Drug Discovery World

FEBRUARY 28, 2024

Induced pluripotent stem cell (iPSC)-derived cells are a significant resource for disease modelling. They allow the use of cells directly from affected patients and have the potential to guide these cells towards multiple lineages.

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Drug Discovery World

APRIL 8, 2024

Their research signals a transformative leap in liver disease management, addressing both inflammation and fibrosis. Metabolic dysfunction-associated steatotic liver disease (MASLD) is a burgeoning global health concern, posing a significant threat to public health and escalating the burden on healthcare resources.

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Drug Discovery World

SEPTEMBER 21, 2023

A research team from VIB-KU Leuven and the UK Dementia Research Institute has discovered how neurons die in Alzheimer’s disease, opening new pathways for potential future treatments. Alzheimer’s disease (AD) has been classified as public health priority by the World Health Organisation (WHO).

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Drug Discovery World

SEPTEMBER 12, 2023

Twist Bioscience and Ono Pharmaceutical will combine their efforts to discover and develop novel antibodies for the treatment of autoimmune diseases. This collaboration… has the potential to lead to future breakthrough treatment options for patients with autoimmune diseases.”

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Drug Discovery World

NOVEMBER 23, 2022

Scientists have found a new drug treatment that can slow the progression of neurodegenerative disease in mice. The breakthrough research may offer fresh hope in tackling currently untreatable conditions such as Alzheimer’s disease (AD). .

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Drug Discovery World

MARCH 21, 2024

Focusing on rare conditions could significantly reduce the burden of kidney disease, according to a new study led by University College London (UCL) and the UK Kidney Association. The study, published in The Lancet to mark World Kidney Day on 14 March, draws on the largest rare kidney disease dataset ever created.

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Drug Discovery World

FEBRUARY 29, 2024

For Rare Disease Day 2024, Diana Spencer provides a round-up of the latest breakthroughs in the treatment of rare disease. million from the French government to advance the development of a gene therapy for the treatment of cerebrotendinous xanthomatosis (CTX), a rare neurodegenerative disease.

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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 15, 2024

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. The findings were presented at the AD/PD 2024 Conference in Lisbon.

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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

APRIL 8, 2024

Class B GPCRs are recognised as important targets for new medicines, particularly in chronic endocrine and metabolic diseases. grant supports drug discovery in metabolic diseases appeared first on Drug Discovery World (DDW). Diana Spencer, Senior Digital Content Editor, DDW The post €1.6m

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Drug Discovery World

JANUARY 31, 2024

In aggregate, the 72-week data with atacicept are consistent with a profile of true disease modification in IgAN. These cytokines are members of the tumour necrosis factor family that promote B-cell survival and autoantibody production associated with certain autoimmune diseases, including IgAN and lupus nephritis. PLOS ONE 2015). “We

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Drug Discovery World

NOVEMBER 22, 2022

Currently, no effective cure or treatment for Huntington’s disease exists. Download this new App Note from bit.bio to learn: How Charles River Laboratories characterised the phenotype of a human iPSC-derived Huntington’s disease model from bit.bio over 38 days in vitro with high-density microelectrode arrays from MaxWell Biosystems.

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Drug Discovery World

FEBRUARY 27, 2024

Arvinas has commenced dosing in the Phase I clinical trial of ARV-102, its first oral PROTAC (PROteolysis-TArgeting Chimera) protein degrader in development to treat neurodegenerative diseases. The post First PROTAC degrader for neurodegenerative disease enters trials appeared first on Drug Discovery World (DDW).

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Drug Discovery World

FEBRUARY 8, 2024

COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune and inflammatory diseases. COUR’s therapies are based on its antigen-specific immune tolerance platform and are designed to reprogramme the immune system to address the underlying root cause of immune-mediated diseases.

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Drug Discovery World

MARCH 7, 2024

Research published in Cell Genomics has shown that prostate cancer includes two different subtypes of the disease, also known as evotypes. This study is really important because until now, we thought that prostate cancer was just one type of disease.

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Drug Discovery World

MARCH 6, 2024

“Their guidance will be invaluable as we soon advance our lead candidate, RNDP-001, into the clinic for the treatment of Parkinson’s disease.” The potentially curative nature of RNDP-001 for Parkinson’s disease could dramatically alter outcomes for patients with very few treatment options.”

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Drug Discovery World

SEPTEMBER 29, 2023

AI-driven drug discovery company Insilico Medicine and the University of Cambridge have discovered an approach to identify therapeutic targets for human diseases associated with protein phase separation (PPS). “Even more difficult has been to clarify the exact nature of its association with human disease.

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Drug Discovery World

APRIL 4, 2024

The US Food and Drug Administration (FDA) has approved Vafseo (vadadustat) Tablets for the treatment of anaemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months.

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Drug Discovery World

APRIL 19, 2024

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA.

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Drug Discovery World

FEBRUARY 5, 2024

One-third (32%) of rare disease patients are not aware they can access unlicensed medicines through Early Access Programmes, new research has revealed. The new data was released by pharma company Clinigen to mark the start of Rare Disease Month.

