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

ces and ambulate independently.  All school-aged patients enrolled in the trial were able to transfer from Braille classrooms to sighted classrooms.

In addition to Spark's Phase 3 recruitment achievement, Marrazzo shared several corporate updates at the J.P. Morgan conference, including: 

  • The company is well-positioned to be the first to obtain U.S. FDA approval for a gene therapy product and is preparing to launch the first in a series of therapies to address the significant unmet need of patients with inherited retinal dystrophies.
  • Spark expects to file an investigational new drug (IND) application for a second inherited retinal dystrophy this year.
  • The company presented encouraging results from the first subject in its Phase 1/2 hemophilia B program, in which a study participant has been able to go without factor for six months since vector infusion.
  • Two new team members have joined Spark: Carol Greve-Philips, senior vice president, business development and Joseph La Barge, senior vice president of business administration and general counsel. Greve-Philips previously served as vice president of corporate development at Genzyme; La Barge played a critical role as general counsel at Tengion, Inc., developing intellectual property strategies for first-in-class, cell-based therapies. 

The company continues to enroll its Phase 1/2 trial for hemophilia B and is advancing toward the clinic with gene therapy programs to address multiple, fatal neurodegenerative diseases.

For more information on Spark Therapeutics please visit www.sparktx.com or follow on Twitter @spark_tx.

About Spark Therapeutics
Spark Therapeutics is developing potentially curative, one-time gene therapy products to transfo
'/>"/>

