Business Wire

Vivet’s First Gene Therapy Product, VTX 801 for Wilson’s Disease, Receives European and US Orphan Drug Designation

Jaa

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 lead gene therapy product, VTX-801, for the treatment of Wilson’s Disease (WD), a chronically debilitating and life-threatening condition if not treated.

“This designation validates Vivet’s efforts to treat the copper metabolism disorder, Wilson’s Disease. The FDA and EC have both recognized the unmet need for a safe and effective treatment of Wilson’s Disease by physiological restoration of copper homeostasis and the potential of VTX 801 to address such need" stated Jean-Philippe Combal, CEO of Vivet Therapeutics.

VTX-801 is Vivet’s first gene therapy product to be granted Orphan Drug Designation. This is an important value driver for Vivet, in particular through the 10 & 7-years market exclusivity in the EU & US respectively if VTX-801 is approved for the treatment of Wilson’s Disease. Initiation of clinical development is planned for Q4 2018 in both the US and the EU.

“Through our strategic collaboration with Fundación para la Investigación Médica Aplicada (FIMA) at the Centro de Investigación Medica Aplicada (University of Navarra, Spain), we are building an unparalleled platform to address key challenges in gene therapy, notably improving sustainability and immune tolerance, targeting Wilson’s Disease and other hepatic rare metabolic genetic disorders. A critical asset to this platform is a new liver targeting AAV-Anc80 serotype, licensed from Massachusetts Eye and Ear (MEE), a teaching hospital of Harvard Medical School, Boston" added Jean-Philippe Combal.

For more information, visit www.vivet-therapeutics.com

About VTX-801: VTX-801 is an adeno-associated virus (AAV) containing a gene coding for a functional mini-ATP7B copper transporter, that is intended to restore copper metabolism through liver cell targeting.

About WiIson’s Disease: Wilson’s disease is an inherited orphan disorder affecting approximately 1:40,000 individuals. In this condition, the physiological copper biliary elimination pathway is disrupted, leading to tissue accumulation of toxic copper levels, most notably in liver and CNS. Onset of symptoms typically occurs in teenagers or in young adults and left untreated, the condition uniformly progresses to severely debilitating complications and death. The disease is due to inactivating mutations in the gene encoding the copper transporter ATP7B.

About Vivet Therapeutics: Vivet Therapeutics is a gene therapy biotech company with headquarters in Paris, France, dedicated to the research, development and future commercialization of gene therapy products for inherited liver disorders with high medical need.

Contact information

Vivet Therapeutics
Media & Investor:
Thomas Daniel, +33 6 68 88 38 60
Business Development Director
info@vivet-therapeutics.com

Tietoja julkaisijasta

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Tilaa tiedotteet sähköpostiisi

Haluatko tietää asioista jo ennen kuin ne uutisoidaan? Kun tilaat tiedotteemme tältä julkaisijalta, saat ne sähköpostiisi yhtä aikaa suomalaisen median kanssa. Tilauksen voit halutessasi perua milloin tahansa.

Lue lisää julkaisijalta Business Wire

Integral Expands Cryptocurrency Market Data Services to Include 14 Cryptocurrencies and 16 Exchanges24.1.2018 01:00Tiedote

Integral (www.integral.com), the financial technology partner trusted by leading banks, brokers, and investment managers to design, deliver and grow their FX businesses, announced today that it has expanded its market data offering for cryptocurrencies to include 14 major cryptocurrencies connecting to all major exchange sources across the U.S., Europe and Asia including Japan and Hong Kong. “An accurate, stable, and reliable reference price is a requirement for any market maker,” said Harpal Sandhu, CEO of Integral. “The Cryptocurrency Market Data Service connects to the most price sources and applies the most advanced price discovery algorithm available.” Integral has years of experience as a technology leader and innovator in foreign exchange and is now applying this ingenuity to the cryptocurrency market. The Market Data Service uses algorithms developed with Stanford University for Integral FX Benchmark to provide the most precise reference prices for cryptocurrencies. “Integral’s

