Cystic fibrosis and sickle-cell anemia could be corrected in embryos

https://www.geneticliteracyproject.org/2017/03/08/cystic-fibrosis-sickle-cell-anemia-could-be-corrected-in-embryos-with-new-crispr-variant/

Cystic fibrosis, sickle-cell anemia could be corrected in embryos with new CRISPR variant

Since the discovery of the genome-editing tool CRISPR/Cas9, scientists have been looking to utilize the technology to make a significant impact on correcting genetic diseases. Technical challenges have made it Continue reading Cystic fibrosis and sickle-cell anemia could be corrected in embryos

Nanoparticle-delivered CRISPR tools could treat cystic fibrosis

Nanoparticle-delivered CRISPR tools could treat hemophilia, cystic fibrosis and muscular dystrophy

More and more scientists are using the powerful new gene-editing tool known as CRISPR/Cas9, a technology isolated from bacteria, that holds promise for new treatment of such genetic diseases as cystic Continue reading Nanoparticle-delivered CRISPR tools could treat cystic fibrosis

Gene Therapy for Treating CF

http://www.news-medical.net/news/20160920/Gene-therapy-may-be-viable-approach-for-treating-CF-lung-problems.aspx

Gene therapy may be viable approach for treating CF lung problems

Two new studies from the University of Iowa suggest that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis Continue reading Gene Therapy for Treating CF

Development of New Gene Therapy Treatments

Exemplar Genetics ExeGen® Cystic Fibrosis Research Model Utilized in Development of New Gene Therapy Treatments

http://www.prnewswire.com/news-releases/exemplar-genetics-exegen-cystic-fibrosis-research-model-utilized-in-development-of-new-gene-therapy-treatments-300326909.html

Exemplar Genetics, a wholly owned subsidiary of Intrexon Corporation (NYSE:  XON) committed to enabling the study of life-threatening human diseases, today announced two publications in the current issue of the Journal of Clinical Continue reading Development of New Gene Therapy Treatments

Top 10 Lessons On Running A Disruptive Foundation — Guest Blog By Emily Kramer-Golinkoff

Originally posted on Emily’s Entourage, on July 21, 2016
http://www.emilysentourage.org/top-10-lessons-on-running-a-disruptive-foundation/

Today’s guest blog post was written by Emily and adapted from a speech delivered at the Global Genes and Penn Orphan Disease Center’s Rare Patient Advocacy Symposium.

Like so many of you, I’ve been thrust into this role of disruptor out of desperation.

I’m learning on the job every moment of everyday. It’s a 24/7 job. It is painstaking, relentless work; there are no breaks; and then there’s this fatal disease to manage on top of it.

It’s a crazy, dizzying reality.

And so while I’m certainly no expert, I’d like to share with you a few things I’ve picked up along the way.

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#1: Stories are powerful.

You don’t realize the power of your story until you start telling it. It might be uncomfortable, but it’s a means to an end, and nothing touches people’s hearts like stories. It’s that thread of humanity that binds us together, and it’s pretty spectacular to see how much people connect and care.

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#2: It’s all about relationships.

Everything that has happened for EE, every major development, every pivotal connection, every advance in research, it’s all been because of relationships. When the test tube says Emily and not some random digital code, it matters.

So go to the labs, talk to the scientists, SHOW UP. Make it painfully personal. There’s nothing in the world more motivating.

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#3: Nobody can be a better or more tireless advocate than you.

I’m participating in a study where I’m be the first and only person in the world to try the therapy. The idea? It came from me and my mom. We have no scientific background, just a huge vested interest in the outcome, some creativity, and logic.

Of course we vetted it with tons of scientists and did all of our homework, but the original idea was all ours — ordinary, everyday Emily and Liza’s. Don’t underestimate your voice, your brain, your creativity, and your ideas.

#4: Be relentless.

When there’s a roadblock, find a creative way around and keep at it. Push and push and push and don’t accept no for an answer if you believe in what you’re fighting for.

#5: People really care, but you have to ask for them to step up and help.

If you don’t ask, the answer is always no. I’m naturally a very private person, a total introvert who hates asking for things. The only thing that has kept me sharing such personal details of my life and medical journey is the outpouring of love and support that results–and seeing how people rise.

After our inaugural event in NYC, I can’t tell you how many people came up to me and THANKED ME for the opportunity to get involved.

People want to be part of something bigger. They want to do good things. They want to make impact. It’s up to us to give them the opportunities. And it takes guts.

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#6: You need a scientist champion and a respected Scientific Advisory Board.

When we first started making phone calls to labs and biotechs, people wouldn’t even answer the phone (or they promptly hung up!).

