever Duchenne muscular dystrophy drug, Cambridge-based Sarepta Therapeutics is broadening its approach to treating the disease through a pair of gene therapy programs. Sarepta (Nasdaq: SRPT) announced Tuesday that it had reached an agreement with Ohio-based Nationwide Children’s Hospital to exclusively license a pre-clinical program that seeks to treat DMD through gene therapy. The company also said it had entered into a research agreement with the hospital to develop a second pre-clinical gene therapy program, with an exclusive option to license it later.
New Gene Therapy Can Reverse Aging Process
Posted on: 18 December 2016, source: University Herald
The results of the latest research on gene therapy done by a team of scientists at the Salk Institute in La Jolla, California looked like it's part of a science fiction movie. Better yet it's reminiscent of a Mel Gibson movie, Forever Young, where the main character has remained young over the decades. What the team has discovered was a new form of gene therapy which showed signs that it can reverse the aging process. They conducted the experiment on aging lab rats which showed signs of rejuvenation after undergoing the said treatment for six weeks. The most notable effects they noticed were improved cardiovascular health, straighter spines, and fast regeneration when hurt.
The results of the latest research on gene therapy done by a team of scientists at the Salk Institute in La Jolla, California looked like it's part of a science fiction movie. Better yet it's reminiscent of a Mel Gibson movie, Forever Young, where the main character has remained young over the decades. What the team has discovered was a new form of gene therapy which showed signs that it can reverse the aging process. They conducted the experiment on aging lab rats which showed signs of rejuvenation after undergoing the said treatment for six weeks. The most notable effects they noticed were improved cardiovascular health, straighter spines, and fast regeneration when hurt.
After one dose of gene therapy, hemophilia B patients maintain near-normal levels of clotting factor
Posted on: 6 December 2016, source: Science Daily
Researchers are reporting the highest and most sustained levels to date of an essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental gene therapy in a clinical trial, patients with hemophilia produced near-normal levels of clotting factor IX, allowing them to stop clotting factor infusions and to pursue normal activities of daily life without disabling bleeding episodes.
Researchers are reporting the highest and most sustained levels to date of an essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental gene therapy in a clinical trial, patients with hemophilia produced near-normal levels of clotting factor IX, allowing them to stop clotting factor infusions and to pursue normal activities of daily life without disabling bleeding episodes.
American Society of Gene & Cell Therapy Issues ‘White Paper’ Explaining, Supporting Treatment
Posted on: 1 December 2016, source: ASGCT
People interested in knowing more about gene editing and its therapeutic applications can now download a document prepared by the American Society of Gene & Cell Therapy (ASGCT). The document, titled 'Therapeutic Gene Editing: An ASGCT White Paper.' is intended as background information for policymakers, patients, and the general public to help them better understand the concept and its related therapies.
Its release is intended to assist people in understanding a report on human gene editing by the National Academy of Sciences and National Academy of Medicine. That report, expected in early 2017, will likely cover the ethical, legal, and social implications of gene editing processes and, possibly, to guide policy.
People interested in knowing more about gene editing and its therapeutic applications can now download a document prepared by the American Society of Gene & Cell Therapy (ASGCT). The document, titled 'Therapeutic Gene Editing: An ASGCT White Paper.' is intended as background information for policymakers, patients, and the general public to help them better understand the concept and its related therapies.
Its release is intended to assist people in understanding a report on human gene editing by the National Academy of Sciences and National Academy of Medicine. That report, expected in early 2017, will likely cover the ethical, legal, and social implications of gene editing processes and, possibly, to guide policy.
Researchers take step toward gene therapy for sickle cell disease
Posted on: 8 November 2016, source: MedicalXpress
A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder. The team went on to demonstrate that the mended cells could make a functioning hemoglobin molecule, which carries oxygen in normal red blood cells, and then successfully transplanted the stem cells into mice. The researchers say the study represents a proof of concept for the repair of blood-borne genetic diseases, such as sickle cell disease and thalassemia.
A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder. The team went on to demonstrate that the mended cells could make a functioning hemoglobin molecule, which carries oxygen in normal red blood cells, and then successfully transplanted the stem cells into mice. The researchers say the study represents a proof of concept for the repair of blood-borne genetic diseases, such as sickle cell disease and thalassemia.