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Drug Discovery World

FEBRUARY 13, 2024

Systemic sclerosis is a chronic, progressive, autoimmune disease characterised by inflammation and fibrosis, such as uncontrolled scar tissue formation, in skin and various internal organs. Around 100,000 people in the EU are affected by SSc and up to 80% of these may develop interstitial lung disease (ILD).

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Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . There are currently no approved disease modifying therapies for Huntington’s Disease. In 2019, BV-101 was granted orphan drug designation in the European Union. .

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Drug Discovery World

NOVEMBER 8, 2023

Over 19 million 1 hearts succumb to cardiovascular disease yearly, while around 10 million 2 lives are claimed by cancer every year. By prioritising treatments for diseases with high mortality rates a standardised approval process can be established. This was the challenge embraced by researchers at John Hopkins University.

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Drug Discovery World

NOVEMBER 14, 2023

DDW’s Megan Thomas looks at how different diseases will benefit from the success of therapeutic antibodies. The work is continuous for this disease target. On 6 January 2023, the FDA approved lecanemab-irmb via the Accelerated Approval pathway for the treatment of Alzheimer’s disease.

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Drug Discovery World

SEPTEMBER 5, 2023

The UK’s National Institute for Health and Care Excellence (NICE) has recommended Elfabrio (pegunigalsidase alfa) as an option for treating Fabry disease (or alpha-galactosidase deficiency). Fabry disease is a rare genetic disease affecting approximately 1,150 people in England.

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Drug Discovery World

DECEMBER 6, 2022

CAR-T therapy can be used to eliminate unwanted cells that cause autoimmunity, according to the results of a new study in mice with an autoimmune disease similar to multiple sclerosis (MS). . I believe that this is a fully treatable disease, and CAR-T cells may be the way toward much better therapeutics.” .

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Drug Discovery World

FEBRUARY 16, 2024

AMPLY works with a network of disease and biotech partners to help advance its programmes. to tackle complex diseases appeared first on Drug Discovery World (DDW). Its MDR-TB project is a collaboration with St George’s University London and its AML project is being run with a Swiss-based biotech partner.

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Drug Discovery World

OCTOBER 31, 2023

Professor Andy Whiting and Dr Tony Lockett discuss how specific levels of a protein could be used to advance drug development in neurodegenerative diseases. These parameters aim to measure the progression of disease over time by looking at the functional or observable change in a patient.

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Drug Discovery World

DECEMBER 11, 2023

MC2 Therapeutics has completed enrolment in its Phase II MC2-25 Ph2 chronic kidney disease-associated pruritus trial, with topline results expected in Q2 2024. MC2-25 CKD is a first-in-class drug candidate and could represent a potential breakthrough in the understanding and treatment of urea associated skin diseases.

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Drug Discovery World

OCTOBER 3, 2023

The US Food and Drug Administration (FDA) has announced a pilot programme with the aim of accelerating the development of novel drug and biological products for rare diseases. The post FDA pilot programme to boost rare disease therapies appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 8, 2023

The UK lags comparable countries, including France and Germany, on the degree of availability of treatments for rare diseases, according to a new report. The report by the BIA and consultancy firm PwC has highlighted the progress that has been made in improving access to rare disease treatments in the UK, and the challenges that remain.

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On Medicine

FEBRUARY 28, 2024

What is Perthes’ disease? Perthes’ Disease is a rare condition that affects the developing femoral head (the “ball” of the “ball and socket” hip joint) of a child due to poor blood supply. This in turn can cause significant pain for children with Perthes’ disease and limit their activity levels significantly.

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Drug Discovery World

DECEMBER 7, 2023

Loss-of-function variants of HSD17B13 are known to be associated with reduced incidence risk and severity of multiple liver diseases. This is the first example of an OOC provider’s data supporting clinical progression of a drug for metabolic, fibrotic liver disease.

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Broad Institute

APRIL 2, 2024

Scientists link certain gut bacteria to lower heart disease risk By Allessandra DiCorato April 2, 2024 Breadcrumb Home Scientists link certain gut bacteria to lower heart disease risk Study finds several species of cholesterol-metabolizing bacteria in people with lower cholesterol levels. Online April 2, 2024. DOI:10.1016/j.cell.2024.03.014.

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Drug Discovery World

FEBRUARY 28, 2023

A new strategic guide , intended to help manufacturers bring rare disease products to market, has been launched to mark Rare Disease Day (February 28). Craig Caceci, Terebellum’s Managing Director, commented: “Bringing any product to market, particularly those used to treat rare and orphan diseases is a challenge enough.

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Science Daily: Pharmacology News

MARCH 28, 2024

Young to middle-aged women who reported drinking eight or more alcoholic beverages per week--more than one per day, on average--were significantly more likely to develop coronary heart disease compared with those who drank less, finds a study presented at the American College of Cardiology's Annual Scientific Session.

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