Kimbal Riley to Become Vista Group CEO24.1.2018 00:57Tiedote

Vista Group International (NZX & ASX: VGL) has today announced that Kimbal Riley will take over as Group CEO from Murray Holdaway. Mr Holdaway will take up the position of Chief Product Officer (CPO) for Vista Group, and will continue in his role as an Executive Director on the Vista Group board. This has taken place as part of a succession planning process and will deliver a seamless transition within the business. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180123006545/en/ Kimbal Riley becomes CEO of Vista Group International (Photo: Business Wire) Mr Riley has had an extensive career in senior executive roles in the IT and Services industries in New Zealand and overseas. He joined Vista Group four years ago, with the past two years as CEO of Vista Entertainment Solutions (VES), which is responsible for the largest proportion of the Group’s revenue. He has been focused on building scale in the business and under his lea

IFF Breaks Ground on Flavors/Fragrances Site in Sri City, India23.1.2018 20:00Tiedote

Regulatory News: International Flavors & Fragrances Inc. (NYSE: IFF) (Euronext Paris: IFF), a leading innovator of sensory experiences that move the world, has broken ground on a new flavors and fragrances plant located in Sri City, in the State of Andhra Pradesh in India. Part of a larger investment into the Greater Asia region, the plant is expected to be commercialized in December 2019. The new facility, which will be the largest flavor and fragrance manufacturing facility in India and the largest IFF site in Greater Asia, occupies a 32-acre/130,000m2 plot and will serve IFF flavors and fragrances customers across India and the region. “In this economically vibrant country, it is critical that we invest to provide our customers with winning tastes and scents,” said IFF Chairman and CEO Andreas Fibig. “This new facility is fitted with capabilities tailored for our customers’ needs. Coupled with our excellent consumer insights teams and their deep understanding of the local markets, w

Optiv Security Names Simon Church as General Manager and Executive Vice President, Europe; Accelerates International Growth Strategy with Expansion in Europe23.1.2018 17:05Tiedote

Optiv Security, a market-leading provider of end-to-end cyber security solutions, today announced it is continuing to accelerate its international growth strategy by naming Simon Church as general manager and executive vice president, Europe. In his newly created role, Church will establish and grow Optiv’s presence in Europe, enabling the company to serve locally-based organizations and global clients with expanded skills, knowledge and presence. Optiv serves more than 60% of the Fortune 1000, and this appointment will increase its ability to provide US and international clients with worldwide expertise and experience, assisting them in reducing the complexity of their cyber security programs while maximizing the efficacy and value of their security investments. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180123005315/en/ Optiv Security Names Simon Church as General Manager and Executive Vice President, Europe; Accelerate

Ad-tech Company Calldorado Acquires App Publishing House CIAmedia23.1.2018 17:00Tiedote

Danish-based ad tech firm Calldorado has expanded its reach in the app economy by acquiring the Austrian app publisher CIAmedia for an undisclosed amount. The acquisition comes after a successful period of cooperation between the two companies and is a natural next step on Calldorado’s path of accelerated growth. “By acquiring CIAmedia and its portfolio of high quality apps, we are extending the Calldorado value chain. Calldorado and CIAmedia have been in cooperation before, and CIAmedia’s focus on Android apps within the Communication and Tools categories is a perfect fit with our mobile advertising technology,” says Claudia Dreier-Poepperl, CEO and Founder of Calldorado. “Calldorado’s vision is to be the world’s number one provider of call-based advertising technology, and by owning and operating a portfolio of relevant apps we are better positioned to realize this vision.” CIAmedia was established 2013 in Vienna, Austria. CIAmedia owns and operates the highly successful app acquisit

LEGO® Education Launches New Maker Activities for Schools23.1.2018 17:00Tiedote

LEGO® Education Maker, a continuum of activities for early learning through middle school, is available today to schools around the world. The activities are centered around the iconic LEGO brick and inspired by the open-ended, playful creativity of making. The new, free LEGO Education Maker activities include stimulating challenges for every student at every education level and incorporate LEGO Education solutions including LEGO Education STEAM Park, LEGO Education Simple Machines, LEGO Education WeDo 2.0, LEGO® MINDSTORMS® Education EV3 and LEGO Education Simple and Powered Machines. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180123005927/en/ LEGO® Education Launches New Maker Activities for Schools (Photo: Business Wire) New LEGO Education Maker activities provide unlimited learning for all ages The new LEGO Education Maker activities place educators in the role of facilitators, as they guide their students through a s

Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.

Tutustu uutishuoneeseemme