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Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinicaltrials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
Clinicaltrials are expensive, slow and often limited by outdated design constraints. They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data. Accelerating timelines and de-risking development decisions across the pipeline.”
Advanced strategies and tools are being implemented to establish the safety and efficacy of new therapeutic modalities, with the development of new biomarkers becoming one of the most relevant approaches for enhancing the precision and utility of early-stage studies.
Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA ClinicalTrials Deals Drug Pricing Gene Therapy An article from Dive Brief Actithera draws new investors to radiopharma drug pitch The four-year-old biotech raised about $75 million in a Series A round that involved nine venture capital firms.
The journey of cell and gene therapies from preclinical discovery to clinicaltrials is complex and challenging, impacting every team member involved, from researchers in the lab to patients receiving treatment. These digital platforms are designed to manage and automate critical trial processes, especially related to drug management.
As clinicaltrials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment.
Its ability to maneuver persistent drug development challenges, like patient recruitment, trial complexity and rising costs, will ultimately determine its success. This blog explores the value of functional service provider (FSP) models and how they help biotech companies augment their clinicaldevelopment needs.
It involves a dynamic and often unpredictable process where every stage, from target identification to clinicaltrials, generates vast amounts of data. Additionally, AI-driven predictive modelling can shorten the preclinical phase by simulating biological responses, leading to more targeted and efficient clinicaltrials.
“Protocol development can be a complex and meticulous process,” he explains, “as it’s crucial to ensure that trial candidates meet the necessary inclusion criteria.” Prior treatments and patient histories often introduce variables that can affect outcomes, making thoughtful trial design essential for generating reliable data.
In just two years, CTMC has advanced eight therapies into clinicaltrials, harnessing genetic engineering to enhance T-cell effectiveness in the fight against cancer. Ive been involved in therapeutics development for over 25 years, working with small, medium, and large biotech companies.
Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. This trial will build on previous positive data from the earlier Phase 1/2 HALOS study and is designed to rigorously assess the potential of ION582 as a disease-modifying therapy. “We
The complexity of diagnosing IPF, understanding its root causes, and translating preclinical findings to clinical success make it a challenging disease for drug development. However, recent advancements and strategic approaches in clinicaltrials offer hope that additional treatments are on the way.
Clinical genomics, powered by NGS, enables more precise target validation, improved patient stratification and optimised trial design, ultimately aiming to increase PoS. If it’s not mixable, the data value chain (from R&D phase to production phase) is neither interpretable or reproducible. Highlighting data integration.
Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.
Biopharma and biotech industries are facing remarkable challenges as the cost of developing new drugs has surged dramatically and clinicaltrial timelines have extended significantly. For example, over the past decade, the average time required to complete a clinicaltrial has increased by approximately 20-30%.
The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., The trial is evaluating NXT007 in people with hemophilia A without factor VIII inhibitors who have not previously been treated with Hemlibra.
Director of Lymphoma Research at Sarah Cannon Research Institute and principal study investigator for the trial. Vice President of Early ClinicalDevelopment and Translational Research at Johnson & Johnson Innovative Medicine. “As formerly Cellular Biomedicine Group, Inc.).
Unlike traditional royalty deals, where investors purchase rights to a portion of revenue from marketed products, this synthetic royalty structure allows Royalty Pharma to provide upfront capital in exchange for a defined share of future revenue, subject to agreed terms and caps.
Our goals are to progress our pipeline and position each asset for clinical and commercial success. The tight-knit structure of the Poolbeg team allows Tremble to contribute not only to early-stage drug development but also to clinicaltrial design, patient stratification, and biomarker strategy.
Biopharmaceutical and biotech drug developers have faced increasing challenges in recent years. In the past decade alone, the time needed to complete a clinicaltrial has increased by 20–30%, and the cost to bring a new drug to market has risen to an average of about $2.6
Regulatory bodies such as the FDA oversee clinicaltrials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. Any delays or missteps in bioanalysis during a Phase I trial can derail the trajectory of a promising drug.
The CRO Perspective From a Contract Research Organization (CRO) specializing in the design and execution of studies for innovative therapies, the REMS requirements have significantly complicated the design and execution of CAR T clinicaltrials.
This significant financial infusion is designed to fuel Revolution Medicines’ ambitious clinicaldevelopment and commercialization plans, with a particular focus on its groundbreaking RAS(ON) inhibitor programs for patients with RAS-addicted cancers. This aligns both parties’ interests toward long-term success in the market.
It provides a range of incentives to the sponsoring company, including seven years of market exclusivity upon approval, tax credits for clinicaltrial costs, fee waivers, and eligibility for expedited regulatory pathways. VITALIZE (NCT06290141): Evaluates the drug in CIDP patients currently receiving intravenous immunoglobulin (IVIg).
4 Essential Topics to Cover in Your First CRO Meeting pmjackson Fri, 11/15/2024 - 19:22 Starting a partnership with a contract research organization (CRO) is a sometimes intimidating—yet exciting—first step in your drug development journey. Get in touch with Altasciences today to see how we can help you with your drug development journey.
Its end-to-end R&D model integrates in-house discovery, development, and manufacturing capabilities—enabling rapid iteration, lower development costs, and sustained pipeline productivity. To date, the company has conducted more than 170 clinicaltrials across 40 countries, enrolling over 25,000 patients.
Balancing generative AI opportunities and risks Generative AI offers unparalleled opportunities for transforming various aspects of health care and clinical research. In clinicaltrials, generative AI accelerates trial designs through protocol optimization and enrollment forecasting, leading to faster and more effective studies.
Over the years, the collaboration has matured into one of the most productive academic-pharma partnerships in China. The company currently operates two global research and development (R&D) centers and four world-class production facilities across China.
Developing a new drug takes an average of 10–15 years and costs upwards of $2 billion, yet the majority of drug candidates fail before reaching regulatory approval. The failure rate in clinicaltrials exceeds 90%, often due to insufficient safety data, efficacy concerns, or regulatory non-compliance.
Exelixis Reports Positive Phase 3 Results for Zanzalintinib and Atezolizumab Combo in Metastatic Colorectal Cancer Exelixis , has announced promising topline results from the STELLAR-303 phase 3 pivotal clinicaltrial, a major milestone in the development of its investigational therapy zanzalintinib. Amy Peterson, M.D.,
Nonclinical safety assessment serves as a cornerstone in drug development, providing essential data to identify early signs of potential toxicity and inform subsequent clinicaltrial design. One important aspect of toxicity studies is determining whether the drug produces undesired immune effects.
Less clear is whether we can productively and safely inhibit TGF- activity at all given the toxicity issues associated with TGF- inhibition. We recently viewed quite positive updates from Scholar Rock who have a TGF--1 antagonist in the clinic. No chemotherapy regimen is used in the trial.
The initiation of this new trial brings much-needed hope for a future in which progression might be delayed or even halted. This further strengthens the biologicial rationale for proceeding into a larger and more definitive Phase III trial. Comments from Roche’s Chief Medical Officer Levi Garraway, M.D.,
FDA Grants Priority Review to Merck’s WINREVAIR™ Based on Landmark ZENITH Trial Showing Dramatic Reduction in Morbidity and Mortality in PAH Patients Merck (NYSE: MRK), operating as MSD outside the U.S. label of WINREVAIR based on compelling new evidence from the Phase 3 ZENITH trial. risk score of 9 or greater.
ClinicalTrials: Pritelivir is currently in phase II clinicaltrials, with ongoing research into its effectiveness and safety. Pritelivir (development codes AIC316 or BAY 57-1293 ) is a direct-acting antiviral drug in development for the treatment of herpes simplex virus infections (HSV).
Groundbreaking Data from the Phase III ARANOTE Trial The CHMP’s recommendation is founded on data from the pivotal Phase III ARANOTE clinicaltrial, which demonstrated that darolutamide, when used in combination with ADT, significantly reduced the risk of radiological progression or death in patients with mHSPC.
Over the past five decades, it has grown steadily, earning global recognition for developing therapeutic solutions for some of the most challenging and underserved conditions in medicine. The company actively seeks partnerships with academic institutions, research organizations, and biopharma peers to co-develop transformative therapies.
Grifols’ BT524 Fibrinogen Therapy Shows Promise in Phase 3 Trial Published in The Lancet’s eClinicalMedicine Grifols , a globally recognized healthcare company and a leader in the production of plasma-derived therapies, has marked a significant advancement in the treatment of acquired fibrinogen deficiency (AFD).
A final lipophilicity adjustment led to the discovery of CDZ173 (leniolisib), a potent PI3K selective inhibitor with suitable properties and efficacy for clinicaldevelopment as an anti-inflammatory therapeutic. Structurally, CDZ173 differs significantly from the first generation of PI3K and PI3K-selective clinical compounds.
With a particular focus on biologics, cell, and gene therapies, Galbraiths work is centred on equipping drug developers with the tools, insights, and support needed to advance their therapies from concept to clinic. His team works closely with clients, helping them navigate the increasingly complex regulatory and analytical landscape.
This strategic collaboration aims to enable the continued clinicaldevelopment and eventual delivery of a low-cost, effective Shigella vaccine to children in low- and middle-income countries (LMICs), where the disease’s health impact is most severe.
The drug’s clearance came after an unusual clinicaldevelopment journey and, in the ensuing months, has swelled Soleno’s market value past $4 billion amid optimism about the drug’s sales prospects. This database now covers the 50 best-selling pharmaceutical products and has been updated to reflect current patent expiry expectations.
This expansion is creating opportunities for clinicaltrials related to a range of new therapy areas and their subpopulations. The SELECT trial set out to understand whether the drug has similar effect on patients without diabetes.
Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance states that only one 90-minute pre-BLA meeting will typically be granted for a specific product or indication planned for an original marketing application.
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