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25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows. THURSDAY, Sept. People with hemophilia B saw their bleeding episodes.
Before a potential new therapy can be administered to patients, it must go through several clinical trial phases designed to test the drug or device for safety and effectiveness. For many new treatments, it will take approximately ten years for the therapy to progress from initial discovery to being approved.
25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows.People with hemophilia B saw their bleeding episodes. THURSDAY, Sept.
2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. 7 Recent clinical studies highlight promising developments in c-MET-targeting ADCs for NSCLC.
In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.
Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. Credit: Neumann EN, Bertozzi TM, et al.
Before any new treatment or therapy is able to be used by the patient population, it must go through the process of a clinical trial — this is the case for any new prescription drug, but also true for over-the-counter medications, medical devices, and more.
14, 2023 -- For people with severe alcohol use disorder, a new gene therapytrial could lead to an effective treatment that would involve chemically rebalancing the area of the brain associated with addiction. MONDAY, Aug. With alcohol alone,
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.
14, 2023 -- For people with severe alcohol use disorder, a new gene therapytrial could lead to an effective treatment that would involve chemically rebalancing the area of the brain associated with addiction. MONDAY, Aug. “With alcohol.
22, 2024 -- At-home brain stimulation therapy can safely and effectively treat severe to moderate depression, a new clinical trial shows.Rates of treatment response and depression remission were three times higher in people receiving. TUESDAY, Oct.
Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.
TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.“It’s It’s encouraging that this gene-editing treatment con.
An efficacy endpoint in oncology is a characteristic or variable that measures how beneficial a treatment is to a patient’s feeling, function, and survival in a clinical trial. They are essential for assessing whether new cancer therapies are safe and efficacious [1].
They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. Only three of the CAR-NK studies were for the treatment of solid tumours.
Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?
The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it.
What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? Despite their potential, ADSC therapy faces several challenges in preclinical studies. Is there a certain disease or condition that you believe stem cell therapy holds the most promise for?
In rheumatoid arthritis (RA) clinical trials, accurately measuring the effectiveness of treatments is critical for determining their value in managing this chronic and debilitating condition.
Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Before becoming available to patients, every medication, therapy, and intervention must undergo a rigorous approval process: the clinical trial. Clinical trials are divided into phases that are aimed at testing the safety and efficacy of every potential new treatment before it is able to enter the market.
What innovative approaches and technologies are anticipated to reshape immuno-oncology treatments in 2024? Immuno-oncology, notably through checkpoint inhibitors, has significantly reshaped cancer treatment over the past decade.
WEDNESDAY, May 29, 2024 -- People battling advanced colon cancers might have a new treatment option that could extend their survival, a new trial finds.A combination of two experimental immunotherapy drugs plus standard chemotherapy led to a median.
Dr David Baram from gene therapy company EmendoBio provides a snapshot of the history of therapeutic genetic engineering, explaining the early pitfalls and reasons for recent renewed optimism.
A group of independent experts wasn't convinced by clinical trial data from company Lykos Therapeutics, which is seeking FDA approval of MDMA-assisted treatment for post-traumatic stress disorder.
Four children have remained free of detectable HIV for more than one year after their antiretroviral therapy (ART) was paused to see if they could achieve HIV remission, according to new research.
1 Although pancreatic cancer is rarer than the abovementioned cancers, it claims over 50,000 lives each year in the United States because of a lack of effective treatments. Since then, two therapies have been approved for use in lung and breast cancer but it did not advance pancreatic cancer treatment.
Could you provide insights into the driving factors behind exploring novel treatments for cancer types? Based on this rationale we selected skin cancer, triple-negative breast cancer, and head and neck cancers as our initial indications for our Phase 1 trials. What aspects prompted this research direction?
Most of these conditions are genetic in origin and the majority have no effective treatment. 1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Symptoms were reversed in mouse models and a clinical trial is planned for later this year.
Before any new treatment is able to be approved, it is required to go through the vigorous testing process known as clinical trials. These trials are aimed at determining whether or not a drug, medical device, therapy, or other type of intervention is safe and effective for the public.
TUESDAY, June 6, 2023 -- Radiation therapy might not be necessary in treating some forms of rectal cancer and lymphoma, sparing patients from the toxic treatment, a pair of new clinical trials shows. One trial found that rectal cancer patients.
THURSDAY, June 27, 2024 -- Acupuncture can help breast cancer patients deal with the hot flashes that often accompany hormone therapy, a new clinical trial says.Nearly 2 in 3 women who got acupuncture during hormone therapy reported fewer and less.
The findings, from two trials involving tens of thousands of women, could influence guidelines around the use of hormonal treatment for menopausal symptoms.
They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. Only three of the CAR-NK studies were for the treatment of solid tumours.
This innovative approach involves the extraction of a patient’s own stem cells from either their bone marrow or blood , followed by a course of chemotherapy and antibody treatment. This study sought to shed light on the safety and effectiveness of aHSCT in routine healthcare settings, moving beyond the confines of clinical trials.
In the world of medical research, experts are constantly looking for new drugs, treatments, and therapies for a variety of conditions — but the process of getting these treatments approved is only possible if they are tested through clinical trials.
The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations. As therapies for the treatment of NASH in adult patients go this year to the U.S.
Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.
By: Christine Moore, PhD, Vice President, Neuroscience, Scientific Solutions Patients with borderline personality disorder (BPD) face a heightened risk of substance abuse and suicide, as well as substantial delays in receiving treatment.
Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.
FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis.
Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?
Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.
A new therapy is on the horizon for patients with metastatic colorectal cancer who have run out of treatment options. Credit: Vanderbilt University Medical Center A new therapy is on the horizon for patients with metastatic colorectal cancer who have run out of treatment options.
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