← All organizations
Highest phase reached
Phase 3
First clinical trial
Jul 1, 2013First marketed drug
Dec 18, 2017First NDA
Dec 18, 2017Last known activity
Mar 27, 2025
Acquired by Roche on Feb 14, 2019
Timeline
2010s
- Jul 1, 2013
Spark Therapeutics— Founded
- Dec 18, 2017
voretigene neparvovec— NDA Organization
- Feb 14, 2019
Roche— Acquired By
Drugs by trial phase (12 distinct, 16 trials)
Phase 3 (2 own, 0 via acquisition)
Phase 1 (2 own, 0 via acquisition)
Other trials (2 own, 0 via acquisition)
Indications
Studied for
- Blindness
- Blood Coagulation Disorders
- Blood Coagulation Disorders, Inherited
- Choroideremia
- Coagulation Protein Disorders
- Disease
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Glycogen Storage Disease
- Glycogen Storage Disease Type II
- Hematologic Diseases
- Hemophilia A
- Hemophilia B
- Hemorrhagic Disorders
- Hemostatic Disorders
- Leber Congenital Amaurosis
- Lysosomal Storage Diseases
- Metabolic Diseases
- Retinal Dystrophies
Marketed drugs (1)
Recent trials (20)
- NCT06846684Lead sponsorPhase 1Mar 27, 2025
- NCT06826612Lead sponsorPhase 1/Phase 2Feb 21, 2025
- NCT06747923Lead sponsorPhase 2Dec 30, 2024
- NCT06297486Lead sponsorPhase 3Mar 13, 2024
- NCT05795192Lead sponsorPhase 1May 24, 2023
- NCT05522868Lead sponsorPhase 1Oct 17, 2022
- NCT05522868Study directorPhase 1Oct 17, 2022
- NCT05053503Lead sponsorN/ANov 1, 2021
- NCT04093349Lead sponsorPhase 1/Phase 2Dec 31, 2019
- NCT03734588Lead sponsorPhase 1/Phase 2Jan 30, 2019
- NCT03003533Lead sponsorPhase 1/Phase 2Dec 1, 2016
- NCT03003533Study directorPhase 1/Phase 2Dec 1, 2016
- NCT02484092Lead sponsorPhase 1/Phase 2Sep 1, 2015
- NCT02484092Study directorPhase 1/Phase 2Sep 1, 2015
- NCT02341807Lead sponsorPhase 1/Phase 2Jan 1, 2015
Details
- Founded
- Philadelphia, PA, US Jul 1, 2013
- First NDA
- Dec 18, 2017
- Last known activity
- Mar 27, 2025
Designations held
- Orphan drug
- Breakthrough therapy
Notes
Acquired Genable Technologies 7 Mar 2016