Navigation Links
Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness
Date:1/14/2014

SAN FRANCISCO, Jan. 14, 2014 /PRNewswire/ -- Spark Therapeutics, a late-stage gene therapy company, has reached its patient recruitment goal for its most advanced clinical program, a Phase 3 study for inherited retinal dystrophies caused by mutations in the RPE65 gene. Spark's CEO, Jeffrey D. Marrazzo, is presenting this and other corporate updates today, at the 32nd Annual J.P. Morgan Healthcare Conference in San Francisco. Marrazzo said Spark was able to achieve its recruitment goal a year after the study's initiation and anticipates filing a BLA on the final data in 2015. Currently, there are no pharmacologic treatments for this or any form of inherited retinal dystrophy, which ultimately causes irreversible blindness.

"The fact that we were able to enroll patients in our lead clinical program so quickly and seamlessly reflects the critical, unmet medical need associated with inherited retinal dystrophies as well as the deep expertise of our founders and the hard work of the clinical teams at The Children's Hospital of Philadelphia and the University of Iowa," said Jeffrey D. Marrazzo, co-founder, president and chief executive officer. "In order to realize our vision of transforming the lives of patients in a single therapeutic dose, we must continue to operate at the highest standard, as we have in achieving patient recruitment for Phase 3 of our lead candidate right on the heels of our corporate launch."

Spark's patient recruitment goal for the Phase 3 study was a minimum of 24 patients. The open-label, randomized, controlled study builds on a Phase 1/2 clinical trial in which 12 patients with RPE65-related blindness demonstrated notable improvements in functional vision, moving in some cases from being profoundly blind to being able to recognize fa
'/>"/>

SOURCE Spark Therapeutics
Copyright©2012 PR Newswire.
All rights reserved

Page: 1 2 3

Related medicine technology :

