NEUROLOGY AND OPHTHALMOLOGY

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1 NEUROLOGY AND OPHTHALMOLOGY OPPORTUNITIES AT INSERM BD & L: Anne Cochi September

2 WE PROPOSE A SELECTION OF PROJECTS AT DIFFERENT STAGES 4 Neurodegeneration patents 4 Alzheimer Disease patents 2 Multiple Sclerosis patents 2 Demyelinating Diseases patents 2 Neurovascular patents 1 Neurodevelopment patent 1 Pain patent 1 CNS - Rare diseases patent 1 Aging patent 3 Psychiatry patents 2 CNS - Other patents 7 Ophthalmology patents 2

3 4 NEURODEGENERATION OPPORTUNITIES Parkinson Diseases / ALS (BIO11382) Treatment of Andermann Syndrome / Rare Diseases (BIO15327) Bornavirus-derived peptide to prevent Neurodegenerescence Use of carbamazepine for the treatment of Andermann syndrome Peptide validated in Parkinson mouse model POC in vivo in mouse model (Method patent) Amyotrophic Lateral Sclerosis (ALS) (BIO15549) A new mechanism to reduce symptoms in Amyotrophic Lateral Sclerosis (ALS) POC in vivo (Target patent) Drug repositionning / Rare Diseases (BIO16340) Methods and pharmaceutical compositions for the treatment of neurodegeneration with brain iron accumulation Drug validated in vitro (Second Use patent) 3

4 4 ALZHEIMER DISEASE OPPORTUNITIES Gene Therapy (BIO15161) Gene Therapy (BIO15162) Progression model (MECA16023) Antibodies to detect a new Tau isoform / Diagnostic (BIO17097) Soluble APPsα-based Gene Therapy in Alzheimer's Disease Interleukin-2-based Gene Therapy in Alzheimer's Disease A progression model that estimates a normative scenario of the progressive impairments of neurodegenerative diseases New Tau isoform in AD patients POC vivo in AAP/PSIΔE9 mice POC vivo in AAP/PSIΔE9 mice Correlation with genetic, clinical and cognitive variables (Method patent) POC in vivo 4

5 2 MULTIPLE SCLEROSIS OPPORTUNITIES Anti-NMDAR mabs (BIO13193) Antibody Protecting from BSCB leakage induced CNS damages POC in vivo in EAE model IRM contrast agent (BIO16015) Imaging method to detect MS relapses POC in vivo 5

6 2 DEMYELINATING DISEASES OPPORTUNITIES Gene or cell therapy product enabling efficient peripheral neuron targeting (BIO15279) Treatment for peripheral demyelinating diseases (BIO15042) Method for expressing a polynucleotide of interest in the peripheral nervous system VDAC1 inhibition for the treatment of peripheral demyelination Product validated in vivo Target validated in vivo in rodent models (Target patent) 6

7 2 NEUROVASCULAR OPPORTUNITIES Method for clinical efficacy follow-up (MECA16457) Imaging method to follow neurovascular coupling evolution Method validated in human (Method patent) Antibody for CADASIL treatment (BIO11607) Anti Notch3 antibody for CADASIL treatment Product validated in vivo 7

8 1 NEURODEVELOPMENT OPPORTUNITY Targeting YIF1B (BIO15284) Yif1b for the diagnosis, prevention and / or treatment of ciliopathies Mutations identified in human (Target patent) 8

9 1 PAIN OPPORTUNITY Treatment of neuropathic pain (BIO14109) Fxyd2 as new target for pain management Target validated in vitro (Target patent) 9

10 1 CNS - RARE DISEASES OPPORTUNITY Friedreich ataxia - neurologic syndromes - gene therapy (BIO14346) Treatment and the prevention of neurological phenotype associated with Friedreich Ataxia Target validated in vivo 10

11 1 AGING OPPORTUNITY Sarcopenia (BIO11315) Apelin analogs for the treatment of dysfunction associated with aging Product validated in vivo in HFD mice 11

12 3 PSYCHIATRY OPPORTUNITIES Specific electrical stimulation of parvalbuminexpressing interneurons (MECA15165) Treatment of NMDA-related diseases (BIO14076) Treatment of NMDA-related diseases (CHIM16005) 4Hz oscillations to control anxiety behaviour Allosterically targeting mglur2 with conformational single domain antibodies A new NMDA receptor allosteric modulator for use in the treatment of central nervous system diseases POC in vivo in PTSD rodent models (Method patent) Product validated in Schizophrenia mouse model SME validated in Schizophrenia mouse model 12

13 2 CNS - OTHER OPPORTUNITIES Botulism diagnosis (BIO15089) In Vitro assay for detecting or measuring a biological activity of a botulinum neurotoxin POC in vivo (Biomarker patent) Blood Brain Barrier (EB15249) A novel target for Blood Brain Barrier integrity conservation mab generation ongoing 13

14 7 OPHTHALMOLOGY OPPORTUNITIES Gene Therapy for retinal detachment (BIO15004) Stargardt disease (small molecules) (CHIM14044) Use of Transferrin as a neuroprotective therapy of retinal degeneration New lipo-phenolic derivatives as anti-carbonyl stressors in retina pathologies POC in vivo slow P23H rat model of retinal degeneration Target validated in vivo Macular Edema (BIO09477) Rare disease (BIO15148) Use of Mineralocorticoids for the treatment of macular edema Undisclosed target for the treatment of Wolfram Syndrome POC in human + current clinical trial Target under in vivo validation (Target patent) Glaucoma_Gene Therapy (BIO14389) Gene Therapy (BIO16491) Product repositionning for diabetic retinopathy (BIO15371) Neuroglobin gene therapy for use in the treatment or prevention of a mitochondrial ophthalmic disease associated with respiratory chain complex Non-invasive methods of delivering a polynucleotide of interest in cone photoreceptors A novel target for the treatment of retinal capillary non-perfusion 14 POC in vitro and in vivo Method tested in mouse models and human retinal explants (Method patent) Target validated in vivo both in rodent models and patient samples (Method patent)

15 Inserm Transfert - Paris Biopark 7 Rue Watt Paris Tel: / Fax: