EUPATI’s Post

🔬 AGTC Genomics PreciseDx™ Comprehensive Gene Profiling (CGP) for Cancers service. PreciseDx™ is a comprehensive gene profiling tool that provides a comprehensive view of genomic alterations, designed to shorten the duration of treatment decision-making, empowering you to make informed and confident decisions for your patients. It analyzes hundreds of clinically actionable biomarkers simultaneously, and measures TMB and MSI. The PreciseDx™ CGP can replace multiple single-gene tests or small hotspot panels with one comprehensive test, decreasing the need to re-biopsy to obtain more data. It also offers a faster turnaround time than sequential iterative testing. Using CGP to match patients with targeted or immunotherapies has been linked to improved clinical outcomes, including increased objective response rate, overall survival, and progression-free survival. #ngs #genomics #precisionmedicine #precisiononcology #cancer

We're thrilled to announce that Dr. Byron Lam one of our investigators from our breakthrough clinical trial for Retinitis Pigmentosa patients will be presenting Endogena Therapeutics, Inc.’s latest clinical data at the Ninth Annual Retinal Cell and Gene Therapy Innovation Summit, jointly organized by the Foundation Fighting Blindness and the Oregon Health & Science University and OHSU Casey Eye Institute. 🔬 Presentation Details: 📅 Date: Friday, May 3 🕒 Time: 3:40pm – 3:55pm 📍 Location: Hyatt Regency Seattle, USA 👨⚕️ Presenter: Dr. Byron Lam Dr. Lam will be sharing insights from our first-in-human study evaluating the safety, tolerability, and exploratory efficacy of our photoreceptor regeneration treatment with EA-2353 in patients with retinitis pigmentosa. This is a pivotal moment for Endogena as we continue our mission to revolutionize the treatment of retinal diseases. Dr. Lam's presentation promises to offer invaluable insights for researchers and the science community into our cutting-edge research and the potential impact on patients' lives. Join us in celebrating this milestone and witnessing the future of retinal therapy unfold. Don't miss Dr. Lam's presentation at the summit - it's sure to be a highlight of the event! For more information about the summit and to register, visit: @retina Innovation Summit 2024 #Endogena #RetinalTherapy #InnovationSummit #ClinicalData #RetinitisPigmentosa #MedicalResearch #HealthcareInnovation

Today Vivet Therapeutics shares the exciting news that it has dosed the first patient with lead program, VTX-801 in Cohort 2 of its ongoing Phase 1/2 #GATEWAY trial for the treatment of #WilsonDisease (WD). WD is a disorder which leads to a build-up of toxic levels of copper in the liver and brain and causes debilitating neurological and hepatic morbidity. Cohort 2 commenced following the observance of encouraging safety, tolerability and early pharmacodynamic changes in Cohort 1 and approval to proceed from an independent Data Monitoring Committee (DMC). Learn more about #Vivet and its potential disease-modifying gene therapy treatment for Wilson Disease in the first comment below! #AAVGeneTherapy #GenomicMedicines #GeneTherapy #GenomeEditing 

At the British Inherited Metabolic Disease Group (BIMDG) Annual Conference in Wales earlier today, our collaborator at UCL, Dr Berna Seker Yilmaz, presented the preliminary findings from the UK’s largest OTCD patient follow-up study, which she is leading at UCL. Dr Yilmaz illustrated current management and metabolic and neurological outcomes of both liver transplanted and non-transplanted OTCD patients, highlighting the important unmet medical needs still faced by these patients today. The findings will be the subject of a future scientific publication. Bloomsbury is actively developing BGT-OTCD, a best-in-class AAV gene therapy treatment for ornithine transcarbamylase deficiency (OTCD), which is currently being evaluated in HORACE, a Phase 1/2 clinical trial initiated in November 2023. Find out more about HORACE: https://lnkd.in/dFVkTnWQ Read the latest about BGT-OTCD: https://lnkd.in/e_SQp8sR #genetherapy #rarediseases #orphandrugs #aav #metabolicdisorders #advancedtherapies #clinicalresearch #OTCdeficiency #ureacycledisorder #ucd #ThinkAmmonia