Gene therapy for blistering skin disease appears to enhance healing in clinical trial
Posted on: 3 November 2016, source: ScienceBlog
Grafting sheets of a patient’s genetically corrected skin to open wounds caused by the blistering skin disease epidermolysis bullosa appears to be well-tolerated and improves wound healing, according to a phase-1 clinical trial conducted by researchers at the Stanford University School of Medicine. The results mark the first time that skin-based gene therapy has been demonstrated to be safe and effective in patients. The findings will be published Nov. 1 in JAMA. Associate professors of dermatology Peter Marinkovich, MD, and Jean Tang, MD, PhD, share senior authorship of the study. Senior scientist Zurab Siprashvili, PhD, is the lead author.
Grafting sheets of a patient’s genetically corrected skin to open wounds caused by the blistering skin disease epidermolysis bullosa appears to be well-tolerated and improves wound healing, according to a phase-1 clinical trial conducted by researchers at the Stanford University School of Medicine. The results mark the first time that skin-based gene therapy has been demonstrated to be safe and effective in patients. The findings will be published Nov. 1 in JAMA. Associate professors of dermatology Peter Marinkovich, MD, and Jean Tang, MD, PhD, share senior authorship of the study. Senior scientist Zurab Siprashvili, PhD, is the lead author.
HIV Cure: Portable 'Gene Therapy In A Box' A Breakthrough In Fight Against AIDS, Cancer?
Posted on: 24 October 2016, source: iTechPost
Human Immunodeficiency Syndrome, better known as the HIV virus is one of the world's most notorious sexually-transmitted diseases. If left untreated, it can lead to AIDS or Acquired Immunodeficiency Syndrome. Going back to its history, HIV infection has existed in the US at least since the mid-to-late 1970s. Recently, it has been found that gene therapy can now be made possible even for the poorest of countries. Through the help of a handheld portable device that has been discovered, medical personnel can manipulate a patient's blood to carry out potential new medications for cancer, HIV and other types of diseases.
Human Immunodeficiency Syndrome, better known as the HIV virus is one of the world's most notorious sexually-transmitted diseases. If left untreated, it can lead to AIDS or Acquired Immunodeficiency Syndrome. Going back to its history, HIV infection has existed in the US at least since the mid-to-late 1970s. Recently, it has been found that gene therapy can now be made possible even for the poorest of countries. Through the help of a handheld portable device that has been discovered, medical personnel can manipulate a patient's blood to carry out potential new medications for cancer, HIV and other types of diseases.
Smallest-reported artificial virus could help advance gene therapy
Posted on: 10 October 2016, source: EurekAlert
Gene therapy is a kind of experimental treatment that is designed to fix faulty genetic material and help a patient fight off or recover from a disease. Now scientists have engineered the smallest-reported virus-like shell that can self-assemble. It could someday carry potentially therapeutic DNA or RNA and transfer it to human cells. The report appears in the Journal of the American Chemical Society.
Gene therapy is a kind of experimental treatment that is designed to fix faulty genetic material and help a patient fight off or recover from a disease. Now scientists have engineered the smallest-reported virus-like shell that can self-assemble. It could someday carry potentially therapeutic DNA or RNA and transfer it to human cells. The report appears in the Journal of the American Chemical Society.
New gene therapy technique can stop the spread of malignant cancer cells
Posted on: 20 September 2016, source: India Times
MIT scientists have developed a new gene therapy technique that can stop the spread of malignant cells around the body - the leading cause of mortality in women with breast cancer. The treatment uses microRNAs - small noncoding RNA molecules that regulate gene expression - to control the spread of cancer cells around the body, known as metastasis. The therapy could be used alongside chemotherapy to treat early-stage breast cancer tumours before they spread, according to Natalie Artzi, a principal research scientist at Massachusetts Institute of Technology (MIT) in the US.
MIT scientists have developed a new gene therapy technique that can stop the spread of malignant cells around the body - the leading cause of mortality in women with breast cancer. The treatment uses microRNAs - small noncoding RNA molecules that regulate gene expression - to control the spread of cancer cells around the body, known as metastasis. The therapy could be used alongside chemotherapy to treat early-stage breast cancer tumours before they spread, according to Natalie Artzi, a principal research scientist at Massachusetts Institute of Technology (MIT) in the US.
Novartis Drops Gene Therapy Research
Posted on: 8 September 2016, source: Reuters
Healthcare company Novartis has either fired or reassigned roughly 400 researchers previously tasked with developing cell and gene therapies. While most of those scientists will be employed by the company elsewhere, 120 positions will be eliminated, according to Reuters. The Cell and Gene Therapies Unit, led by Usman Azam, attracted attention for a promising experimental therapy based on chimeric antigen receptor T-cells (CAR-T), called CTL109, designed to treat lymphomas in children, reports BusinessInsider.
