PIPELINE

PIPELINE

hEN1 for Amyotrophic Lateral Sclerosis

The intrathecal administration of recombinant hEN1 protein bears the potential to be a cure for ALS. hEN1 is expected to halt the progression of ALS through its rejuvenating activity on alpha motor neurons, those that degenerate in the disease. Preclinical evidence has been obtained in human motor neurons – derived from ALS patients iPSCs-, and in murine pharmacological models. BREN02 is in late preclinical phase.

hEN1 for Parkinson’s disease

The continuous delivery (AAV-based gene therapy) of hEN1 bears the potential to be a cure for Parkinson’s disease. It is expected to halt Parkinson’s disease progression and improve motor functions by protecting and rejuvenating midbrain dopaminergic neurons which degenerate in PD patients. Extensive evidence has been gathered in PD preclinical models with the recombinant protein. hEN1 for PD therapy is in late discovery phase, selecting a viral delivery system for intracerebral long-term local expression and conducting both activity and safety preclinical studies.

hOTX2 for Age-related Macular Degeneration

An OTX2-based therapy has a potential to become a treatment for AMD. OTX2 plays a key role in the differentiation of photoreceptors, bipolar cells and Retinal Pigment Epithelium during development. In the adult, Otx2 isexpressed in the retinal pigmented epithelium and in cone & rod photoreceptors that degenerate in AMD.

hOTX2 for Glaucoma

An OTX2-based therapy has a potential to become a treatment for glaucoma. OTX2 has been shown to be amaintenance and survival factor for retinal ganglion cells, those that degenerate in glaucomaand are responsible for the vision loss. Preliminary proof-of-concept has been established in preclinical models (ref).