VIVET-THERAPEUTICS
1.6.2020 13:02:07 CEST | Business Wire | Press release
Vivet Therapeutics announced today that both the Food and Drug Administration (FDA) and the European Commission (EC) have granted Orphan Drug Designation (ODD) for Vivet’s second gene therapy product, VTX-803, for the treatment of Progressive Familial Intrahepatic Cholestasis type 3 (PFIC3). PFIC3 is a rare life-threatening and chronically debilitating condition due to progressive severe liver dysfunction, accompanied by jaundice, portal hypertension, hepatosplenomegaly and failure to thrive. The symptoms of the disease usually appear first in childhood with progressive cholestasis, evolving to hepatic failure, cirrhosis and need for liver transplantation.
“We are very pleased to receive these designations which further validate Vivet’s efforts to treat rare inherited metabolic disorders. The FDA and EC have both recognized the unmet need for a safe and effective treatment of PFIC3 and the potential of VTX-803 to address such need. The field of Progressive Familial Intrahepatic Cholestasis has been gaining increased attention in the medical field with numerous ongoing clinical trials. There is a high unmet medical need, especially in the paediatric segment, for this condition, for which liver transplantation remains the only truly curative option today” stated Jean-Philippe Combal, co-founder & CEO of Vivet Therapeutics.
VTX-803 is Vivet’s second gene therapy product to be granted Orphan Drug Designation. This is an important value driver for Vivet which would provide 10 & 7 years of market exclusivity in the EU & US respectively if VTX-803 is approved for the treatment of PFIC3. VTX-803 is currently under IND-enabling studies and initiation of clinical development is planned for the second part of 2021 in both the US and the EU.
“We are very happy about this recognition of our scientific achievement for a complex liver disease and further validation of our strategic collaboration with Fundación para la Investigación Médica Aplicada (FIMA). This program could be a game-changer in the gene therapy field for PFIC disorders” added Dr. Gloria González-Aseguinolaza, Vivet Therapeutics co-founder & CSO and deputy director at CIMA.
VTX-803 was recently the subject of an oral presentation at 2019 ASGCT (American Society of Gene and Cell Therapy), a lecture at the 2019 ESGCT (European Society of Gene and Cell Therapy) annual meetings and a publication in Nature Communications (Weber et al. Nature Communications, 2019, 10(1):5694).
About Vivet Therapeutics
Vivet Therapeutics is an emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.
Vivet is building a diversified gene therapy pipeline based on novel adeno-associated virus (AAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada, University of Navarra based in Pamplona, Spain.
Vivet’s lead program, VTX-801, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation by the Food and Drug Administration and the European Commission.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200601005221/en/
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Nadia Karkar Joins 500 Global as Managing Partner7.4.2026 19:43:00 CEST | Press release
Former TPG Rise executive joins as Managing Partner as the firm expands its global investment platform. 500 Global today announced the appointment of Nadia Karkar as Managing Partner as the firm expands its global investment platform. Nadia’s appointment follows the recent announcement of the appointment of Atul Mehta — former Chief Investment Officer of the International Finance Corporation — to 500 Global's Board of Directors, and reflects the firm's continued build-out of the leadership and capabilities required to deliver on its next stage of global growth. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260407719214/en/ Nadia Karkar Joins 500 Global as Managing Partner Nadia joins from TPG Rise, the $31 billion impact investing platform of TPG Inc., where she most recently served as Head of Business Development, responsible for product innovation, strategic partnerships and corporate development. She played a central rol
Mouser Electronics Explores How Artificial Intelligence Shapes Everyday Technologies and Experiences7.4.2026 17:11:00 CEST | Press release
Mouser Electronics, Inc., the authorized global distributor with the newest electronic components and industrial automation products, today announced the first 2026 installment of its Empowering Innovation Together (EIT) technology series, Engineering AI for Daily Life. This installment explores how artificial intelligence is increasingly embedded in everyday products and services, from assisted search and messaging tools to healthcare wearables that monitor personal well-being. As AI capabilities expand across consumer and connected devices, engineers continue to design systems that make these technologies more useful, intuitive, and trustworthy in real-world applications. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260407524737/en/ This installment of Empowering Innovation Together explores how AI is increasingly embedded in everyday products and services, from assisted search and messaging tools to healthcare wearables
Umrah and Ziyarah Forum Concludes in Madinah with Landmark Achievements in Pilgrim Services7.4.2026 16:38:00 CEST | Press release
Under the generous patronage of His Royal Highness Prince Salman bin Sultan bin Abdulaziz, Governor of the Madinah Region, the third edition of the Umrah, Hajj, and Ziyarah Forum concluded its activities, marking a significant milestone in the Kingdom's mission to enhance the experience for the "Guests of Allah." This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260407456369/en/ Umrah and Ziyarah Forum Concludes in Madinah with Landmark Achievements in Pilgrim Services (Photo: AETOSWire) The final harvest of the forum resulted in exceptional numbers and a massive turnout, with more than 50,000 visitors flocking to the accompanying exhibition, which attracted over 150 exhibitors from global and local companies across an area exceeding 11,000 square meters. To enrich the knowledge aspect, the three days of the forum featured more than 160 speakers and trainers who contributed to discussions across 25 main sessions and 40 workshop
Poland's Galaxy Systemy Informatyczne Becomes First Private Enterprise to Buy Quantum Computer from IQM7.4.2026 15:00:00 CEST | Press release
IQM will deliver and install the 54-qubit quantum computer at Galaxy in the fourth quarter of 2026. The deployment builds Galaxy’s quantum capabilities, supporting talent development and innovation across sectors, while reinforcing Poland’s growing quantum ecosystem. This will be another Radiance 54-qubit computer deployed this time in Poland by IQM, following installations at LRZ in Germany, VTT in Finland, CINECA in Italy, and CESGA in Spain, underscoring the company’s leading market position. IQM Quantum Computers, a global leader in superconducting quantum computing, today announced a commercial milestone to deploy a hybrid integrated quantum computer at GalaxySystemy Informatyczne Sp. z o.o., marking its first deployment in a private enterprise worldwide. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260407351934/en/ IQM Radiance quantum computer Galaxy has been implementing innovations in security, digitisation, and h
Variational AI Releases Enki 4: Major Update to Foundation Model for Small-Molecule Drug Discovery7.4.2026 15:00:00 CEST | Press release
Variational AI introduces Enki™ 4: an improved algorithm and architecture designed to scale, expanding the pre-trained target coverage from 592 to 760 and now applicable to proximity-based therapeutics and novel payloads for antibody drug conjugates Variational AI today announced the release of Enki™ 4, a major update to its generative AI platform for small-molecule drug discovery. The release of the fourth generation of Enki™ delivers significant improvements in algorithmic performance and target coverage through platform architecture design, as well as extending its application to degraders, PROTACs, glues, and novel payload design for antibody drug conjugates (ADCs) and related modalities. “Enki 4 is a massive step forward for our platform,” said Ali Saberali, Co-Founder & Head of Platform at Variational AI. “We’ve re-architected Enki and improved the underlying algorithms to expand target and modality coverage, while operating faster to deliver even better performance for our partn
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom