bluebird bio Statement on European Regulatory Status of LentiGlobin™
A third party press release was issued today stating that the EMA (European Medicines Agency) issued an approval for the conditional Marketing Authorization Application (MAA) for LentiGlobin™, bluebird bio’s investigational gene therapy for the treatment of transfusion dependent β-thalassemia (TDT). LentiGlobin for TDT is scheduled to be reviewed as part of the CHMP (Committee on Human Medicinal Products) meeting from March 25 – 28, however no opinion has been issued by the CHMP. If the CHMP’s opinion is issued it would then be reviewed by the European Commission (EC), which has the authority to grant approval for the use of LentiGlobin in the EU.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, we’re developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, we’re working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.
bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s plans and expectations for the development, regulatory approval, and commercialization for the LentiGlobin™ in TDT to treat transfusion-dependent β-thalassemia, and the potential implications of clinical data for patients. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that our MAA submitted for LentiGlobin may not be approved by the European Commission when expected, or at all; the risk that the efficacy and safety results from our prior and ongoing clinical trials of LentiGlobin will not continue or be repeated in our ongoing or planned clinical trials of LentiGlobin; the risk that the current or planned clinical trials of LentiGlobin will be insufficient to support regulatory submissions or marketing approval in the US and EU; the risk that the production of HbA T87Q may not be sustained over extended periods of time; and the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of LentiGlobin following regulatory approval. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.
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 ensimmäisten joukossa? Kun tilaat mediatiedotteemme, saat ne sähköpostiisi välittömästi julkaisuhetkellä. Tilauksen voit halutessasi perua milloin tahansa.
Lue lisää julkaisijalta Business Wire
Immersion Signs Agreement With TDK Electronics Bringing Haptics Design and Marketing to PowerHap Piezo Actuators24.4.2019 15:30:00 EEST | Tiedote
Immersion Corporation (NASDAQ:IMMR), the leading developer and licensor of touch feedback technology, announced that it has signed an agreement with TDK Corp. (TSE:6762) subsidiary TDK Electronics for the design and marketing of haptic feedback for TDK PowerHap™ piezo actuators. Under the agreement, Immersion will include TDK’s actuators in reference designs for automotive, industrial and other markets. Immersion will also certify the components for use with its software products. This will enable TDK’s customers in a wide variety of markets to rapidly implement cutting-edge haptic solutions with its high-performance actuators. With 25 years in the field of touch technology, Immersion brings together the art of design and the precise science behind haptic feedback in actuators and ICs. The company connects the haptic ecosystem through the certification of haptic components, helping partners like TDK and their customers maintain consistency and high-fidelity haptic experiences across de
Kite Announces Plans for New State-of-the-Art Facility to Expand Cell Therapy Production Capabilities24.4.2019 15:30:00 EEST | Tiedote
Kite, a Gilead Company (Nasdaq: GILD), today announced plans for a new facility in Frederick County, Maryland, which will produce innovative cell therapies for people with cancer. The 20-acre site will significantly expand Kite’s ability to manufacture a variety of chimeric antigen receptor T (CAR T) therapies, including Yescarta® (axicabtagene ciloleucel), Kite’s first commercially available CAR T cancer therapy, and investigational T cell receptor (TCR) cell therapies being evaluated in solid tumors. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190424005257/en/ “This new facility in Frederick County builds on our substantial technical capabilities and rapid progress in making personalized CAR T and TCR cell therapies for people with cancer. As we advance our industry-leading cell therapy pipeline and seek to help a growing number of people with cancer, expanding and investing in our manufacturing capabilities is essentia
SugarCRM Announces Game-Changing Product Enhancements to Accelerate Growth, Improve Business Performance and Deliver Exceptional Customer Experiences24.4.2019 15:00:00 EEST | Tiedote
SugarCRM Inc.®, the company that helps organizations build better business relationships, today announced several new platform capabilities to help companies to accelerate their business growth. These new capabilities will enable them to resolve customer issues quickly, collaborate more effectively, make faster and more informed decisions about their customers, eliminate time-consuming manual processes, expand real-time mobile access to critical customer information – and do all of this on the new, blazing-fast Sugar 9 platform. “With these significant enhancements, our customers can now collaborate and communicate more effectively, know more about their customers to quickly make better decisions and confidently transact more business with faster quotes, more automation and rock-solid SOC 2 compliance. Most importantly, we have boosted system performance to new heights, enabling customers to process up to 70% more transactions without investing in new hardware,” says Rich Green, chief
USD$90 Million Initiative between A*Star, Aptorum Group and Aeneas Capital to Drive Healthcare Innovations24.4.2019 15:00:00 EEST | Tiedote
Aptorum Group Limited (Nasdaq: APM), Aeneas Capital Limited, and A*ccelerate Technologies Pte Ltd, the enterprise office of the Agency for Science, Technology and Research (A*STAR), have signed a USD$90 million agreement to co-create local deep tech start-ups in the healthcare and life sciences sector. This agreement, is the latest in a series of venture co-creation (VCC) agreements signed by A*ccelerate to strengthen the start-up ecosystem in Singapore. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190424005535/en/ (Photo: Business Wire) Through this agreement, Aptorum Group and A*ccelerate are looking at creating up to 20 deep tech ventures in Singapore over the next five years. These enterprises will leverage technologies co-developed by A*STAR research institutes and Aptorum Group. As part of this agreement, the parties involved will also support the start-ups’ growth through actively nurturing the entrepreneurs. This w
IONITY Fast Charging Network Relies on has·to·be Know-How for the Long Term24.4.2019 13:18:00 EEST | Tiedote
The IONITY joint venture aims to help electromobility achieve a breakthrough in Europe with a nationwide network of super-fast charging stations. The Austrian electromobility provider, has·to·be, is playing an important role in this. The red-white-red company will take care of the reliable and trouble-free operation of the charging stations. The two companies have agreed on a long-term cooperation so that the expansion of the Europe-wide network can proceed according to plan. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190424005465/en/ has·to·be and IONITY focus on long-term cooperation (Graphic: Business Wire) has·to·be, based in Radstadt (Salzburg), specializes in smart solutions for e-charging stations. For this purpose, the company has developed intelligent software for the management of electrical charging infrastructures. The product is called be.ENERGISED and is used successfully at more than 14,000 charging statio
Knopp Biosciences Receives FDA Orphan Drug Designation for Dexpramipexole for Treatment of Hypereosinophilic Syndrome24.4.2019 13:00:00 EEST | Tiedote
Knopp Biosciences LLC, a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases with high unmet needs, announced today that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation to dexpramipexole, its oral drug candidate for the treatment of hypereosinophilic syndrome (HES). HES is a rare and often incapacitating disorder with limited treatment options. The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose, or prevent diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees. “We are very pleased to receive orphan drug designation for dexpramipexole for HES,” said Michael Bozik, M.D., CEO of Knopp Biosc
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