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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.
In a recent panel discussion at DIA Global , our experts explored how these elements are reshaping clinicalresearch and drug discovery. This can be converted to a clinical trial simulator, which can model a clinical trial before the trial design is finalized and initiated.
In every early phase clinical trial, the transportation, chain of custody, environmental monitoring and subsequent analysis of biological samples are the cornerstones of a successful, robust and quality outcome. Within cell therapy clinical trials, apheresis collections contribute to the specific constituents of the given therapy.
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. This will, in turn, slow the progression of, or even halt, the disease.
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.
As clinical trials 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.
While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.
Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.
By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets.
Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. His mother had a presentation of the disease that suggested her immune system was already on the job.
Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.
By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare diseaseresearch.
Since the advent of modern medicine, the scientific community has developed more than 3,000 drugs for thousands of diseases. Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. There are so many patients suffering from diseases that have no treatments.
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.
to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. Christian Rommel, Member of the chief Committee of Bayer AG’s Pharmaceutical Division and Head of Research and Development. 1 in 3 adults is at risk for the disease. In the U.S.,
Baynes, head of Global ClinicalDevelopment (GCD) and Chief Medical Officer, Merck Research Laboratories (MRL), will be retiring from Merck in July. Li, president, Merck Research Laboratories, until his retirement. Li, president, Merck Research Laboratories, until his retirement.
In the quest to combat and eradicate diseases, scientists and researchers are increasingly turning to the immense potential of artificial intelligence (AI) as a tool. Her focus initially consisted of preclinical development strategies , clinicaldevelopment optimisation, target discovery and validation.
It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.
To keep a pulse on this rapidly changing industry, the PPD clinicalresearch business of Thermo Fisher Scientific surveyed 150 leaders in the biopharma and biotech industries for the second consecutive year. Differences in size, resources and agility have led the two groups to take different approaches to clinicaldevelopment.
Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare diseaseresearch and therapeutic development. The study identified 8 groups based on symptom presentation, and found over 70 disease-associated genes, including 4 patients with KIF1A mutations.
Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare diseaseresearch and therapeutic development. KIF1A-Related Research In the rare disease space, a common and straightforward question is: How rare is the disease?
Orphan drug designation is a regulatory status granted to pharmaceuticals developed for the treatment of rare diseases. It provides incentives to encourage the research, development, and approval of therapies targeting small patient populations.
Combinatorial analytics approaches identify combinations of features that together are associated with the disease phenotype in patient sub-groups, capturing the non-linear effects of interactions between multiple genes. The combinatorial approach is considerably more sensitive than GWAS and requires much smaller patient populations.
Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?
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 clinicaldevelopment, offering promising advancements in innovative cancer treatments.
During my academic career, I had the opportunity to examine the underlying causes of neurodegenerative diseases like Parkinson’s disease and develop novel animal models to investigate potential disease-modifying therapeutics. For instance, we know ageing is a primary risk factor for many neurodegenerative diseases.
Written by Angela Vinken and Patti Arsenault Key Opinion Leaders (KOLs) — i.e., trusted, well-respected experts — are crucial in clinicalresearch, especially in rare diseases. The challenge, however, is how to use KOLs wisely — they are often clinicians first and researchers second, and their time is in high demand.
Sandoz, a Novartis division, today announced progress in the late-stage clinicaldevelopment program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. About aflibercept.
By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare diseaseresearch.
(Nasdaq: GILD) today announced that Flavius Martin, MD will join the company as Executive Vice President, Research, and will become a member of the company’s senior leadership team, reporting to Chairman and Chief Executive Officer Daniel O’Day. Dr. Martin will assume responsibility for Gilead’s research organization effective April 12, 2021.
I am a pharmacist by training and continued with a PhD in Clinical Pharmacology. However, my goal was all the time to work with drug development in the pharma industry, so I moved on and started that journey in 1992 when I took on a role as ClinicalResearch Manager at AstraHässle, a mid-size Swedish pharma company.
For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.
By staying up to date with these developments, readers will be able to anticipate future advancements and understand their potential impact. Disease-Specific Applications Each chapter of the report focuses on specific therapeutic areas, such as cancer, Alzheimer’s disease, HIV infection, and immunotherapy.
Back in 2019, he was working in full-service ophthalmology when he learned he was going blind due to a rare and orphan disease called Wolfram syndrome. Earlier this year, McNary participated in a panel discussion at a rare disease conference that focused on the importance of including patients during FDA engagement.
Could you give us an overview of IRLAB’s current drug development pipeline? 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).
What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer.
By adjusting the activity of this receptor, these drugs can influence brain functions associated with mood, cognition and pain, positioning them as potential treatments for conditions like depression, cognitive disorders and neurological diseases.
One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.
Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Now, FDA’s CBER is setting its sights on making ambitious strides toward tackling rare diseases.
Obesity is a major factor in the prevalence of T2D, and it is vital to develop T2D treatments with this in mind. Evolving sentiments recognize obesity as a medical condition and lifestyle choice as well as a chronic disease with genetic, psychological, cultural, and environmental causes (Figure 1).
Novel chronic kidney disease (CKD) target from the partnership enters AstraZeneca’s portfolio.
CKD is a complex, and debilitating disease which affects around 10% of the world’s population. BenevolentAI is headquartered in London with a research facility in Cambridge (UK) and a further office in New York.
The field of nephrology clinicalresearch has been steadily adding new interventional trials over the last five years at an average pace of 176 trials per year. This activity reflects the … The post Enabling better access to kidney disease trials appeared first on Insights From Our Labs to Yours.
Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.
The association between excessive alcohol consumption and liver disease is well documented. In fact, the effect on alcohol consumption levels during the COVID-19 pandemic has more recently been investigated.
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