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Zongertinib potently and selectively blocks HER2, while sparing EGFR, and inhibits the growth of cells dependent on HER2 oncogenic driver events, including HER2-dependent human cancer cells resistant to trastuzumab deruxtecan. Significance: HER2-mutant NSCLC poses a challenge in the clinic due to limited options for targeted therapies.
Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies. Among patients who had previously undergone two or more lines of therapy (n = 12), the ORR was 92% and the CRR was 75%.
Some researchers hypothesized that the primary event associated with the cholinergic neurotransmitter (acetylcholine) is memory loss and cognitive impairment. Due to the disease's complicated pathogenesis, long-term therapy with a single target candidate is futile. As a result, multitargeted and multifunctional therapies have emerged.
Simply remembering events can trigger brain rhythms, even more so than when people are experiencing the actual event, says a new study. The findings could lay foundations for cognitive impairment therapy and help improve memory.
The 7th World Conference on Targeting Phage Therapy 2024, two-day event dedicated to advancing the field of phage research and therapy will be hosted at Corinthia Palace Malta on June 20-21.
Recent studies have yielded controversial results on the long-term effects of statin treatment on the risk of cardiovascular (CV) events. The incidence of first CV events was 6.0% Taken together, our data demonstrate that a 7-year stable control of LDL-C induces a forty percent reduction in the incidence of CV events.
Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.
Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Abstract Noncoding RNAs (ncRNAs) are engaged in key cell biological and pathological events, and their expression alteration is connected to cancer progression both directly and indirectly. However, there are several limitations, including delivery, uptake, and short half-life, in the application of ncRNAs in cancer treatment.
Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance describes various methods to assess either cell distribution for cell therapy products or vector biodistribution for gene therapy products.
Rullo Molecular Therapy 2024 DOI: [link] Proximity-induction of cell-cell interactions via small molecules represents an emerging field in basic and translational sciences. We determined that covalent linkage enforced early T cell activation events in a manner that was dependent upon each SARs biology and signaling threshold.
Myeloproliferative Neoplasms (MPNs) are hematological malignancies that result from acquired driver mutations in hematopoietic stem cells (HSCs), causing overproduction of blood cells and an increased risk of thrombo-hemorrhagic events. The most common MPN driver mutation affects the JAK2 gene ( JAK2 V617F ).
Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.
a principal investigator for the TRANSCEND FL study and a lymphoma and cell therapy specialist at Memorial Sloan Kettering Cancer Center in New York. This high durability highlights the sustained Benefit provided by liso-cel in a population typically faced with poor prognosis after multiple lines of standard therapy.
The DSMB’s primary responsibilities include : Monitoring patient safety: Ensuring participants are not exposed to undue risk and adverse events are promptly identified and addressed. For example, consider a global Phase III, double-blind study, in a novel new therapy.
While these therapies hold great promise for improving cancer treatment outcomes, their development presents significant challenges, especially in achieving the optimal balance between efficacy and safety. Most patients are given combination therapies to enhance efficacy, explains Wang.
• Face-to-face event, taking place in London, UK, 26-27th April, will feature two dedicated tracks: ‘Scientific’, focused on seven core therapy areas and ‘Life Sciences Business’ Programme features keynote from George Freeman MP, with an update on the UK Government Life Sciences VisionRegistration for BioTrinity 2022 now available online
Rullo, Molecular Therapy: Oncology , 2024 , 200842, [link] Proximity-induction of cell-cell interactions via small molecules represents an emerging field in basic and translational sciences. Nickolas J. Serniuck, Eden Kapcan, Duane Moogk, Allyson E. Moore, Benjamin P.M. Lake, Galina Denisova, Joanne A. Hammill, Jonathan L. Bramson, Anthony F.
Durable Benefit and Positive Clinical Outcomes “Today’s results underscore the promising potential of Intellia’s approach to genome-editing therapy — a one-time treatment that has been well tolerated and offers a highly differentiated, durable effect for patients suffering from a serious disease,” said John Leonard, M.D.,
These results further validate the potential of Ascendis’ proprietary TransCon technology to deliver long-acting therapies that improve both efficacy and patient experience. Importantly, the combination therapy maintains a safety profile that aligns with what we’ve observed in monotherapy studies.” years in prior clinical studies.
The double-blind treatment period lasted 24 weeks, during which both efficacy and adverse events (AEs) were closely monitored. Primary Endpoint Met: Improved Tolerability with Atogepant At the core of the TEMPLE study was the investigation of treatment discontinuation rates due to adverse events, a key measure of real-world drug tolerability.
