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An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells.
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).
Why brain cancer is often resistant to immunotherapy By Allessandra DiCorato February 26, 2025 Breadcrumb Home Why brain cancer is often resistant to immunotherapy Researchers find four coordinated gene expression programs in immune cells from glioma tumors, including two that could lead to immunotherapy resistance. Online February 26, 2025.
Our primary focus is to design and develop RNAtherapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.
Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development pmjackson Wed, 02/19/2025 - 21:30 The unique physicochemical properties of oligonucleotides require the use of specialized bioanalytical approaches, with key considerations including selectivity and specificity, sensitivity, stability, and matrix effects.
Advancing the Future of Biomarker-Driven Immunotherapy swheeler Fri, 03/14/2025 - 13:43 Mon, 04/21/2025 - 12:00 Resource Type Webinar Eduard Porta-Pardo Can spatial biomarkers revolutionize patient stratification for immune checkpoint inhibitors?
These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. TfR1 is almost becoming table stakes for Pharma and biotech looking to deliver to the brain and / or muscle.
But, regardless of which was first, they all operated with the same core data as their mechanism for understanding life: messenger RNA ( mRNA ). It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life. As such, they were quietly abandoned.
Using the 10X Genomics Chromium platform, we conducted ribonucleic acid (RNA) sequencing on the midbrain’s PAG region in animals treated with SRP-001, ApAP and a vehicle control, focusing on gene expression changes related to pain processing. was founded in 2016 to develop safer, non-opioid therapies for acute and chronic pain.
Made from a Cas9 nickase (the histidine at amino acid position 840 is swapped for an alanine) fused to a reverse transcriptase enzyme (which makes DNA from RNA; transcription in reverse), prime editing clinical trials are expected to begin by 2025. Gene Therapy. Molecular Cell. Nature Biotechnology. Cell Systems. Cell Systems.
Made from a Cas9 nickase (the histidine at amino acid position 840 is swapped for an alanine) fused to a reverse transcriptase enzyme (which makes DNA from RNA; transcription in reverse), prime editing clinical trials are expected to begin by 2025. Gene Therapy. Molecular Cell. Nature Biotechnology. Cell Systems. Cell Systems.
Astra Zenecas purchase of Esobiotec with a $425M upfront has rekindled interest in CAR-T cell therapy technologies, and in particular, the in vivo cell therapy companies. In vivo CAR-T cell therapy requires that genetic material be delivered to the desired immune cells while those cells are still inside the patient.
Research & Innovation 2025 Keynote Speakers Announced Cambridge, UK, 24 January 2025: The European Laboratory Research & Innovation Group (ELRIG), a not-for-profit, volunteer-led organisation for the drug discovery community, today announced the keynote speakers for Research & Innovation 2025.
Advances in biotechnology are driving significant progress in the treatment of rare diseases, making it possible to develop targeted therapies for previously untreatable conditions. Founded in 2021, SynaptixBio is working to develop therapies for TUBB4A leukodystrophies – a group of rare and severe neurological disorders.
A genome-wide atlas of cell morphology reveals gene functions By Allessandra DiCorato January 27, 2025 Breadcrumb Home A genome-wide atlas of cell morphology reveals gene functions PERISCOPE, a technique for genome-wide imaging screens, is helping Broad scientists understand the connections between genes and traits. Online January 27, 2025.
By Allessandra DiCorato January 16, 2025 Credit: Scott Sassone, Broad Communications Tara McDonald (left) and Steve McCarroll (right) are coauthors of a new paper describing a biological mechanism underlying Huntington's disease. Online January 16, 2025. Funding This work was supported by CHDI Foundation, Inc., DOI: 10.1016/j.cell.2024.11.038.
Our first Fellows Spotlight of 2025 goes to Keystone Symposia Fellow Dr. Abigael Cheruiyot! Studying RNA surveillance mechanisms, they identified nonsense mediated decay as a potential target for novel cancer therapies. She is very passionate about both science and promoting equity within the research community.
Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.
ESM is a protein language model that captures certain aspects of protein structure and function, for example, but is blind to DNA or RNA-level features that drive cell behavior. Evo 2 was trained on DNA sequences but can make predictions about myriad aspects of biology, including RNA stability and protein structures. & McCarty N.
For example, the European Medicines Agency (EMA), in its Regulatory Science Strategy to 2025 , has highlighted the critical role of biomarker discovery, qualification and utilisation in accelerating precision medicine.
The researchers also ran follow-up experiments in the laboratory; cell lines carrying the same mutations as those found in the monkey had similar RNA expression patterns as cells taken from Parkinson’s patients. ” Asimov Press (2025). If you’re interested, please contact him at trevor[at]highwaypharm.com.
After decades of research into therapies and preventive measures, though, global AIDS deaths have now fallen to about 600,000 annually. In January 2025, the incoming Trump administration issued a 90-day freeze on PEPFAR while it conducts a “programmatic efficiencies” review. ” Asimov Press (2025).
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