Neuroscience, ophthalmology and rare diseases



neuroscience compounds in the development pipeline


million people worldwide suffering from Alzheimer’s Disease (AD)

We are pursuing several strategic focus areas for neuroscience partnering. These are: neurodegeneration, neuroimmunology, psychiatry, and neurodevelopmental disorders.

We are particularly interested in programmes, including early-stage, that focus on priority targets and disease modifying pathways, provided that preclinical proof of concept is available and the compound has suitable physico-chemical characteristics.

We are open to treatments that are either disease-modifying or have a superior impact on those symptoms that are sub-optimally controlled with existing treatments.

In neurodegenerative diseases like Alzheimers’s disease , Parkinson’s disease (PD), and Huntington’s disease (HD), we focus on the correction of those pathologies that are associated with misfolded proteins. We also look into therapies that target the associated neuroinflammation. In multiple sclerosis (MS), we are very interested in therapies offering the prospect of remyelination and ameliorating neuroinflammation in the CNS. Better modulation of fatigue and enhancement of cognition are also of interest.

Schizophrenia is a condition with established therapeutics for stage one disease but very little is available for the negative symptoms in chronic disease which include apathy, blunted emotion and depression. There have been recent advances in the understanding of disease pathology and we are interested in oral or biologic compounds that have agents which address unmet medical need, positive residual symptoms and approaches in Personalised Healthcare (PHC) enabling better patient stratification. Genetic evidence and circuit relevance of target are important information and relevance should be demonstrated by target modulation and circuit engagement in translational models.

Autism spectrum disorder (ASD) constitutes a spectrum of heterogeneous types including autism, Asperger syndrome and pervasive developmental disorder (PDD) – for which no current therapies exist. Our focus is currently on the heterogenous ASD phenotype and genetic forms of autism.

We continue to seize opportunities as they arise in areas such as schizophrenia positive symptoms, schizophrenia maintenance, bipolar disorder, and depression, and typically require evidence of a clinical signal before we further evaluate drug candidates in these indications.


Our strategy in ophthalmology focuses on serious diseases affecting the back of the eye which result in blindness. They include: age-related macular degeneration (AMD), geographic atrophy (GA), diabetic retinopathy (DR), and diabetic macular edema (DME)

Dry AMD and related GA have no currently approved therapies. The scientists at Roche are committed to the development of therapies for these retinal diseases and are seeking drug candidates with neuro-protective and retinal epithelial-preserving properties. In addition, we are also actively seeking novel therapies for the wet form AMD, diabetic macular edema, and other related diabetic retinal complications that would complement our Lucentis (ranibizumab) franchise.

Other diseases where we seize opportunities as they arise include glaucoma, severe dry eye and rare inherited retinal dystrophies (e.g. retinitis pigmentosa). In glaucoma, we are particularly interested in therapies that have demonstrated the ability to protect retinal neurons, and compounds that lower intraocular pressure to a greater extent than standard therapies.

Rare diseases

Apart from the neuro-rare and ophthalmology rare diseases mentioned above, we are keen to partner in therapeutics for monogenic diseases affecting various other organ systems such as blood, lung, heart and liver. The focus is on late pre-clinical or clinical stage assets, either large or small molecules.

If you have a promising drug candidate in these areas, why not get in touch with us?

Partner with us partnering contact

Tom Zioncheck Head of Neuroscience, Ophthalmology & Rare Diseases Partnering partnering

If you have an idea, we would like to hear from you.

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