Phuturdex › Diseases › Genetic disease
Drugs in development · updated 30 October 2026
Genetic disease: drugs in development
As of 30 October 2026 there are 40 active clinical trials (recruiting or ongoing, phases I–III) on 33 drugs for genetic disease. 23 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 |
|---|---|---|---|
| AOC 1044 | III | 1 | NCT07587242 |
| BMN 333 | II–III | 1 | NCT07441876 |
| Deramiocel | III | 1 | NCT05126758 |
| ECUR-506 | III | 1 | NCT06255782 |
| Etavopivat | III | 1 | NCT04624659 |
| GNR-055 | II–III | 1 | NCT05208281 |
| NGN-401 | III | 1 | NCT05898620 |
| Zeleciment Rostudirsen | III | 1 | NCT07608432 |
| Zodasiran | III | 1 | NCT07473843 |
| 4D-310 | I–II | 2 | NCT05629559 |
| MRNA-3927 | I–II | 2 | NCT05130437 |
| VX-670 | I–II | 2 | NCT06185764 |
| Anumigilimab | II | 1 | NCT07224360 |
| EDIT-301 | I–II | 1 | NCT04853576 |
| FBX-101 | I–II | 1 | NCT05739643 |
| GS010 | II | 1 | NCT07303296 |
| ILT101 | I–II | 1 | NCT07697118 |
| MRNA-3705 | I–II | 1 | NCT05295433 |
| MRNA-3745 | I–II | 1 | NCT05095727 |
| Senicapoc | II | 1 | NCT07282210 |
| AIR-001 | I | 1 | NCT07431112 |
| ATI-105 | I | 1 | NCT07869654 |
| RM-004 | Early I | 1 | NCT06107400 |
Sorted by most advanced phase and number of active trials.
Approved drugs in new trials
Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| CTX001 | III | 3 | NCT04208529 |
| InfigratinibTruseltiq®, Febseltiq® | II–III | 3 | NCT06873035 |
| VosoritideBMN 111, Voxzogo® | II–III | 3 | NCT07441876 |
| DCR-PHXC | III | 1 | NCT04042402 |
| Valoctocogene roxaparvovecRoctavian® | III | 1 | NCT06224907 |
| AlpelisibBYL719, Vijoice® | II | 1 | NCT05392608 |
| FulvestrantCligavyx®, Faslodex® | II | 1 | NCT05392608 |
| N-AcetylcysteineAcetylcysteine, Acetadote | II | 1 | NCT04381897 |
| RifampicinRifampin, RIFADIN | II | 1 | NCT03301038 |
| ThalidomideThalomid®, Thalidomide Lipomed® | II | 1 | NCT06490627 |
Sorted by most advanced phase and number of active trials.
All 33 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
- Atopic dermatitis 91 drugs
- Sickle-cell disease 73 drugs
- Duchenne muscular dystrophy 35 drugs
- Hemophilia A 26 drugs
- Graves' ophthalmopathy 19 drugs
- Neurofibromatosis 1 28 drugs
- Cystic fibrosis 30 drugs
- Retinitis pigmentosa 25 drugs
- Eczema 27 drugs
- Wilms Tumor 37 drugs
- Autosomal dominant polycystic kidney 30 drugs
- Beta thalassemia 34 drugs
Broader categories
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Nutritional and metabolic diseases 5 drugs
- Metabolic disease 30 drugs
- Metabolism, Inborn Errors 6 drugs
- Congenital hemolytic anemia 4 drugs
- Nervous system heredodegenerative disease 3 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: D030342 “Genetic Diseases, Inborn”.