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Genetix Biotherapeutics clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine — current trials in our USA and UK registers with Genetix Biotherapeutics as lead sponsor. Synced 26 Sep 2026
6Current trials
2Recruiting now
0Drugs in these trials
2Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

Genetix Biotherapeutics is the lead sponsor of 6 current clinical trials in our USA and UK registers. 2 are recruiting patients now. The most common phase is Observational. The most-studied condition is Cerebral Adrenoleukodystrophy (CALD).

ClinicalTrials.gov trials by status

  • Recruiting233%
  • Active, closed350%
  • By invitation117%

Trials by phase

  • Observational583%
  • Phase 3117%
01

Clinical trials sponsored by Genetix Biotherapeutics

6 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
NCT06271512 · ClinicalTrials.gov ↗ · Beta-Thalassemia
Observational Recruiting 2024-01 USA
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
NCT06224413 · ClinicalTrials.gov ↗ · Cerebral Adrenoleukodystrophy (CALD)
Observational Recruiting 2024-03 USA
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
NCT04628585 · ClinicalTrials.gov ↗ · Sickle Cell Disease
Observational By invitation 2020-10 USA
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
NCT02698579 · ClinicalTrials.gov ↗ · Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
Observational Active, closed 2016-01 UK, USA
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
NCT02633943 · ClinicalTrials.gov ↗ · Transfusion-dependent Beta-Thalassemia
Observational Active, closed 2014-01 UK, USA
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
NCT04293185 · ClinicalTrials.gov ↗ · Sickle Cell Disease
Phase 3 Active, closed 2020-02 USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Conditions in Genetix Biotherapeutics trials

6 conditions
Cerebral Adrenoleukodystrophy (CALD)2Sickle Cell Disease2Adrenoleukodystrophy (ALD)1X-Linked Adrenoleukodystrophy (X-ALD)1Transfusion-dependent Beta-Thalassemia1Beta-Thalassemia1
03

By trial register

RegisterTrialsRecruiting
Clinical trials in USA62
Clinical trials in UK20

A trial with sites in more than one country appears in each of those registers.

04

Questions about Genetix Biotherapeutics trials

How many clinical trials does Genetix Biotherapeutics sponsor?

Genetix Biotherapeutics is the lead sponsor of 6 current trials on ClinicalTrials.gov in our USA and UK registers, as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are Genetix Biotherapeutics trials recruiting?

Yes. 2 of its 6 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.