Phuturdex › Diseases › X-linked disease
Drugs in development · updated 30 October 2026
X-linked disease: drugs in development
As of 30 October 2026 there are 9 active clinical trials (recruiting or ongoing, phases I–III) on 8 drugs for x-linked disease. 8 are experimental, that is not yet approved in the United States.
Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.
Sign in free →Experimental drugs
Not yet approved by the US FDA for any indication.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| TSHA-102 | III | 2 | NCT07480564 |
| Bionetide | III | 1 | NCT06840496 |
| Deramiocel | III | 1 | NCT05126758 |
| ECUR-506 | III | 1 | NCT06255782 |
| GNR-055 | II–III | 1 | NCT05208281 |
| NGN-401 | III | 1 | NCT05898620 |
| Zeleciment Rostudirsen | III | 1 | NCT07608432 |
| CTH120 | II | 1 | NCT07654114 |
Sorted by most advanced phase and number of active trials.
All 8 drugs, with the details
In Phuturdex every drug has all its trials, sponsors and countries, FDA and EMA approvals, orphan designations and safety alerts. Free until 31 March 2027.
Open in Phuturdex →Specific forms
- Duchenne muscular dystrophy 35 drugs
- Mucopolysaccharidosis II 15 drugs
- Hemophilia B 10 drugs
- Fabry disease 10 drugs
- Chronic granulomatous disease 21 drugs
- Fragile X syndrome 10 drugs
- Dyskeratosis congenita 15 drugs
- Rett syndrome 8 drugs
- Hemophilia A 26 drugs
- Ornithine carbamoyltransferase deficiency 6 drugs
- Bone marrow failure 26 drugs
- Bulbo-Spinal Atrophy, X-Linked 8 drugs
Broader categories
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Nervous system heredodegenerative disease 3 drugs
- Genetic disease 33 drugs
- Mental disorder 24 drugs
- Nervous System Diseases 39 drugs
- Neurobehavioral manifestation 8 drugs
Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D040181 “Genetic Diseases, X-Linked”.