[INTERNATIONAL CRIGLER-NAJJAR DAY] 🌻This Friday, June 21, our teams are supporting #CriglerNajjarDay, an worldwide day dedicated to Crigler-Najjar syndrome, a rare genetic liver disease. It is characterized by the abnormal accumulation in all body and brain tissues of bilirubin, a yellow-pigmented substance produced by the liver, which can lead to severe neurological damage and death. Only phototherapy, lasting from 10 to 12 hours a day, remains the mainstay of long-term  treatment for patients suffering from this syndrome and thus enables  bilirubin levels to be reduced, but it considerably impairs patients' quality of life. 💙An international gene therapy clinical trial, sponsored by Genethon, began in 2018. The first results published in 2023 in the New England Journal of Medicine demonstrated the efficacy of this #GeneTherapy treatment designed by the «Immunology and liver diseases team» led by Giuseppe Ronzitti. The pivotal part of the trial has been initiated enabling the inclusion children 10 years old and over. 👉 https://vu.fr/BbKwx Giuseppe Ronzitti Fanny Collaud #genethon #raredisease #clinicaltrials #Liver #GeneTherapy

Our recently accepted article entitled “ MicroRNA-141-regulated KLK10 and TNFSF-15 gene expression in hepatoblastoma cells as a novel mechanism in liver carcinogenesis" is now online 😀 https://lnkd.in/duZ-TQY4

Today is World Parkinson's Day – a day dedicated to raising public awareness of, and supporting those affected by, Parkinson’s disease (PD). Parkinson's disease is the fastest growing and the second most prevalent neurological condition, affecting an estimated 10 million people worldwide. Despite significant advancements in treatment, there is still no known cure for the disease. Levodopa-induced dyskinesia (LID) is a prevalent and debilitating side effect of chronic levodopa treatment, the current gold-standard for managing PD motor symptoms. Around half of PD patients are affected by LID after 5 years of levodopa treatment and up to 90% after 10 years, with current treatment options providing only partial therapeutic benefit on LID.   We are developing BGT-PD, an investigational AAV2 gene therapy candidate for the treatment of LID in Parkinson’s disease. First preclinical data on our BGT-PD program will be presented at the upcoming American Society of Gene & Cell Therapy 27th Annual Meeting.    Learn more about BGT-PD here: https://lnkd.in/e2tZTHQm #WorldParkinsonsDay #ParkinsonsAwarenessMonth #ASGCT2024 #genetherapy #rarediseases #aav #NeurodegenerativeDisorders #advancedtherapies #Parkinsonsdisease 

The ATTC Networks webinar 'Evaluation of the Patient Referral Pathways for Gene Therapy and CAR-T in the UK' which was hosted last week is now available to view on-demand! Tune in to learn more about the existing patient referral pathways for advanced therapies in the UK and how these can be improved. In this webinar, speakers provided an expert overview of two projects that assessed patient referral pathways for different advanced therapies and the resulting best practice recommendations. Watch it here: https://buff.ly/49guJA0 #ATTC #PatientReferral #CellTherapies #GeneTherapies

Spark’s co-founder, Beverly Davidson, announced a $54M Series A raise that allows for the officially-launched-today AAV gene therapy company Latus to move to Boston and enter clinic in 2025. What I like most about this announcement is that Latus is developing its CLN2, Huntington’s and other CNS therapies to be administered outpatient, in smaller doses, directly to the brain, which could reduce immunogenicity issues with the added bonus of perhaps sidestepping the complexity of inpatient DRG and NTAP considerations that can slow adoption and make reimbursement even more cumbersome.   Regardless of whether inpatient or outpatient administration is better (and for whom), I love the idea that being an outpatient gene therapy is already part of Latus’ investor narrative, as it shows they have done their market access homework and they are thinking ahead about commercialization. Contributing to my chronic FOMO when it comes to CGT learning opportunities, Latus announced that its big, detailed reveal will come at ASGCT next week in Baltimore where Davidson will be a Keynote speaker. One more reason I envy those folks headed to ASGCT next week. #asgct #genetherapy #cgt #huntingtons #AAV

💡 Exciting News from SparingVision at the Macula Society 47th Annual Meeting!   We are proud to announce that we have presented clinical data for our pioneering gene therapy, SPVN06, for the first time. This represents a significant milestone in our journey towards transforming the treatment of retinal disease.   Our abstract provides information on the preliminary safety data of SPVN06, our gene-agnostic investigational gene therapy, which shows that no severe adverse events were reported in the initial groups of patients who received treatment. Most adverse events were procedural, transient, and quickly resolved.   This achievement demonstrates our commitment to advancing genomic medicines and represents a step forward in our mission to lead the way in bringing new hope to millions affected by retinal diseases, for which there are currently no viable treatments.   👁️ To learn more about our findings and the impact of our research, please read the full press release here: https://lnkd.in/dN4ZTSMY   #GenomicMedicines #Ophthalmology #GeneTherapy