Methylmalonic Acidemia (MMA) Gene Therapy – Charles Venditti and Randy Chandler – Video

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Methylmalonic Acidemia (MMA) Gene Therapy - Charles Venditti and Randy Chandler
Dr. Charles Venditti and Dr. Randy Chandler discuss recent developments in gene therapy for methylmalonic acidemia (MMA) - a group of inherited disorders in which the body is unable to process...

By: GenomeTV

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Methylmalonic Acidemia (MMA) Gene Therapy - Charles Venditti and Randy Chandler - Video

Regenxbio nets $30M to bring its gene therapy to clinical trial

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Washington, D.C.-basedgene therapy outfit Regenxbio just raised $30 million to bringits platforminto the clinic.

The dollars will help Regenxbio generate clinical proof of concept data, as well as work toward in-licensing new programs. The companys also using the money to beef up its clinical and manufacturing processes, it said in a statement.

Regenxbio has developed what it calls NAV Technology a form of adeno-associated viral gene therapy that treats lysosomal storage disorders and ocular disease. Its got drugs in the pipeline that treat Hurler syndrome, Hunter syndrome, wet age-related macular degeneration and X-linked retinitis pigmentosa.

Heres how it works: In Hurler syndrome, for instance, children dont carry a gene that develops an enzyme called IDUA that breaks down complex sugars. These build up, and ultimately impairmental development, organ function, physical abilities and appearance. The NAV platform delivers a normal copy of the IDUA-producing gene which ultimately embeds itself into a patients DNA in a one-time doze so that patients can produce the enzyme. The research comes out of the University of Pennsylvania, and has been successful in vivo so its a matter of testing its efficacy in a real patient pool.

The companys also out-licensing this technology for other indications, with companies like Baxter, Dimension Therapeutics and Lysogene developing viral vector-based gene therapy for a number of other indications.

The Series C funding was led by Venrock and Brookside Capital, with other investors like Deerfield Management, FoxKiser and Fidelity Biosciences.

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Regenxbio nets $30M to bring its gene therapy to clinical trial

U.S. Gene Therapy Clinical Trial to treat Choroideremia initiated

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PHILADELPHIA, Penn., January 20, 2015- Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today it has initiated a Phase 1/2 clinical trial for the potential treatment of patients with choroideremia (CHM) utilizing its gene therapy product SPK-CHM. The Phase 1/2 trial is an open-label, dose-escalating trial designed to assess the safety and preliminary efficacy of sub-retinal administration of SPK-CHM. The Phase 1/2 trial will be conducted at The Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania, and has plans to enroll up to 10 patients afflicted with the CHM genetic mutation.

"Spark's groundbreaking announcement today brings real hope of a treatment for blindness caused by choroideremia, and promises to pave the way for treatments of other retinal diseases impacting millions of people around the World," said Dr. Chris Moen, President of the Choroideremia Research Foundation (curechm.org), the leading advocacy and fundraising organization focused on finding a cure for CHM. "The Choroideremia Research Foundation is proud to have provided key pre-clinical funding to Jean Bennett, MD, PhD and her team atthe Perelman School of Medicine at the University of Pennsylvania,that has helped bring us to the gene therapy human clinical trials being announced today."

Dr. Jean Bennett's pre-clinical work, which was funded in part by consistent financial support from the Choroideremia Research Foundation (curechm.org), demonstrated the ability of the SPK-CHM gene therapy to restore REP-1 protein production, membrane trafficking and retinal structure.

"Throughout my career's work developing genetic therapies for inherited retinal dystrophies I have had my target set on a number of different conditions, in particular, choroideremia," said Dr. Bennett, who is also one of Spark's scientific co-founders and a scientific advisor on the SPK-RPE65 clinical trials being conducted at CHOP. "The SPK-CHM program, for the first time, creates the potential for patients to use their vision for longer and see more things."

In addition to evaluating safety, the trial will help define the dose required to achieve stable or improved visual function and identify appropriate endpoints for subsequent clinical trials. With SPK-CHM, Spark is leveraging the experience and technology utilized in the development of its gene therapy for Leber's Congenital Amaurosis (LCA), SPK-RPE65, including the same vector, target cells and route of administration, as well as the same manufacturing process. SPK-RPE65 is currently in a fully-enrolled pivotal Phase 3 clinical trial.

