Sentences with phrase «trial of its cell therapy»

[38] Although the results of the trials of cell therapy for PD were mixed, with only a minority of patients showing clear - cut and durable gains, researchers have subsequently gained substantial insight into the reasons underlying differential outcomes at different centers and amongst different protocols, and for the troubling dyskinesias that were suffered by some patients.

Not exact matches

Kite Pharma, one of the companies chasing a new generation of cancer drugs called chimeric antigen receptor T - cell (CAR - T) therapies, announced a patient death in a clinical trial of its experimental KTE - C19.

These risks and uncertainties include: Gilead's ability to achieve its anticipated full year 2018 financial results; Gilead's ability to sustain growth in revenues for its antiviral and other programs; the risk that private and public payers may be reluctant to provide, or continue to provide, coverage or reimbursement for new products, including Vosevi, Yescarta, Epclusa, Harvoni, Genvoya, Odefsey, Descovy, Biktarvy and Vemlidy ®; austerity measures in European countries that may increase the amount of discount required on Gilead's products; an increase in discounts, chargebacks and rebates due to ongoing contracts and future negotiations with commercial and government payers; a larger than anticipated shift in payer mix to more highly discounted payer segments and geographic regions and decreases in treatment duration; availability of funding for state AIDS Drug Assistance Programs (ADAPs); continued fluctuations in ADAP purchases driven by federal and state grant cycles which may not mirror patient demand and may cause fluctuations in Gilead's earnings; market share and price erosion caused by the introduction of generic versions of Viread and Truvada, an uncertain global macroeconomic environment; and potential amendments to the Affordable Care Act or other government action that could have the effect of lowering prices or reducing the number of insured patients; the possibility of unfavorable results from clinical trials involving investigational compounds; Gilead's ability to initiate clinical trials in its currently anticipated timeframes; the levels of inventory held by wholesalers and retailers which may cause fluctuations in Gilead's earnings; Kite's ability to develop and commercialize cell therapies utilizing the zinc finger nuclease technology platform and realize the benefits of the Sangamo partnership; Gilead's ability to submit new drug applications for new product candidates in the timelines currently anticipated; Gilead's ability to receive regulatory approvals in a timely manner or at all, for new and current products, including Biktarvy; Gilead's ability to successfully commercialize its products, including Biktarvy; the risk that physicians and patients may not see advantages of these products over other therapies and may therefore be reluctant to prescribe the products; Gilead's ability to successfully develop its hematology / oncology and inflammation / respiratory programs; safety and efficacy data from clinical studies may not warrant further development of Gilead's product candidates, including GS - 9620 and Yescarta in combination with Pfizer's utomilumab; Gilead's ability to pay dividends or complete its share repurchase program due to changes in its stock price, corporate or other market conditions; fluctuations in the foreign exchange rate of the U.S. dollar that may cause an unfavorable foreign currency exchange impact on Gilead's future revenues and pre-tax earnings; and other risks identified from time to time in Gilead's reports filed with the U.S. Securities and Exchange Commission (the SEC).

Cambridge, MA — March 30, 2017 — Aura Biosciences, a biotechnology company developing a new class of therapies to target and selectively destroy cancer cells using viral nanoparticle conjugates, announced today that it has enrolled and dosed the first patient in its Phase 1b clinical trial of light - activated AU - 011, an investigational, first - in - class targeted therapy in development for the treatment of ocular melanoma, a rare and life - threatening disease.

Dr. Newman added that rival CAR T cell therapy developer Kite Pharma was still likelier to generate more revenue than Juno with KTE - C19, which last week completed patient enrollment in the Phase II portion of the ZUMA - 1 trial in patients with diffuse large B - cell lymphoma.

No. 4: CAR - T takes a backseat Research into chimeric antigen receptor T - cell (CAR - T) therapy has captured the attention of investors over the past two years, but safety risks could emerge as trial sizes increase, and that could help shift attention to other promising anti-cancer approaches, including NantKwest's (NASDAQ: NK) natural killer cell approach and soon - to - IPO Editas» CRISPR / Cas approach.

