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CHOP celebrates EC approval of first and only gene therapy for patients

first_imgReviewed by James Ives, M.Psych. (Editor)Dec 6 2018Children’s Hospital of Philadelphia (CHOP) celebrates a pivotal moment in medicine: approval by the European Commission (EC) of LUXTURNA® (voretigene neparvovec), the first and only gene therapy for patients with an inherited retinal disease, last month. This also makes LUXTURNA the first gene therapy for a genetic disease that has received regulatory approval in both the U.S. and European Union (EU).The EC approved LUXTURNA, a one-time gene therapy for the treatment of vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations, in pediatric and adult patients who have sufficient viable retinal cells. RPE65 -mediated inherited retinal disease is a progressive condition that leads to total blindness in most patients.The authorization is valid in all 28 member states of the EU, as well as Iceland, Liechtenstein and Norway. In December 2017, the U.S. Food and Drug Administration (FDA) approved LUXTURNA for use in patients in the U.S.”The European Commission’s approval of LUXTURNA highlights the vital role of pediatric research in developing breakthrough cures,” said Bryan Wolf, MD, PhD, Chief Scientific Officer and Chair of the Department of Biomedical and Health Informatics at Children’s Hospital of Philadelphia. “The research conducted as a collaborative effort between CHOP’s Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) and investigators at the Perelman School of Medicine at the University of Pennsylvania laid the groundwork for this revolutionary gene therapy, which was developed and is now manufactured by Spark Therapeutics. Today, we are thrilled to see LUXTURNA approved as a therapy for children and adults outside the U.S.”Related StoriesGuidelines to help children develop healthy habits early in lifeResearchers identify gene mutations linked to leukemia in children with Down’s syndromeRevolutionary gene replacement surgery restores vision in patients with retinal degenerationCHOP founded Spark Therapeutics in 2013 in an effort to accelerate the timeline for bringing new gene therapies to market. Spark’s mission, to create a world where no life is limited by genetic disease, was to build on the foundational research conducted over a multi-year period by the CHOP and Penn Medicine teams.Beginning in 2000, the initial research for LUXTURNA was conducted by Jean Bennett, MD, PhD, F.M. Kirby professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania’s Scheie Eye Institute, and Albert M. Maguire, MD, a professor of Ophthalmology at Penn’s Perelman School of Medicine and an attending physician at CHOP. Bennett and Maguire joined forces with then-CHOP researcher Katherine A. High, MD, a gene therapy pioneer who directed the CCMT and who is now Spark’s President and head of research and development. Dr. Maguire served as a Principal Investigator of the therapy’s clinical trials.In the U.S., the gene therapy is currently administered at 10 treatment centers by leading retinal surgeons who receive training provided by Spark Therapeutics on the administration procedure.In January 2018, Spark Therapeutics entered into a licensing and supply agreement with Novartis covering development, registration and commercialization rights to LUXTURNA in markets outside the U.S. Upon the transfer of the marketing authorization from Spark Therapeutics to Novartis. Novartis can commercialize LUXTURNA in the EU/EEA. Novartis already has exclusive rights to pursue development, registration and commercialization in all other countries outside the U.S., and Spark Therapeutics will supply the gene therapy to Novartis.Source: https://www.chop.edu/news/children-s-hospital-celebrates-european-commission-approval-first-its-kind-gene-therapylast_img read more