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U.S. Gene Therapy Clinical Trial to treat Choroideremia initiated

Future Trendspots: Futurist Perspective on Netflix, the German Football Team, and Big Data – Video

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Future Trendspots: Futurist Perspective on Netflix, the German Football Team, and Big Data
In this video, global futurist Anders Sorman-Nilsson dives into the world of Big Data and recounts his experiences at the 2014 World Cup, when Big Data enter...

By: Anders Sorman-Nilsson

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Future Trendspots: Futurist Perspective on Netflix, the German Football Team, and Big Data - Video

Dr. Amy Zalman, World Future Society President @ Brooklyn Futurist Meetup – Video

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Dr. Amy Zalman, World Future Society President @ Brooklyn Futurist Meetup
Brooklyn Futurist Meetup presents Dr. Amy Zalman, the President of The World Future Society. at Brooklyn Law School, New York, on Wednesday, January 21, 2015.

By: Brooklyn Futurist Meetup

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Dr. Amy Zalman, World Future Society President @ Brooklyn Futurist Meetup - Video

3D Printing Visionaries from Autodesk, 3D Systems-Gentle Giant Studios to Deliver Keynote Addresses at 3D Printer …

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BURBANK, Calif.--(BUSINESS WIRE)--Already revolutionizing industry, arts, entertainment and education, 3D printing is the technology of today and the future. Keynote sessions will touch on both at 3D Printer World Burbank, which commences Jan. 29-31.

Keynote Address: Educating the Generative Generation

by Jordan Brandt, PhD and Futurist, Autodesk

Friday, Jan. 30, 10a.m. PST

New generative design and additive manufacturing technologies are accelerating the convergence of traditional disciplines. Engineers and architects of the future will speak a common computational vocabulary, with the skills to coax millions of design iterations from artificially intelligent machines on the path to optimal solutions. Likewise, the education of our next gen(erative) designers will shift away from explicitly drawing ideas to framing the problem and learning how to ask the right questions.

Keynote Address: From Screen to Print: The Physical Birth of Digital Art

by Louie Tucci, 3D Systems Gentle Giant Studios

Saturday, Jan. 31, 10a.m. PST

Take an inside look at how 3D Systems and Gentle Giant Ltd. are helping digital artists push the boundaries of art and design. The historical divide between traditional and digital art is under constant scrutiny as technology blurs the line between the virtual and real. Will the USB stick of today be the Monet of tomorrow? See how 3D Systems is working to solve real-world problems while helping to birth a new age of digital art.

Attendees at the 3D printing industrys main event also will have front-row seats for an epic battle between 16 leading entertainment and gaming industry artists as they square off head-to-head in a LIVE Sculpt-Off on Friday, January 30. Other attractions include multiple 3D scanning and printing demonstrations, SeeMeCNCs 16-foot-tall Deltabot called The PartDaddy, a 3D printed unmanned aerial vehicle (UVA), 3D printed guitars, lamps and shoes, and a 3D print art gallery featuring renowned artists Joshua Harker, Gil Bruvel and Academy Award winner Kevin Mack.

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3D Printing Visionaries from Autodesk, 3D Systems-Gentle Giant Studios to Deliver Keynote Addresses at 3D Printer ...

A Futurist's Prescription for the Vision Council Executive Summit: Foresight 20/20

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Palm Beach, FL (PRWEB) January 22, 2015

This year's theme of the Vision Councils Executive Summit is Vision 2020. They are asking, If you could see the future today, how much more effective could you be? What would you be focusing on? What would you change first? Global futurist Jack Uldrich, their keynote speaker is helping to address these questions with his presentation: Foresight 2020: How to Future-Proof Your Business.

Based on his latest book, Foresight 20/20: A Futurist Explores the Trends Transforming Tomorrow Uldrich will draw on the scenarios he and fellow futurist Simon Anderson have developed to help highlight just how fast the world is changing, and what leaders and practitioners in the vision industry need to do in order to future-proof themselves in the face of this sea of change.

In his blog post: Seeing What You Cant See, he says, As a visual learner, Ive become fond of analogies or metaphors that help keep me humbleoften, by reminding me of my ignorance. While ignorance may not be bliss, it may hold the key to wisdom. What you cant see is often times just as important, if not more important, than what you can see.

