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The negative results from the Phase 3 study, called Ciffreo, come a little more than a month after Pfizer said a patient died in another trial of the gene therapy.
The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.
A new clinical trial of five children with inherited deafness found administering gene therapy in both ears led to restored hearing and speech, and additional gains including sound source localization, ability to hear in noisy environments, and for two children, abillity to appreciate music.
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.
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.
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.
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.
Before it is released onto the market, the development of any new drug or medical device must undergo rigorous testing , part of which involves clinical trials. Clinical trials are integral to making sure that any new therapy is both safe and effective for individuals, and volunteers are a vital part of the process.
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,
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. “As
Since a landmark paper in 2022, drugmakers have begun nearly a dozen trials of cell therapies for lupus, with more set to start. Here’s why their efforts are worth watching.
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.
MONDAY, May 6, 2024 -- An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says.Researchers recruited 14 people with Leber Congenital Amaurosis.
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.
Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.
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?
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?
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. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients.
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].
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.
The 7th World Conference on Targeting Phage Therapy 2024, two-day event dedicated to advancing the field of phage research and therapy will be hosted at Corinthia Palace Malta on June 20-21.
The company is working with trial researchers to investigate further. Pfizer said the patient, a young boy who was treated earlier last year, had died suddenly.
The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.
TUESDAY, June 25, 2024 -- An experimental stem cell therapy can essentially cure type 1 diabetes by restoring insulin production in some patients, early clinical trial results show.Seven out of 12 patients no longer needed daily insulin shots after.
Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?
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.
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.
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.
This month features many of the big reveals from the ACS fall 2023 first disclosures including Nurix’s BTK degrader and RAPT’s CCR4 autoimmune therapy. III clinical trials such as Vertex’s sodium channel inhibitor for acute pain and Denali Therapeutics’ CNS-penetrant eIF2B activator for ALS.
MONDAY, June 12, 2023 -- A clinical trial that’s attempting to discover a cure for sickle cell disease has found a new gene therapy to be safe and successful in four patients. Two of the patients were treated at Cleveland Clinic Children’s in.
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.
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.
An experimental Alzheimer’s therapy from Cassava Sciences is still being tested in two Phase 3 studies, even as the company has come under regulatory scrutiny.
How can they engage providers and raise awareness of gene therapies for the patients who need them most? png Listing Introduction Explore how incorporating genetic services can increase identification of patients who meet trial-specific inclusion/exclusion criteria, helping more patients benefit from life-changing therapeutics.
Discover how incorporating genetic counseling and genetic services can unlock larger, more diverse and better qualified patient populations for your clinical trials. Join us as we explore real-world examples of how biopharma organizations have leveraged genetic services to identify, engage, and enroll diverse clinical trial participants.
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. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients.
The study provides both mechanistic and preclinical evidence supporting the rapid initiation of clinical trials for patients with acute myeloid leukemia (AML) to test an existing medicine that inhibits the complex, called eganelisib, both alone and in combination with the most used AML chemotherapy, cytarabine.
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.
Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.
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.
Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.
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.
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.
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.
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