Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

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Drug Discovery World

MARCH 6, 2024

This second ODD allows research and development of a therapy for another form of the disease, Isolated Hypomyelination. Dan Williams, CEO at SynaptixBio, said; “This ODD is a huge boost to our efforts in tackling these devastating, life-limiting rare diseases.

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Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The post First gene therapy trial for Hunter syndrome opens in Manchester, UK appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 13, 2022

PacBio, developer of sequencing solutions, has announced its HiFi sequencing technology will be used in a pilot project for the Children’s Rare Disease Cohorts Initiative (CRDC) at Boston Children’s Hospital. . As causative variants are identified by CRDC researchers, results are returned to participants.

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Drug Discovery World

JANUARY 17, 2023

Genoscience Pharma, a clinical-stage biotech company developing lysosomotropic drug candidates for the treatment of cancer, fibrosis and auto-immune diseases through autophagy modulation has launched the ABE-LIVER study, along with trial sponsor Grenoble University Hospital. . The trial . The primary criteria is PFS.

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Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? 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.

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Drug Discovery World

SEPTEMBER 8, 2022

Under the partnership, Brainomix will leverage its e-ILD technology (automated artificial intelligence software) which has been trained to process high resolution chest CT (HRCT) data in patients with interstitial lung diseases. The post Phase II IPF trial to benefit from AI imaging appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JULY 5, 2024

The Division of Clinical Neuroscience at Oslo University Hospital (OUS) and the Swedish drug development company TikoMed have signed a research collaboration agreement focused on amyotrophic lateral sclerosis (ALS).

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Drug Discovery World

JANUARY 30, 2023

The primary objectives of MOVE were to evaluate the efficacy of palovarotene in reducing new HO volume, as assessed using whole-body computed tomography. “There is a significant need for a treatment specifically developed to help manage the progression of this disease.

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”

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Drug Discovery World

OCTOBER 10, 2023

Rejuvenate Biomed, a clinical-stage company advancing therapeutics to delay the onset of age-related diseases, has completed a six-week clinical proof-of-mechanism study with lead candidate RJx-01 for the treatment of sarcopenia.

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Drug Discovery World

SEPTEMBER 15, 2022

Findings from the SUNLIGHT trial have revealed that the combination of trifluridine/tipiracil plus bevacizumab showed a statistically significant improvement in overall survival compared to trifluridine/tipiracil alone. The trial was funded by Taiho Pharmaceutical in collaboration with Servier.

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Drug Discovery World

MAY 17, 2024

Deepika Khedekar , Associate Centralized Clinical Trial Lead at IQVIA Inc, looks at the challenges of utilising artificial intelligence (AI) in oncology clinical research. MIT research 4 tells us a hard truth: about 95% of these trials fall short, leaving behind a trail of broken hopes for cancer patients each year.

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Drug Discovery World

OCTOBER 10, 2022

A new clinical trial, led by the University of Glasgow and NHS Greater Glasgow and Clyde (NHSGGC), hopes to improve treatment options for patients with Crohn’s disease. . Crohn’s disease is a chronic condition which currently affects more than 115,000 people in the UK and causes inflammation of the lining of the digestive system.

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Drug Discovery World

JUNE 19, 2024

These dramatic improvements – if confirmed by further studies – may eventually provide renewed hope for a much-needed paradigm shift in the treatment of debilitating autoimmune diseases.” KYV-101 is a product candidate for use in B cell-driven autoimmune diseases.

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Drug Discovery World

JULY 12, 2023

Sequana Medical, a company targeting fluid overload in liver disease, heart failure and cancer, has enrolled the first patient in its MOJAVE study. The post Sequana announces first patient enrolled in heart failure trial appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JULY 5, 2024

Amgen has reported positive topline results from its randomised, double-blind, multicentre, placebo-controlled Phase III clinical trial evaluating the efficacy and safety of its treatment for Immunoglobulin G4-related disease (IgG4-RD).

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Drug Discovery World

AUGUST 28, 2023

DDW’s Diana Spencer speaks to Peter Hamley , Chief Scientific Officer of Samsara Therapeutics, to understand the role autophagy plays in neurodegenerative diseases and how boosting this process could increase healthspan. DS: Samsara’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials.

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Drug Discovery World

SEPTEMBER 17, 2024

Where do you work, and can you tell us what you’re working on at the moment?  I work at Poolbeg Pharma, where we are developing novel treatments for diseases with unmet medical needs and large addressable markets, with programmes in oncology, infectious diseases and endocrinology.

