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4c00622 The STAT family of transcription factors are important signaling hubs, with several of them, particularly STAT3, being emerging oncotargets already investigated in clinical trials. Keserű ACS Medicinal Chemistry Letters 2025 16 (6), 991-997 DOI: 10.1021/acsmedchemlett.4c00622
Accelerate Clinical Trials with Methylation-Based ctDNA Detection Download now to learn from biopharma industry leaders about how methylation-based ctDNA detection for precise MRD and response monitoring can accelerate your clinical trials.
Accelerating drug development with basket trials One of the strategies that Alltrna is employing to accelerate the development of its tRNA therapeutics is the use of basket trials. While commonly used in oncology, the basket trial approach is novel for rare genetic diseases.
“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.
Traditionally, scientists discover enzymes in nature and adapt them through trial and error. They synthesized DNA encoding each protein variant, expressed the proteins in E. These molecular machines power various cellular processes, from converting sugar into energy to switching genes on and off.
Motivated by our success, we are actively working to enhance the automation and optimization of sample dilution and reagent addition for ELISA and ECLIA assays to support large pharmacokinetic clinical trials. Automated workflows ensure higher quality, better efficiency, and consistency across projects of all sizes.
The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.
Related groups Liu Group Related news New CRISPR genome editing system offers a wide range of versatility in human cells Researchers extend power of gene editing by developing a new class of DNA base editors In May 2025, researchers announced that K.J. Some of these treatments, like K.J.’s, This unprecedented feat required diagnosing K.J.’s
The guidance ensures that these targeted evaluations help identify and mitigate safety concerns early in the development process, supporting the safe progression of oligonucleotide therapies into clinical trials. An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy.
“Biogen’s antisense ALS drug shows promise in early clinical trial” FierceBiotech. Tofersen , sold under the brand name Qalsody , is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [3] January 2024. Archived from the original on 10 January 2024. Retrieved 9 January 2024. ^ Liu A (1 May 2019).
For example, once considered incurable and terminal, patients with sickle cell disease may reach new summits in their lives with gene editing technologies such as CRISPR to repair affected DNA and, in some cases, functionally cure the condition. Operationalizing these trials requires proactive and flawless management at every stage.
Clinical Trials: Pritelivir is currently in phase II clinical trials, with ongoing research into its effectiveness and safety. Clinical Trials: Pritelivir mesylate is currently under extensive study to evaluate its efficacy and safety profile, with promising results in early clinical trials.
Certain CRISPR components can add short DNA sequences from the genomes of defeated viruses into the bacterium’s own genome, creating a type of protective “memory.” If the same virus invades the cell a second time, the gRNA’s spacer sequence will bind to the matching viral DNA sequence, then be cut by the Cas protein.
The ability of modifying RNA enabled us to sidestep many of the potential risks associated with permanent DNA editing. We aimed to achieve this by modifying and modulating proteins by precisely changing a single alphabet on RNA with the use of an oligonucleotide (think short stretches of chemically modified RNA or DNA).
Art and technology often intersect in this way: a brush, a chisel, a 3D printer, DNA encoded with the image of a Germanic Rune , a biocompatible implant that induced the growth of an ear on a forearm, a bioprinter, CRISPR editing that induces pigmentation in bacteria.
Still, while these developments provide cause for hope, each drug or vaccine faces a variety of challenges, ranging from financial incentives for clinical trials to the difficulty of discovering new antimicrobials that pass safety and efficacy tests. A phase 3 clinical trial for M72/AS01E, funded by the Gates Foundation, began this year.
Findings can explain sets of symptoms and illnesses in other family members, which DNA Science recently covered in A Genetic Crystal Ball: When Newborn Genome Sequencing Findings Explain Illnesses in Relatives. The post Nixing the Newborn Screening Advisory Committee Is Ill-Advised appeared first on DNA Science.
In IDH-driven gliomas, the mutated IDH enzyme fosters the addition of methyl groups to the cells’ DNA. These chemical changes alter the expression of genes without modifying the underlying DNA sequence. Clinical trials are now underway to test IDH inhibition alongside immunotherapy.
Of course the larger markets would be in non-NRG1 fusion cancers where HER3 is overrexpressed but Partner Therapeutics does not appear to be supporting additional clinical trials. Once bound to HER3-expressing tumor cells, the ADC is designed to be internalized and the cytotoxic payload released to induce DNA damage and cell death.
DNA sequencing and synthesis costs have dropped precipitously, in part, because maintaining Carlson curves became a rallying point for the field. Learning requires trial, error, and revision. For starters, we not only need faster, cheaper options (supply) but also people excited to use them (demand). Intuitively, this makes sense.
Although Amodei does acknowledge some real-world issues limiting scientific progress — such as the slow growth of organisms and tedious clinical trials — he mostly passes over the more general tools that will be required to accelerate research in the near term. This essay focuses on how we might do both, specifically for the cell.
Have you optimized your trial designs to employ precision medicine approaches for your novel solid tumor intervention? Integrate Biomarkers Where Possible Biomarker integration is crucial in solid tumor clinical trials, though more research is needed before fully validated assays become the norm across solid tumor indications.
Thats why Worldwide Clinical Trials was committed to ensuring the rare disease community still had a space to share knowledge, resources, and support on Rare Disease Day 2025. The post Rare Disease Day 2025: Rally for Rare Event Recap appeared first on Worldwide Clinical Trials.
Clinical trials assessing the impact of Radiotherapy (RT) in combination with inhibitors of the DNA Damage Response Pathway (DDRi) and/or immune checkpoint blockade are currently ongoing. The model was fitted to datasets which assessed the impact of RT in combination with the DNA Protein Kinase inhibitor (DNAPKi) - AZD7648.
Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.
Clinical trials are now underway for various genetic subtypes of both rare and common diseases, on the understanding that medications utilising genetic biomarkers have a significantly higher chance of success. There are clinical trials underway for genetic subtypes of rare and common diseases.
Cancer Research UK, the University of Southampton and Touchlight Genetics, a London based biotechnology company, today (Wednesday) announce a new clinical development partnership to progress a therapeutic DNA vaccine, TGL-100, into an early phase clinical trial targeting head and neck squamous cell carcinoma (HNSCC).
The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis. million DNA bases. It delivers a shortened dystrophin gene, just 4,558 DNA bases. What’s an HDAC Inhibitor?
Instead of the black, printed stripes of the Universal Product Codes (UPCs) that we see on everything from package deliveries to clothing tags, they used short, unique snippets of DNA to label cells. DNA barcoding has already empowered single-cell analysis, including for nerve cells in the brain. PRISM consists of two key components.
Pharmacogenomics (PGx), the study of how genes affect a person’s response to drugs, has brought significant changes to the clinical trial industry. At Worldwide, our recent study delved into the intricacies of PGx, comparing two common methods of DNA sample collection: buccal swabs and blood samples. for buccal swabs and 93.2%
When a mutation causes a deletion or duplication of larger areas of DNA, we call it Copy Number Variation. DNA deletion and duplication may impact large sections of genes, or multiple neighboring genes. The altered expression of so many genes has profound consequences for the nervous system. She graduated from UC San Diego with a B.S.
Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). The two conditions and the therapeutic approaches differ, but their clinical trials illustrate the importance of selecting patients whose characteristics suggest that they are the most likely to respond.
Some participants will receive a kit in the mail that they can use to send a saliva sample for DNA analysis. Participants will answer questions about their background, lifestyle, and medical history. Participants will be updated as the study progresses on how their contributions are enabling new discoveries.
UK drug developer Scancell said it has chosen a COVID-19 vaccine candidate, SN14, from more than a dozen potential products to advance to a clinical trial. . SN14 works by targeting the coronavirus’ nucleocapsid and spike proteins to prevent viral replication using the company’s ImmunoBody DNA vaccine technology.
Here, the specificity of the protein is built into the gene-editing protein itself and, unlike earlier generation gene-editing systems, this system is not designed to create double-strand breaks in genomic DNA, but rather is designed to create nicks or single-strand breaks in genomic DNA.
Joseph Kim , President and CEO of INOVIO, said, “We are very pleased to share peer-reviewed Phase 1 clinical data for INO-4800 published in The Lancet’s EClinicalMedicine , and are grateful for the support of all participants and investigator staff involved in the clinical trial.” .
PLYMOUTH MEETING, Pa. ,
The underlying assumption of most gene therapy procedures, irrespective of the disease being targeted, is that restoration of a wild-type DNA sequence will alone be sufficient to normalise the phenotype of the individual. Surely, then, a correct set of DNA plans will ensure a correctly functioning cell?
Genetic testing provides patients with a diagnosis for their illness, helps patients and family members to understand risks of developing new diseases, and can be used to support clinical trial advancement. Can prior genetic testing results be utilized in a trial, or should a new genetic sample be taken before participating?
Beyond simply backing great science (separating the wheat from the chafe), setting a company up properly is critical, and early choices can get locked into the DNA of the company. Some continue to do so today, unfortunately, but there appears to be less of it happening. Second, great teams of truly experienced leaders are scarce.
Transforming Genomics Research Next-generation sequencing (NGS) enables comprehensive genome sequencing and has transformed genomics research, offering vastly improved capabilities for analyzing DNA and RNA in a high-throughput and cost-effective manner. Research through WGS and WES has provided novel insights into biological processes.
This approach can be personalized for each patient by sequencing the tumor's DNA and RNA to identify unique mutations. Its individualized neoantigen-specific immunotherapy platform has demonstrated promising results in early clinical trials for melanoma and other cancers.
We remain committed to further progressing our trial preparations, as we look forward to initiating a U.S. Phase 2 trial for Berubicin during the first quarter of 2021.”
In addition to its manufacturing efforts, the Company has also made progress in its clinical trial preparations.
By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.
Throughout the course of our lives, natural and environmental factors can break DNA. Breaks in both DNA strands are a particularly dangerous threat to genome stability. At a DNA double-strand break (DSB), potentially lost sequence information cannot be recovered from the same DNA molecule.
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