Spark Therapeutics

Closed portfolio with an early-phase archive tilt

According to ClinicalTrials.gov-derived indexes, Spark Therapeutics sits in the top decile of US sponsors by registered trial count (#1,049 of 15,742 sponsors by registered trial count).

13 total trials none currently recruiting 8 completed early-phase tilt top decile by trial count

Spark Therapeutics: 13 sponsored US clinical trials, none currently recruiting.

Spark Therapeutics sponsors 13 registered US clinical trials on ClinicalTrials.gov, none currently recruiting, and 8 completed. 1 of these trials are in Phase 3-4 (later-stage) and 7 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Leber Congenital Amaurosis, with 2 trials.

Top-decile sponsor footprint

Spark Therapeutics ranks in the top decile of sponsors by registered trial count (#1,049 of 15,742 sponsors by registered trial count). Volume at this scale usually reflects a long institutional pipeline, read phase tilt and therapeutic areas below before treating it as current-year activity.

Indexed trials (enrollment closed)

13 total, page 1 of 1

ACTIVE NOT RECRUITING Phase 1

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349
ACTIVE NOT RECRUITING

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

NCT03432520
ACTIVE NOT RECRUITING Phase 3

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCT00999609
ACTIVE NOT RECRUITING Phase 1

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

NCT01208389
COMPLETED NA

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

NCT03893240
COMPLETED Phase 1

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

NCT03734588
COMPLETED

Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

NCT03876301
COMPLETED

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

NCT03597399
COMPLETED Phase 1

A Gene Transfer Study for Hemophilia A

NCT03003533
COMPLETED Phase 1

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

NCT02341807
TERMINATED Phase 1

Hemophilia B Gene Therapy With AAV8 Vector

NCT01620801
COMPLETED Phase 1

Safety Study in Subjects With Leber Congenital Amaurosis

NCT00516477
COMPLETED

LTFU for Gene Transfer Subjects With Hemophilia B

NCT00515710

Phase Distribution

PhaseTrial count
Phase 1 7
Phase 3 1

Therapeutic Areas

Leber Congenital Amaurosis 2
CHM (Choroideremia) Gene Mutations 1
Factor VIII (FVIII) Protein 1
Factor VIII (FVIII) Gene 1
Factor VIII (FVIII) Deficiency 1
Coagulation Protein Disorders 1
Adeno-Associated Virus (AAV) 1
Inherited Retinal Dystrophy Due to RPE65 Mutations 1
Acid Maltase Deficiency 1
Lysosomal Storage Diseases 1

How the Spark Therapeutics early-phase archive is shaped

13 indexed studies, none listed as recruiting (0% open); 8 completed (62%).

Phase mix: 1 late (III/IV) vs 7 early (I/II) (early-phase heavy).

Top condition: Leber Congenital Amaurosis (2 trials), 9 others listed.

How to read a closed sponsor archive

When recruiting_count is zero, the indexed set is completed or otherwise not open. Phase tilt still describes where archived studies sat on the investigational pathway. Re-check ClinicalTrials.gov before acting.

Spark Therapeutics: archived-pipeline peers nationwide

Peers matched on 13 indexed studies with no current recruiting: volume and archive shape, not condition overlap.

Spark Therapeutics sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together. Source: ClinicalTrials.gov (2026-08-08 snapshot). 2×2 strategic matrix plotting 9 entities by Total trials (X) and Recruiting share (Y), with a crosshair dividing the plot into four quadrants. Higher volume & more openFocused & more openHigher volume, mostly closedSmaller & mostly closed 051015 0%2%4%6%8%10% Total trials Recruiting share Spark TherapeuticsAdaptimmuneAgennixAkili Interactive LabsAlumis10xBio22nd Century Group23andMe3-C Institute for Social Development
Spark Therapeutics sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together. Source: ClinicalTrials.gov (2026-08-08 snapshot).

Spark Therapeutics sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together.

Sponsors near 13 archived trials

Sponsors with similar archive density (± trial count)

Related

Read our methodology - how this data is sourced, computed, and verified.

What to do with this Spark Therapeutics page

13 registered trials is a research-attention snapshot, not an endorsement of Spark Therapeutics or any single study.

  • None of Spark Therapeutics's trials are currently recruiting in this registry; that can change as new trials post, or check the broader research index. Browse research indexes
  • Leber Congenital Amaurosis is the condition Spark Therapeutics studies most, compare against other sponsors active in the same condition before evaluating a specific study. See Leber Congenital Amaurosis trials
  • Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

Spark Therapeutics pipeline card: 13 registered trials from official public datasets.Report a data error.