Healthcare company Novartis has either fired or reassigned roughly 400 researchers previously tasked with developing cell and gene therapies. While most of those scientists will be employed by the company elsewhere, 120 positions will be eliminated, according to Reuters. The Cell and Gene Therapies Unit, led by Usman Azam, attracted attention for a promising experimental therapy based on chimeric antigen receptor T-cells (CAR-T), called CTL109, designed to treat lymphomas in children, reports BusinessInsider.
Gene Therapy 2016: The Pipeline Is Swelling
Posted on: 2 September 2016, source: GEN
Door Has Been Opened to a Range of Novel Biological Therapies for Human Diseases. Twenty-seven years after Steven A. Rosenberg, M.D., Ph.D., at the National Cancer Institute Bethesda campus performed the first human gene transfer experiment, gene therapy is poised to take its place in the armamentarium of biologic therapies available to treat human diseases.
Some examples: the EMA-approval in November 2012 of Glybera® (Uniqure), an intramuscular recombinant adeno-associated virus (rAAV) product for treatment of lipoprotein lipase deficiency; and the recently announced Phase II trial results showing efficacy of another rAAV product for a genetic cause of blindness (LCA2)—for which a Biologics Licensing Application is anticipated in the U.S. this year.
Door Has Been Opened to a Range of Novel Biological Therapies for Human Diseases. Twenty-seven years after Steven A. Rosenberg, M.D., Ph.D., at the National Cancer Institute Bethesda campus performed the first human gene transfer experiment, gene therapy is poised to take its place in the armamentarium of biologic therapies available to treat human diseases.
Some examples: the EMA-approval in November 2012 of Glybera® (Uniqure), an intramuscular recombinant adeno-associated virus (rAAV) product for treatment of lipoprotein lipase deficiency; and the recently announced Phase II trial results showing efficacy of another rAAV product for a genetic cause of blindness (LCA2)—for which a Biologics Licensing Application is anticipated in the U.S. this year.
Novel Gene Therapy Drug for Huntington's Disease
Posted on: 25 August 2016, source: PRNewswire
Data from Vybion on a novel treatment for Huntington's disease has been published in the Journal of Neurodegenerative diseases. The published study links the ability of Vybion's proprietary, novel Intrabody (INT41) blocking of cellular gene dysregulation to the delay of cognitive and motor function loss in the well-validated vR6/2 animal model. INT41 interferes with direct binding of toxic N-terminal huntingtin fragments to DNA, as well as their transport into the nucleus. The data support a direct gain of function of N-terminal huntingtin protein fragments that may lead to neuron dysfunction and brain atrophy as well as a novel therapeutic modality.
Data from Vybion on a novel treatment for Huntington's disease has been published in the Journal of Neurodegenerative diseases. The published study links the ability of Vybion's proprietary, novel Intrabody (INT41) blocking of cellular gene dysregulation to the delay of cognitive and motor function loss in the well-validated vR6/2 animal model. INT41 interferes with direct binding of toxic N-terminal huntingtin fragments to DNA, as well as their transport into the nucleus. The data support a direct gain of function of N-terminal huntingtin protein fragments that may lead to neuron dysfunction and brain atrophy as well as a novel therapeutic modality.
FDA Grants Breakthrough Designation to AveXis's Gene Therapy
Posted on: 24 July 2016, source: DDD-Mag
AveXis, a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS-101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients. The Breakthrough Therapy Designation is based on preliminary clinical results from the ongoing trial of AVXS-101, conducted in collaboration with The Research Institute at Nationwide Children's Hospital and The Ohio State University.
AveXis, a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS-101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients. The Breakthrough Therapy Designation is based on preliminary clinical results from the ongoing trial of AVXS-101, conducted in collaboration with The Research Institute at Nationwide Children's Hospital and The Ohio State University.
Ziopharm Confirms Three Patient Deaths in Gene Therapy Trial
Posted on: 16 July 2016, source: GEN
Zopharm Oncology has disclosed the deaths of three patients in a Phase I study of its gene therapy candidate Ad-RTS-hIL-12 plus oral veledimex for a form of brain cancer. The third patient died of an intracranial hemorrhage that occurred sometime following discharge from a treating center for the Phase I study, Ziopharm said today in a statement. The study is designed to assess the combination of Ad-RTS-hIL-12 with orally administered veledimex in patients with recurrent or progressive glioblastoma (GBM) or grade III malignant glioma.
Zopharm Oncology has disclosed the deaths of three patients in a Phase I study of its gene therapy candidate Ad-RTS-hIL-12 plus oral veledimex for a form of brain cancer. The third patient died of an intracranial hemorrhage that occurred sometime following discharge from a treating center for the Phase I study, Ziopharm said today in a statement. The study is designed to assess the combination of Ad-RTS-hIL-12 with orally administered veledimex in patients with recurrent or progressive glioblastoma (GBM) or grade III malignant glioma.
Gene-therapy trials must proceed with caution
Posted on: 1 July 2016, source: Nature
The perils of the past must not be allowed to happen again. Jesse Gelsinger was 18 and healthy when he died in 1999 during a gene-therapy experiment. He had a condition called ornithine transcarbamylase deficiency (OTC), but it was under control through a combination of diet and medication. Like others with the disorder, Gelsinger lacked a functional enzyme involved in breaking down ammonia, a waste product of protein metabolism that becomes toxic when its levels become too high. The gene therapy that he received used a viral vector to introduce a normal gene for the enzyme. Gene therapy remains an obvious route to treat OTC. Simply adding the missing gene has been shown to repair metabolism in mice. But the memory of what happened to Gelsinger has slowed progress in gene therapy for any condition.
The perils of the past must not be allowed to happen again. Jesse Gelsinger was 18 and healthy when he died in 1999 during a gene-therapy experiment. He had a condition called ornithine transcarbamylase deficiency (OTC), but it was under control through a combination of diet and medication. Like others with the disorder, Gelsinger lacked a functional enzyme involved in breaking down ammonia, a waste product of protein metabolism that becomes toxic when its levels become too high. The gene therapy that he received used a viral vector to introduce a normal gene for the enzyme. Gene therapy remains an obvious route to treat OTC. Simply adding the missing gene has been shown to repair metabolism in mice. But the memory of what happened to Gelsinger has slowed progress in gene therapy for any condition.
Gene therapy drug approval granted to GSK
Posted on: 29 May 2016, source: BBC
Regulators have given one of the world's largest drug companies approval to sell a new gene therapy. The treatment is for an illness called ADA-SCID which prevents babies from fighting off everyday infections. This is the first approval for a genetic therapy granted to a large multinational drug company, GSK. Commentators say the development marks the beginning of many more genetic medicines from so-called "Big Pharma". The condition is extremely rare and affects around two dozen babies each year. Approval of the gene therapy paves the way for the development of treatments for more widespread illnesses such as thalassemia and sickle cell disease. Hundreds of inherited disorders such as cystic fibrosis, muscular dystrophy and many types of blindness are caused by faulty genes.
Regulators have given one of the world's largest drug companies approval to sell a new gene therapy. The treatment is for an illness called ADA-SCID which prevents babies from fighting off everyday infections. This is the first approval for a genetic therapy granted to a large multinational drug company, GSK. Commentators say the development marks the beginning of many more genetic medicines from so-called "Big Pharma". The condition is extremely rare and affects around two dozen babies each year. Approval of the gene therapy paves the way for the development of treatments for more widespread illnesses such as thalassemia and sickle cell disease. Hundreds of inherited disorders such as cystic fibrosis, muscular dystrophy and many types of blindness are caused by faulty genes.
Federal advisory committee greenlights first CRISPR clinical trial
Posted on: 22 June 2016, source: Nature
CRISPR, the genome-editing technology that has taken biomedical science by storm, is finally nearing human trials. On 21 June, an advisory committee at the US National Institutes of Health (NIH) approved a proposal to use CRISPR–Cas9 to help augment cancer therapies that rely on enlisting a patient’s T cells, a type of immune cell. “Cell therapies for cancer are so promising but the majority of people who get these therapies have a disease that relapses,” says study leader Edward Stadtmauer, a physician at the University of Pennsylvania in Philadelphia. Gene editing could improve such treatments and eliminate some of their vulnerabilities to cancer and the body’s immune system, he says.
CRISPR, the genome-editing technology that has taken biomedical science by storm, is finally nearing human trials. On 21 June, an advisory committee at the US National Institutes of Health (NIH) approved a proposal to use CRISPR–Cas9 to help augment cancer therapies that rely on enlisting a patient’s T cells, a type of immune cell. “Cell therapies for cancer are so promising but the majority of people who get these therapies have a disease that relapses,” says study leader Edward Stadtmauer, a physician at the University of Pennsylvania in Philadelphia. Gene editing could improve such treatments and eliminate some of their vulnerabilities to cancer and the body’s immune system, he says.