The RedirecTT-1 trial shows the power of this novel combination approach as a potential treatment option for these patients, many of whom have limited options after multiple lines of therapy.” 20% had previously received BCMA-directed CAR-T therapy , adding further resistance to standard treatments. Among these: 84.4% Among these: 84.4%
Prof Rory Johnson, Associate Professor, University College Dublin, and Dr Shalini Andersson, Vice President Nucleic Acid Therapeutics, AstraZeneca will lead this years event focussed on drugging the undruggable.
Among the 85 relapsed or refractory patients, 36% had previously received a single line of treatment, 42% had undergone two lines, and 12% had gone through as many as three lines of prior therapy. Importantly, there were no cases of QTc prolongation of Grade 3 or greater, and only 6% of patients experienced Grade 1 prolongation.
Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.
Based on previous studies, we hypothesized that vascular senescence increases the risk of bleeding during warfarin therapy. The vascular senescence group tended to experience more bleeding or thromboembolic/ischemic events during one year of follow-up, while there was no statistical difference.
The L2C Accelerator is focused on vastly shortening the time to develop therapies for rare disease patients by grouping together hundreds (or even thousands) of rare genetic mutations in parallel and identifying many candidate therapeutic targets (i.e. L2C2023 to view the full recording of the event and learn more about our research.
Cardiovascular diseases : Atherosclerosis : ADCs could be engineered to target and deliver drugs to atherosclerotic plaques, potentially stabilising them and preventing cardiovascular events. Liver Fibrosis : Targeted therapies using ADCs could help reduce liver fibrosis and prevent the progression to cirrhosis.
The investigational therapy, an Fc-free anti-CD40L monoclonal antibody, is being developed for the treatment of systemic lupus erythematosus (SLE), a chronic autoimmune disease with limited treatment options and significant unmet need. Most adverse events were consistent with expectations for an immunomodulatory therapy in an SLE population.
Hybrid Event May 17-19, 2022 | Boston, MA www.informaconnect.com/rare. Come together with experts from across the rare disease community, in-person or virtually, to unite in areas of unmet medical need and create life-transforming therapies and breakthroughs. Rare Disease Innovation & Partnering Summit.
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.
The Food and Drug Administration (FDA) recently released new guidance regarding cellular and gene therapy products, one of which may significantly impact early-phase clinical trials of such products. This article explores the implications of this guidance, including new approaches for studies focused on cellular or gene therapies.
The therapy is administered via subcutaneous injection and is being studied for its ability to produce and sustain significant weight loss over extended periods. The findings are expected to inform healthcare systems and providers about adherence gaps and optimization strategies in statin and non-statin therapy use. to 3:00 p.m.
He suggests that lessons learned from the COVID-19 pandemic can inform both innovation in AMR therapies and our understanding of AMR. This virtual event is presented by OptimizeRx in collaboration with Melina Therapeutics. Read on for Matt’s insights.
Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.
Through a series of experiments, they successfully developed a personalised stem cell therapy using a data-driven, single-cell technique based on swift subcellular proteomic imaging. Our recent work proposes the use of an intracellular toolkit to map organelle bio-geography in stem cells that could lead to more precise therapies.”
Xywav: A Low-Sodium Alternative with FDA Approval Xywav is a uniquely formulated, low-sodium oxybate therapy, and remains the only product of its kind approved by the U.S. of participants reported treatment-emergent adverse events (TEAEs), all of which were mild or moderate and consistent with previous safety data on Xywav.
The most important drug discovery event of the year? 18-19 October 2023 The most important drug discovery event of the year? It’s with great excitement that we announce the return of ELRIG’s FREE TO ATTEND Drug Discovery 2023 (DD 2023); Europe’s leading event for the drug discovery community! Save the date! Save the date!
Delivered as a Hybrid Event. This important event convenes key stakeholders including manufacturers, specialty pharmacies, hub providers and more for unrivaled collaboration to leverage innovation and optimize access. To find out how you can promote an event on Drug Channels, please contact Paula Fein (paula@drugchannels.net).
All patients received their respective biologic therapy in addition to standard background treatment with mometasone furoate nasal spray (MFNS). Serious adverse events were somewhat lower in Dupixent (2%) than in omalizumab (4%).
My Attendance at the 2024 Boston Society Cell and Gene Therapy Conference pmjackson Tue, 06/11/2024 - 20:49 , via Wikimedia Commons" data-entity-type="file" data-entity-uuid="c7a7fa8b-b2fe-4d84-a75e-d1ba3a4e2caf" src="[link] width="742" height="249" loading="lazy" /> Ecm85, CC BY-SA 3.0
It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure. However, there are significant challenges associated with the successful development of these complex, leading-edge therapies. what is the altascientist?
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