Vesiculab’s Post

Harnessing synthetic biology for advancing #RNA therapeutics and #vaccine design:- •Understanding RNA-protein interactions is crucial for deciphering the cellular functions and molecular mechanisms of regulatory RNAs. •Consequently, there is a constant need to develop innovative and cost-effective methods to uncover such interactions. •We developed a simple and cost-effective technique called Multiple Oligo assisted RNA Pulldown via Hybridization (MORPH) to identify proteins interacting with a specific RNA. •#MORPH employs a tiling array of antisense oligos (#ASOs) to efficiently capture the RNA of interest along with proteins associated with it. •Unlike existing techniques that rely on multiple individually biotinylated oligos spanning the entire RNA length, MORPH stands out by utilizing a single biotinylated oligo to capture all the ASOs. •To evaluate MORPH’s efficacy, we applied this technique combined with mass spectrometry to identify proteins interacting with #lncRNA NEAT1, which has previously been studied using various methods. •Our results demonstrate that despite being a simple and inexpensive procedure, #MORPH performs on par with existing methods. #rnatherapeutics #oligonucleotides #vaccinedevelopment #vaccineresearch

Drug Discovery News has published a very interesting article about #circularRNA and its significant advantages and promising #therapeutic future in comparison to #linearRNA and #mRNA. This results from a Q&A interview between Luisa Torres, PhD, Science Editor and Erik Digman Wiklund, CEO of Circio Holding ASA . As the co-discoverer of circular #RNA, Erik is the equal first most qualified person to discuss this, alongside the other co-discoverer, Thomas Hansen, Circo's CTO. Erik discusses the discovery, why #circRNA is so important, why Circio chose #AATD as a lead target, and the future of the circRNA and wider RNA fields. Really interesting to work with Circio and be part of the examination into the future of RNA and #genetherapy. https://lnkd.in/eANY7nN5

💡 Unlocking Potential: Risdiplam (discovered and developed by PTC Therapeutics, Inc. and Roche) marks a milestone in medical history as one of the first small molecule therapies designed to target RNA. More specifically, it was developed as a treatment for Spinal muscular Atrophy (SMA) and works by targeting the RNA splicing mechanism affected in this disease. Using Risdiplam as a case-study, learn more about the science behind this game-changing innovation and its implications for future treatments. This is a continuation of a special 3-part series that we started at the end of 2023, to introduce the emerging field of using small molecule drug compounds to target RNA, as well as EDDC's in-house efforts in this field. As was presented in the previous installment, the illustrations in this were meticulously drawn by our talented chemist Juliana Mohammad. #EDDCsg #DrugDiscovery #DrugDevelopment #Risdiplam #MedicalInnovation #RNATherapy #SpinalMuscularAtrophy

With all the publicity around cell and gene therapies for genetic disorders, a less known strategen is to use small molecules to target gene products. With this approach, the disease process is alleviated and relief provided to the patient. This has the benefit that the cost of goods will be typically much lower than traditional cell and gene therapies, and also unlike CGT for which the effects can be permanent, there is an "off switch" in that cessation of treatment should reverse its effects. EDDC is pursuing efforts here, and this is an exciting space to watch!

Get along to Elrig here in Manchestwr tomorrow to learn about CryoLogyx #assayready cells to accelerate your drug discovery, toxicology and more.

We have made a good start at the 50th #DMDG Open meeting in York today. Three #TNO posters were presented during the Poster Blitz/Early Career and Student Session. Together with colleagues Marlies Aten Emily Rayner Joanne Donkers and Evita van de Steeg I am looking forward to another day with inspiring presentations and exhibit representing TNO’s in vitro and ex vivo DMPK platforms and Peregrion’s microtracer AMS capabilities Selected posters: -> Unlocking the secrets of the human gut: Studying satiety hormone release and host-microbe interactions using the ex vivo intestinal tissue models InTESTine and Intestinal Explant Barrier Chip by Joanne Donkers -> Utilizing normothermic machine perfusion of liver for preclinical testing of RNA therapeutics by Emily Rayner -> Intestinal enteroids to study ADME: Proof of concept study for enteroids derived from pediatric and adult donors by Evita van de Steeg #DMPK #DrugMetabolism #DrugDevelopment #DrugAbsorption

From SONOKINE Bioscience- making intratumoral histotripsy to all organ sites a clinical reality.

Join us for the RNA Therapeutics Symposium: Unlocking the RNA’s Potential in Medicine, scheduled to take place from September 25-27, 2024, in the beautiful city of Porto, Portugal.    RNA Horizons 2024 will be held in collaboration with CANCERNA, is set to be an impactful event that will facilitate the exchange of research, acceleration and development efforts in RNA therapeutics. The Symposium2024 will feature interactive workshops, panel discussions, and poster presentations, providing a comprehensive and immersive experience. This is a unique opportunity to learn from and engage with the pioneers and innovators shaping the future of RNA therapeutics.  Share your research with the RNA Community by submitting an abstract for oral or poster presentation. Submission Deadline: June 15, 2024 Click here for abstract submission guidelines >> https://bit.ly/3yLM5HT   Early Bird Registration is Open! Take advantage of our exclusive early bird rates and secure your spot for RNA Horizons 2024. Join us in shaping the future of RNA Therapeutics Early Registration Deadline: August 15, 2024 Click here to register early and save >> https://bit.ly/4aUoCl3 #RNAtherapeuticssymposium2024 #RNA #MolecularBiology #RNAWorld #event #medical #science #research #wondersofscience #simplyscience

There are so many rodent models available for preclinical target and drug discovery for #MASH. But which model offers the highest resemblance of the human disease?   The answer is simple. In the recent issue of Nature Metabolism, the renowned #LITMUS consortium concludes that Gubra’s GAN DIO-MASH mouse model ranks #1 for human proximity, a key indicator of clinical translatability, in terms of both metabolic relevance and ability to induce MASH-fibrosis. LITMUS compared a wide range of the most commonly used preclinical MASH models, evaluating their metabolic phenotype, liver histopathology and transcriptomics against corresponding MASH patient data.    We are very proud and grateful to be recognized by LITMUS as the provider of the most translational preclinical model.   Check out the paper and explore the objective ranking of preclinical models of MASH in the industry. #PreclinicalModel #PreclincalResearch #ClinicalTranslatability #MASLD #Publication #PreclinicalCRO #ResearchModels

🔎 Company focus: Vesper Bio Vesper is a clinical stage biotech and world leader in sortilin receptor biology. Its lead program uses a sortilin inhibitor to rebalance levels of progranulin in patients where the sortilin receptor would otherwise reduce circulating and extracellular progranulin, contributing to disease. Find out more about Vesper Bio here: #vesperbio #biotechnology #companyfocus