“Wow.” That’s pretty much the word first time attendees at BIO’s annual convention use, a lot. With over 17,000 people from 49 states and 67 countries in attendance, and expo booths bigger and more plentiful than you can likely imagine, “wow” is what the convention does. 

The BIO International Convention is hosted by Biotechnology Innovation Organization (BIO).  BIO represents more than 1,100 biotechnology companies, academic institutions, biotechnology centers and related organizations across the United States and in more than thirty other nations.

This year, the massive convention was held in Philadelphia, home of CRBF President, Kristin Smedley. Kristin documented her attendance to give you an up close look at the convention and a bit of a window into how the CRBF networks to advance our mission.

First, the fun part of the convention: the Expo Center!  Kristin’s says, “It.  Was.  Massive.”  You have likely been to an expo style event before with booths big and small, right?  The BIO Expo Center was filled with massive structures representing every country!  Some were so big they had a stage in the “booth” for presentations.  Others had configurations of couches, tables, and chairs for meetings.  Many had snack bars and drink buffets.  And much to Kristin’s delight, many had coffee stations!

Since Pennsylvania was the host of the convention, Life Sciences PA, the only life sciences trade association in Pennsylvania, hosted one of the biggest “pavilions” in the Expo Center.  In the picture you can see the massive space full of functional meeting areas for people to get together.  Kristin held several meetings there and it ended up being her “base camp” to meet up with rare disease advocacy colleagues from all over the country.

One of the most informative meetings Kristin held at that pavilion was with Advanta, a life sciences branding and marketing company.  Kristin was captivated by Advanta’s knowledge of the life sciences work and passion to help companies and organizations build a brand and tell their story effectively to spread their missions more efficiently.  Kristin and Steph (Advanta’s CEO) talked at length about using things like websites, media and literature to effectively spread a mission.  Stay tuned for more on this meeting.

The biggest opportunity Kristin had while at the Life Sciences PA pavilion was only about  3 minutes long.  Kristin got to meet and chat with the Governor of Pennsylvania, Tom Wolf.  Kristin reports that that 3 minutes of incredible face time with the Governor was a testament to the power of being a part of an extraordinary organization.  Imagine the scene: hundreds of people are in and around the pavilion when the Governor walks in with his staff, security, photographers, etc.  A swarm of people gather as the CEO of Life Sciences PA welcomes him and they start chatting.  Tons of people from the industry want a few minutes with the Governor.  Many of them got that time. 

But an interesting thing happened.  You see, there is a lot of talk in this field about all of the work being “about the patient.” But so many times the patient is left out of pretty much everything.  Things are getting better, and there are organizations and companies that are models of true patient centered work.  Life Sciences PA is one of those organizations.  CEO Chris Molineux saw Kristin Smedley standing in the crowd trying to get a minute with the Governor.  He politely steered a few folks away and announced that while he was so proud of all the work being done in Pennsylvania, it is nothing without the patients we serve.  Then, he introduced the Governor to Kristin, and she had a great conversation with him, thanking him for what is going well in PA in terms of the education of blind children.  She then offered to talk with him at a later time about where she sees the need for a few improvements.  The Governor was quite interested to learn more.  Stay tuned for that follow up meeting.

There were thousands of presentations, meet ups and connections at BIO 2019 and CRBF was involved in a lot of them.  You’ll be seeing the impact of this convention on our work in the coming months.  It was an extraordinary opportunity to be there and connect our mission to those that can help us grow.

If you would like to discuss ways to help our mission grow in research, fundraising, advocacy or outreach to the CRB1 patients, please send an email to connect@crb1.org

FOR IMMEDIATE RELEASE: 

The Curing Retinal Blindness Foundation Joins the Rare Eye Disease Community in Celebrating the First Ever FDA Approved Treatment for Blindness

December 19, 2017 Bucks County, PA – The Curing Retinal Blindness Foundation is proud to be a part of this historic day today as Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced that the U.S. Food and Drug Administration (FDA) has approved LUXTURNA™ (voretigene neparvovec-rzyl), a one-time gene therapy product indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. LUXTURNA should only be administered to patients with mutations on both copies of the RPE65 gene who have sufficient viable retinal cells as determined by their treating physicians.

LUXTURNA is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the U.S.

“During the more than 12 years of innovative research with dedicated collaborators near and far, I’ve witnessed the dramatic improvement in vision in many patients who would have otherwise lost their sight,” said Jean Bennett, the F.M. Kirby Professor of Ophthalmology in the Perelman School of Medicine at the University of Pennsylvania and Penn’s Scheie Eye Institute. “I believe that the success of the LUXTURNA clinical development program will pave the way for the development of other gene therapies, that may help the millions of patients with genetic diseases who currently have limited or no treatment options.”

“The FDA approval of Luxturna just gave patients living with rare eye disease more than hope – it has given them a concrete example that treating blindness is possible and effective,” said Kristin Smedley, President and Co-Founder of the Curing Retinal Blindness Foundation. “The decades long timeline from basic research to this approval day is proof that hope can indeed carry you a long way.”

About Gene Therapy
Gene therapy is an approach to treat or prevent genetic disease by seeking to augment, replace or suppress one or more mutated genes with functional copies. It addresses the root cause of an inherited disease by enabling the body to produce a protein or proteins necessary to restore health or to stop making a harmful protein or proteins, with the potential of bringing back function in the diseased cells and/or slowing disease progression. To deliver the functional gene into the cell, a vector is used to transport the desired gene and is delivered either intravenously or injected into specific tissue. The goal is to enable, through the one-time administration of gene therapy, a lasting therapeutic effect.

About Spark Therapeutics 
At Spark Therapeutics, a fully integrated company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia and neurodegenerative diseases. We have successfully applied our technology in the first FDA-approved gene therapy in the U.S. for a genetic disease, and currently have three programs in clinical trials, including product candidates that have shown promising early results in patients with hemophilia. At Spark, we see the path to a world where no life is limited by genetic disease. For more information, visit www.sparktx.com

.About Curing Retinal Blindness Foundation (CRBF)

The CRBF is a 501(c)(3) organization dedicated to funding research to promote treatments and cures for CRB1 degenerative retinal disease and connecting patients to resources for living with blindness.  The children in this foundation are blind or visually impaired due to mutations in the CRB1 gene causing a rare genetic disease, a degenerative retinal disease (Leber’s Congenital Amaurosis, Retinitis Pigmentosa, or cone-rod dystrophy) Some of the children have already lost almost 100% of their sight. Many of the children still have some useable vision, but it will deteriorate if nothing is done to stop the degeneration.

FOR MORE INFORMATION CONTACT:

Kristin Smedley

curingretinalblindness@gmail.com

267.566.9068

www.crb1.org