First-in-human studies
HORNBILL Trial
A first-in human trial to study the safety and tolerability of single rising intravitreal dOses (open label, non-randomised, uncontrolled) and, in addition, the early biological response of multiple intravitReal dosing (single-masked, raNdomized, sham-controlled) of BI 764524 in panretinaL photocoagulation (PRP) treated proLiferative diabetic retinopathy (PDR) patients with diabetic macular ischemia (DMI) – the HORNBILL Study.
BI 1418 0001
Safety, tolerability and pharmacokinetics of single rising intravitreal doses and multiple intravitreal dosing of BI 754132 in patients with geographic atrophy secondary to age-related macular degeneration (open label, non-randomised, uncontrolled).
PARTRIDGE Study
A first-in-human trial to study safety and tolerability of single rising intravitreal doses (oPen label, non-randomized, uncontrolled) and, in addition, the early biological Response of mulTiple intravitreal doses (double-masked, RandomIzed, shamcontrolleD) of BI 765128 in panretinal photocoaGulation (PRP) treated diabetic rEtinopathy (DR) patients with diabetic macular ischemia (DMI) – the PARTRIDGE Study.
Stem cell studies
The London Project to Cure Blindness
The London Project LogoPhase 1, open-label, safety and feasibility study of implantation of TLPCB-05206388 (human embryonic stem cell derived retinal pigment epithelium (RPE) living tissue equivalent) in subjects with acute wet agerelated macular degeneration and recent rapid vision decline
Our latest gene therapy trials
Two current studies in the gene therapy portfolio are:
A study in normally sighted participants and in participants with X-linked RetinitisPigmentosa (XLRP) associated with variants in the retinitis pigmentosa guanosinetriphosphatase regulator (RPGR) gene (RPGR-XLRP) to validate the Vision-Guided Mobility Assessment for use in natural history studies and therapeuticclinical trials of adult, adolescent and child patients with Inherited Retinal Diseases
An open label, multi-centre, Phase I/II dose escalation trial of arecombinant adeno-associated virus vector (AAV2/8-hG1.7p.cohCNGA3for genetherapy of children with Achromatopsia owing to defects in CNGA3