Videoondemand and the myth of endless choice

first_img Film industry eyes Internet future at Venice fest If you like independent, art-house films or other specialised movies, you may have heard of the Romanian comedy-drama Sieranevada, which was released in 2016. The film was formally premiered as part of the main competition programme of the prestigious Cannes Film Festival and was subsequently shown at other international film festivals, including Toronto, New York and London. Due to its success on the festival circuit, Sieranevada was reviewed by 48 international film critics, and received a positive rating from 92% of them. Among these were UK-based trade journals, such as Sight and Sound and Screen International as well as mainstream newspapers The Guardian and The Telegraph. But while this publicity generated audience interest in the film, it has yet to secure distribution that would allow UK audiences to actually watch the film – it’s not in cinemas, on DVD/Blu-ray, nor on online video-on-demand platforms (VOD).The development of VOD has provided new opportunities for films to reach audiences. In particular, specialised films with traditionally limited distribution opportunities have taken advantage of this development. But are online audiences presented with an endless choice? Not really. So why is this?The digital film revolutionIn the mid-2000s, digital utopians such as Chris Anderson were already arguing that an endless choice of specialised and niche content would become available to online audiences. And more than a decade later, it’s true that distribution opportunities have increased for such content in the online market. Film audiences are able to browse through catalogues on transactional VOD platforms such as Amazon Video, Microsoft and iTunes where they can find tens of thousands of films. Provided by The Conversation Why can’t we get everything?In the film business, sales companies have an important role to play in the process of enabling access for films because they negotiate distribution deals with a range of distributors in international markets. But if sales companies are unable to sell distribution rights, they retain control over the distribution and release of those films.The development of the online market, in this respect, has opened up opportunities for them to work directly with VOD platforms or with content aggregators, who work as intermediaries between rights holders and VOD platforms. Examples of such content aggregators include The Movie Partnership, Juice Worldwide and Gunpowder & Sky.For instance, the comedy-drama Dreamland (2016), directed by Robert Schwartzman, premiered in the US Narrative Competition of the Tribeca Film Festival. The US sales company FilmBuff (now named Gunpowder & Sky) acquired worldwide distribution rights. In the UK market, the film was not released in cinemas or on DVD or Blu-ray, but FilmBuff made it available in the online market through direct connections with Microsoft and iTunes rather than via a UK distributor.Despite such opportunities, sales companies do not always work with content aggregators or directly with VOD platforms to make specialised films available if they are not picked up by distributors. Making films available online requires organisational effort and a low-cost investment in digital formatting, but returns on investment can be very modest. That explains why some films remain inaccessible to audiences – as demonstrated for the UK market in the table below. This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only. A selection of films released by distributors in the UK market. BFI weekend box office figures, IMDb, Amazon, Microsoft, iTunes, Author provided Endless choiceThe politics behind the process of providing access to specialised films ultimately affects producers and audiences. In the new digital economy of attention, producers demand wider distribution for their films, while audiences demand endless choice.This issue needs to be resolved. First, it needs to be addressed in film industry discussions between film producers and sales companies. In particular, sales companies should make a stronger commitment to making films available on transactional VOD platforms.Second, policymakers can intervene in the process of making specialised films available online. Public funding agencies, such as the British Film Institute (BFI) in the UK, provide substantial financial support for the production of specialised films. They can provide more distribution incentives to support cultural diversity in the online market for films in the UK. This would help to support greater cultural diversity, democratisation of access to films and enhancement of consumer choice. While cinema goers have always had limited options when it comes to the number of screens they can see their favourite art house movie on, the internet era was supposed to bring with it an endless choice. But what is becoming clear is that this utopian dream is still far from being realised. But there is still a significant proportion of films that remain inaccessible for audiences, even if – like Sieranevada – they have been selected for prestigious international film festivals.center_img This article was originally published on The Conversation. Read the original article. Explore further Number of specialised films released in the UK market. BFI weekend box office figures, Amazon, Microsoft, iTunes, Author provided What is available?In an effort to identify the proportion of well-regarded specialised films that reach audiences in the UK market, I analysed a sample of 119 such films shown at prestigious European and US film festivals in 2016. My analysis in the graphic below confirms that the online market creates distribution opportunities for a greater number of films than the theatrical cinema and DVD/Blu-ray markets:88 specialised films (74%) were given an online release on Amazon, Microsoft or iTunes71 specialised films (60%) were given a DVD/Blu-ray release61 specialised films (51%) were given a theatrical cinema releaseBut while access to specialised films has increased, 26% of specialised films remain inaccessible for audiences on any format. That is a remarkably high percentage – given that it is relatively easy to secure online access for films. My analysis includes a sample of specialised films selected for some of the most prestigious festival programmes, but it is likely that online availability is more limited among specialised films selected for less prestigious competitions. So why can’t online audiences see any film they want? It’s to do with the way the industry works. Credit: Shutterstock Citation: Video-on-demand and the myth of ‘endless choice’ (2018, August 16) retrieved 18 July 2019 from https://phys.org/news/2018-08-video-on-demand-myth-endless-choice.html Example of Amazon’s transactional VOD platform as of July 6, 2018 (Amazon’s catalogue includes 50,000 films). Credit: Amazon.co.uk, Author providedlast_img read more

