Genetix Biotherapeutics
Trial Pipeline
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 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
NCT01896102
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 2 | 1 |
| Phase 3 | 1 |
What the Pipeline for Genetix Biotherapeutics Shows
According to the ClinicalTrials.gov registry, Genetix Biotherapeutics is linked to 33 US clinical trials across every stage of research activity. Of those, 29 studies are currently recruiting — about 88% of the sponsor's indexed portfolio — and 1 are already marked complete, representing roughly 3% of the total. 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.
The phase mix for Genetix Biotherapeutics reports 1 late-stage studies (Phase 3 and Phase 4 combined) and 1 earlier-phase studies (Phase 1 and Phase 2). 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.
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. 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.