Sentences with phrase «of gene therapy clinical trials»

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).

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.

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.»

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 plgene 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 tooktherapy 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 plGene Therapy, where the test tookTherapy, 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 Chiltherapy 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 ChilTherapy at Nationwide Children's.

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 AVXStherapy 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 AVXSTherapy 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.

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.

Despite expectations of a rapid breakthrough, no cystic fibrosis gene therapy trial so far has been able to show long - term clinical improvement.

«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.

«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.

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.»

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

Weill Cornell is the birthplace of many medical advances — including the development of the Pap test for cervical cancer, the synthesis of penicillin, the first successful embryo - biopsy pregnancy and birth in the U.S., the first clinical trial of gene therapy for Parkinson's disease, and most recently, the world's first successful use of deep brain stimulation to treat a minimally conscious brain - injured patient.

CRISPR Therapeutics is pioneering a new class of medicines, to conduct the first company - sponsored clinical trial of a CRISPR gene - edited therapy.

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 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 faiTrial 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 clinical trial of an investigational drug for the treatment of advanced heart faitrial of an investigational drug for the treatment of advanced heart failure.

Using a disabled form of HIV to deliver the gene therapy, researchers were able to halt the progression of the disease in 15 out of 17 boys who participated in the clinical trial.

Gene and cell therapies have made important medical advances over the past three decade, developing technologies and testing novel therapies in multiple human clinical trials of many diseases.

Yesterday a team of University of Pennsylvania researchers — led by Dr Pablo Tebas, Professor Carl June, and Dr Bruce Levine — announced the successful conclusion of a clinical trial to evaluate the safety of a new gene therapy technique for treating HIV.

The program is unique in its concentration of nationally and internationally recognized leaders in the areas of new drug development, cell and gene therapy and design and conduct of both local and national clinical trials through the Children's Oncology Group (COG).

Doudna believes a clinical trial of CRISPR - based gene therapy could begin in less than a decade.

Paris, France, April 3, 2018, 5.35 pm CET — GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA - PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced topline results from the REVERSE Phase III clinical trial evaluating the safety and efficacy of a single intravitreal injection of GS010 (rAAV2 / 2 - ND 4) in 37 subjects whose visual loss due to 11778 - ND4 Leber Hereditary Optic Neuropathy (LHON) commenced between 6 and 12 months prior to study treatment.

That clearance would be a high - water mark for gene therapy research, which suffered a major setback in 1999 with the death of a patient in a clinical trial for a liver disorder.

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.

Indeed, Leber Congenital Amaurosis (LCA) was one of the first recessive disorders to undergo clinical trials for gene replacement therapy.

The research team also found mutations in the PIK3CA gene, which has been well studied in cancer and is already the focus of several clinical trials of targeted therapies.

The treatment, developed at the University of Pennsylvania with clinical trials carried out at Children's Hospital of Philadelphia and the University of Iowa, is the first time gene therapy is used to treat an inherited disease and could lead to treatments of other currently untreatable conditions.

Overall, SR - Tiget represents a multi-disciplinary research environment, which provides a unique blend of scientific expertise in the development of innovative gene and cell therapy strategies, access to relevant preclinical models to evaluate their efficacy and safety, as well as competence in conducting early phase clinical trials.

Despite the marginal improvements in survival of patients suffering from malignant glioma treated with gene therapy vectors, the clinical trials conducted so far using viral vectors, in particular adenoviral vectors, have proven that the use of adenoviral vectors is a safe therapeutic approach, even in large, multicenter, phase 3 clinical trials.

The technique is also inefficient, correcting only a small percentage of gene mutations, and transplantation success has proven rare in clinical trials testing gene therapy to treat beta thalassemia.

Auburn University College of Veterinary Medicine, UMass Medical School to test gene therapy treatment for Tay - Sachs and Sandhoff diseases Researchers at Auburn University College of Veterinary Medicine and UMass Medical School are nearing human clinical trials on a genetic therapy for two rare neurological diseases that are fatal to children.

New treatments are being tested to tackle the crippling difficulties of vertigo, including a clinical trial of prosthetic ear implants and ear gene therapy, with initial work revealing novel aspects of brain anatomy linked to balance which could be used as targets for future treatments.