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A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.
A tiny, four-fingered 'hand' folded from a single piece of DNA can pick up the virus that causes COVID-19 for highly sensitive rapid detection and can even block viral particles from entering cells to infect them, researchers report.
Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.
Researchers map communities of single cells in metastatic breast cancers By Corie Lok October 30, 2024 Breadcrumb Home Researchers map communities of single cells in metastatic breast cancers The team compared different single-cell and spatial profiling methods to explore the diversity of cells in metastatic breast cancer biopsies.
Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health
But its impact goes far beyond research. From Research to Real-World Care 🌎 Understand how AI-driven advancements are shaping personalized treatments and patient outcomes. AI is transforming clinical trials—accelerating drug discovery, optimizing patient recruitment, and improving data analysis.
With AI, researchers predict the location of virtually any protein within a human cell By Corie Lok May 19, 2025 Breadcrumb Home With AI, researchers predict the location of virtually any protein within a human cell Trained with a joint understanding of protein and cell behavior, the model could help with diagnosing disease and developing new drugs.
Alltrna’s unique approach focuses on addressing genetic diseases at a fundamental level, shifting the paradigm from a one-disease, one-therapy model to a mutation-specific strategy that could transform the treatment of hundreds of rare diseases. This will mark a critical step in validating the potential of engineered tRNA therapeutics.
By Count Me In Communications March 11, 2025 Count Me In , a patient-partnered research initiative led by the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute, is launching a new project focused on translocation renal cell carcinoma (tRCC), a rare and aggressive form of kidney cancer.
His approach focused on integrating key components of the process: research, clinical, regulatory, and manufacturing into a cohesive, unified system. My focus has always been on advancing novel medicines from research to clinical development. The specificity of the treatment for the cancer is already inherent in these lymphocytes.
Researchers reviewing nearly 300 top-tier ADHD drug trials found that half skipped the rigorous, expert-led evaluations needed to rule out other conditions like depression or schizophrenia.
Expert panel discusses malaria drug resistance, AI-driven discovery, long-acting treatments, and African research capacity. Learn about CDD's access program.
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. The Natural History Studies Grants Program funds natural history studies that collect gather data on how rare diseases progress over time without treatment.
The potential therapeutic efficacy of luteolin in the treatment of cervical cancer has been identified. Therefore, we aim to elucidate the mechanism of action of luteolin in the treatment of cervical cancer through a comprehensive approach that integrates metabolomics with bioinformatics.
Creating replicas of organs in vitro has been a goal of researchers for over a century. 1 This early work laid the foundation for a century of research on finding the optimal conditions for growing parts of organs in in vitro cultures, testing their functions and even mimicking many diseases.
CRISPR-CAS9 is an innovative gene editing technology to precisely remove or incorporate into the gene of interest; CRISPR-CAS9 shows promising results in HIV treatment. This review article also discusses its mechanism of action and potential applications in the treatment of HIV/AIDS.
The aim of this research was to design a pre-clinical hepatic safety assessment strategy for TCIs considering risk associated with electrophilic warhead reactivity and reactive metabolites formation at clinically-relevant plasma concentrations. The calculated hepatotoxicity margin towards plasma exposure of 2.5
These twins simulate how a patients condition might evolve without treatment, enabling researchers to compare the real-world effects of an experimental therapy against predicted outcomes. He was a postdoctoral researcher in mathematics at the University of Waterloo. Aaron received his Ph.D.
These digital twins are created for each trial participant using their baseline data – regardless of whether they are assigned to the placebo or treatment arm – and simulate how that individual would have responded under control conditions. AI with a human purpose Herne speaks about AI in clinical research with pragmatic focus.
This flaw may be one reason treatments like CAR-T don’t work as well on solid tumors. Now, researchers are exploring ways to block the enzyme and give our immune system its power back. The change affects a protein used by immune cells to kill tumors—except in humans, it’s vulnerable to being shut down by an enzyme that tumors release.
Research by Markets and Markets projects the market to grow from $12.13 1 Emergen Research attributes this to the advantages of high-throughput sequencing technologies and declining sequencing costs. 2 This trend reflects the increasing demand for genomic sequencing in research, clinical diagnostics and other applications.
Therefore, Zinc finger protein 726 (ZNF726) can be considered an attractive therapeutic target for the treatment of breast cancer. ABSTRACT Studies documented by our lab established ZNF726 to potently augment the tumorigenic behavior of breast cancer cells by increasing cellular cholesterol levels.
Our shared commitment is to explore novel technologies and therapeutic targets in various cardiovascular and renal diseases to help deliver new treatment options to patients in need,” said Andrea Haegebarth, Ph.D., Global Head of Research and Early Development for Cardiovascular, Renal, and Immunology at Bayer’s Pharmaceuticals Division.
In an interview with our team, Delpassand shared his perspective on how targeted radiopharmaceuticals are reshaping cancer treatment and what lies ahead for this evolving field. This dual role enables him to bridge research and clinical practice, ensuring scientific innovation is directly informed by patient care. “As
Oncology drug approvals in H1 2025 In the first half of 2025, the FDA’s Center for Drug Evaluation and Research (CDER) approved a total of 16 novel drugs , with half of these drugs related to the treatment of cancer.
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The integration of AI in drug discovery is revolutionizing the way researchers approach the development of new treatments for various diseases. The integration of AI in drug discovery is revolutionizing the way researchers approach the development of new treatments for various diseases. Traditional.
Exciting new research is exploring carprofen's broader therapeutic possibilities, extending beyond its original anti-inflammatory role. With ongoing research pushing the boundaries of its potential, carprofen remains a promising candidate for innovation in drug development and treatment strategies.
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.
In the vast realm of pharmaceutical research and development, there’s a fascinating intersection between ancient wisdom and modern science. The Discovery Process: Unearthing Nature’s Secrets The journey from plant to patent begins with extensive research. What is a Drug Patent?
Our findings highlight key structure-activity relationships, paving the way for flavonoid-based inhibitors as safer alternatives to conventional treatments. ABSTRACT Last few decades, extensive research efforts have been dedicated to uncovering novel cancer treatments.
Combining genomics with transcriptomics, proteomics, metabolomics, spatial profiling and cellular imaging, will enable researchers to move beyond single-variable analysis and begin to see the biological system as a whole. Enter multiomics Multiomics is designed to solve this problem by observing multiple molecular layers simultaneously.
Targeting leucine-rich repeat kinase 2 (LRRK2) has been identified as a potential strategy for the treatment of PD. Combined with predicted favorable drug-like properties, this hit can be used as a starting point for further structural optimization, probably offering insight into targeting LRRK2 for PD treatment in the future.
Researchers from around 29 universities and institutions across North America have teamed up to create a large, versatile, and effective arsenal of new biological tools that will play a critical role in the battle against brain disease. Ben-Simon et al.) and striatum ( Hunker et al. Ben-Simon et al.) and striatum ( Hunker et al.
These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. The treatment was generally well tolerated, and adverse events aligned with the known effects of the drug class and prior studies. ZENITH, the second pivotal Phase 3 trial, presented at ACC.25
FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.
With a background spanning drug development, clinical strategy and translational research, Eleni brings more than two decades of experience to the role. mCRPC is a particularly aggressive form of the disease, and patients currently have limited treatment options,” said Eleni. “As
As the limitations of current treatments become increasingly clear, researchers are exploring new, safer approaches. As co-founder and chief executive of South Rampart Pharma , and a practicing academic vascular surgeon, Dr Bazan combines hands-on clinical experience with a strong track record in scientific research.
Designed using the same fundamental mechanism as Biogen’s approved SMA treatment SPINRAZA (nusinersen), salanersen represents a next-generation approach aimed at greater potency and less frequent dosing—potentially requiring only once-yearly administration. Treatment-naïve individuals, to evaluate efficacy as a first-line therapy.
Metastatic castration-resistant prostate cancer represents the most advanced and lethal form of the disease, defined by progression despite androgen deprivation therapy and subsequent ARPI treatment. This has created an urgent need for more effective and tolerable treatments for this population.
The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. It is characterized by early-onset, treatment-resistant seizures, beginning as early as six weeks of age, and profound global neurodevelopmental impairment.
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.
However, getting essential treatments to patients quickly and safely requires more than just technological innovation. A global network with local expertise To ensure the delivery of treatments to patients worldwide, biotech and biopharma companies also need partners that can provide comprehensive solutions from a geographic perspective.
Additionally, treatment led to improvements in height-adjusted total kidney volume (htTKV) , an established clinical marker that correlates with disease progression in ADPKD. Importantly, farabursen showed consistent modulation of urinary polycystin (PC) levels—a key biomarker reflecting therapeutic engagement with the target pathway.
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