Disease, International and Treatment

Data support one-year efficacy of ND0612 in Parkinson’s disease

Drug Discovery World

OCTOBER 3, 2024

New data from the Phase III BouNDless trial and its ongoing open-label extension (OLE) support the long-term efficacy of ND0612 in people with Parkinson’s disease (PD) experiencing motor fluctuations. The new clinical trial data was shared at the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS).

UK lags behind on access to treatments for rare diseases

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.

Disease

Disease Treatment International Pharmaceuticals

Dual-action treatment for fibrotic disease secures orphan drug status

Drug Discovery World

FEBRUARY 13, 2024

AM1476, a first-in-class, peripheral-acting serotonin receptor antagonist, has been granted US and EU Orphan Drug Designation as an anti-fibrotic treatment for systemic sclerosis. Around 100,000 people in the EU are affected by SSc and up to 80% of these may develop interstitial lung disease (ILD).

Disease

Disease Treatment Small Molecule Pharmacokinetics

New treatment slows progression of neurodegenerative disease

Drug Discovery World

FEBRUARY 16, 2023

Minoryx Therapeutics has revealed the findings from the first and largest international study to enroll adult male X-linked adrenoleukodystrophy patients. Treatment with leriglitazone also significantly reduced plasma levels of MMP-9, a marker of blood-brain barrier integrity.

Treatment

Treatment Disease Clinical Trials International

Flagship and Quotient partner on cardiovascular and renal disease therapies

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.

Disease

Disease Therapies International Drug Development

International Women’s Day: Female life science leaders

Drug Discovery World

MARCH 7, 2023

For International Women’s Day, DDW’s Diana Spencer celebrates some of the women in leadership making an impact on the drug discovery and life sciences sector. She has managed multinational projects for charities and not-for-profits to find new treatments for Malaria and led Innovate UK grants through to successful completion.

International

International Science Pharmaceuticals DNA

Actigen moves closer to clinical trial for rare disease treatment

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 .

Clinical Trials

Clinical Trials Treatment Trials Disease

Treatment potential for immunotherapy-induced cytokine release syndrome

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.

Treatment

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Research reveals therapeutic potential of mRNA in rare diseases

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.

Disease

Disease Research RNA Therapies

“Highly encouraging” results for treatment-resistant schizophrenia drug

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.

Treatment

Treatment Drugs Laboratories Pharmaceuticals

AI reveals prostate cancer is not just one disease

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 discovery was made by using artificial intelligence (AI) to help unlock new discoveries about the evolution of prostate cancer which could lead to the development of new treatments.

Disease

Disease Medical Schools DNA International

Add-on treatment for severe uncontrolled asthma approved

Drug Discovery World

APRIL 11, 2023

Owing to the complexity of severe asthma, many patients have unclear or multiple drivers of airway inflammation resulting in some patients not responding to the current standard of treatment, and being at increased risk of asthma attacks, hospitalisations, and a poor quality of life 3,4,5,6. Final draft guidance is subject to appeal.

Treatment

Treatment Clinical Trials Trials Science

New dermatitis treatment shows promising long-term results

Drug Discovery World

DECEMBER 13, 2022

Rocatinlimab inhibits OX40 – an immune molecule involved in activating inflammatory cells that play a key role in the development of atopic dermatitis and other inflammatory diseases. . All active dose cohorts also continued improving after week 16, and most patients maintained the response for at least 20 weeks off-treatment. .

Treatment

Treatment Clinical Trials Laboratories Therapies

Could NfL accelerate drug development for neurodegenerative diseases?

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.

Drug Development

Drug Development Disease Drugs Clinical Trials

Donanemab significantly slowed decline in early Alzheimer’s disease trial

Drug Discovery World

JULY 19, 2023

Eli Lilly has announced positive results of the TRAILBLAZER-ALZ 2 Phase III study showing that donanemab significantly slowed cognitive and functional decline in people with early symptomatic Alzheimer’s disease.

Disease

Disease Trials Clinical Trials Therapies

Macrophage cell therapy: a new hope for chronic liver disease patients

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.

Therapies

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FDA approves Sarclisa combination for multiple myeloma

Drug Discovery World

SEPTEMBER 27, 2024

The efficacy of the treatment was evaluated in the IMROZ trial in patients with newly diagnosed multiple myeloma who were not eligible for ASCT. The main efficacy outcome measure was progression-free survival (PFS) as assessed by an independent review committee based on International Myeloma Working Group criteria.

