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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.
Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms. A biomarker is a measurable indicator of a biological process, disease state, or response to a treatment.
His approach focused on integrating key components of the process: research, clinical, regulatory, and manufacturing into a cohesive, unified system. Ive been involved in therapeutics development for over 25 years, working with small, medium, and large biotech companies.
The convergence of real-world data (RWD), technology and artificial intelligence (AI) is playing a vital role in accelerating drug development. In a recent panel discussion at DIA Global , our experts explored how these elements are reshaping clinicalresearch and drug discovery.
Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.
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.
Like many aspects of life in the 21st century, this era of scientific advancement and increased partnership is largely driven by next-generation technologies that are streamlining workflows, breaking down silos and empowering scientists to extract the most prescient insights from their data throughout the drug development process.
More than 7,000 rare diseases – conditions that affect fewer than 1 in 2,000 people – have been identified, and1 in 17 people will develop a rare disease in their lifetime, with children disproportionately affected. Most of these conditions are genetic in origin and the majority have no effective treatment.
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. It was already becoming clear that some cancers responded better than others to immunotherapy.
2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. 15 Figure 1: A schematic illustrating a bispecific antibody-drug conjugate (ADC).
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.
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.
Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials.
Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. As a provider of end-to-end clinicaldevelopment solutions, we plan to leverage our expertise to refine drug-disease matches identified by Every Cure’s use of AI and data analysis.
Instead of the standard approach where studies are designed to address a primary focus, a parallel study method — a format that incorporates multiple endpoints and objectives, modifications, treatment arms and dose selections into a single protocol — is being rapidly adopted.
Recent Applications of Ring-Closing Metathesis in Drug Discovery and Development A selection of representative macrocycles in drug discovery where ring-closing metathesis has been applied to their synthesis are shown below, highlighting chemically diverse macrocycles from programs that have reached clinicaldevelopment.
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.
For clinicalresearch professionals, this journey often involves reconciling differing feedback from regulatory agencies, each with unique standards, priorities, and expectations. If you’re facing regulatory challenges or simply looking for guidance on your clinicaldevelopment strategy, meet us at the upcoming ASCO conference.
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. The post Looking for Opportunities to Accelerate ClinicalResearch in Rare Diseases appeared first on LifeSciVC.
Jayaprakash Kotha, MBBS, PhD, ASCP (SH), Vice President, Bioanalytical Laboratory Satish Kumar, MBB, Head of Process Improvement Continuous Innovation is a Cornerstone of Bioanalysis Approximately 80% of drugs that begin the research process fail to reach approval. What is one contributing factor that sets the 20% that do apart from the rest?
#ScienceSaturday posts share exciting scientific developments and educational resources with the KAND community. Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. What’s a pre-print?
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.
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.
#ScienceSaturday posts share exciting scientific developments and educational resources with the KAND community. Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development.
Boehringer Ingelheim and Gubra today announced a new research and licensing agreement focused on the identification and validation of targets and innovative peptide compounds for the treatment of obesity. Senior Vice President and Global Head of Cardiometabolic Diseases Research, Boehringer Ingelheim.
In addition, as part of the label update, this indication has been revised to be for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for any platinum-containing chemotherapy. Source link: [link].
Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.
In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches. Sponsors can bolster their reach to diverse patient populations by expanding beyond these city centers and by training research sites new to cell therapies in other parts of the country.
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.
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. Aflibercept is a key treatment in ophthalmology.
PRA Health Sciences selected for the study which will position Awakn at the Forefront of Psychedelic Research for Addiction Treatment. While there are current treatment methods available, which are effective for some segments of the population, relapse rates are high. ” The study team is chaired by Prof.
A new protein-based antiviral nasal spray developed by researchers at Northwestern University, University of Washington and Washington University at St. Louis is being advanced toward Phase I human clinical trials to treat COVID-19. Food and Drug Administration (FDA). 2 omicron subvariant. 2 omicron subvariant.
Bayer will present new research across its oncology portfolio at the virtual American Association for Cancer Research (AACR) Annual Meeting 2021 , taking place over two weeks on April 10-15 and May 17-21, 2021. and China for marginal zone lymphoma (MZL) based on clinical data of MZL patients enrolled in the CHRONOS-1 study.
The high rate of unvaccinated people will continue to put a strain on hospitals and healthcare systems around the world, furthering the need for effective treatments for patients hospitalised with COVID-19,” said Levi Garraway, M.D., Chief Medical Officer and Head of Global Product Development, Roche.
Could you share an example of a specific project or research that you have worked on and the impact it has had in your field? I am currently working as Chief Scientific Officer at CatalYm, where we translate the latest scientific findings into clinical applications.
We are at the forefront of drug development in an area of research called cellular rejuvenation, which is an approach that has the potential to address many diseases of ageing by restoring aged and injured cells to a more youthful and resilient state. Our lead program utilises OSK for the treatment of age-related optic neuropathies.
Food and Drug Administration (FDA) has lifted the clinical hold placed on the company’s Investigational New Drug Application (IND) to evaluate injectable lenacapavir for HIV treatment and HIV pre-exposure prophylaxis (PrEP). There is no cure for HIV or AIDS. About Lenacapavir.
Additionally, Boehringer has been granted the responsibility for all non-clinical as well as clinicaldevelopment. Enara is also eligible for an upfront payment from Boehringer, in addition to research and preclinical milestones as well as licensing fees for each tumor type that is investigated. Source link.
Food and Drug Administration (FDA) and a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval of the novel combination of the cancer treatments copanlisib and rituximab. submission is for the treatment of patients with relapsed indolent B-cell non-Hodgkin’s lymphoma (B-iNHL). Fields, M.D.,
We will explore how GDF-15 impacts the tumour microenvironment and hinders the infiltration of T cells into the tumour, as well as the implications of neutralising GDF-15 to reverse its inhibitory effects and sensitise tumours to anti-PD-1 treatment. T-cell infiltration is a prerequisite for responses to checkpoint inhibitors.
A review by the external Data Monitoring Committee (eDMC) determined that this effect was related to treatment with the combination of ISL08507; the greatest decreases were seen in the arms of the study receiving the highest doses of MK-8507 (200 mg and 400 mg). The company has notified investigators and paused development of MK-8507.
Opdivo in combination with Cabometyx was generally well tolerated, with a low rate of treatment-related discontinuations. The combination of Opdivo and Cabometyx was well tolerated, with safety reflective of the known profiles of both medicines and a low rate of treatment-related adverse events (TRAEs) leading to discontinuation.
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.
We plan to discuss these data with health authorities, and we will continue to expand our tislelizumab clinicaldevelopment program in pursuit of novel, synergistic combinations with the ultimate goal of extending survival for more patients.”. About 108,000 people of more than 140 nationalities work at Novartis around the world.
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