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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 clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.
By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinicalresearch, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?
As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. However, getting essential treatments to patients quickly and safely requires more than just technological innovation. billion in 2023 to 1.2
Obesity treatment is undergoing a major shift, much like the advances seen in cancer care. Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Phenomix Sciences, built on over a decade of clinicalresearch at the Mayo Clinic, is disrupting this outdated approach.
In response, the PPD clinicalresearch business of Thermo Fisher Scientific conducted its third global survey of 150 drug developers to capture a comprehensive view of these evolving trends. Furthermore, the development of innovative therapies often necessitates intricate protocols, adding layers of complexity to trial management.
Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. This limited pool poses significant barriers to conducting statistically significant studies and validating the efficacy and safety of new treatments.
Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.
Fortunately, advances in clinicalresearch are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
In the ever-evolving landscape of immuno-oncology, managing the side effects of advanced therapies has become just as important as enhancing their efficacy. I take responsibility for identifying pre-clinicalresearch that can support our clinical programs, as well as designing and overseeing its execution, he explains.
Dr Pooja Hingorani, Senior Medical Director of Oncology Early Development at AbbVie , shares her journey in STEM, from her early days in New Delhi to her impactful career in oncology research and drug development. Opportunities in clinicalresearch and drug development were not always easy to come by.
While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF. However, recent advancements and strategic approaches in clinical trials offer hope that additional treatments are on the way.
The data, which encompass up to 3 years of follow-up after a single infusion of the treatment, were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held from June 13–16 in Glasgow, United Kingdom. President and Chief Executive Officer of Intellia Therapeutics.
The drug development industry faces rising costs and increasingly complex protocols as top challenges, with 49% of developers citing costs as their primary concern and 39% pointing to protocol complexity as a key hurdle, according to a recent survey of 150 drug developers worldwide by the PPD clinicalresearch business of Thermo Fisher Scientific.
Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.
One of the most important factors to consider during the course of your early-phase clinicalresearch is drug-drug interactions (DDIs), which occurs when one drug alters the effect of another, either by reducing its effectiveness or elevating systemic concentrations to potentially dangerous levelsultimately causing side effects.
1] It’s a privilege to participate in such a prestigious gathering of vision science professionals, and to gain insight into the latest innovations and treatments for complex ocular diseases. These technologies are helping pave the way toward more objective, reliable tools for clinicalresearch.
The data, presented at the annual European Alliance of Associations for Rheumatology (EULAR 2025) congress in Barcelona, Spain, show that dapirolizumab pegol resulted in significant and clinically meaningful improvements in disease activity while addressing a major symptom that profoundly impacts patients’ daily lives — fatigue.
Institutional review boards (IRBs) play a crucial role in the ever-evolving field of clinicalresearch. Innovations in fields such as immunotherapy, cancer and chronic diseases owe their existence to clinical trials, which judiciously test and evaluate safety and efficacy.
In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.
This milestone marks a potential turning point in the treatment landscape for patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) who are ineligible for stem-cell transplant—a patient population with limited and often suboptimal options. Rates of Grade 3–4 adverse events were comparable between arms (58.5%
It is being investigated as a potential treatment for various herpes infections, including those resistant to traditional antivirals like acyclovir. 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).
Millions of people around the world each year face serious illness, with the promise of groundbreaking treatment being just out of reach. They wait for the therapies that could significantly improve their quality of life or even save it knowing approval could be years or decades away.
Initially approved for treatment of Type 2 diabetes and later for obesity studies in GLP-1 are now expanding in other indications such as obstructive sleep apnea (OSA), heart failure with preserved ejection fraction (HFpEF) and chronic kidney disease (CKD).
Clinicalresearch is a vital driver of medical progress. Yet, many people are unaware of the essential role of institutional review boards (IRBs) in protecting clinical trial participants by ensuring compliance with human subject protection requirements. This process ensures that new therapies meet the U.S.
In a recent webinar, Challenges of Drug Development in Early Parkinsons Disease , Tom Babic, MD, PhD , our Vice President of Scientific Solutions in Neuroscience, uses his 40+ years of experience to share invaluable insights into the current state and future directions of early Parkinsons disease (PD) research.
Fortunately, advances in clinicalresearch are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
Solid tumors present a significant challenge to clinicalresearch due to their complex and heterogeneous nature. Proactively Address Key Recruitment Challenges in Solid Tumor ClinicalResearch Solid tumor clinicalresearch is challenging, especially because of tumor heterogeneity.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. In the trial, 59% of people who got a 1.2 milligram shot, the company said. .
This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.
Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.
Organoid technologies are becoming an invaluable solution for preclinical research, with the ability to augment the development of personalised medicine, drug discovery and gene therapies. Organoids for cell therapy and drug discovery. Stem cell-derived organoids and their application for medical research and patient treatment.
Reaching the end goal of delivering transformational new treatment options for patients can take a decade or more even when companies execute successfully and are well capitalized. We already have the first-ever clinical-stage NBD1 stabilizer and a second series of highly potent NBD1 stabilizers that are advancing to Phase 1 this summer.
Despite these advancements, challenges for widespread effective care remain due to the heterogeneous patient population, opening the door for further development of treatments for T2D. Obesity is a major factor in the prevalence of T2D, and it is vital to develop T2D treatments with this in mind.
The recent surge in GLP-1 treatments has ignited a profound shift in the pharmaceutical landscape, with more studies being announced than ever before. However, the treatment journey does not end with weight reduction, as maintaining weight loss overall is equally crucial.
Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). How does the DCI mechanism compare to the design of other drugs for cancer treatment?
The field of clinicalresearch is a dynamic and rapidly evolving one. One way to ensure that you are at the forefront of these developments is by attending clinicalresearch conferences. AACR, founded in 1907, is the world’s oldest and largest professional organization dedicated to advancing cancer research.
At Olympian ClinicalResearch we’ve gotten a chance to work with countless Schizophrenia patients over the years through our clinical trials, and we know just how untrue many of the common assumptions about Schizophrenia really are. . Myth #2: People with Schizophrenia are violent. Learn More.
Vitiligo, characterized by the loss of skin pigment and the formation of white patches, has been a subject of medical intrigue and research for decades. The Evolution of Vitiligo Treatments From ancient remedies to modern medicine, the treatment of vitiligo has undergone a remarkable transformation.
The journey toward effective treatments has been long and evolving, with recent breakthroughs offering new hope to those living with this challenging condition. From Coal Tar to Biologics: A Historical Perspective The history of psoriasis treatment is as old as the condition itself, with records dating back to ancient times.
Before any new medical treatment, therapy, or device is approved, it must first be tested through clinicalresearch studies. While some research studies seek participants with illnesses or conditions to be studied in the clinical trial, it is not always necessary to have a specific illness or condition to participate.
However, advancements in treatments and ongoing clinicalresearch offer new hope. The complexity of HS means that treatment is often multifaceted, aiming not only to manage symptoms but also to address the underlying causes of inflammation. Surgery: In severe cases, surgical removal of affected skin may be necessary.
NCCRED was funded through the Strategy, and commenced in 2018, to build clinicalresearch capacity and work towards establishing evidence-based treatment approaches for both methamphetamines and other emerging drugs of concern. Why do a clinicalresearch priority setting study?
For clinicalresearch professionals, this journey often involves reconciling differing feedback from regulatory agencies, each with unique standards, priorities, and expectations. Ultimately, our goal is to bring safe and effective new therapies to patients.
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