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

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. In response, the BioIndustry Association (BIA)’s Rare Disease Industry Group (RDIG) is calling for the UK to improve access to new treatments for patients.

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Drug Discovery World

MARCH 24, 2023

Israeli researchers have developed a drug that is effective in treating a rare genetic muscle disease. The team demonstrated that the disease is caused by a mutation in the gene encoding HMG CoA reductase, inhibiting the enzyme’s activity. Other patients, some of whom are already in late stages of the disease, are awaiting treatment.

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Drug Discovery World

MARCH 14, 2023

Teitur Trophics, a biotech company developing new treatments for patients suffering from neurodegenerative diseases, has complete a €28M Series A financing. The financing was co-led by Sunstone Life Science Ventures and Sound Bioventures, with participation from new investors, Industrifonden, Innovestor’s Life Science Fund and P53 Invest.

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Drug Discovery World

AUGUST 20, 2024

The US Food and Drug Administration (FDA) has approved axatilimab-csfr (Niktimvo), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD).

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Drug Discovery World

JULY 9, 2024

The Scottish Medicines Consortium (SMC) has published final advice on two rare disease therapies. Elfabrio (pegunigalsidase alfa), a novel enzyme replacement therapy (ERT), has been approved as a treatment option for Fabry disease in adults. Birch bark extract gel EB is the name for a group of rare, genetic skin disorders.

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Drug Discovery World

FEBRUARY 16, 2023

X-linked adrenoleukodystrophy (X-ALD) is an orphan inherited neurodegenerative disease. Disease progression X-ALD male patients (paediatric and adult) develop cerebral lesions that can evolve to progressive cerebral ALD (cALD), which is devastating and leads to death within two to four years.

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Drug Discovery World

AUGUST 19, 2024

The discovery of a new regulator affecting immune cells could lead to new treatments to reduce inflammation in diseases including arthritis and severe Covid-19. This activity triggers responses which are finely balanced, to protect against disease and infection, and to reduce cell-damaging inflammation.

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Drug Discovery World

SEPTEMBER 22, 2022

Nefecon has become the first and only approved treatment in the EU for primary immunoglobulin A nephropathy (IgAN), a rare, progressive kidney disease. Primary IgAN, also known as Berger’s disease, is a chronic, debilitating and life-threatening kidney disease.

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Drug Discovery World

AUGUST 20, 2024

Positive results from multiple in vivo studies have shown the impact of sphingosine kinase-2 (SPHK2) inhibition in various models of metabolic disease. It has also received orphan drug designation from the FDA for the treatment of cholangiocarcinoma and has undergone studies in advanced cholangiocarcinoma (Phase IIa) and prostate cancer.

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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

AUGUST 28, 2024

Under the collaboration, the companies will analyse somatic mutations that occur in diseased patient tissue to inform the discovery and development of therapies for cardiovascular and renal diseases. The collaboration was the first initiated under Flagship’s Pioneering Medicines strategic partnership with Pfizer.

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Drug Discovery World

JANUARY 30, 2023

Cell and gene therapies (CGT) are transforming the way diseases are treated. The post Webinar: Transforming disease treatment with CGT appeared first on Drug Discovery World (DDW). As research progresses it is hoped that cell and gene therapies will offer innovation for the future.

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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

AUGUST 19, 2024

Researchers have uncovered how Flaviviruses like Dengue and Zika propagate, suggesting the possibility of new treatment strategies. With the global risk of such virus-borne diseases increasing, DENV alone is known to cause approximately 390 million infections and 100 million symptomatic cases annually, and JEV around 70,000 cases each year.

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Treatment Virus Vaccine Disease 148

Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. Most of these conditions are genetic in origin and the majority have no effective treatment.

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Drug Discovery World

JANUARY 5, 2024

KYV-101 is an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate for use in B cell-driven autoimmune diseases. KYV-101, specifically targets CD19, a protein expressed on the surface of B cells, which is involved in various types of autoimmune diseases.

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Drug Discovery World

JUNE 26, 2024

Scientists at the University of Bristol have uncovered a new disease driving mechanism which they are aiming to target to help treat the development of atherosclerotic plaques. This unique award will allow us to develop a therapeutic to target this novel non-coding RNA, and validate its use in patients with heart disease.”

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Treatment Research Medical Schools RNA 148

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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Treatment Medical Schools Trials Clinical Trials 148

Drug Discovery World

AUGUST 8, 2024

Additionally, MTX-463 was granted orphan drug and fast track designations by the FDA, underscoring the potential of WISP1 to address significant unmet needs in treating rare fibrotic diseases, including idiopathic pulmonary fibrosis (IPF).

