Sentences with phrase «other gene therapy trial»

Although the study's approach wasn't a sure bet, the researchers hoped to go further than any other gene therapy trial yet for this relatively common inherited disease that fills people's lungs with sticky mucus that promotes deadly infections.

What's more, none of the patients experienced macrophage activate syndrome or cytokine release syndrome, an infusion reaction observed in other gene therapy trials characterized by fever, nausea, chills, hypotension or a rash.

Not exact matches

So far, its trials have shown it can improve outcomes when used alongside other multiple myeloma drugs and that could offer it some insulation if the market gets disrupted by new treatment approaches, such as gene therapy.

But he contends that other gene therapies already in clinical trials suggest that the treatment could work.

AAV1 is considered safe as a viral vector and is already in use in human gene therapy trials for blindness, heart disease, muscular dystrophy and other conditions.

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 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.

Formerly a research leader at CHOP, High pursued groundbreaking preclinical investigations in hemophilia B gene therapy and provided scientific expertise to previous gene therapy trials in hemophilia and other genetic disorders at CHOP before moving to Spark Therapeutics, which was spun off from CHOP in 2013.

His story inspired a book, The Forever Fix, which heralded the LCA trials as part of the wave of successes for gene therapy, which was recovering from several setbacks — including the death of a patient and cancer in others — in the late 1990s and early 2000s.

These are skills that we implement for other international trials of gene therapy for rare genetic diseases of the immune system, blood, muscle, vision or liver... We will continue the current study with the objective of providing treatment for patients.»

Other blood disorders that have shown significant benefit from targeted gene therapy in small trials include hemophilia (specifically, factor IX deficiency), severe beta - thalassemia (deficiency for the adult beta - globin gene) and leukemia, where the patient's immune cells were treated to enable them to recognize cancer cells and destroy them.

One virus he selected for his experiments, known as an adeno - associated virus, proved to be promising in human gene - therapy trials in other labs.

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.

AGTC plans to use the net proceeds of the offering to fund preclinical investigation and Phase 1/2 trials of potential product candidates for treatment of wet AMD; to expand its manufacturing capabilities and create a pilot manufacturing group; to in - license, acquire or invest in complementary gene therapy, technologies, products or assets; and for working capital and other general corporate purposes.