Genetix Biotherapeutics

Open enrollment across a mixed-phase pipeline

According to ClinicalTrials.gov-derived indexes, Genetix Biotherapeutics sits in the top decile of US sponsors by registered trial count (#1,137 of 15,742 sponsors by registered trial count).

12 total trials 2 currently recruiting 6 completed top decile by trial count

Genetix Biotherapeutics: 12 sponsored US clinical trials, 2 recruiting.

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.

Top-decile sponsor footprint

Genetix Biotherapeutics ranks in the top decile of sponsors by registered trial count (#1,137 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.

Open and recent trials

12 total, page 1 of 1

RECRUITING

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

NCT06224413
RECRUITING

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

NCT06271512
ACTIVE NOT RECRUITING Phase 3

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

NCT04293185
ACTIVE NOT RECRUITING

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

NCT02698579
ACTIVE NOT RECRUITING

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

NCT02633943
COMPLETED Phase 3

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT03852498
COMPLETED Phase 3

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

NCT03207009
COMPLETED Phase 3

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
TERMINATED

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

NCT02204904
COMPLETED Phase 1

A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease

NCT02140554
COMPLETED Phase 2

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
COMPLETED Phase 1

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

NCT01745120

Phase Distribution

PhaseTrial count
Phase 1 2
Phase 2 1
Phase 3 4

Therapeutic Areas

Cerebral Adrenoleukodystrophy (CALD) 3
Transfusion-dependent Beta-Thalassemia 1
X-Linked Adrenoleukodystrophy (X-ALD) 1
Adrenoleukodystrophy (ALD) 1
Beta-Thalassemia 1
Sickle Cell Disease 1

What is open in the Genetix Biotherapeutics pipeline

2 recruiting of 12 indexed (17% open); 6 completed (50%).

Phase mix: 4 late (III/IV) vs 3 early (I/II) (balanced across stages).

Top condition: Cerebral Adrenoleukodystrophy (CALD) (3 trials), 5 others listed.

How open-pipeline share is computed

Recruiting share is recruiting_count divided by trial_count for this sponsor in the public registry export. Status can change between pulls; treat the figure as a research-attention snapshot, not product availability.

Genetix Biotherapeutics: open-pipeline peers nationwide

Peers matched on 12 registered trials and 17% open enrollment, not the therapeutic-area sidebar.

Genetix Biotherapeutics 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 468101214 0%5%10%15%20% Total trials Recruiting share Genetix BiotherapeuticsAlachua Government ServicesAllergy TherapeuticsAmpio Pharmaceuticals.AngiodynamicsAdageneAgendiaAkamis BioAnthos Therapeutics
Genetix Biotherapeutics 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).

Genetix Biotherapeutics 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 12 trials (± recruiting share)

Sponsors near 17% open enrollment (± trial volume)

Related

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

What to do with this Genetix Biotherapeutics page

12 registered trials is a research-attention snapshot, not an endorsement of Genetix Biotherapeutics or any single study.

  • 2 of Genetix Biotherapeutics's trials are currently recruiting, check each one's own eligibility criteria before contacting a site. Browse recruiting trials
  • Cerebral Adrenoleukodystrophy (CALD) is the condition Genetix Biotherapeutics studies most, compare against other sponsors active in the same condition before evaluating a specific study. See Cerebral Adrenoleukodystrophy (CALD) 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.

Genetix Biotherapeutics pipeline card: 12 registered trials from official public datasets.Report a data error.