Vivet Therapeutics’ Post

As Co-founder and Chief Scientific Officer of #VivetTherapeutics, Dr Gloria Gonzalez-Aseguinolaza will be presenting 📢 at the upcoming #CTX Scientific & Family Meeting on the 28 June at 13.25 CST in Itasca, Illinois! Join Gloria where she will provide valuable insights into 'potential gene therapy for Cerebrotendinous Xanthomatosis (CTX) from CYP27AI KO mouse model' to the families affected by leukodystrophy and the world's most respected and knowledgeable leukodystrophy experts. CTX is a rare autosomal recessive genetic disorder that affects the body’s ability to metabolize fats known as cholesterols. #Vivet is accelerating development of gene therapy product, VTX-806 as an effective treatment option to stop or reverse disease progression over the long-term, or potentially cure CTX in patients. Initial proof of concept data has shown solid efficacy with VTX-806 in a relevant animal model. Learn more about VTX-801 here https://bit.ly/3XqiChb. The annual family conference presented by CTX Alliance in collaboration with United Leukodystrophy Foundation (#ULF) provides a well-rounded educational and networking experience aimed at empowering families effected by leukodystrophies around the world. The event provides a voice for members of the community and advocates for continued research and development of safe and efficient long-lasting therapies for leukodystrophies. #AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT

We are delighted to share our findings in a peer reviewed paper published in the World Journal of Hepatology, titled ‘Characteristics of patients with Wilson Disease in the United States: An insurance claims database study.’   #WilsonDisease (WD) is a progressive, potentially fatal degenerative disease affecting the liver and central nervous system. The worldwide prevalence of WD has been estimated to occur in one in 30,000 to 40,000 people.   The retrospective cohort study revealed its findings on patients in the US with WD based on a comprehensive insurance claims database due to the ongoing challenges of collecting large cohorts of patients with rare diseases and the low prevalence of WD.   The study evaluating 5,376 patients who had submitted documented claims and identified as suffering with WD displayed increasing amounts of psychiatric and neurological manifestations over a 5-year follow-up period. The study ultimately concluded that patients treated with WD in the US had an excess early mortality ratio of 2.19 when compared with the US population, with a mean age of death at 52.7 years. These findings indicate that there is a significant unmet medical need for more effective treatments for WD in the US, alongside corroborating data published from France, Sweden, Poland, South Korea and the UK.    The statistics in the infographic further highlight the prevalence and ongoing burden of patients with WD. Read the full paper 📰 here https://bit.ly/4bURpY6.   #AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT

June 13 is Rare Chromosome & Gene Disorder Awareness Day, a day that we at #VivetTherapeutics support to raise awareness of unique genetic variations! Today we celebrate all the unique people around the world and their fantastic achievements by shining a spotlight on everyone affected by rare chromosome and gene disorders. At Vivet we are dedicated to developing transformational therapies for those living with rare inherited liver metabolic conditions, including #WilsonDisease, #CTX, #citrullinemiatypeI (#CTLN1) and #ProgressiveFamilialIntrahepaticCholestasis (#PFIC). To learn more about our platforms focused on #genetherapies, follow us on LinkedIn and @Vivet_Tx on X. #RareChromoDay #WilsonsDisease #AAVGeneTherapy

📰 News alert 📰Vivet Therapeutics presents interim data on its Phase 1/2 GATEWAY trial, evaluating the safety, pharmacodynamics, and efficacy of its lead program VTX-801 for the treatment of #WilsonDisease (WD) at EASL | The Home of Hepatology Congress 2024! Key highlights: 📢 VTX-801 increased ceruloplasmin ferroxidase activity and improved liver histology 📢 Encouraging safety and tolerability with no serious adverse events reported #Vivet has successfully dosed the first patient with a higher intravenous dose of lead program, VTX-801 in Cohort 2 and is hoping for a more robust, pharmacodynamic response at a much higher magnitude. Stay up to date on the trial by following Vivet Therapeutics! Read the press release linked in the comments below 📜 #EASLCongress #EASLCongress2024 #AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT

At Vivet Therapeutics we understand that living with a #raredisease such as #WilsonDisease can be physically, mentally, financially, and emotionally exhausting. Key findings published in the international peer-reviewed medical journal - Current Medical Opinion & Research (CMRO), indicate that an unmet need exists among patients with #WilsonDisease (WD) and their care partners, which underscores the need for more effective treatments to reduce the burden of disease and improve the outcomes of patients with WD. Read the full article in the link in the comments below! #Vivet is committed to working with research and development teams to create a diversified pipeline of novel gene therapies that provide treatment options to clinicians, with long lasting benefits that will improve the quality of life of those living with a rare disease. #AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT

Vivet Therapeutics highlights the need for new therapies for #WilsonDisease in a peer reviewed paper titled ‘Treatment of Wilson Disease in Japan: An Insurance Claims Database Study’ in the Japanese Journal of Gastroenterology and Hepatology.   Key figures illustrate the need for more effective treatment paradigms for this progressive, fatal disease in Japan, where treatment rates of patients with Wilson Disease are higher than any other country and life expectancy is curtailed even when current standard of care treatments are available.   #Vivet recently dosed its first patient with its lead program VTX-801 in Cohort 2 of its ongoing GATEWAY clinical trial for the treatment of Wilson Disease, following the successful completion of Cohort 1.   To learn more about Wilson Disease and study statistics in Japan, read the full press release linked below in the comments!

Join Sonia VALERO, Head of Clinical Operations and Patient Advocacy Representative at Vivet Therapeutics' at the 4th Annual Gene Therapy Patient Engagement Summit in Boston, 29-31 May! The summit aims to join gene therapy engagement professionals and patient advocacy groups to gain actionable insights and develop solutions to the current challenges that are unique to the gene therapy space. At #Vivet, we believe that patient engagement is crucial as we continue to develop safe, effective patient-centric gene therapies for rare inherited liver metabolic disorders. See you there! #AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT #GeneTherapy #AAVectors 

Join us in London next week at the 5th Annual Gene Therapy Analytical Development Europe Summit, 22-24 May, where our Chief Operating Officer, Anne DOUAR will be participating in a panel discussion, entitled 'Unpicking the Complexities of Batch-Batch Variability to Enable the Setting of Feasible Thresholds & Avoid Regulatory Obstacles.’   The Panel takes place on 🗓️ 23 May at 🕒 15:15 BST, where Anne will share her insights, along with other key industry leaders as they delve into regulatory guidance on analytical development.   The summit brings together experts in analytical development and will highlight the latest innovations in analytical methods across various delivery modalities (such as AAV, lentiviral, and non-viral approaches) and disease indications, showcasing safety and efficacy to regulatory bodies.   Get in touch if you would like to learn more about Vivet Therapeutics and its gene therapy platform which uses recombinant adeno-associated viruses (#rAAVs) as vectors for rare inherited liver metabolic conditions by using the link in the first comment below!   #AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT #GeneTherapy #AAVectors 

👀Check out our CEO Jean-Philippe Combal on stage! Jean-Philippe wraps up a busy first day at #BioEquity 2024 with a Company presentation updating attendees on Vivet Therapeutics’ latest milestones for its #GATEWAY study for lead program, VTX-801, a novel gene therapy for the treatment of #WilsonDisease.   Do reach out if you would like to connect with Jean-Philippe or #Vivet General Counsel and Head of Finance Susan Coles to find out more about #Vivet and its novel and long-lasting #rAAV based gene therapies for rare inherited liver metabolic disorders including #WilsonsDisease and #CTX   #BioEquityEurope #BioEquity2024 #AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT