Cure in Sight tops $29k!

Attendees sampling a vast wine selection
Attendees sampling a vast wine selection
On Saturday, October 10, over 100 people gathered at the Delaware Center for Horticulture in Wilmington, DE to participate in the Moen Family’s 5th Annual “Cure in Sight” Wine Tasting & Silent Auction. Attendees enjoyed live music, samples of several fine wines, snacked on gourmet foods, bid on auction items and were treated to a viewing of the CRF’s “Saving Sight” documentary.

Chris & Alis Moen
Chris & Alis Moen
Several CHMers attended to support the event. Three of them, who all spoke at the event, are participants in the ongoing gene therapy trials at UPenn. CRF President Chris Moen & his wife Alis also addressed the packed house. It was an enjoyable and inspirational evening for the attendees, which resulted in the Moen’s raising over $29,000!

Our thanks to the Moen family, the volunteers and all those who sponsored and attended “Cure in Sight”! We’ll see you next year!

CRF2016 Tips On Travel

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Booking Your Flight

Use the following link to go to GoodShop.com and book your flight. When the page loads, scroll down and click on Choroideremia Research Foundation. It will then ask you to sign in. A percentage of your purchase will go directly to the CRF!

Click here to visit the Goodshop website and support the CRF!

Car Rental

If you wish to rent a car, there are several car rental agencies at the airport. Again, use the GoodShops link above to book your car rental and help support the CRF. Hotel parking is complimentary!

Hotel Shuttle Service

The hotel has free shuttle service to and from the airport. After you arrive and collect your baggage, call the hotel at 215-365-4500, advise them of the airline and terminal you are in and they will come pick you up. For service to the airport, the shuttle leaves the hotel every half hour. Please be in the lobby at least 5 minutes before you leave.

CRF2016 Things To Do

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Below you will find a listing of restaurants, historic sites and interesting places you may want to visit during your stay in Philadelphia. Our thanks to the Philadelphia locals who helped to compile this listing!

Restaurants

Dinner Buffet
Ralph’s Italian Restaurant– 760 S 9th St, Philadelphia, PA 19147
• Oldest family owned restaurant in the country, located in the Italian market
Victor’s Café– 1303 Dickinson St, Philadelphia, PA 19147
• Filmed Rocky 6 here/Italian opera singers play while eating fun atmosphere
El Vez– 121 S 13th St, Philadelphia, PA 19107
• Best Mexican food in the city
Ting Wong– 138 N 10th St, Philadelphia, PA 19107
• Best Chinese Food in the city
Vic Sushi Bar– 2035 Sansom St, Philadelphia, PA 19103
• Highly rated sushi bar
Reading Terminal Market– 12th and Arch St
• Great Pub Food
Independence Beer Garden– 100 S Independence Mall W, Philadelphia, PA 19106
• Near Liberty Bell, American food & 40+ regional craft beers on tap.
Geno’s Steaks & Pat’s King of Steaks-1219 S 9th St, Philadelphia, PA
• Who was first? Who is better? Try them both & decide for yourself!
Prohibition Taproom– 501 N 13th St, Philadelphia, PA
Sampan– 124 S 13th St, Philadelphia, PA 19107
• Center City neighborhood, fine Asian cuisine
Frankford Hall– 1210 Frankford Ave, Philadelphia, PA 19125
• Beer garden specializing in German food & craft beer

Historical Sites

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Independence Visitor Center– 6th Ave & Market St, Philadelphia, PA
• Information on everything in the area
Independence National Historical Park– 1 N Independence Mall W, Philadelphia, PA 19106
• National Park, near Liberty Bell, Independence Hall, etc.
Independence Hall– 520 Chestnut St, Philadelphia, PA 19106
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Liberty Bell– 6th St & Market St, Philadelphia, PA 19106
Betsy Ross House– 239 Arch St, Philadelphia, PA 19106
• American Flag house & Betsy Ross Memorial
Philadelphia Mint– 151 N Independence Mall E, Philadelphia, PA 19106
• Daily tours
Eastern State Penitentiary– 2027 Fairmount Ave, Philadelphia, PA 19130
• Daily tours

Attractions

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Philadelphia Zoo– 3400 W Girard Ave, Philadelphia, PA 19104
• One of the top in the country, great to take the kids
Citizens Bank Park– 6th St & Market St, Philadelphia, PA 19106
• Phillies Games
Lincoln Financial Field– 1 Lincoln Financial Field Way, Philadelphia, PA
• Eagles Games
Xfinity Live– 1100 Pattison Ave, Philadelphia, PA 19148
• Fun night life
• Daily tours
Love Park– 1599 John F Kennedy Blvd, Philadelphia, PA 1910
Boat House Row– 1 Boathouse Row, Philadelphia, PA 19130
Rocky Balboa Statue– 2600 Benjamin Franklin Pkwy
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The Bourse– 111 S Independence Mall E, Philadelphia, PA
• Food Court & Specialty Shops
Franklin Square– 200 N 6th St, Philadelphia, PA
• Carousel, Mini-Golf, SquareBurger
Fright Factory– 38 Jackson St, Philadelphia, PA
• Haunted House Attraction
Philadelphia’s Magic Gardens– 1020 South St, Philadelphia, PA
• Mosaic Gallery & Community Arts Center
Spirits of ’76 Ghost Tours of Philadelphia– 325 Chestnut St, Philadelphia, PA 19106 (Outside of Mrs. K’s Koffee Shop)
• Haunted History & Folklore. Audio Tours available.
Yards Brewing Company– 901 N Delaware Ave, Philadelphia, PA
• Sat-Sun Tours, 12-4, Tasting Room open 7 days
Lucky Strike Philadelphia– 1336 Chestnut St, Philadelphia, PA
• Bowling, bar & food
Museums

