Drugs / 4D-110
last change Oct 2021 re-read 3 minutes ago

4D-110

Gene therapy (AAV / viral vector) targets CHM

Developed for
choroideremia
Investigated by
4D Molecular Therapeutics

Trials 1

2021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1 NCT04483440 Jun 2020 → May 2027 expected choroideremia 4D Molecular Therapeutics Active not recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 2

DateIssuerRelease
2021-10-25 4D Molecular Therapeutics Results 4D Molecular Therapeutics Reports Interim Results from the 4D-310 Phase 1/2 Clinical Trial in Patients with Fabry Disease and Provides Clinical Data Update from the 4D-110 Phase 1/2 Clinical Trial in Patients with Choroideremia 4dmoleculartherapeutics.com ↗
4D-110 was generally well-tolerated, with initial evidence of clinical activity observed
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 clinical trial indicate that 4D-110 was well-tolerated and did not result in any dose-limiting toxicity (n=6; all patients followed between one and nine months)

Evidence & citations 4 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as 4D-110 ClinicalTrials.gov intervention name — accepted as the source's own label NCT04483440 ↗
Modality Gene therapy (AAV / viral vector) “recombinant adeno-associated virus (AAV) gene therapy” NCT04483440 ↗
Route Intravitreal “single intravitreal (IVT) injection” NCT04483440 ↗
Target CHM “transgene encoding a codon-optimized human CHM gene” NCT04483440 ↗