1. New Snoring Therapy Awarded The Prestigious Spark Award
2. Kalyspo Awarded Best of Ann Arbor SPARK Boot Camp
3. Next Generation Sequencing Bioinformatics Heats up: Nearing $1000 Genome Sparks Soaring Data Output
4. Spark Therapeutics to Present at 32nd Annual J.P. Morgan Healthcare Conference
5. Cell Therapeutics, Inc. (CTI) to Report First Quarter Financial Results on April 20
6. Faruqi & Faruqi, LLP Encourages Investors Who Suffered Substantial Losses Investing in Chelsea Therapeutics International, Ltd. to Contact the Firm
7. Argos Therapeutics Secures $25 million Series D Financing to Commence Phase 3 ADAPT Study in Patients with Metastatic Renal Cell Carcinoma (mRCC) in Mid-2012
8. Novelos Therapeutics Announces Successful Completion of First Cohort In Lung Cancer Trial With I-124-CLR1404 (LIGHT) Cancer-Targeted PET Imaging Agent at UW Carbone Cancer Center
9. Zacks Industry Outlook Highlights: Eli Lilly, Forest Labs, ViroPharma, Novartis and United Therapeutics
10. Oxford BioTherapeutics Appoints Senior Industry Leader to Head its New Clinical Development Operations in Basel, Switzerland
11. Nektar Therapeutics Reports Financial Results for the First Quarter of 2012
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/1/2015)... , May 1, 2015 The breakthrough ... its high price but manufacturer Gilead Sciences overcame initial ... pricing, and effective public relations campaigns. Sovaldi,s ... less severe side effects propelled the drug to $2.3 ... the therapy,s first quarter on the market. But Sovaldi,s ...
(Date:5/1/2015)... 1, 2015 CANTEL MEDICAL CORP. (NYSE: CMN ... Executive Officer, will be presenting at the Bank of America ... Wynn in Las Vegas, NV. The ... 10:00 AM PT/1:00 PM ET. In addition, management will be ... An audio webcast will be available via the investor ...
(Date:5/1/2015)... 2015 Research and Markets ( http://www.researchandmarkets.com/research/pjbxjd/us_aco_it ... ACO IT Spending Outlook 2018" report to their ... lives covered by ACO would grow at a CAGR ... ACO IT spending has been studied on three main ... spending on services. Our research provides current market estimation ...
Breaking Medicine Technology:New Study Examines Success Lessons of Sovaldi's and Harvoni's Blockbuster Launches 2United States ACO IT Spending Outlook 2014-2018 2
(Date:5/2/2015)... (PRWEB) May 02, 2015 Ticket Down has ... for the long awaited event today, Saturday, May 2nd ... Diehard boxing fans will be able to secure tickets for ... The fight that boxing fans have wanted for more than ... ago. Floyd “Money” Mayweather will step inside the ring to ...
(Date:5/2/2015)... (PRWEB) May 02, 2015 “ ActOn ” ... which takes a look at the latest and coolest applications ... the host of AppWatch and technology expert, conducted the app ... to get connected with organizations to make a difference. , ... are very few things that aren’t about money, which is ...
(Date:5/2/2015)... York, NY (PRWEB) May 02, 2015 ... when a deadly natural disaster strikes anywhere on this ... to leverage human sympathy and compassion for their own ... (United States Computer Emergency Readiness Team) issued an ... users “of potential email scams citing the earthquake in ...
(Date:5/2/2015)... 2015 “ Peachtree Audio ” was ... Report, which features the latest and coolest technology products ... special reporter for NewsWatch, conducted the review and shared ... , Music is an important part of people’s ... and given the royal treatment. Peachtree Audio does just ...
(Date:5/2/2015)... 2015 Kare Visits LLC. launched in Chicago ... caregivers. Kare Visits offers relief for caregivers by visiting ... According to the Caregiver Action Network, eight in ... 20 hours a week caring for an elderly or disabled ... hire a nurse, a caregiver is now empowered to schedule ...
Breaking Medicine News(10 mins):Health News:Cheap Floyd Mayweather vs. Manny Pacquiao Tickets: Ticket Down Has Last Minute Tickets for Mayweather vs. Pacquiao at the MGM Grand Garden Arena in Las Vegas 2Health News:Cheap Floyd Mayweather vs. Manny Pacquiao Tickets: Ticket Down Has Last Minute Tickets for Mayweather vs. Pacquiao at the MGM Grand Garden Arena in Las Vegas 3Health News:An Application to Help People Make a Difference Was Featured on NewsWatch Television 2Health News:Cyber Aftershocks from Nepal’s Earthquake Can Cripple Businesses Right Here at Home 2Health News:Cyber Aftershocks from Nepal’s Earthquake Can Cripple Businesses Right Here at Home 3Health News:Cyber Aftershocks from Nepal’s Earthquake Can Cripple Businesses Right Here at Home 4Health News:Affordable High-End Audio Equipment was Featured on NewsWatch Television 2Health News:Chicagoan Seeks to Transform Industry by Launching an Innovative Elderly Care Solution 2
... come across, feel residents of the west Garfield County, near ... everyday, in the search of natural gas. Indeed this area ... the lure of big bucks blinds them to the repercussions ... skin irritation, nausea and dizziness from breathing noxious fumes that ...
... can influence a persons diet. Those who dont get enough ... instead, opt to eat fast food. It is the lack ... cause health problems for these people in the long-run. ... York, surveyed nine females and 12 males, all undergraduates who ...
... presented Monday at SLEEP 2007, a sleep-related breathing disorder ... than expected in this population, and at higher rates ... by Hawley E. Montgomery-Downs, PhD, of West Virginia University, ... attending a neonatal follow-up clinic to a research questionnaire. ...
... -of -its-kind case, a teenager died due to an overdose ... Arielle Newman, 17, from Staten Island, New York was examined ... the active ingredient found in such creams, was discovered in ... and Johnson produce such creams meant to ease muscle strain ...
... now they are out to prove to doubting scientists that the ... pain. ,A team of yoga teachers, practitioners and ... scientifically determine whether or not it can help alleviate back pain. ... assess moves from the two most popular types of yoga - ...
... Researchers have found that functional brain scans and tests of ... children will have ongoing reading problems. ,The study ... by Fumiko Hoeft at Stanford University School of Medicine. ... Pittsburgh-area children of ages 8 to 12, who were identified ...
Cached Medicine News:Health News:Residents Of Gas Patch In Colorado Suffering In Silence 2Health News:Residents Of Gas Patch In Colorado Suffering In Silence 3Health News:American Sports Star Teenager Dies Due To Overdose Of Muscle Cream 2Health News:Yoga to Be Scientifically Tested as Therapy for Chronic Low Back Pain 2Health News:Combination of Brain Scans, Reading Skill Tests can Predict Kids Reading Problems 2
... management software for eyecare professionals- ... multi-tasking, multi-office practice management system, ... technology- Winx Pro is a ... you in effectively managing the ...
... over 2,500 ophthalmologists nationwide, ... serve one of the ... in the country. With ... we are setting new ...
... EMR Ophthalmology is a comprehensive electronic ... for ophthalmology practices. Intuition EMR Ophthalmology ... provides the tools to evaluate your ... to make better decisions and to ...
... the latest in advanced cartridge mixing and ... mixes and mechanically injects all types of ... ,The Revolution&trade line-up includes a large selection ... even in difficult to access areas. A ...
Medicine Products: