Ultragenyx’s Post

Calling all those interested in learning about how the patient referrals pathways for #GeneTherapies in the UK can be improved! Next month the ATTC Network are hosting a webinar titled 'Evaluation of the Patient Referral Pathways for Gene Therapy and CAR-T in the UK', which will provide an overview of two projects that assessed patient referral pathways for different #AdvancedTherapies and the resulting best practice recommendations. Learn more: https://buff.ly/3NnbYCm #CellTherapy #GeneTherapy #AdvancedTherapy #Patient #Access #ATMPs

ASGCT has started! Check out poster #56 and #58 on the use of a humanized liver mouse to test epigenetic editing.

Having trouble with preclinical assessments for gene and cell therapies because traditional animal models lack human genes and pathways? Learn how our #PXBmouse and #PXBcells can change that at ASGCT 2024. Book a meeting with our attending team members in the link below. #preclinicalresearch #humanizedmodels

🔍 Insights on Industrial Partnerships at Carnot Imagine : We are thrilled to share the latest communication of our partner Cellectis in Nature Communications, including results obtained in partnership with Dr Annarita Miccio and Pr Marina Cavazzana's teams from Carnot Imagine. This study unveils a non-viral gene therapy approach for sickle cell disease using TALEN® technology, achieving over 50% normal hemoglobin expression in patient-derived cells. The edited hematopoietic stem and progenitor cells show promising long-term engraftment in preclinical models, paving the way for future clinical applications. 📰 https://lnkd.in/e4AZC9zc #GeneTherapy #Biotech #SickleCellDisease #TranslationalResearch #CarnotImagine #Cellectis

The ATTC Network has today released a paper which provides a consolidated view of recommendations to improve patient referrals pathways for #GeneTherapies in the UK. Key highlights in the infographic 👇 The recommendations have been drawn from work on gene therapy patient referrals by the Duchenne’s Muscular Dystrophy (DMD) Hub and other relevant work on patient referrals from NHS England, Pfizer and the Royal College of General Practitioners. Read here: https://lnkd.in/eTH8bYPz #AdvancedTherapies #ATMPs #GeneTherapy #ATTCnetwork #PatientReferrals

Whether you are attending ASGCT in person or virtually, you’ll want to sit in on our presentation titled “Validating EPI-321 Safety for FSHD: A Comprehensive Off-target Characterization Platform for Epigenetic Gene Therapies” where Epic Bio scientist, Linsin Smith, PhD., will showcase our pioneering work in establishing the standard for off-target characterization in epigenetic editing. Read the abstract here: https://ow.ly/cgEn50RtYmH #EpicBio #ASGCT2024 #GeneticMedicine #epigenetics

People living with devastating disease want to be part of the solution. Being a patient living with a condition is not what defines them. This is why patient engagement is critical. This is why health literacy is so important. #patientengagement

How will the widespread adoption of liquid biopsy impact the utilization of other non-invasive tests? Dr. Erica Barnell (Co-Founder & Chief Medical Officer of Geneoscopy) provides insights on The Scope Forward show. #scopeforward #gastroenterology #liquidbiopsy

🔬 Insightful Research Alert: The Lymphatic System 🔬 A recent study highlights how genetic mutations, particularly in the KRAS gene, can profoundly impact the lymphatic system. This research, using a mouse model, offers valuable insights into rare lymphatic disorders and opens doors for targeted therapies and early interventions. As healthcare professionals, let's discuss how these findings can shape future treatments and patient care strategies. Read more: https://bit.ly/48LnuPR #MedicalResearch #Genetics #HealthcareInnovation #GeneticResearch #lymphaticmalformation #CLAawareness #ComplexLymphaticAnomalies David Williams

🧪 Lentiviral vs Retroviral – Which Viral Vector Is Right For My Research? 🧬 Our viral vector team breaks down some of the differences between lentivirus and retrovirus when it comes to cell and gene therapy applications, manufacturing differences and similarities, and plasmid DNA requirements between the two vectors. 🧫https://lnkd.in/eZBUnq3T ⚗️ Let's accelerate your team's discovery! 🔬