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Drug Discovery World

JULY 30, 2024

A new partnership between NHS Scotland, AstraZeneca and the Universities of Dundee and Glasgow hopes to accelerate research into treating chronic kidney disease. The partners will work together on new medicines to slow the progression of chronic kidney disease, reducing the risk that patients might need dialysis or kidney transplantation.

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Disease Research Government Clinical Trials 130

Drug Discovery World

AUGUST 28, 2024

It is approved for the first-line treatment of adult patients with unresectable or metastatic cancer, who are eligible for platinum-containing chemotherapy. I look forward to seeing the treatment combination implemented as a first-line regimen in the clinical setting.” months compared to 16.1 The median PFS of 12.5

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Drug Discovery World

JUNE 28, 2024

A new Phase II trial, REGENERATE-PD, will study AB-1005, an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of moderate-stage Parkinson’s disease. The trial will include an estimated 87 subjects with study sites located in the US, UK and Europe.

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Therapies Disease Medical Schools Trials 148

Drug Discovery World

JULY 14, 2022

Biotech company Actigen has moved closer to clinical trials for rare disease treatment for Hunter syndrome, following a pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA). ? . Official comments .

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Clinical Trials Trials Treatment Disease 130

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

JUNE 11, 2024

Researchers have discovered a new biological pathway in inflammatory bowel disease (IBD) and related conditions that can be targeted using existing drugs. About 5% of the world’s population are currently affected by an autoimmune disease, such as IBD, the umbrella term for Crohn’s disease and ulcerative colitis.

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Chemical Biology and Drug Design

DECEMBER 15, 2023

The figure depicts the neuroprotective role of exendin-4 in Alzheimer's disease and Parkinson's disease. Exendin-4's diverse therapeutic targets suggest its potential therapeutic uses in neurodegenerative ailments like Alzheimer's disease and Parkinson's disease and have garnered an increasing amount of attention.

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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

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

APRIL 30, 2024

Four new LifeArc Translational Centres for Rare Diseases have been created to give people living with rare medical conditions access to improved tests and treatments. Globally, there are more than 300 million people living with rare diseases. However, rare disease research can be fragmented. The £9.4 ($11.7)

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Drug Discovery World

JULY 24, 2024

ALSFRS-R score, a predictor of survival time, and ALSAQ-40 score, a disease-specific health-related quality of life instrument, changed minimally over the 48-week reporting period which may indicate a slowing of disease progression in this small patient cohort. Read the article in PLoS ONE.

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Trials Disease Hospitals Clinical Trials 130

Drug Discovery World

JANUARY 18, 2024

Poolbeg Pharma, a biopharmaceutical company focused on the development and commercialisation of medicines targeting diseases with a high unmet medical need, has announced promising in vivo results for POLB 001 in addressing cancer immunotherapy-induced cytokine release syndrome (CRS). million cases of cancer by 2030 1,2.

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SCIENMAG: Medicine & Health

FEBRUARY 9, 2024

CLEVELAND—University Hospitals (UH) Brain Health & Memory Center is now treating patients with LEQEMBI® (lecanemab), a Food and Drug Administration-approved medication for the treatment of Alzheimer’s disease.

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Drug Discovery World

NOVEMBER 28, 2023

An experimental drug has shown potential as a disease-modifying therapy for Parkinson’s disease, according to a new study published in Nature Communications. Parkinson’s disease is highly associated with mitochondrial dysfunction. Mission is planning to initiate a MTX325 Phase I trial in humans in early 2024.

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Drug Discovery World

FEBRUARY 19, 2024

PrecisionLife and the Metrodora Institute have agreed a partnership to jumpstart the diagnosis and treatment options for a range of complex, chronic diseases. The diseases include long Covid, myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), amyotrophic lateral sclerosis (ALS), Sjögren’s syndrome, and many more.

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Drug Discovery World

JANUARY 8, 2024

Milan-based Newron Pharmaceuticals has reported “exceptional” one-year results from study 014/15 into evenamide as an add-on to antipsychotics for the management of treatment-resistant schizophrenia (TRS). More than 70% of the patients experienced clinically important reduction in disease severity.

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Chemical Biology and Drug Design

FEBRUARY 2, 2024

Abstract Existing chemotherapy for neglected tropical diseases (NTDs) can often be toxic and ineffective, highlighting the necessity for new treatments. Their use in combination therapy decreases the concentration of the reference drug used. Terpenes are secondary metabolites with pharmacological potential.

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