Uldrichs aim when speaking to the Vision Council Executive Summit is to help them with their Vision 2020 mission by helping them see both, what trends are coming, and helping them embrace what they cant see, and discussing in depth how to prepare for that paradox.

Jack Uldrich is a highly acclaimed global futurist, business speaker, and bestselling author. Uldrich, who has been a keynote speaker for the American Medical Association, United Healthcare, Fairview Hospitals, and St. Jude Medical is noted for his ability to deliver stimulating, new perspectives on competitive advantage, organizational change, and transformational leadership.

In addition to his expertise in the transformative trends of healthcare, Uldrich also speaks to audiences in agriculture, education, energy, finance, retail and manufacturing. His most recent presentation clients include Signal Hill, Informatica, Verizon Wireless, Emerson, and ABB/Thomas and Betts.

Parties interested in learning more about Jack Uldrich, his books, his daily blog or his speaking availability are encouraged to visit his website. Media wishing to know more about this event or interviewing Jack as a futurist or trend expert can contact Amy Tomczyk at (651) 343.0660.

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A Futurist's Prescription for the Vision Council Executive Summit: Foresight 20/20

From The 'Quantified Self' To The 'Quantified Workplace'

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Many of us have heard of this idea of the quantified self, the idea that we can measure pretty much anything we want about ourselves. Fitness trackers, wearable devices and the internet of things are allowing us to explore whatwe want to know about how we live. All of this data is then neatly viewed in dashboards that show us areas we can improve on. Fitness is the most obvious example with food and activity tracking. However, what if we took some of these concepts and applied them to the workplace to get a better understanding of how we work?

My guest this week on The Future of Work Podcast is Kris Duggan, and his company BetterWorks, does just that. As the former CEO of gamification company Badgeville, Dugganhas a unique understanding into what engages employees and what gets them to share.

Some people will say that the idea of the quantified workplace oversteps the personal boundaries that employees have, while others will argue that it poses a unique opportunity to improve organizational effectiveness and employee engagement. We are still in the early stages of both the quantified self and the quantified workplace, but Duggangives listeners plenty to think about around the future of work.

What you will learn in this episode

Jacob Morgan is afuturist, author, and speaker. You can get the first 30 pages of his book for free as well as weekly content on the future of work bysubscribing to his newsletter.

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From The 'Quantified Self' To The 'Quantified Workplace'

Freedom Planet Playthrough – Part 2: Relic Maze [No Commentary] – Video

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Freedom Planet Playthrough - Part 2: Relic Maze [No Commentary]
After the forces of Lord Brevin kill the King of Shuigang and brainwash his son, the evil conqueror sets out to capture the powerful Kingdom Stone to use as an energy source for his ship. These...

By: SuperChaosComms

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Freedom Planet Playthrough - Part 2: Relic Maze [No Commentary] - Video

Freedom Rider: Injustice can't be ignored

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Freedom rider Carol Ruth Silver looks at one of the cells where she and other Freedom Riders were jailed in at the Mississippi State Penitentiary 50 years ago in Parchman, Mississippi, Wednesday, May 25, 2011. Silver spent 30 days at the prison.

Mississippi Freedom 50th Anniversary Reunion

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SALT LAKE CITY While being jailed during her fight for human rights, one woman transcribed her experiences on any scrap of paper she could find.

Carol Ruth Silver, one of the original Freedom Riders, spent 40 days in jail in Jackson, Mississippi, in June 1961 for her involvement with the movement.

After her release, Silver transcribed her notes to family members, who typed them up. Her thoughts sat in storage in her mother's garage in Los Angeles for decades. More than 50 years after her release, "Freedom Riders Diary: Smuggled Notes from Parchman Prison" was published in early 2014.

Silver spoke to a group at the Jewish Community Center on Wednesday about what led her to join the group and her experiences as a Freedom Rider. She also appealed to those gathered to continue to fight for justice.

Silver's speech came days after Martin Luther King Jr. Day and shortly before Black History Month.

The interracial Freedom Riders rode interstate buses into the then-segregated southern states after U.S. Supreme Court rulings in 1946 (Irene Morgan v. The Commonwealth of Virginia) and 1960 (Boynton v. Virginia) came down against segregation in interstate buses, terminals and restrooms.

To test the rulings, a group of civil activists traveled on buses, first through the upper south and later through the lower south.

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Freedom Rider: Injustice can't be ignored