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Please Touch Museum– 4231 Avenue of the Republic, Philadelphia, PA
• Great for kids
The Barnes Foundation– 2025 Benjamin Franklin Pkwy, Philadelphia,PA
• Post-Impressionist Art
The Franklin Institute– 222 N 20th St, Philadelphia, PA
• Science Museum
The Mutter Museum– 19 S 22nd St, Philadelphia, PA
• Medical History, anatomical samples, instruments, etc.
National Constitution Center– 525 Arch St, Philadelphia, PA
• U.S. Constitution, rare artifacts, theater
Philadelphia Museum of Art– 2600 Benjamin Franklin Pkwy, Philadelphia, PA
• One of the largest art museums in the U.S., over 227,000 ojects
Rodin Museum– Franklin Pkwy @ 22nd St, Philadelphia, PA
• Beaux-Arts architecture museum & formal French Garden featuring Auguste Rodin Sculptures

CRF2016 Book Your Room

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The Embassy Suites Philadelphia/Airport hotel is excited and proud to host the 2016 CRF Conference! Located at 9000 Bartram Avenue, Philadelphia, PA 19153, the hotel is located close to the airport with easy shuttle access to public transportation and nearby restaurants. For a listing of local activities, attractions and recreation in the Philadelphia area, please visit our Things To Do page.


Room rates for the conference are only $179 per night (plus applicable taxes)! This rate includes complimentary Wi-Fi in all rooms/public areas, free buffet breakfast and access to all the great amenities the hotel offers. Important: The Embassy Suites is a Smoke-Free Hotel.


The code to get this discounted room rate is CRF. Please refer to this code when booking your room. There are three ways to book your hotel room:

1. Call the hotel directly, 215-365-4500,
2. Click here to reserve your room online, or
3. Call toll free, 1-800-EMBASSY (800-362-2779)

The deadline for booking your room is May 24th, 2016, so book today!

Information on the free hotel shuttle service is on the Travel Tips page.

Spark Announces Phase 3 Data for LCA Trial

20150101_inq_spark01-cAn announcement today by Spark Therapeutics brings the world one step closer toward a commercially available gene therapy treatment.  This morning Spark released positive results from the Phase 3 pivotal trial of its lead gene therapy product candidate, SPK-RPE65, for the treatment of RPE65-mediated inherited retinal dystrophies, more commonly known as LCA.

“We saw substantial restoration of vision in patients who were progressing toward complete blindness,” said Albert M. Maguire, MD, principal investigator in the trial and professor of ophthalmology at the Perelman School of Medicine of the University of Pennsylvania. “The majority of the subjects given SPKRPE65 derived the maximum possible benefit that we could measure on the primary visual function test, and this impressive effect was confirmed by a parallel improvement in retinal sensitivity. If approved, SPK-RPE65 should have a positive, meaningful impact on the lives of patients with this debilitating condition.”

The phase 3 study evaluated 31 patients, 21 of whom were treated and 10 who represented controls.  Patients treated with SPK-RPE65 were able to navigate through a mobility course more effectively at varying levels of light after treatment was provided.  They also were found to have improved full-field light sensitivity threshold testing, which is a measure of physiologic function of the retina.  In addition, there were no serious adverse events or concerning immune responses observed in the clinical trial.  These results show evidence of success of the gene therapy treatment and

“These results are the culmination of more than a decade of work of many dedicated individuals to correct the underlying cause of RPE65-mediated blindness through the one-time administration of a gene therapy,” said Jean Bennett, MD, PhD, professor of ophthalmology and director of the Center for Advanced Retinal and Ocular Therapeutics at the Perelman School of Medicine of the University of Pennsylvania. “We are excited about the potential impact that the results will have on the treatment of this and other blinding conditions.”

Currently, there are no commercially available gene therapy treatments in the United States and only one in the Western world.  These positive results bring Spark one step closer to approval for SPK-RPE65, which would also be the first gene therapy treatment for a retinal degenerative disease.  Successful progress and approval of SPK-RPE65 bodes well for Choroideremia, currently in Phase 1/2 clinical trials by Spark.  With the success and experience from the RPE65 program now established, the Choroideremia community hopes that their disease will follow a similar, if not simpler, path through the clinical trial and regulatory process.

For more information on Spark Therapeutics, you can navigate to their website at www.sparktx.com.  In addition, patients can contact Spark directly at 800-SPARKTX or patients@sparktx.com for more information.