FDA Approval

FDA Approval FDA International Treatment

Potential treatment for patients with acute myeloid leukaemia

Drug Discovery World

MARCH 16, 2023

Overcoming Resistance to FLT3 Inhibitors in the Treatment of FLT3-Mutated AML. International Journal of Molecular Science, 21(4): 1537. Around one third of patients with AML are diagnosed with FLT3-mutations, which are associated with a higher risk of relapse and poor clinical outcome. and Leung, A. and Wang, H-G.

Treatment

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First-line AZ treatment for adults with biliary tract cancer approved

Drug Discovery World

FEBRUARY 9, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation to AstraZeneca for Imfinzi (durvalumab) in combination with gemcitabine and cisplatin in Great Britain for use as a first-line treatment of adults with locally advanced, unresectable or metastatic biliary tract cancer (BTC). versus 11.5%).

Treatment

Treatment Clinical Research International FDA

Drug candidates with blockbuster potential for CNS diseases

Drug Target Review

SEPTEMBER 19, 2024

Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). The pipeline currently includes five drug candidates, all of which have the potential to revolutionise the treatment of PD.

Disease

Disease Drugs Treatment Drug Development

First treatment for sickle cell side-effect in 20 years approved

Drug Discovery World

JULY 13, 2022

The first new treatment in 20 years that prevents a common, painful side-effect in patients with sickle cell disorder (SCD) has been issued interim acceptance by the Scottish Medicines Consortium (SMC). . Many people with sickle cell disease experience recurrent painful crises which can result in hospital admission.

Treatment

Treatment Hospitals Disease International

15% of Parkinson’s cases linked to genetic factors, study says

Drug Discovery World

AUGUST 6, 2024

Centogene has announced data from its Rostock International Parkinson’s Disease (ROPAD) Study revealing the genetic factors and prevalence of Parkinson’s disease (PD). The findings from the study indicate that approximately 15% of PD-related cases are tied to genetic variants, with the majority being linked to LRRK2 and GBA1.

Clinical Trials

Clinical Trials Disease International Trials

Deep dive into the gut unlocks new disease treatments

SCIENMAG: Medicine & Health

OCTOBER 20, 2023

Now an international team led by the Hudson Institute of Medical Research has found a way to determine which species are important and how they interact to create a healthy microbiome. The more diverse species in your gut, the better it is for your health. Credit: Hudson Institute of Medical Research The more diverse species […]

Treatment

Treatment Disease International Research

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

Trials

Trials Disease Clinical Trials Clinical Research

Engineered brain parasites could treat cognitive disorders

Drug Discovery World

JULY 31, 2024

Researchers have discovered a way of using engineered brain parasites to deliver treatments for neurological conditions across the blood-brain-barrier. The study – led by the University of Glasgow in collaboration with Tel Aviv University and an international team of researchers – represents an exciting new field of research.

Engineering

Engineering Treatment Disease International

Protective effect of naringin against radiation?induced heart disease in rats via Sirt1/NF??B signaling pathway and endoplasmic reticulum stress

Chemical Biology and Drug Design

JANUARY 15, 2024

Abstract This study was designed to explore the protective effect and mechanism of naringin (NG) on radiation-induced heart disease (RIHD) in rats. The results showed that NG treatment significantly attenuated the 20 Gy XR-induced decline of LVEF and LVFS and the elevation of LVIDs.

Disease

Disease International Regulations Treatment

Meet the Researcher: Dr Joanne Taylor

Drug Discovery World

AUGUST 28, 2024

JT: We are developing novel small molecule, disease-modifying, allosteric regulators to treat serious diseases such as Parkinson’s disease and other dementias. In Parkinson’s disease, we are focusing on Parkinson’s disease patients that carry mutations in the GBA1 gene that encodes the lysosomal enzyme, glucocerebrosidase.

Research

Research Small Molecule Disease Therapies

Stem cell therapy shows promise in advanced MS

Drug Discovery World

NOVEMBER 28, 2023

An international team of scientists has shown that a stem cell therapy for multiple sclerosis (MS) could prevent further damage to the brain. The stem cells are thought to reduce the inflammation that drives the disease. But this is an encouraging step towards a new way of treating some people with MS.”

Therapies

Therapies Clinical Trials Trials Treatment

Drugs for enlarged prostate could prevent neurodegeneration

Drug Discovery World

JUNE 25, 2024

A new study suggests that certain drugs commonly used to treat enlarged prostate may also decrease the risk for dementia with Lewy bodies (DLB) and potentially other neurodegenerative diseases. One of the most exciting things about this study is that we find that same neuroprotective effect that we saw in Parkinson’s disease.

Drugs

Drugs Disease International Production

This week in drug discovery (21 – 25 August)

Drug Discovery World

AUGUST 26, 2022

A treatment for Monkeypox, spider venom for drug discovery and funding for international start-ups are some of the stories in this week’s news round-up. A new study investigating a potential treatment for people who have been diagnosed with monkeypox has been launched by the same team who helped develop Covid-19 treatments.

International

International Drugs Clinical Trials Treatment

Renaissance Pharma licenses Hu14.18 for neuroblastoma

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%.

Licensing

Licensing Licensing Hospitals Therapies

This week in drug discovery (27 Nov – 1 Dec)

Drug Discovery World

DECEMBER 1, 2023

The key drug discovery news this week focuses on experimental approaches to the treatment of some of the most challenging diseases, including a mathematical model that can simulate Alzheimer’s progression and using CRISPR gene editing to ‘shred’ tumours. Can a plant-based discovery pave the way for new drug treatments?

Therapies

Therapies Drugs Disease International

First therapy for very rare blood clotting disorder gets US greenlight

Drug Discovery World

NOVEMBER 14, 2023

A very rare, inherited blood clotting disorder, cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting. For prophylactic ERT, Adyznma is administered to help reduce the risk of disease symptoms.

Therapies

Therapies FDA Approval Protein Production FDA

Non-viral gene therapy shows early efficacy in lung cancer

Drug Discovery World

OCTOBER 9, 2023

Positive Phase I trial data indicates that Reqorsa (quaratusugene ozeplasmid) has shown early efficacy for the treatment of non-small cell lung cancer (NSCLC). The data will be presented at the 2023 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Therapies

Therapies Clinical Trials Trials International

Patients show 91% response to allogeneic CAR-T therapy

Drug Discovery World

OCTOBER 1, 2024

Investigators saw a 91% overall response rate (ORR) in patients with relapsed/refractory multiple myeloma (RRMM) following treatment with allogeneic CAR-T therapy P-BCMA-ALLO1 and lymphodepletion. P-BCMA-ALLO1 is an investigational non-viral, stem cell memory T cell (TSCM)-rich allogeneic CAR-T cell therapy.

Therapies

Therapies Trials FDA Approval Clinical Trials

GLP-1 receptor agonist slows Alzheimer’s cognitive decline

Drug Discovery World

JULY 30, 2024

The trial data was reported at the Alzheimer’s Association International Conference (AAIC) 2024, in Philadelphia, US. GLP-1 receptor agonists have already been shown to help people manage diabetes, lose weight and lower their risk of heart disease , stroke and kidney disease.

Trials

Trials Clinical Trials Disease Science

This week in drug discovery (1-5 January)

Drug Discovery World

JANUARY 5, 2024

The top stories: China’s NMPA okays maribavir for post-transplant cytomegalovirus Takeda’s Livtencity (maribavir) has been approved in China for the treatment of adult patients with post-hematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT) cytomegalovirus (CMV) infection/disease.

Drugs

Drugs International FDA Regulations

First spaceflight mission for drug development lab

Drug Discovery World

NOVEMBER 1, 2023

Redwire will launch its in-space pharmaceutical manufacturing platform, PIL-BOX, onboard SpaceX’s 29th cargo resupply services mission (SpaceX-29) for NASA to the International Space Station (ISS).

Drug Development

Drug Development Pharmaceuticals Drugs Pharmaceutical Companies

‘Off-the-shelf’ CAR-T secures FDA advanced therapy designation

Drug Discovery World

SEPTEMBER 19, 2024

P-BCMA-ALLO1 is an investigational stem cell memory T cell (TSCM)-based allogeneic CAR-T cell therapy in Phase I/Ib clinical development for the treatment of patients with relapsed/refractory multiple myeloma.

Therapies

Therapies FDA Clinical Development International

AI and transcriptomics identify dual targets for ageing and cancer

Drug Discovery World

NOVEMBER 16, 2023

An international research team is the first to use artificial intelligence (AI) analysis to identify dual-purpose target candidates for the treatment of cancer and ageing. Of these, 22 were proposed as dual-purpose targets for anti-ageing and anti-cancer treatment with the same therapeutic direction. “We

Engineering

Engineering Treatment International Research

WHO, Ghana and Norway agree to accelerate actions to save lives in first International Strategic Dialogue on Noncommunicable Diseases and the Sustainable Development Goals

The Pharma Data

APRIL 12, 2022

WHO welcomes the establishment of a new Heads of State and Government Group to accelerate progress towards the SDG target for noncommunicable diseases ? a one-third reduction in “premature” deaths from diseases like diabetes, cancer, heart and lung disease and the promotion of mental health and well-being.

International

International Disease Government Production

Treating immune-mediated diseases with nanoparticles

Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

Disease

Disease Pharmaceuticals Therapies Production

Study finds dual receptor beta-agonist promotes healthy weight loss

Drug Discovery World

MAY 30, 2024

Atrogi’s CEO, Alexandra Ekman Ryding, said: “This publication highlights strong pre-clinical data relating to ATR-127 and validates our approach for the development of a novel modality for the treatment of obesity. We believe our pioneering dual receptor beta-agonist approach has the potential to transform the obesity treatment landscape.

Small Molecule

Small Molecule Treatment International Production

Condensate biology: advancing drug discovery for complex diseases

Drug Target Review

JULY 13, 2023

Drug discovery is a complex and vital field that continually seeks to identify new therapeutic targets and develop effective treatments. Dewpoint’s breakthrough was the realisation that certain condensates are associated with human diseases. Klein notes the importance of Dewpoint’s disease-agnostic approach.

Disease

Disease Small Molecule Cell Biology Medical Schools

Data support one-year efficacy of ND0612 in Parkinson’s disease

UK lags behind on access to treatments for rare diseases

Trending Sources

Dual-action treatment for fibrotic disease secures orphan drug status

New treatment slows progression of neurodegenerative disease

Flagship and Quotient partner on cardiovascular and renal disease therapies

International Women’s Day: Female life science leaders

Actigen moves closer to clinical trial for rare disease treatment

Treatment potential for immunotherapy-induced cytokine release syndrome

Research reveals therapeutic potential of mRNA in rare diseases

“Highly encouraging” results for treatment-resistant schizophrenia drug

AI reveals prostate cancer is not just one disease

Add-on treatment for severe uncontrolled asthma approved

New dermatitis treatment shows promising long-term results

Could NfL accelerate drug development for neurodegenerative diseases?

Donanemab significantly slowed decline in early Alzheimer’s disease trial

Macrophage cell therapy: a new hope for chronic liver disease patients

FDA approves Sarclisa combination for multiple myeloma

Potential treatment for patients with acute myeloid leukaemia

First-line AZ treatment for adults with biliary tract cancer approved

Drug candidates with blockbuster potential for CNS diseases

First treatment for sickle cell side-effect in 20 years approved

15% of Parkinson’s cases linked to genetic factors, study says

Deep dive into the gut unlocks new disease treatments

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Engineered brain parasites could treat cognitive disorders

Protective effect of naringin against radiation?induced heart disease in rats via Sirt1/NF??B signaling pathway and endoplasmic reticulum stress

Meet the Researcher: Dr Joanne Taylor

Stem cell therapy shows promise in advanced MS

Drugs for enlarged prostate could prevent neurodegeneration

This week in drug discovery (21 – 25 August)

Renaissance Pharma licenses Hu14.18 for neuroblastoma

This week in drug discovery (27 Nov – 1 Dec)

First therapy for very rare blood clotting disorder gets US greenlight

Non-viral gene therapy shows early efficacy in lung cancer

Patients show 91% response to allogeneic CAR-T therapy

GLP-1 receptor agonist slows Alzheimer’s cognitive decline

This week in drug discovery (1-5 January)

First spaceflight mission for drug development lab

‘Off-the-shelf’ CAR-T secures FDA advanced therapy designation

AI and transcriptomics identify dual targets for ageing and cancer

WHO, Ghana and Norway agree to accelerate actions to save lives in first International Strategic Dialogue on Noncommunicable Diseases and the Sustainable Development Goals

Treating immune-mediated diseases with nanoparticles

Study finds dual receptor beta-agonist promotes healthy weight loss

Condensate biology: advancing drug discovery for complex diseases

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