Amazon hits trilliondollar milestone with focus on the long game Update

first_img Citation: Amazon hits trillion-dollar milestone with focus on the long game (Update) (2018, September 4) retrieved 17 July 2019 from https://phys.org/news/2018-09-amazon-tops-trillion-stock.html Amazon Spheres, in Seattle, Washington, is part of the home of the online giant, which is seeking a second North American headquarters Explore further Amazon CEO and founder Jeff Bezos, seen with his wife MacKenzie Bezos, has become the world’s richest person based on his company stake but has also created a private space exploration firm and has purchased the Washington Post newspaper Amazon’s market value hit $1 trillion on Tuesday, the second company after Apple to hit the milestone, following an incredible journey for the internet giant which has kept a long-term focus since launching as an online bookseller two decades ago. For example, Amazon’s lucrative cloud computing business is built on technology infrastructure that the company needed to run its own operations.Investing in warehouses, trucking, drones, shipping and other distribution systems not only enables Amazon to drive down costs they position the company to compete with the likes of FedEx and UPS.Buying the Whole Foods grocery chain last year got Amazon established real world outlets while putting its delivery and retail smarts and systems to work in the brick-and-mortar world.Drugs and digital adsPrescription medicine would be a natural market for Amazon to expand into, according to Enderle. Meanwhile, Amazon is reportedly beefing up its digital advertising business to better compete in an online ad market dominated by Google and Facebook.In the past quarter, Amazon posted its best-ever profit of $2.5 billion as Bezos, whose stake in the company has made him the richest person, highlighted the importance of digital assistant Alexa that powers Amazon electronics along with cars, appliances and other connected devices. According to the research firm eMarketer, Amazon’s e-commerce revenue will grow more than 28 percent this year to reach $394 billion, and will account for 49 percent of US online retail sales and nearly five percent of all retail spending.One of Amazon’s revenue drivers is its Prime subscription service which offers streaming video and music, free delivery and other perks and which has more than 100 million members worldwide.Arrogance trapSome fear Amazon is becoming too dominant a force, especially in retail, sparking antitrust discussion even as the company keeps expanding globally and searches for a second headquarters in North America.”It wasn’t that long ago that people were freaking out about Walmart, and Amazon basically stepped on Walmart,” analyst Enderle said.”What Amazon means is disruption and people don’t like to be disrupted.”Critics of the company include US President Donald Trump, who has expressed ire at the Bezos-owned Washington Post newspaper that has published stories the president didn’t like.Bezos bought the Washington Post five years ago for $250 million from his personal funds.While his skills could be advantageous in the content-oriented business, getting into news comes with the risk of displeasing politicians.”The Post was a mistake because it results in him going to war with people he wouldn’t otherwise go to war with,” Enderle said.Amazon’s huge cloud computing segment powers systems for government clients, and contracts could be influenced by politics.Amazon must also guard against the kind of arrogance that can undo companies that come to dominate markets, according to the analyst.”If Amazon does have a downfall, it will be arrogance in dealing with the customer,” Enderle said. Amazon powers up profits as footprint grows Early gains lifted Amazon’s value to $1 trillion only briefly, with the final close at $2,039.51 giving it a value of $995 billion.Amazon’s journey from an online bookseller in a garage to a global e-commerce powerhouse has centered on obsession with the long road.The company initially incorporated as “Cadabra” by Jeff Bezos in 1994 and backed with money borrowed from his parents joined Apple as the second US technology firm to be valued at $1 trillion on Tuesday.Apple crossed the trillion-dollar value threshold a month ago and has remained above it. Amazon became the second US company at that eye-popping value.GlobalData Retail managing director Neil Saunders called Amazon’s valuation achievement “extraordinary” and considered it a sign of the company’s potential.”Despite its size and scale, there is still something young about Amazon,” Saunders said.”Amazon is really only just getting started.”Created in a garage in a suburb of Seattle, Washington, the company renamed “Amazon” sold its first book—Fluid Concepts and Creative Analogies: Computer Models of the Fundamental Mechanisms of Thought by Douglas Hofstadter—in mid-1995.By the end of that year, Amazon was selling books online throughout the US. Amazon went public in early 1997.The company for more than a decade put growth over profit, investing heavily in warehouses, distribution networks, and data centers.”Every cent they made they put back in the company,” said independent technology analyst Rob Enderle. “They kept their eye on the prize, which was initially to take over most of commerce.”Innovation sans scandalSaunders said Amazon’s success comes from the fact that it innovates unlike any other.”This heady pace of creativity is the key reason why it stays several steps ahead of the market and is able to generate so much growth,” Saunders said.Bezos has kept firm control of Amazon, steering clear of hedge fund investors inclined to short-term tactics aimed at getting share prices to jump.The founder and chief executive also avoided scandals or other distractions, keeping revenue and costs close enough to manage and easing into “adjacent markets” that play into Amazon strengths or interests, according to Enderle. © 2018 AFP Amazon has expanded from its modest origins two decades ago as an online bookseller to a global powerhouse worth $1 trillion This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.last_img read more

GE pushes back Q3 earnings release to October 30

first_img GE shares at 9-year low amid latest power woes The company postponed the release by a week “to allow GE Chairman and CEO Larry Culp to complete initial business reviews and site visits following his appointment on October 1,” it said in a statement The company postponed the release by a week “to allow GE Chairman and CEO Larry Culp to complete initial business reviews and site visits following his appointment on October 1,” it said in a statement.”The company will discuss the results for the third quarter ending September 30, 2018. Culp will share his initial observations, with more detail expected in early 2019.”H. Lawrence “Larry” Culp, 55, is GE’s third CEO in 14 months following the ouster of John Flannery.In announcing Culp’s rise, the company, which was bumped from the prestigious Dow Jones Industrial Average in June, cited his history as chief executive of the industrial and healthcare conglomerate Danaher, where GE said he presided over a quintupling of market capitalization. Culp was named to the GE board earlier this year.GE’s other bombshell was that it planned to write down effectively up to $23 billion in value from its troubled power business, the prime catalyst of its nosedive in stock market valuation. Citation: GE pushes back Q3 earnings release to October 30 (2018, October 13) retrieved 17 July 2019 from https://phys.org/news/2018-10-ge-q3-october.html General Electric, the once-mighty conglomerate which weeks ago announced a new chief executive in a move meant to stem a two-year decline, has said it will move the date of its third-quarter earnings release to October 30.center_img © 2018 AFP Explore further This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.last_img read more