Genetix Biotherapeutics
Genetix Biotherapeutics sponsors 12 registered US clinical trials on ClinicalTrials.gov, 2 of them currently recruiting, and 6 completed. 4 of these trials are in Phase 3-4 (later-stage) and 3 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Cerebral Adrenoleukodystrophy (CALD), with 3 trials.
Trial Pipeline
12 total, page 1 of 1
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
NCT06224413
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
NCT06271512
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
NCT04293185
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
NCT02698579
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
NCT02633943
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
NCT03852498
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
NCT03207009
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
NCT02906202
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
NCT02204904
A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease
NCT02140554
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
NCT01896102
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
NCT01745120
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
| Phase 2 | 1 |
| Phase 3 | 4 |
What the Pipeline for Genetix Biotherapeutics Shows
According to the ClinicalTrials.gov registry, Genetix Biotherapeutics is linked to 12 US clinical trials across every stage of research activity. Of those, 2 studies are currently recruiting, about 17% of the sponsor's indexed portfolio, and 6 are already marked complete, representing roughly 50% of the total.
The phase mix for Genetix Biotherapeutics reports 4 late-stage studies (Phase 3 and Phase 4 combined) and 3 earlier-phase studies (Phase 1 and Phase 2).
The top therapeutic focus area indexed for Genetix Biotherapeutics is Cerebral Adrenoleukodystrophy (CALD) with 3 linked trials, and 5 other condition areas appear in the top list above.
How to read these pipeline numbers
Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.
A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work, safety, dosing, and early signal detection, and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.
That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice, verify current details directly with ClinicalTrials.gov before relying on any figure here.
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.