The truth, of course, is that there are no human embryonic stem - cell therapies even in clinical trial, let alone ready for therapy, and there have been no major treatment....

With about 50 scientists and technicians working furiously to produce cell therapies for trials throughout the University of Pennsylvania (UPenn), CHOP can treat about five children each month.

Based on results of the current study described in a report online June 18 in the journal Cancer Cell, Johns Hopkins researchers say they are planning a phase I clinical trial to test the paclitaxel - fostamatinib combination therapy in patients with recurrent advanced ovarian cancer.

«There are currently no therapies which successfully reverse the damage seen in the more than 12,000 individuals who suffer a spinal cord injury each year in the United States alone,» says Dr. Richard G. Fessler, professor of neurological surgery at Rush University Medical Center and principal investigator for the Phase 1 clinical trial involving AST - OPC1 (oligodendrocyte progenitor cells).

«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.

Now a University of Colorado Cancer Center study published online ahead of print in the journal Oncogene offers compelling evidence explaining this failure and offering a possible strategy for the use of retinoic acid or other retinoids against some breast cancers: Because early clinical trials are often offered to patients who have already tried other more established therapies, breast cancer cells may have been pushed past an important tipping point that offers retinoic acid resistance.

«These are optimistic results for one of the first targeted therapies for cancer stem cells,» says Antonio Jimeno, MD, PhD, investigator at the CU Cancer Center, director of the university's Cancer Stem Cell - Directed Clinical Trials Program, and principal investigator of the clinical trial at the CU Cancer Center site.

But scientists are making progress in refining these therapies, and the first ever trial of fetal stem cells injected directly into the brain is currently under way in children with Batten disease, a rare and fatal illness of the nervous system.

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.

The researchers hope that ultimately human trials will prove the efficacy of the OH14 compound in sensitising tumour cells and cancer stem cells to existing drug - based therapies thus disabling tumours from seeding new growth after treatment.

A phase 3 trial of lenalidomide / dexamethasone with elotuzumab (Empliciti ™), an antibody therapy that attacks myeloma cells directly and spurs the immune system to launch an attack of its own.

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.

Two of 10 children treated with gene therapy for SCID in a French trial develop leukemia, researchers announced in 2002, and it is discovered that the virus had inserted genes in several unexpected places around the genome, leading the cells to become cancerous.

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]

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..

Clinical trials that charge enrollees to participate are ostensibly aimed at giving patients early access to promising therapies — often in the fields of stem cells or aging reversal — that are too unusual or have too little profit potential to get funding from traditional sources such as companies, foundations, or the National Institutes of Health.

Combining radiation therapy with chemotherapy for patients with limited metastatic non-small cell lung cancer (NSCLC) may curb disease progression dramatically when compared to NSCLC patients who only receive chemotherapy, according to a new randomized phase II clinical trial reported today at the 59th Annual Meeting of the American Society for Radiation Oncology (ASTRO).

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.

ACT, which has almost gone under several times in recent years, may now be on the verge of conducting the nation's first trial of an embryonic stem cell therapy, says Chief Scientific Officer Robert Lanza.

Most gene - therapy trials use viruses to deliver genes to a patient's cells, and most of those viruses are retroviruses, which have the ability to neatly splice their genes — and the human gene they're carrying — into a cell's chromosomes.

A clinical trial in the Republic of Korea for patients with degenerative eye diseases is the first to test the safety of an embryonic stem cell therapy for people of Asian descent.

Graham looked to the Internet for treatment options and came across Duke University oncologist Keith Sullivan, who was comparing standard chemotherapy and stem cell therapy as part of a large - scale clinical trial for scleroderma.

A panel of top science administrators and stem cell scientists met on Friday with Davide Vannoni, the president of an Italian foundation providing highly controversial stem cell treatments, in a first effort to hash out the details of a government - sponsored clinical trial of the therapy.