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Broad Institute

OCTOBER 23, 2023

The team aims to uncover how genetics and lifestyle affect cardiometabolic disease risk in South Asian populations and how to potentially decrease that risk, including through the development of new drugs and lifestyle modifications. “A

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Drug Discovery World

APRIL 10, 2023

RESP-X, Infex Therapeutics’ anti-virulence therapy to treat Pseudomonas aeruginosa (Pa) infections in non-cystic fibrosis bronchiectasis (NCFB) patients, has shown a promising interim safety and tolerability profile in its ongoing first-in-human Phase I clinical trial.

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Drug Discovery World

SEPTEMBER 3, 2024

Semaglutide has been found to reduce the rates of all-cause death, including as a result of Covid-19, in those who are overweight or have obesity and established cardiovascular disease without diabetes. Of the 833 deaths in trial participants, 58% were CV related, and 42% were non-CV related. .

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PPD

JUNE 7, 2023

Adults aren’t the only patients affected by the increasing prevalence of liver disease. 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.

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Drug Discovery World

MAY 14, 2024

DDW’s Megan Thomas talks to Dr Henrik Jespersen, Head of Melanoma Oncology, at Oslo University Hospital. HJ: I work as a clinical oncologist at Oslo University Hospital where I am head of melanoma oncology. In the clinic, I am always involved in clinical trials, both industry-initiated and investigator-initiated.

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Drug Discovery World

JULY 17, 2024

A drug commonly used to treat cystic fibrosis improved outcomes for patients with severe Covid-19 pneumonia in a recent clinical trial. The next step will be to conduct larger clinical trials, with the ultimate goal of approving dornase alfa for wider use.

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Conversations in Drug Development Trends

MARCH 20, 2024

One way to ensure customized disease management is to utilize pharmacogenomics (PGx) in your clinical trial. As the study of how an individual’s genes affect their response to drugs, pharmacogenomics is a critical factor in developing effective and safe drugs and dosages in clinical trials.

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Drug Discovery World

MAY 31, 2024

The selected news highlights this week report breakthrough findings from early phase research and development, mainly preclinical studies and Phase I clinical trials, including a drug that can prevent sepsis deaths, a microbiome based immuno-oncology candidate, and a plant-based therapy for diarrhoea.

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Drug Discovery World

AUGUST 24, 2022

The trial will see participants receive either a 14-day course of 600 mg tecovirimat twice daily or a matched placebo treatment. As a community-based trial, participants will take the treatment or placebo in their own homes. The trial looks to include at least 500 participants in the UK. .

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Drug Discovery World

JANUARY 31, 2024

Breye Therapeutics, a clinical-stage biopharmaceutical company developing novel oral therapies for retinal vascular diseases within ophthalmology, has started a Phase Ib/IIa clinical trial to investigate danegaptide, following oral administration, in patients suffering from diabetic macular oedema (DMO).

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Drug Discovery World

JUNE 27, 2024

Novo Nordisk has announced results from the Phase III FRONTIER2 trial of 254 adults and adolescents aged 12 years and over with haemophilia A, with and without inhibitors. The trial assessed both once-weekly and once-monthly prophylactic treatment with the investigational treatment Mim8. with once-weekly and 98.7%

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Drug Discovery World

JUNE 23, 2023

Duchenne is a relentlessly progressive, degenerative disease, robbing children of muscle function,” said Jerry Mendell, Paediatric Neurologist and principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital.

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The Pharma Data

DECEMBER 29, 2020

29, 2020 /PRNewswire/ — Phase 3 program in hospitalized patients to continue based on passing futility analysis on ability to reduce incidence of death or mechanical ventilation. As in earlier outpatient trial, immune status when patients entered the trial was a strong predictor of viral load and clinical outcomes.

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Drug Discovery World

AUGUST 2, 2023

Renaissance Pharma has announced its first development programme focused on Hu14.18, a humanised anti-GD2 monoclonal antibody (mAb), licensed from St Jude Children’s Research Hospital for the treatment of newly diagnosed high-risk neuroblastoma. A novel Phase II trial incorporating Hu14.18 and three-year event-free (EFS) of 73.7%.

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The Pharma Data

MARCH 16, 2022

The Independent Data Monitoring Committee recommended that the EMPA-KIDNEY trial be stopped early, following a formal interim assessment EMPA-KIDNEY is the largest and broadest dedicated SGLT2 inhibitor trial in chronic kidney disease to date Detailed results are expected to be presented later this year.

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PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. There’s little time to lose.

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biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. This New Alternative Model (NAM) provides a platform for High-Content Screening through disease modeling through the transplantation of primary patient tumors into immunocompromised lines.

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Drug Discovery World

MARCH 29, 2024

As the primary investigator for the Phase III SPOTLIGHT clinical trial, I witnessed firsthand the significant improvement in progression-free survival and overall survival for patients treated with Vyloy in combination with chemotherapy compared to those treated with placebo plus chemotherapy. positive population in Japan.”

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