Description
Spark Therapeutics, Inc. is engaged in the field of gene therapy. The Company develops one-time, life-altering treatments for patients suffering from debilitating genetic diseases. The Company is focused on treating rare diseases where no, or only palliative therapies exist. The Company addresses a range of debilitating genetic diseases including inherited retinal dystrophies, hematologic disorders and neurodegenerative diseases. Its pipeline of product candidates is based on AAV vector technology. The Company’s product candidate, SPK-RPE65, is in a Phase III clinical trial for the treatment of inherited retinal dystrophies (IRDs) caused by mutations in the RPE65 gene. It is also conducting other preclinical programs such as SPK-CHM, SPK-FIX and GenableRhoNova. The Company has received both breakthrough therapy and orphan product designation for SPK-RPE65 and orphan product designation for SPK-CHM for the treatment of choroideremia.
Address
3737 Market St Ste 1300
PHILADELPHIA, PA 19104-5543
United States
PHILADELPHIA, PA 19104-5543
United States
Website
http://www.sparktx.comKey stats and ratios
| Q3 (Sep '14) | 2013 | |
| Net profit margin | - | - |
| Operating margin | - | - |
| EBITD margin | - | - |
| Return on average assets | -33.72% | - |
| Return on average equity | - | - |
| Employees | 47 |
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