Vanderbilt - led research, as part of an international, multicenter trial, found regular blood transfusion therapy significantly reduces the recurrence of silent strokes and strokes in children with sickle cell anemia who have had pre-existing silent strokes, according to study results released today in the New England Journal of Medicine (NEJM).

Results from a clinical trial investigating a new T cell receptor (TCR) therapy that uses a person's own immune system to recognize and destroy cancer cells demonstrated a clinical response in 80 percent of multiple myeloma patients with advanced disease after undergoing autologous stem cell transplants (ASCT).

Clinical trials of nitric oxide therapy to ease pain in sickle cell patients have yielded conflicting results, with some patients reporting increased pain.

Over the past two years, investigators from the Perelman School of Medicine at the University of Pennsylvania have reported results from a human trial in GBM using chimeric antigen receptor (CAR) T cell therapy, through which patients» own T cells were engineered to track down and kill cancer cells that express a tumor - specific protein known as EGFRvIII.

Jonathan Appleby, GSK project leader for the ADA - SCID trial and head of cell and gene therapy at the rare diseases unit, says there are many differences between working on this kind of trial and working with one for a common disease.

Genetically modified «hunter» T cells successfully migrated to and penetrated a deadly type of brain tumor known as glioblastoma (GBM) in a clinical trial of the new therapy, but the cells triggered an immunosuppressive tumor microenvironment and faced a complex mutational landscape that will need to be overcome to better treat this aggressive cancer, Penn Medicine researchers report in a new study this week in Science Translational Medicine.

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.

If these preclinical studies are successful, the researchers plan to further develop their CAR T cell therapy and test its safety and efficacy for different types of metastatic cancer in upcoming clinical trials.

In recent clinical trials, CAR T cell therapy has dramatically improved the outcomes of blood cancer patients with advanced, otherwise untreatable forms of leukemia and lymphoma.

The trial, published in Circulation Research, was designed to test the feasibility and side effects of a genetically - enhanced stem cell therapy to repair and regenerate lung blood vessels in PAH.

Canadian researchers have published promising results of the first clinical trial in the world of a genetically - enhanced stem cell therapy for pulmonary arterial hypertension (PAH).

Dr. Stewart is also leading the first clinical trial in the world of a genetically - enhanced stem cell therapy for heart attack.

A number of research teams are putting other experimental stem cell therapies through stringent clinical trials.

After preclinical studies, a gene therapy trial for SCID - X1 was initiated, based on the use of complementary DNA containing a defective γc Moloney retrovirus — derived vector and ex vivo infection of CD34 + cells.

Past clinical trials of stem cell therapies for chronic stroke patients used cells derived from tumors in humans and brain tissue from fetal pigs.

In contrast, the safety record of hematopoietic stem cell gene therapy is less clear, with a significant frequency of gene - therapy induced oncogenesis or clonal outgrowth reported in several hematopoietic stem cell trials [56], [57].

«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.»

Given the ongoing adoptive therapy trial of CD4 + T cells treated with R5 - ZFNs and the anti-viral success of the recent ccr5Δ32 bone marrow transplant in an HIV - infected patient [6], we sought to determine if cxcr4 could be genetically disrupted simultaneously with ccr5.

The vector is a self - inactivating lentivirus — a member of a family of viruses that can insert genes into mammalian cells and drive expression of those genes — that has been engineered to avoid triggering the development of leukemia, a complication seen in previous gene therapy trials for immunodeficiency syndromes, including WAS.

Updated results from a global clinical trial of the CAR T - cell therapy for ALL reveal that children and young adults continued to show high rates of durable, complete remission.

Pr J.A. Sahel (chief of the «Institut de la Vision» — Inserm, CNRS, Université Paris VI), a member of the French Académie des Sciences, then presented a cell therapy phase I clinical trial project for retinitis pigmentosa developed in collaboration with I - Stem.