Abeona Therapeutics Doses First Patient in Phase 1/2 Clinical Trial for Sanfilippo Syndrome Type A
Posted on: 19 May 2016, source: Abeona Therapeutics
Cleveland-based Abeona Therapeutics has dosed its first patient in a Phase 1/ 2 Trial with ABO-102 gene therapy for patients with Sanfilippo syndrome type A (a/k/a Mucopolysaccharidosis Type IIIA or MPS IIIA). ABO-12, a single intravenous injection, is an adeno-associated viral (AAV)-based gene therapy to treat the central nervous system and the peripheral disease manifestations in Sanfilippo patients, representing a new approach to addressing the progressive disease which often prevents children from reaching adulthood. The drug is based on more than 18 years of research and development. There are currently no approved treatments available for Sanfilippo syndrome type A.
Cleveland-based Abeona Therapeutics has dosed its first patient in a Phase 1/ 2 Trial with ABO-102 gene therapy for patients with Sanfilippo syndrome type A (a/k/a Mucopolysaccharidosis Type IIIA or MPS IIIA). ABO-12, a single intravenous injection, is an adeno-associated viral (AAV)-based gene therapy to treat the central nervous system and the peripheral disease manifestations in Sanfilippo patients, representing a new approach to addressing the progressive disease which often prevents children from reaching adulthood. The drug is based on more than 18 years of research and development. There are currently no approved treatments available for Sanfilippo syndrome type A.
Breakthrough Gene Therapy Reverses Vision Loss In Patients With Inherited Blindness
Posted on: 30 April 2016, source: TechTimes
A genetic therapy offers a glimmer of hope for patients with a rare form of blindness. In a clinical study conducted in Oxford University in the United Kingdom, the therapy restored vision in the patients for as long as four years, potentially benefiting not only those with the type of genetic blindness but also those afflicted by common causes of vision loss.
A genetic therapy offers a glimmer of hope for patients with a rare form of blindness. In a clinical study conducted in Oxford University in the United Kingdom, the therapy restored vision in the patients for as long as four years, potentially benefiting not only those with the type of genetic blindness but also those afflicted by common causes of vision loss.
Could gene therapy help you live forever? CEO of controversial firm claims she has successfully carried out first anti-ageing treatment - on herself
Posted on: 27 April 2016, source: DailyMail
Eternal youth could be one step closer. A woman from Seattle claims to have become the first human to have become younger using gene therapy. Elizabeth Parrish, CEO of controversial firm Bioviva, says she has been using her own company's experimental gene therapies to battle ageing - but not everyone is convinced of the results. The self-treatment, which has been ongoing for the past six months, has been met with a mixture of praise for innovation and criticism for a lack of scientific rigour. But now, Parrish claims to have become the first human being to be successfully rejuvenated by reversing 20 years of normal telomere shortening.
Eternal youth could be one step closer. A woman from Seattle claims to have become the first human to have become younger using gene therapy. Elizabeth Parrish, CEO of controversial firm Bioviva, says she has been using her own company's experimental gene therapies to battle ageing - but not everyone is convinced of the results. The self-treatment, which has been ongoing for the past six months, has been met with a mixture of praise for innovation and criticism for a lack of scientific rigour. But now, Parrish claims to have become the first human being to be successfully rejuvenated by reversing 20 years of normal telomere shortening.
U.S. FDA reports rise in gene and cell therapy applications
Posted on: 19 April 2016, source: RAPS
The US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) met in February to discuss updates of research programs in the Office of Cellular, Tissue and Gene Therapy (CTGT), which is part of FDA’s Center for Biologics Evaluation and Research (CBER). The research updates came as Raj Puri, MD, PhD, Director, Division of Cellular, and Gene Therapies (DCGT) at CBER told the committee that although his division has only licensed 12 products in its entire history, more than 1,530 active investigational new drugs (INDs), investigational device exemptions (IDEs) and master files are currently under review, and 106 of those INDs deal specifically with natural killer cells, which play a role in the rejection of tumors and virally-infected cells.
