Biotech · Private company
Spark Therapeutics, Inc.
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Clinical trials144 recruiting or active
Pipeline programs10From trials and the company's site
Disclosed deals0None found yet
Cash runwayNot availablePrivate: no SEC financials
Pipeline10
- AAV2-hRPE65v2,voretigene neparvovec-rzylPhase 3 · Inherited Retinal Dystrophy Due to RPE65 MutationsClinicalTrials.gov · fetched Nov 12, 2026
- SPK-8011Phase 3 · Hemophilia AClinicalTrials.gov · fetched Nov 12, 2026
- AAV2-hCHMPhase 2 · ChoroideremiaClinicalTrials.gov · fetched Nov 12, 2026
- SPK-3006Phase 2 · Pompe DiseaseClinicalTrials.gov · fetched Nov 12, 2026
- SPK-8016Phase 2 · Adeno-Associated Virus (AAV)ClinicalTrials.gov · fetched Nov 12, 2026
- voretigene neparvovec-rzylPhase 2 · Leber Congenital AmaurosisClinicalTrials.gov · fetched Nov 12, 2026
- AAV8-hFIX19Phase 1 · Hemophilia BClinicalTrials.gov · fetched Nov 12, 2026
- AAV2-hRPE65v2,voretigene neparvovec-rzylConfirmed Biallelic RPE65 Mutation-associated Retinal DystrophyClinicalTrials.gov · fetched Nov 12, 2026
- Neutralizing Antibody to SPK-3006 capsidPompe DiseaseClinicalTrials.gov · fetched Nov 12, 2026
- SPK-8016Hemophilia AClinicalTrials.gov · fetched Nov 12, 2026
Clinical trials14
- Study of a Gene Therapy Treatment for Hemophilia APHASE3 · WITHDRAWN · started Mar 13, 2024ClinicalTrials.gov NCT06297486 · fetched Nov 12, 2026
- A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)PHASE1_PHASE2 · ACTIVE_NOT_RECRUITING · started Oct 1, 2020ClinicalTrials.gov NCT04093349 · fetched Nov 12, 2026
- Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe DiseaseNA · COMPLETED · started Jun 12, 2019ClinicalTrials.gov NCT03893240 · fetched Nov 12, 2026
- Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII InhibitorsPHASE1_PHASE2 · COMPLETED · started Jan 30, 2019ClinicalTrials.gov NCT03734588 · fetched Nov 12, 2026
- Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A ParticipantsCOMPLETED · started Jan 21, 2019ClinicalTrials.gov NCT03876301 · fetched Nov 12, 2026
- A Patient Registry Study for Patients Treated With Voretigene Neparvovec in USCOMPLETED · started Jan 10, 2019ClinicalTrials.gov NCT03597399 · fetched Nov 12, 2026
- Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia AACTIVE_NOT_RECRUITING · started Aug 14, 2018ClinicalTrials.gov NCT03432520 · fetched Nov 12, 2026
- A Gene Transfer Study for Hemophilia APHASE1_PHASE2 · COMPLETED · started Jan 26, 2017ClinicalTrials.gov NCT03003533 · fetched Nov 12, 2026
- Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene MutationsPHASE1_PHASE2 · COMPLETED · started Jan 15, 2015ClinicalTrials.gov NCT02341807 · fetched Nov 12, 2026
- Hemophilia B Gene Therapy With AAV8 VectorPHASE1 · TERMINATED · started Oct 1, 2012ClinicalTrials.gov NCT01620801 · fetched Nov 12, 2026
- Safety and Efficacy Study in Subjects With Leber Congenital AmaurosisPHASE3 · ACTIVE_NOT_RECRUITING · started Oct 1, 2012ClinicalTrials.gov NCT00999609 · fetched Nov 12, 2026
- Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2PHASE1_PHASE2 · ACTIVE_NOT_RECRUITING · started Nov 1, 2010ClinicalTrials.gov NCT01208389 · fetched Nov 12, 2026
- Safety Study in Subjects With Leber Congenital AmaurosisPHASE1 · COMPLETED · started Sep 1, 2007ClinicalTrials.gov NCT00516477 · fetched Nov 12, 2026
- LTFU for Gene Transfer Subjects With Hemophilia BCOMPLETED · started Aug 1, 2007ClinicalTrials.gov NCT00515710 · fetched Nov 12, 2026