Many point to the Jesse Gelsinger case, in which a teenager died during
a gene therapy clinical trial.
There are now more than 100
gene therapy clinical trials aimed toward cancer, genetic diseases (such as ADA deficiency, cystic fibrosis and hemophilia A), infectious diseases (including AIDS) and autoimmune diseases (such as rheumatoid arthritis).
As a new generation of
gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist - oncologist from Dana - Farber / Boston Children's Cancer and Blood Disorders Center argues in a commentary published by the journal Science.
Several
gene therapy clinical trials using gene - modified hematopoietic stem cells have shown therapeutic efficacy in multiple disease areas.
Colleen Dansereau, RN, MSN, CPN Program Manager, Gene Therapy Program As manager of the Gene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of
the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (CGD).
11/10/2008 UC San Diego Medical Center Offers
Gene Therapy Clinical Trial for Advanced Heart Failure UC San Diego Medical Center is currently enrolling patients in a Phase 2 clinical trial of an investigational drug for the treatment of advanced heart failure.
He notes that AAV is already being used in
gene therapy clinical trials for Parkinson's disease, hemophilia, and vision disorders.
Reid and his colleagues undertook this study following the death of Jesse Gelsinger, a participant in
a gene therapy clinical trial at University of Pennsylvania for ornithine transcarbanoylase (OTC) deficiency, a metabolic liver disorder.
Lysogene's MPS IIIA
gene therapy clinical trial strategy includes a partnership with the MPS patient networks to ensure families are well informed about our development program and are able to make informed choices concerning the best treatment strategy for their children.
Not exact matches
Katherine High, Spark's president and chief scientific officer, expressed her enthusiasm for the early
clinical data related to SPK - 8011: «The encouraging start of our SPK - 8011
clinical trial reinforces the strength of our
gene therapy platform, delivers human proof - of - concept in a second liver - mediated disease — a significant achievement in the
gene therapy field — and positions us well to potentially transform the current treatment approach for this life - altering disease with a one - time intervention.»
He and his lab, for example, along with the company Neurologix based in Fort Lee, N.J., are conducting a
clinical trial to test a similar
gene -
therapy treatment for Parkinson's disease.
But he contends that other
gene therapies already in
clinical trials suggest that the treatment could work.
These allusions to the past aren't surprising considering how drastically the
clinical trial changed
gene therapy and, in particular, the career of James M. Wilson, the medical geneticist who headed Penn's Institute for Human Gene Therapy, where the test took pl
gene therapy and, in particular, the career of James M. Wilson, the medical geneticist who headed Penn's Institute for Human Gene Therapy, where the test took
therapy and, in particular, the career of James M. Wilson, the medical geneticist who headed Penn's Institute for Human
Gene Therapy, where the test took pl
Gene Therapy, where the test took
Therapy, where the test took place.
«My team at Nationwide Children's has worked with commitment and dedication to develop a
therapy that may subsequently be shown through future clinical trials to potentially alter the course of this unforgiving condition and provide a therapeutic option for the families and infants with SMA1,» says Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide Chil
therapy that may subsequently be shown through future
clinical trials to potentially alter the course of this unforgiving condition and provide a therapeutic option for the families and infants with SMA1,» says Jerry Mendell, MD, principal investigator in the Center for
Gene Therapy at Nationwide Chil
Therapy at Nationwide Children's.
Although
gene therapy research has made great strides in recent years, it has yet to be widely deployed, and no CRISPR - edited
genes have yet been tested for safety or efficacy in human
clinical trials.
AAV is in use in many late - stage
clinical trials in the United States, and has already been approved for use in one
gene therapy drug in the European Union.
This study represents a significant step towards the development of
clinical trials in
gene therapy for the curative treatment of hereditary deafness and balance loss in humans.
AveXis, Inc., a
clinical - stage
gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXS
therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough
Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXS
Therapy Designation for the treatment based on preliminary
clinical results from the
trial of AVXS - 101.
A one - time intravenous infusion of the high dose of
gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1
clinical trial, according to a study published in the New England Journal of Medicine.
«Fibroblast growth factor receptor inhibitors are new
therapies being developed in
clinical trials for patients whose cancer cells have genetic alterations in this family of
genes,» says Roychowdhury, a member of the OSUCCC — James Translational Therapeutics Program.
One
clinical trial involves the drug CGF166, a one - time
gene therapy, which, if proven successful in humans, could regenerate new hair cells within the cochlea that can signal the part of the brain that processes sound.
Another candidate, AVXS - 101 (AveXis), is a
gene therapy approach to increase SMN levels, which is in Phase I
clinical trials.
A U.K. team is designing a
clinical trial even more radical than prenatal stem cell
therapy: the first ever test of
gene therapy in pregnancy.
Priscilla N. Kelly Associate Editor Education: B.Sc., University of Western Australia; Ph.D., University of Melbourne Areas of responsibility: Preclinical development, translational medicine, cancer immunotherapy, drug discovery,
clinical trials,
gene and cell
therapy E-Mail:
[email protected]
After this incident, and the death in 1999 of a young man in a
clinical trial, apparently because of an immune reaction to the treatment,
gene therapy research grounds nearly to a halt.
«Our
gene therapy protocol is not yet ready for
clinical trials — we need to tweak it a bit more — but in the not - too - distant future we think it could be developed for therapeutic use in humans,» says Jeffrey Holt, PhD, a scientist in the Department of Otolaryngology and F.M. Kirby Neurobiology Center at Boston Children's and an associate professor of Otolaryngology at Harvard Medical School.
It showed effectiveness against liver cancer in a phase II
clinical trial and will move into a phase III
trial later this year, David Kirn, an oncologist and the company's president and chief executive officer, said at a recent meeting of the American Society for
Gene & Cell
Therapy in Washington, D.C..
Ultimately, Holt hopes to partner with clinicians at Boston Children's Department of Otolaryngology and elsewhere to start
clinical trials of TMC1
gene therapy within 5 to 10 years.
The proposed
clinical trial, in which researchers would use CRISPR to engineer immune cells to fight cancer, won approval from the Recombinant DNA Advisory Committee (RAC) at the U.S. National Institutes of Health, a panel that has traditionally vetted the safety and ethics of
gene therapy trials funded by the U.S. government and others.
«The good news is that this finding predicts that patients missing either
gene should be sensitive to new
therapies targeting focal adhesion enzymes, which are currently being tested in early - stage
clinical trials,» says Shaw, who is also a member of the Moores Cancer Center and an adjunct professor at the University of California, San Diego.
Dr. Levine directs the
Clinical Cell and Vaccine Production Facility (CVPF), which develops, manufactures, and tests novel cell and gene therapies in clinical trials at Penn and collaborating insti
Clinical Cell and Vaccine Production Facility (CVPF), which develops, manufactures, and tests novel cell and
gene therapies in
clinical trials at Penn and collaborating insti
clinical trials at Penn and collaborating institutions.
Despite expectations of a rapid breakthrough, no cystic fibrosis
gene therapy trial so far has been able to show long - term
clinical improvement.
(Adeno - associated virus or AAV has been approved in Europe as a vector for
gene therapy, and is being explored in the United States for
clinical trials.)
The team envisions that in the future, deaf people might benefit from
gene therapy similar to the approaches currently being tested in
clinical trials for other diseases.
«A
clinical trial in both type 1 and type 2 diabetics in the immediate foreseeable future is quite realistic, given the impressive nature of the reversal of the diabetes, along with the feasibility in patients to do AAV
gene therapy.»
Boston Children's Hospital has offered non-exclusive licenses to for - profit entities on a patent developed by Orkin's laboratory regarding BCL11A, a genetic switch regulating hemoglobin production that is expected to form the basis of
clinical trials for
gene therapy and
gene editing for sickle cell disease and thalassemia.
Woo and Ledley predict that
clinical trials using
gene therapy for metabolic disorders such as PKU could start within eight years.
«Despite extensive preclinical approaches, translation of
gene therapy strategies into
clinical trials is still a difficult and expensive process.»
Nonetheless, the result has renewed concern about the oversight of
clinical trials for
gene therapy.
The U.S. Food and Drug Administration (FDA) has begun proceedings that could disqualify
gene therapy researcher James Wilson of the University of Pennsylvania in Philadelphia from conducting any future
clinical trials.
It is the first time that a
gene therapy based on genetically modified stem cells is tested in a multicenter, international
clinical trial that shows a reproducible and robust therapeutic effect in different centers and different countries.
For very rare diseases such as WAS, multicenter
clinical trials are the only effective way of proving the safety and efficacy of
gene therapy and having it rapidly approuved and made available to all patients.
The research, part of a phase I
clinical trial to test the safety of the treatment, was published as a letter to the editor in The New England Journal of Medicine earlier this week and will be in the September issue of Human
Gene Therapy.
«RNAi
therapies are a unique approach to cancer treatment as they have the potential to «turn off» the
genes» coding for proteins involved in cancer cell division,» said Ramesh K. Ramanathan, M.D., medical director of the Virginia G. Piper Cancer Center
Clinical Trials Program at Scottsdale Healthcare and deputy director of the
Clinical Translational Research Division of the Translational Genomics Research Institute (TGen) in Phoenix, Ariz. «Using a lipid nanoparticle, the RNAi drug can be delivered to a cancer cell to block the expression of specific proteins involved in tumor growth.»
Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler - Najjar Synd
Gene therapy: Généthon starts a
clinical trial to test a
gene therapy treatment for a rare liver disease, Crigler - Najjar Synd
gene therapy treatment for a rare liver disease, Crigler - Najjar Syndrome
Lysogene is currently preparing to initiate a Phase II / III pivotal
clinical trial with its next generation
gene therapy formulation.
Lysogene successfully completed its phase I / II
clinical trial of
gene therapy in MPS IIIA and plans its phase II / III registrational
trials... Read More
UC San Diego Health joins national
clinical trial testing a
gene therapy treatment for individuals with hemophilia B.
Although this field is still relatively young, evidence for
clinical efficacy has been observed and continued progress seems assured, as
clinical trials continue to yield insights into how
gene therapy can be applied and improvements are made in
gene therapy tools.
Within the past 10 years or so, lentiviruses have shown promise in
clinical trials (1 — 3), and adenoassociated viruses (AAVs) have been used in the first approved
gene therapies in the Western world (4).