The US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) met in February to discuss updates of research programs in the Office of Cellular, Tissue and Gene Therapy (CTGT), which is part of FDA’s Center for Biologics Evaluation and Research (CBER). The research updates came as Raj Puri, MD, PhD, Director, Division of Cellular, and Gene Therapies (DCGT) at CBER told the committee that although his division has only licensed 12 products in its entire history, more than 1,530 active investigational new drugs (INDs), investigational device exemptions (IDEs) and master files are currently under review, and 106 of those INDs deal specifically with natural killer cells, which play a role in the rejection of tumors and virally-infected cells.
Chinese Scientists Defy Ethics, Double Down on Editing Human Embryos
Posted on: 14 April 2016, source: GEN
In April of 2015, Chinese researchers announced to the world that they had used the new genome-editing technology CRISPR/Cas9 on human preimplantation embryos to modify the HBB gene, mutations of which have been implicated in the disease β-thalassemia. The news had sparked a media firestorm and eventually led scientists from across the globe to discuss intently the ethics of such studies, ultimately leading to the decision for an international moratorium on the use of CRISPR with human embryos.
In April of 2015, Chinese researchers announced to the world that they had used the new genome-editing technology CRISPR/Cas9 on human preimplantation embryos to modify the HBB gene, mutations of which have been implicated in the disease β-thalassemia. The news had sparked a media firestorm and eventually led scientists from across the globe to discuss intently the ethics of such studies, ultimately leading to the decision for an international moratorium on the use of CRISPR with human embryos.
EU Regulatory Panel Recommends Approval Of Glaxo's Gene Therapy For Bubble Boy Disease
Posted on: 3 April 2016, source: TechTimes
An EU regulatory panel has recommended the approval of a gene therapy from GlaxoSmithKline (GSK) for the treatment of Bubble Boy Disease. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), together with the Committee for Advanced Therapies (CAT) issued a positive opinion last April 1 with regards to marketing the drug called Strimvelis for the treatment of ADA-SCID (Adenosine Deaminase Deficiency-Severe Combined Immunodeficiency).
An EU regulatory panel has recommended the approval of a gene therapy from GlaxoSmithKline (GSK) for the treatment of Bubble Boy Disease. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), together with the Committee for Advanced Therapies (CAT) issued a positive opinion last April 1 with regards to marketing the drug called Strimvelis for the treatment of ADA-SCID (Adenosine Deaminase Deficiency-Severe Combined Immunodeficiency).
Dal researcher develops technique for enhancing gene therapy
Posted on: 5 March 2016, source: Dalhousie University
Using his knowledge of how genes are organized and repaired in human cells, Dr. Graham Dellaire, Dalhousie Medical School’s Cameron Research Scientist in Cancer Biology, has developed a technique that could make gene therapy more effective and safer to use. His work was recently published in Nucleic Acids Research and Nature.
Using his knowledge of how genes are organized and repaired in human cells, Dr. Graham Dellaire, Dalhousie Medical School’s Cameron Research Scientist in Cancer Biology, has developed a technique that could make gene therapy more effective and safer to use. His work was recently published in Nucleic Acids Research and Nature.
Gene therapy in the womb for ill babies: Plan to give injections to mothers whose unborn children have stopped growing due to failure of the placenta
Posted on: 11 March 2016, source: Daily Mail
Desperately ill babies could have their lives saved by a pioneering treatment given while they are still in the womb. In a world first, doctors at a British hospital plan to give injections of genes to mothers-to-be whose unborn children have stopped growing. It is hoped that the genes will boost blood flow to the placenta, the lifeline between mother and baby, providing the child with the oxygen and nutrients needed to thrive and survive.
Desperately ill babies could have their lives saved by a pioneering treatment given while they are still in the womb. In a world first, doctors at a British hospital plan to give injections of genes to mothers-to-be whose unborn children have stopped growing. It is hoped that the genes will boost blood flow to the placenta, the lifeline between mother and baby, providing the child with the oxygen and nutrients needed to thrive and survive.
Researchers to use nano-tech in gene therapy of cancer cells
Posted on: 5 March 2016, source: Mehr News
Iranian researchers of Tabriz University of Medical Sciences managed to apply laboratory synthesis of nano-polymeric carriers which can be used in gene therapy of cancer cells. Gene therapy is an experimental technique through which defective genes are modified and sent into the cell by a carrier. Vectors used to transfer genes are divided into two Viral and Non-viral categories.
Iranian researchers of Tabriz University of Medical Sciences managed to apply laboratory synthesis of nano-polymeric carriers which can be used in gene therapy of cancer cells. Gene therapy is an experimental technique through which defective genes are modified and sent into the cell by a carrier. Vectors used to transfer genes are divided into two Viral and Non-viral categories.