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.

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. Spark Therapeutics pipeline card: 13 registered trials from official public datasets. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.