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

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). ? . Hunter syndrome .

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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. One diagnostic example that I discussed in my presentation is autism.

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Drug Discovery World

AUGUST 29, 2023

A stem cell derived investigational therapy for treating Parkinson’s disease has shown promise in early clinical trials. BlueRock Therapeutics, a subsidiary of Bayer, has announced details of the positive data from a Phase I clinical trial for bemdaneprocel (BRT-DA01).

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Drug Discovery World

FEBRUARY 23, 2024

Efficacy was evaluated in the Phase III trial FLAURA2 in 557 patients with EGFR exon 19 deletion or exon 21 L858R mutation-positive la/mNSCLC and no prior systemic therapy for advanced disease. The post Combination therapy reduced risk of lung cancer progression by 38% appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP). For prophylactic ERT, Adyznma is administered to help reduce the risk of disease symptoms.

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Drug Discovery World

MARCH 2, 2023

The Avacta Therapeutics Science Day brought together senior company scientists and international oncology experts to provide an overview of the current development of the company’s cancer drug pipeline and the latest innovations in targeted oncology. The company anticipates the drug will enter Phase I clinical trials in 2024.

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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

SEPTEMBER 16, 2022

A biologic therapy for very young children with moderate to severe eczema (or atopic dermatitis) has been shown to be safe and effective in an international trial. The therapy is already licensed in the UK for adults and children aged six to 18, and was approved by the FDA in June 2022 for use in younger children.

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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.

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Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? Curbishley explains that as the wave of new therapies in development continues to increase, global pressures on manufacturing availability will become more acute.

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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.

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Drug Discovery World

JULY 30, 2024

In new revelations about glucagon-like peptide-1 (GLP-1) drugs like Ozempic, a study has demonstrated the therapies could protect against dementia. The trial data was reported at the Alzheimer’s Association International Conference (AAIC) 2024, in Philadelphia, US.

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Drug Discovery World

NOVEMBER 3, 2023

News round-up for 30 October – 3 November by DDW Editor Reece Armstrong My news highlights this week feature a variety of exciting clinical trial results and launches that are examining therapies for much-needed disease areas, new drug approvals and even a space mission with a pharma twist.

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Drug Discovery World

AUGUST 2, 2023

Fifty percent of neuroblastoma patients have high-risk (HRNB) disease which has an overall survival of ~50% over five years. A novel Phase II trial incorporating Hu14.18 into induction therapy and additionally, within post-consolidation therapy, demonstrated outstanding patient outcomes with overall survival (OS) of 86.0%

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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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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

JUNE 28, 2023

Takeda has revealed favourable interim results from its Phase III crossover trial evaluating the safety and efficacy of TAK-755 (recombinant ADAMTS13) replacement therapy for the prophylactic treatment of congenital thrombotic thrombocytopenic purpura (cTTP). Acute TTP events have a mortality rate of >90%, if left untreated.

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Drug Discovery World

FEBRUARY 2, 2024

Sunday 4 February is World Cancer Day, an international awareness day led by the Union for International Cancer Control (UICC), so the round-up this week will focus on oncology, including the UK approval of a rare blood cancer drug and a novel production process for CAR-T therapies.

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Conversations in Drug Development Trends

APRIL 3, 2024

By: Sue Batchelor, Executive Director, Oncology Project Management Radiopharmaceuticals represent a cutting-edge frontier in oncology treatment, offering the promise of highly targeted therapy with the potential to revolutionize cancer care. Are you aware of the challenges you must address for a successful radiopharmaceutical trial?

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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. Bayer to advance Huntington’s Disease gene therapy trial . International start-ups receive funding from BioInnovation .

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Drug Discovery World

APRIL 25, 2023

University of California researchers have dosed the second participant in their clinical trial to identify a potential cure for HIV utilising CAR-T cell therapy. The trial is the first-in-human clinical study investigating the duo CAR-T cell therapy for the treatment of HIV. “We In 2021 there were an estimated 1.5

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Drug Discovery World

SEPTEMBER 16, 2022

A slightly quieter week for news in light of national events in the UK, but the vital work of the international drug discovery community goes on and new discoveries are being made all the time. . First biologic therapy for young children with severe eczema . Trial sheds light on combined treatment for colorectal cancer .

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Vial

JUNE 18, 2024

The success of clinical trials hinges on increasing access to participation by all eligible patients, including populations that have been underrepresented due to the barriers highlighted below. Representation, Underrepresentation & Diversity in Clinical Trials Studies indicate a need to increase patient accessibility to clinical trials.

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Drug Discovery World

DECEMBER 14, 2023

However, 50% of responding patients will relapse either due to evolution of CD19 negative disease or early loss of CAR T-cells so that only around 40% of patients are cured by the currently licensed CAR T-cell therapies. Genetically modified CAR T-cells targeting CD19 have shown impressive outcomes in relapsed/refractory ALL.

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H1 Blog

SEPTEMBER 26, 2023

Breaking Down Barriers to International Clinical Trials Global Disruptions, Health Equity & Data Sharing As the U.S. When time is of the essence, like in a global pandemic, how do you scale trials internationally for success? Additionally, clinical trials are no longer confined to one country or region.

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Drug Discovery World

MAY 6, 2024

billion on R&D during 2021, with Germany and Switzerland being the biggest contributors, and around one fourth of clinical trials worldwide were performed in Europe 1. The city is host to many innovative biotech companies, including relative newcomers Orexa, a company that recently launched its first Phase II clinical trial.

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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.

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Vial

MARCH 25, 2024

Introduction Worldwide Clinical Trials vs. Vial. Worldwide Clinical Trials is a mid-size, full-service global contract research organization (CRO) that works with biotech and pharma to advance new medications. With an international presence in nearly 60 countries, Worldwide is supported by over 3,400 team members.

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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.

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Drug Discovery World

SEPTEMBER 7, 2023

Over 100 subtypes of childhood cancer exist today and it represents the most common cause of disease-related death in children. 4 Recent regulatory initiatives in the United States and Europe have aimed to increase the study of novel anti-cancer therapies in children.

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Drug Discovery World

DECEMBER 13, 2022

A novel, patient-tailored monoclonal antibody therapy has shown promise in treating moderate to severe atopic dermatitis. . Patients who participated in a clinical trial of rocatinlimab showed positive results while taking the drug and up to 20 weeks after stopping therapy, Mount Sinai researchers have reported in The Lancet. .

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Drug Discovery World

DECEMBER 7, 2023

New guidance for the motor neuron disease (MND) drug discovery community has been published to ensure that future drug discovery and development efforts have the best chance of successfully translating from the lab to the clinic. Despite recent leaps forward in our understanding of MND biology, treatment options remain limited.

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Drug Discovery World

JANUARY 27, 2023

Enhertu has been approved in the EU as the first HER2 directed therapy for patients with HER2 low metastatic breast cancer. The drug is approved as monotherapy for adult patients who have received prior chemotherapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy.

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Vial

DECEMBER 11, 2023

Pathophysiological evidence links HCV infections to the risk of liver diseases such as hepatocellular carcinoma (HCC) and liver cirrhosis. Other inhibitors, currently in different stages of development (including clinical trials ), mainly target viral non-structural proteins or the internal ribosome entry site (IRES).

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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Drug Discovery World

MAY 30, 2024

Unlike existing anti-obesity therapies that compromise lean muscle mass, ATR-127 induces significant weight loss while preserving crucial lean body mass. Atrogi is also conducting trials into ATR-258, a novel b2adrenoceptor agonist for the treatment of type 2 diabetes and comorbidities.

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