Sentences with phrase «therapy safety trial»

«First embryonic stem cell therapy safety trial in Asian patients.»

Not exact matches

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).

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 researchers caution that the booster therapy used in their new study will not be available on the market or even for use in human trials anytime soon; it must await years of animal testing for safety and effectiveness first.

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.

The CO-STAR (Hepatitis C Patients on Opioid Substitution Therapy Antiviral Response) trial sought to evaluate the efficacy and safety of elbasvir - grazoprevir for injection drug users.

The trial he conducted tested the safety of a therapy for ornithine transcarbamylase (OTC) deficiency, a rare disorder in which the liver lacks a functional copy of the OTC gene.

It typically takes many years to initiate such trials because of the stringent safety testing that must be done before testing in humans begins, but Reynolds said it may be possible to move faster as the therapy only involves modifying a patient's dietary intake and supplementing with a medium - chain triglyceride oil, both of which have no known side effects.

Research groups at universities and hospitals need to meet strict safety guidelines for clinical trials but some small private clinics are offering therapies to people without research or marketing approval.

These results are especially remarkable given that Cannata was participating in a Phase I safety trial that delivered the therapy at a relatively low dose.

The phase I and phase I / II trials aimed to measure the safety of adding HCQ to either chemotherapy, radiation therapy or targeted therapies, its effectiveness at inhibiting autophagy, and the potential clinical benefit of HCQ combination therapies.

The data safety monitoring board of the trial, as mandated by the study protocol, continues to monitor outcomes in patients with a Recurrence Score of 11 to 25 randomized to chemo - endocrine therapy or endocrine therapy alone.

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.

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.

The therapy was found to have an acceptable safety profile in all patients, with no clinical or laboratory signs of systemic cytokine release syndrome, a potentially serious toxicity that has been observed in other CAR trials.

In the current trial, two patients experienced an immune response to the gene therapy that did not result in safety concerns, and were treated with steroids.

Two new phase III clinical trials investigating the efficacy and safety of bitopertin, a glycine uptake inhibitor considered to be a promising new add - on therapy for treating negative symptoms in schizophrenia, failed to show a benefit of the drug over placebo.

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.

Dr. Timmerman, who holds the Effie Marie Cain Distinguished Chair in Cancer Therapy Research, has championed the use of SABR globally and has served as the lead investigator in several national trials designed to evaluate the efficacy and safety of SABR to treat cancer in the lung, liver, spine, and prostate.

Stricter safety procedures and new ways to weaken pathogens to reduce their risks are leading investigators in industry, universities, and government to take a new look at human challenge trials, which offer a powerful tool for studying diseases and potential therapies.

George notes, «Our goal in this trial was to evaluate the safety of the gene therapy product and secondarily, to determine if we could achieve levels of factor IX that could decrease bleeding events in patients.»

Women were not included in the first trial of gene therapy, which was mainly concerned with proving the safety of the technique.

The ENGOT - OV16 / NOVA trial evaluated the efficacy and safety of the PARP inhibitor niraparib as maintenance therapy in patients with recurrent ovarian cancer who respond to platinum - based chemotherapy.

Fischer also suspended his gene therapy trials to solve safety issues.

«The first year results from the CoreValve US Pivotal Trial support the safety and efficacy of this therapy in patients unsuitable for surgical aortic valve replacement,» said lead investigator Steven Yakubov, MD..

But many projects come under public and federal scrutiny — the recent fracas over gene therapy trials is a case in point (Science, 24 March, p. 2163), often because the manner in which they were conducted led to concerns over the safety of the recruited participants or the protection of their confidentiality.

To create a longer - lasting preventive therapy for severe hemophilia A-associated bleeding, a group of Japanese researchers launched a first - in - human Phase I trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ACE910, a bispecific antibody that mimics FVIII and works with other clotting factors to make the blood clot properly.

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.

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].

If their phase I clinical trial to test the safety and preliminary efficacy of this therapy gains approval, University of Pittsburgh researchers will test the therapy on 10 patients who suffer from chronic ischemic stroke — the most common form, in which clots block blood flow.

A Randomized, Phase III Trial to Evaluate The Efficacy and Safety of MK - 3475 (Pembrolizumab) as Adjuvant Therapy for Triple Receptor - Negative Breast Cancer with ≥ 1 cm Residual Invasive Cancer or Positive Lymph Nodes (yPN +) After Neoadjuvant Chemotherapy

The CBER is already utilizing in silico techniques to predict clinical outcomes and product safety, inform clinical trial designs, and optimize dosing, and is also implementing the Regenerative Medicine Advanced Therapy (RMAT) designation which provides resources to expedite promising regenerative medicines that address unmet needs.

«With so few patients aged 75 or older enrolled in clinical trials, critical information on the safety and effectiveness of new therapies in this age group is greatly lacking,» she said.

The goal of phase - I trials is to determine the safety and toxicity of a new therapy.

The first - in - human STEP ONE * trial is evaluating the safety and efficacy of ViaCyte's PEC - Encap (a.k.a. VC - 01 ™) product candidate, a stem cell - derived, encapsulated cell replacement therapy.

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.

«We have been very concerned about reports of patients using unproven medical therapies whose safety and efficacy have not been tested in systematic clinical trials,» he said.

ViaCyte's VC - 01 ™ product candidate, a first - in - class cell replacement therapy for the treatment of type 1 diabetes, is currently being evaluated in a Phase 1/2 trial called STEP ONE, or Safety, Tolerability, and Efficacy of VC - 01 Combination Product in Type One Diabetes.

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.

The ISSCR believes that the safety and efficacy of all stem cell and regenerative medicine therapies should be proven in systematic clinical trials, reviewed by the FDA, before they are sold to patients.

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.

These centers specialize in conducting clinical trials to evaluate the safety and effectiveness of new CF therapies.

Immune Design entered into clinical collaboration agreements with Merck, known as MSD outside the U.S. and Canada, to evaluate the safety and efficacy of two Immune Design immuno - oncology investigative agents, G100 and LV305, separately combined with KEYTRUDA ® (pembrolizumab), Merck's anti-PD-1 therapy, in Phase 1 trials in patients with non-Hodgkin's lymphoma (NHL) and melanoma, respectively.

Eugene Fine was also there with a poster on his just published pilot study on ketogenic diets and cancer — «Targeting insulin inhibition as a metabolic therapy in advanced cancer: A pilot safety and feasibility dietary trial in 10 patients.»

Targeting insulin inhibition as a metabolic therapy in advanced cancer: a pilot safety and feasibility dietary trial in 10 patients.

Seeking Safety therapy for adolescent girls with PTSD and substance use disorder: A randomized controlled trial.

This randomized clinical trial tests the safety and efficacy of daily vs intermittent antituberculosis therapy in HIV - positive patients.