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:1/15/2014)... YORK , January 15, 2014 ... Ltd., the exclusive worldwide distributor of highly sophisticated agro-breeding ... on January 7, 2014, the Company,s board of directors ... Weiner as its new member of the board. ...
(Date:1/15/2014)... 15, 2014  In an unprecedented effort to curb the spread of ... other transport vehicles, an advanced and portable UV germicidal lamp manufactured ... first time. In order to prevent EMS paramedics ... West Palm Beach Fire Rescue is the ...
(Date:1/15/2014)... 15, 2014 MultiCell Technologies, Inc. (OTC Bulletin Board: ... patent application concerning composition of matter, biological targets, mechanism ... cell death.  This patent application describes ... RNA molecules (VSRNAs) which interfere with the basic homeostatic ...
Breaking Medicine Technology:BreedIT (OTC: BRDT) Appoints Chemistry and Pharmaceutical Industry Expert Dr. Ben-Zion Weiner to Its Board of Directors 2BreedIT (OTC: BRDT) Appoints Chemistry and Pharmaceutical Industry Expert Dr. Ben-Zion Weiner to Its Board of Directors 3Superbugs No Longer Ride In Rescue Vehicles 2MultiCell Technologies Files Patent Application Covering New Class of RNA Therapeutics 2MultiCell Technologies Files Patent Application Covering New Class of RNA Therapeutics 3
... 14 from 1 to 5 p.m., Children,s Hospital Boston, will ... 17 short sessions , cutting-edge approaches to improving care delivery ... innovative leaders at Children,s Hospital Boston. The event ... an 18-month-old program at Children,s Hospital Boston charged with building ...
... Va., Feb. 2, 2012 The head of ... over the proposed mega-merger of prescription drug middlemen ... Community Pharmacists Association (NCPA) saluted the lawmaker,s ... Senator Herb Kohl, (D-Wis.), chairman of ...
Cached Medicine Technology:Innovation Day 2012: a Time To Inform and Inspire 2Innovation Day 2012: a Time To Inform and Inspire 3Innovation Day 2012: a Time To Inform and Inspire 4Innovation Day 2012: a Time To Inform and Inspire 5Leading Antitrust Senator Raises Serious Doubts About Proposed Express Scripts-Medco PBM Merger 2Leading Antitrust Senator Raises Serious Doubts About Proposed Express Scripts-Medco PBM Merger 3Leading Antitrust Senator Raises Serious Doubts About Proposed Express Scripts-Medco PBM Merger 4
(Date:7/9/2014)... common type of lung cancer, researchers have uncovered mutations ... forming tumors. The new knowledge may expand treatments for ... already are available or are in clinical trials. , ... The Cancer Genome Atlas (TCGA), including researchers at Washington ... School and other institutions, studied tumors from 230 patients ...
(Date:7/9/2014)... The Miriam Hospital have found that people with ... depressed have difficulty sticking to a pulmonary rehabilitation ... women, and screening and brief treatment of depression ... study and its findings are published in print ... obstructive pulmonary disease is a common and often ...
(Date:7/9/2014)... have been linked to an emerging fungal disease ... Institute for Mathematical and Biological Synthesis (NIMBioS) shows ... , In a series of mathematical models, researchers ... internal organs in frogs, could cause extinction of ... exposed to the virus every few years, a ...
(Date:7/9/2014)... 9, 2014) A retrospective study conducted by researchers ... reports that among adults with intellectual and developmental disabilities, ... of years receiving dental care increased. The findings, published ... Dentistry , may help improve interventions designed to address ... , The researchers reviewed the dental records of 107 ...
(Date:7/9/2014)... allergic diseases has risen dramatically in Western societies. One ... to microorganisms and have fewer infections than previous generations, ... study by researchers at Sahlgrenska Academy, University of Gothenburg, ... maturation of the immune system in relation to allergic ... of the Vstra Gtaland Region, half of them on ...
Breaking Medicine News(10 mins):Health News:Lung cancer study hints at new treatments 2Health News:Miriam Hospital study examines effect of depressed mood on pulmonary rehab completion 2Health News:Study predicts ranavirus as potential new culprit in amphibian extinctions 2Health News:Adults with special needs see gains, challenges with long term oral care 2Health News:Adults with special needs see gains, challenges with long term oral care 3
... Male high school athletes more likely to drink and fight ... News) -- When it comes to teaching healthy behaviors, boys, ... thought. , New research suggests that for teenage boys, participation ... and binge drinking. , Girls, on the other hand, ...
... Study finds medication works as well in certain people, ... -- People suffering from blocked renal arteries fare just ... by having the narrowed artery opened and a stent ... chronic kidney disease -- people with severe hypertension -- ...
... ... employment and networking portal for people with disabilities and renews its commitment to leading the ... ... recently passed a milestone, the one year anniversary of the launch on November 7, 2008 ...
... Announces VSO Support For Lung Cancer Legislation , ... all veterans today on Veterans Day and announces the ... veterans on lung cancer -- the leading cause of ... ) , LCA also announces that major ...
... Near 9% Unemployment Leaves More Idaho Families Struggling with ... , BOISE, Idaho, Nov. 11 As Idaho,s jobless ... the over 67,300 unemployed Idahoans finding themselves without employer benefits. ... is calling on Idaho,s Congressional delegation to set politics aside ...
... Nov. 11 Accelify is proud to announce the addition of ... of Development in the State of Florida and will also join ... Accelify, Sandra served for eight years as a member of the ... During her time in the Florida legislature, she personally sponsored ...
Cached Medicine News:Health News:Boys' Team Sports May Encourage Bad Behavior 2Health News:Boys' Team Sports May Encourage Bad Behavior 3Health News:Surgery Not Always Best for Narrowed Kidney Arteries 2Health News:Surgery Not Always Best for Narrowed Kidney Arteries 3Health News:GettingHired.com Recognizes First Year Anniversary, Renews Commitment to Helping Lead the Effort to Reduce Employment Participation Gap for People with Disabilities 2Health News:GettingHired.com Recognizes First Year Anniversary, Renews Commitment to Helping Lead the Effort to Reduce Employment Participation Gap for People with Disabilities 3Health News:Lung Cancer Alliance Salutes Veterans 2Health News:Idaho's Rising Jobless Rate Fuels State's Growing Health Care Woes 2Health News:Accelify Announces the Addition of Sandra L. Murman to its National Board of Advisors 2Health News:Accelify Announces the Addition of Sandra L. Murman to its National Board of Advisors 3
... is a national reference laboratory and a ... development. ARUP offers an extensive test menu ... in clinical and anatomic pathology. Owned by ... include more than half of the nation's ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... reference laboratory and a worldwide leader in ... an extensive test menu of highly complex ... anatomic pathology. Owned by the University of ... half of the nation's university teaching hospitals ...
Medicine Products: