This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My
In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.
Understanding health and disease requires more than reading the genomic code. The result is a more holistic, actionable view of cellular and tissue dynamics that is essential for uncovering causal mechanisms in disease. Biology, however, presents a unique challenge: it is deeply contextual, dynamic and multilayered.
At this premier global meeting focused on thrombosis, hemostasis, and bleeding disorders, Sanofi will present compelling data across 18 scientific abstracts, including five oral presentations. In addition, two poster presentations will offer deeper insights into the LUNA 3 trial.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. These fields explore highly precise biological processes related to RNA, specific proteins and gene expression mechanisms.
“We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun. Scrutinising these distinctions aids scientists in comprehending cellular operations, thereby fostering enhanced therapeutic approaches for diverse diseases.
This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).
The interim data, presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, California, offer a strong signal of therapeutic potential. These reductions were sustained through one year , highlighting salanersen’s potential to meaningfully slow disease progression. a leader in RNA-targeted therapeutics.
Ras- G TPase-activating protein (SH 3 domain)- b inding p roteins (G3BP) are RNA binding proteins that plays a critical role in stress granule (SG) formation. Recent evidence suggests that G3BPs can also regulate mRNA expression through interactions with RNA outside of SGs.
“I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. Today’s broad-spectrum drugs often miss the mark, especially for chronic diseases like COPD or heart failure.
In nature, proteins take millions and trillions of years to evolve, 1 but scientists do not have this time when designing drugs for life-threatening diseases. Therefore, researchers use directed protein evolution technologies , to evolve and design proteins with specific functions under well-defined conditions and a practical time frame.
Abstract Chronic rhinosinusitis (CRS) is an inflammatory disease of paranasal sinuses. CRS rabbit models were constructed by Staphylococcus aureus infection and rabbits were injected with lentiviral vectors of short hairpin RNA-targeting Chemerin (shChemerin), followed by micro-computed tomography (CT) scan.
Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. This is also important when newborns present as having a “common” disorder that may have a rare genetic component, like KIF1A in genetic cerebral palsy.
Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs. However, mRNA technology is not limited to infectious diseases. Once delivered into the body, the mRNA instructs cells to produce these antigens, which are then presented to the immune system.
First data to be presented from the phase II coopERA Breast Cancer study evaluating neoadjuvant giredestrant treatment for oestrogen receptor (ER)-positive, HER2-negative breast cancer. Breast Cancer Highlights. Lung Cancer Highlights.
Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Jude’s, the team identified a group of genes involved in processing uLCFAs that are present only in the non- Lactobacillus iners species. At the Ragon Institute, Kwon’s lab is working to move this research toward a human clinical trial. “We
So we took lots of reference single-cell RNA sequencing data from atlases and used our scalable machine learning algorithms to embed all of the gene expression data on these cells into compact vector representations — you can think of these as a signature for each cell. We wanted to build a tool like that for cell biology.
Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the presentation of data from the company’s HIV clinical development program at the 11th International AIDS Society Conference on HIV Science (IAS 2021) from July 18-21. Late Breaking Oral Presentation OALC01LB03. On-Demand Oral Presentation OAB0304.
In the dynamic world of drug discovery, the notion of "undruggable" targets presents both a significant challenge and an intriguing frontier for researchers and pharmaceutical companies. Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.
Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.
Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. Please summarise Eterna Therapeutics’ ($ERNA) new research at ASGCT on a cell therapy approach that integrates multiple cell types to emulate the natural immune system’s fight against diseases?
Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. To investigate these proteins, the authors used RNA interference to silence Nup153 and KIF1A.
The data were presented at the 28th Conference on Retroviruses and Opportunistic Infections ( virtual CROI 2021). In patients with chronic kidney disease, assess serum phosphorus. In patients with chronic kidney disease, assess serum phosphorus. Pregnancy and lactation. Source link:[link].
This is a game-changer, especially in the fight against cancer and other complex diseases. The versatility and potential of PROTACs are vast, paving the way for new therapies that could transform how we treat diseases. This can potentially correct the root causes of some diseases at the genetic level.
These preliminary findings were presented today during Science Spotlights TM at the 2021 Conference on Retroviruses and Opportunistic Infections (CROI 2021). Findings from the primary efficacy and safety endpoints and additional secondary objectives will be presented at an upcoming medical meeting.
Regulatory T cells (Treg) cells are specialised immune cells that are essential for maintaining peripheral tolerance, preventing autoimmune diseases, and limiting chronic inflammatory diseases. However, they also limit beneficial responses by suppressing sterilising immunity and limiting antitumour immunity.
How has nucleolar stress (NS) been linked to age-related diseases, and what approach did researchers use to investigate its toxicity? The nucleolus (and nucleolar stress) had been previously linked to aging and age-related diseases such as cancer or degeneration. However, the mechanism of this observation was not fully understood.
A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. A Scant Protein with Outsize Importance Slowing the muscle decline of DMD presents two huge challenges: the ubiquity of muscle and a giant gene. The gene presents a complex landscape.
Published June 24, 2025 Gwendolyn Wu Senior reporter post share post print email license Lexeo Therapeutics and two life sciences investors are working together to launch a startup to develop cardiac RNA therapies. Earlier this year, it laid off 15% of its workforce.
5 Here, the original oncogene is still present but the transdifferentiated cancer cells are less dependent on it. Investigating non-oncogene resistance as a parallel defense mechanism used by cancer cells to evade therapies could therefore present a significant opportunity to address drug resistance more comprehensively.
Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Ridgeback Biotherapeutics announced today the presentation of previously announced Phase 2 interim results from two Phase 2/3 clinical trials (MOVe-OUT and MOVe-IN) of molnupiravir (MK-4482/EIDD-2801), an investigational oral antiviral therapeutic. About Molnupiravir.
Morphological profiling of cells is commonly employed in drug discovery across many disease areas. However, there has been limited study on its application specifically in the development of therapeutics for heart diseases. The Broad Institute almost doesn’t feel real to me, it is special and unlike any place I've ever visited.
US10071079, Example 15 Ref WO2018005586 [link] The invention further pertains to pharmaceutical compositions containing at least one compound according to the invention that are useful for the treatment of conditions related to TLR modulation, such as inflammatory and autoimmune diseases, and methods of inhibiting the activity of TLRs in a mammal.
Live Webcast : accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or with this link: [link]. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.
New research from first-in-class marketed and investigational therapies in hemophilia, immune thrombocytopenia and acquired thrombotic thrombocytopenic purpura will be presented. Oral presentation. These data support the revised fitusiran dose and dosing regimen implemented in the ongoing adult and adolescent studies.
Each of these mechanisms represents a distinct strategy to address the underlying genetic perturbations contributing to disease pathogenesis. In sickle cell disease, for instance, a missense mutation in the HBB gene , encoding the beta-globin subunit of hemoglobin, leads to the production of abnormal hemoglobin S.
Here are ten key tutorials, presentations and posters designed to help you achieve fast, accurate and reproducible results in precision medicine, next generation sequencing, homogenous assays and more. Time away from the lab is limited and valuable. Here we describe a cloud-enabled AI platform for analyzing and visualizing HCS data.
They needed reference materials for the disease in order to develop and validate diagnostic tests. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays. NGS tools can be used to ‘read’ DNA to identify a disease subclass and detect specific mutations.
We are incredibly grateful for the selfless commitment of the individuals with ALS who participated in the study, and the community’s dedication to advancing research for this devastating disease,” said Toby Ferguson, M.D., The companies will present the BIIB078 Phase 1 data at a future scientific forum. Biogen Inc.
The company’s commercial priorities are three-fold: (1) Initially focusing its commercial efforts on rare diseases within its prolific neurology and cardiology franchises (2) pioneer new markets where there are no available treatments (3) create new standards of care where there has been a lack of innovation to optimize patient care.
Overall, the vaccine protected against symptomatic disease in 70% of cases, according to a team led by researchers from Oxford University in England. Both employ a newer technology that uses messenger RNA to prime the immune system to attack the new coronavirus. TUESDAY, Dec. 8 in The Lancet.
The CureVac-GSK COVID-19 collaboration announced in February 2021 extends the existing strategic mRNA technology partnership both companies started in July 2020, which focuses on the development of new products based on CureVac’s second-generation RNA-technology for different targets in the field of infectious diseases.
Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. In this regard, AI presents itself as a valuable ally, offering the potential to bolster our endeavours and ensure successful outcomes.
We organize all of the trending information in your field so you don't have to. Join 15,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
Let's personalize your content