A scientist champion is your key into the scientific world and a respected Scientific Advisory Board is essential for vetting research projects and securing major gifts and grants. People need to know their money is going to reputable work.

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#7: Build a nest of trusted advisors.

It truly takes a village.

We were thrust into this role of running a foundation. We still have no idea what we’re doing, we just know what we want and that we are willing to do whatever it takes to get there.

By building a nest of advisors, we draw on the expertise of so many different, brilliant people and pull them onboard.

We’ve had the privilege of meeting some of the most remarkable individuals. We call them our guardian angels and our progress is a testament to — and totally dependent on — them

#8: Don’t underestimate the value of YOUR creativity and ideas.

Sometimes the greatest innovation comes from outside of the biomedical institution.

Nobody has a bigger vested interest in your future. Desperation spurs creativity and innovation. You don’t need a PhD to have great ideas. Believe in them and tenaciously pursue them.

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#9: Digital technology is your best friend.

Technology is democratizing; content is king. Use technology to spread your content far and wide. The world is at your fingertips and they’re eager to hear your story. Make it concise. Make it compelling. Make it easily sharable.

#10: Know what you want.

You need a clearly defined research goal to raise money and effectively mobilize a community. Do your homework, identify the leading researchers, labs, biotechs, pharma and all the key stakeholders, and bring them together. It’s essential to draw on their expertise to clearly articulate your research strategy.

People want to fund good work, but they need to know the targets. And so do you! Otherwise, your wheels will be spinning, and let’s be honest, we don’t have time or energy to waste.

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Emily’s Entourage is a work in progress. We are learning as we go. We constantly have to realign, refocus on our end goal, and troubleshoot — and it is HARD.

I think about EE every second of the day, with every breath I take. Literally.

What’s next? How do we grow? How do we make the research go faster? How do we add more revenue streams? How do we make the organization sustainable? How do I balance running EE and managing my health?

So many questions. So much to do. So little time — with a disease that rages on and robs me of my life, my energy, my breath.

I feel the crunch of time, the terror of disease progression, ALL the time. And that is the propeller.

At Emily’s Entourage, we pledge to keep at this, with this same vigor until there is a cure for EVERYONE with CF. And then, we pledge to take these lessons learned and apply them to another disease, to help another disease community get to the finish line. That is our greatest wish. That would be our dream come true.

Continue reading Top 10 Lessons On Running A Disruptive Foundation — Guest Blog By Emily Kramer-Golinkoff

Moderna, Vertex to collaborate on cystic fibrosis

https://www.bostonglobe.com/business/2016/07/06/moderna-vertex-collaborate-cystic-fibrosis/rEmPx5Ll746RmQL0oUNIiO/story.html

Moderna Therapeutics Inc. has emerged as a partner of choice for drug makers eager to supplement their own research.

Vertex Pharmaceuticals Inc. became the latest established biopharma company Continue reading Moderna, Vertex to collaborate on cystic fibrosis

UI strikes agreement with Pfizer Inc. to develop potential cystic fibrosis gene therapy

The University of Iowa Research Foundation recently finalized a license and sponsored research agreement with Pfizer Inc. to support the development of potential gene therapies for cystic fibrosis (CF) by the laboratories of the Continue reading UI strikes agreement with Pfizer Inc. to develop potential cystic fibrosis gene therapy

Cystic Fibrosis Phase 2 Trial of Drug Aiming to Stabilize Key CF Gene Underway

http://cysticfibrosisnewstoday.com/2015/12/02/cystic-fibrosis-phase-2-trial-drug-aiming-stabilize-key-cf-gene-underway/

Published originally in Cystic Fibrosis News Today
By Charles Moore

Nivalis Therapeutics, Inc., a Boulder, Colorado-based clinical stage pharmaceutical company, announced that the first patient has been dosed in the Phase 2 clinical trial of its lead investigational drug, N91115, a stabilizer Continue reading Cystic Fibrosis Phase 2 Trial of Drug Aiming to Stabilize Key CF Gene Underway

Spraying new genes into your lungs could ease misery of cystic fibrosis

For Oli Dillon, an effective therapy for cystic fibrosis can’t come soon enough. Thanks to a groundbreaking development, he’s already had a tantalising ‘taster’ of a treatment that could transform lives such as his. Continue reading Spraying new genes into your lungs could ease misery of cystic fibrosis

Galapagos advances triple combination therapy in cystic fibrosis

GLPG2665, a next-generation (C2) corrector, together with the other two components of the triple combination, show up to six-fold greater chloride transport than Orkambi1) in vitro

– Triple combination therapy of C2 with GLPG2222 and GLPG1837 expected to Continue reading Galapagos advances triple combination therapy in cystic fibrosis