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QurAlis presents! QurAlis' head of development Emma Bowden, PhD, will present an overview of our Phase 1 ANQUR clinical trial evaluating QRL-201 at the 3rd ALS Drug Development Summit. QRL-201 is a first-in-class #STATHMIN-2 (#STMN2) #precisionmedicine for #ALS. ANQUR is the first-ever study to evaluate a therapy that rescues STMN2 expression in ALS patients and could halt disease progression. STMN2 is a well-validated protein important for neural repair and axonal stability, the expression of which is significantly decreased in nearly all ALS patients. QurAlis is exploring the use of the Unlearn.AI Digital Twin Generators in ANQUR with the goal of supporting both ANQUR and future studies for QRL-201. #neurodegenerativediseases https://bit.ly/3WLZB8l

If you're at the #ALS Drug Development Summit this week, don't miss QurAlis' presentation covering how our Digital Twin Generators can support their ANQUR study and future studies for QRL-201. #clinicaltrials #ALSdrugdevelopmentsummit2024 #AI #machinelearning

QurAlis presents! Emma Bowden, PhD, QurAlis' head of development, recently presented an overview of our Phase 1 ANQUR clinical trial - the first-ever study to evaluate a therapy that rescues #STATHMIN-2 (#STMN2) in #ALS - at the 3rd ALS Drug Development Summit. ANQUR is evaluating QRL-201, a first-in-class STMN2 #precisionmedicine for ALS that rescues STMN2 expression in ALS patients and could halt disease progression. QurAlis is exploring the use of the Unlearn.AI Digital Twin Generators in ANQUR. #neurodegenerativediseases

On #LimbGirdleAwarenessDay, we’re proud to announce the launch of C-Path’s new task force dedicated to advancing drug development for limb-girdle muscular dystrophies. Under the Rare Disease Cures Accelerator-Data and Analytics Platform, this collaborative effort unites experts from leading organizations to tackle the unmet medical needs in the LGMD community. This task force includes pivotal members: ML Bio Solutions (BridgeBio), Coalition to Cure Calpain 3, CURE LGMD2I FOUNDATION, Jain Foundation Inc, LGMD2D Foundation, LGMD2i Research Fund and The Speak Foundation. Together, we’re leveraging patient-level data, shared knowledge, and a neutral platform to expedite therapeutic advancements for those affected by LGMDs. As we work toward accelerating meaningful progress, our commitment remains to ensure patient voices and data drive innovation. Read the full details, here: https://lnkd.in/gFiffMB3 Alexandre Bétourné, PhD, PharmD, PMP Heidi Grabenstatter Douglas Sproule Kat Bryant Knudson Ramona Belfiore-Oshan Rachel DeConti Adora Ndu, PharmD, JD Anna Wade Cybele Gouverneur Jean-Pierre Laurent Jennifer Levy Jessica Evans Kelly Brazzo Laura Rufibach Patrick Moeschen #CPath #LimbGirdleMuscularDystrophy #RareDiseaseResearch #DataCollaboration #DrugDevelopment #DataSharing #RDCADAP #PatientVoice #Collaboration

“Why should you prefer an IVDR-certified NIPT test for your patients?” Listen to this short webinar featuring Jennifer Stobbs from Illumina 👉https://lnkd.in/ew_AxQg5 Register now and figure out more about: Reliable Test Results Extensive Clinical Validation External Review and Assessment Regulated Post-Market Surveillance Locally performed tests #NIPT #NGS #illumina #CEIVDR

Milestone Achieved: 8th Clinical Trial Emerges from Target ALS funded consortia QurAlis has completed dosing the first participant cohort in a Phase 1 clinical trial for QRL-101, a first-in-class precision therapy targeting hyperexcitability in ALS. This breakthrough treatment has the potential to benefit both familial and sporadic ALS patients by addressing motor neuron degeneration caused by hyperexcitability—affecting nearly 50% of all cases. Target ALS is proud to have supported the foundational research that led to this clinical trial, as well as providing additional grants, marking a significant step toward effective treatments for ALS. Read the full press release here: https://prn.to/47KVTiX #ALSResearch #ClinicalTrial #EveryoneLives #MilestoneAchieved

Will you be in Boston for #NextGenOmicsUS24 this week? Don't forget to stop by booth 14 to meet Paul Reid and learn how Genseq's multiomic services can help you maximise the potential of your samples to elevate your research, discovery, and clinical trial efforts. Please feel free to message us or Paul Reid before the event to arrange a chat! #OGOmics #NextGenOmicsUS24 #multiomics

By integrating a clinical-grade Knowledgebase with the Clinical Genomics Workspace (CGW), you can quickly make informed decisions regarding patient treatment. The Velsera Knowledgebase offers current information on clinical trials, medical literature, drug labels, and additional relevant data. Utilizing this information, CGW facilitates the automatic assessment of variant significance, enabling the creation of reports that present the most relevant therapeutic options to the treating clinician. This integration not only saves your team time but also ensures the delivery of high-quality clinical results, ultimately leading to the best possible treatment for your patients. Learn more: https://lnkd.in/guXky8s7 #PrecisionMedicine #NGS #Genomics #EraOfVelsera

Are you navigating the complexities of preclinical and clinical trials? Let’s make it simpler together! Meet us at #ASGCT24, booth 2172, where Accelevir and Noble Life Sciences unite to accelerate your path from discovery to delivery. Dive into our full-service offerings in drug, vaccine, and medical device development, enhanced by precise cell and biomarker analytics. 🧬💡 👋 Swing by for a chat, learn more about our exciting collaboration, and enter our iPad raffle! #baltimore #ASGCT24 #cellandgene

Last week, Kuros Biosciences sponsored a presentation at Base to Summit entitled 'Favorable Results of MagnetOs as Standalone Alternative to Autograft in a Prospective, Multi-center, Randomized, Intra-patient Controlled Trial.' Pictured below are images of Dr. Robert Eastlack presenting, featuring a slide of a case report from the investigator-led, level 1 clinical trial. For more information about Kuros or this level 1 clinical trial, click here to meet with one of our experts → https://lnkd.in/gTV-zSvJ #spine #basetosummit #kurosbiosciences