Drugs / 4D-125
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04517149 | Jun 2020 → Jun 2026 overdue | retinitis pigmentosa | 4D Molecular Therapeutics | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 3
| Date | Issuer | Release |
|---|---|---|
| 2022-01-10 | 4D Molecular Therapeutics | Regulatory 4D Molecular Therapeutics Announces FDA Fast Track Designation Granted to 4D-125 for the Treatment of X-linked Retinitis Pigmentosa 4dmoleculartherapeutics.com ↗
4D Molecular Therapeutics (NASDAQ: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for 4D-125 for treatment of patients with inherited retinal dystrophies due to defects in the RPGR gene, including X-linked Retinitis Pigmentosa (XLRP). |
| 2021-10-10 | 4D Molecular Therapeutics | Results 4D Molecular Therapeutics Presents Interim Results from the Ongoing 4D-125 Phase 1/2 Clinical Trial in Patients with Advanced X-linked Retinitis Pigmentosa at the ASRS Annual Meeting 4dmoleculartherapeutics.com ↗
4D-125 was well tolerated in all patients treated to-date (n=8), with no dose-limiting toxicities, no serious adverse events and no chronic inflammation |
| 2021-06-24 | 4D Molecular Therapeutics | Results 4D Molecular Therapeutics Announces Rare Disease Ophthalmology Product Candidate Portfolio Update, Including Initial Clinical Safety and Tolerability Data for 4D-110 for Choroideremia and 4D-125 for XLRP, and Termination of Roche Collaboration and License 4dmoleculartherapeutics.com ↗
Initial clinical safety data at both of the two dose levels in the Phase 1 portion of a Phase 1/2 clinical trial indicate that 4D-125 was well-tolerated and did not result in any dose-limiting toxicity (n=6; all patients followed between four and nine months) |
All press releases naming this drug 5 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | 4D-125 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04517149 ↗ |
| Action | Restore | “4D-125 has been developed as a gene replacement therapy for XLRP.” NCT04517149 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “comprises an AAV capsid variant (4D-R100)” NCT04517149 ↗ |
| Route | Intravitreal | “a single intravitreal (IVT) injection” NCT04517149 ↗ |