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Page 1: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);
Page 2: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

Thank You

Venue Host

Title Sponsor

Presented By

Page 3: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

Neuroscience Partnering at NIH

Sue Ano, Ph.D.

Technology Development CoordinatorNational Institute of Neurological Disorders and Stroke (NINDS)

Page 4: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

About Sue

Licensing and Patenting ManagerInfectious Diseases

NIH OTT

2002

2008

2015

Branch ChiefInfectious Diseases and Medical

EngineeringNIH OTT

DirectorTechnology Transfer

NINDS

Page 5: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

NINDS Technology Transfer Mission

Support Our

Investigators

Build

Partnerships

Facilitate Scientific

Innovations

NINDS mission: to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease

Page 6: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

NINDS Intramural Research Program (IRP)

• $169M Fiscal Year 2017 (~10% of NINDS overall budget)

• Basic and translational neuroscience, neurology and neurosurgery

Neuromuscular Diseases

Clinical Neurosciencee.g., Parkinson’s, epilepsy, multiple sclerosis, dystonia, neuropathies, and gene therapy

Cell/Membrane Biochemistry/Biophysics

Neuroimmunology

Neurogenetics

Functional and Molecular Imaging

Page 7: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

NIN

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Agr

eem

ents

Exe

cute

d

New

Inve

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on

s

Agr

eem

ent

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reat

ed

NIN

DS

TT T

eam

>320

24024 CDAs

32 collab

10 licenses

6 CRADAs

5740 PrincipalInvestigators

14 Staff Clinicians

3 Associate Clinical Investigators

12

5

NINDS Tech Transfer - 2018 Numbers

Page 8: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

NIDDK

NIAID

NIDCR

NINDS

NEI

NICHDNIMH

NICHD

NIA

CCB

NCCIH

NIEHSNHGRI

NIDA

Neuroscience at NIH

Page 9: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

Porter Neuroscience Research Center• 85 research groups from 10+

Institutes/Centers at NIH

• World-class researchers

• Imaging and microscopy cores

• Transgenic animal cores

• Mark O. Hatfield Clinical Center (PBS special “First in Human”)

• Induced pluripotent stem cells (iPSC)

Page 10: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

Example Successful Partnerships

• CRADA with GeNeuro SA

• Test and evaluate several monoclonal antibodies provided by Geneuro SA • Anti-HERV-K envelope protein

(Env) • Evaluated for their properties to

neutralize Amyotrophic Lateral Sclerosis (ALS)-related biological and pathological effects induced by HERV-K envelope expression in various model systems

Page 11: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

Example Successful Partnerships

• Multi-IC CRADA with General Electric (GE) Healthcare

• >10 years and still going

• Improve quality and breadth of GE proprietary MRI technology• Hardware

• Acquisition pulse sequences

• Software for MR signal processing

Page 12: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

Example Successful Partnerships

• Two clinical CRADAs with Audentes Therapeutics• one site in a multi-site pre-Phase 1 prospective, non-interventional clinical

assessment study in X-linked Myotubular Myopathy (XLMTM) subjects aged 3 years and younger (INCEPTUS)

• one site in a multi-site clinical trial, entitled “A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients (ASPIRO)”

• Gene therapy following our role in GAN trial• www.cbsnews.com/news/a-mothers-quest-to-find-cure-for-rare-genetic-defect

Page 13: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

Sample Partnering Opportunities

• Licensing: >100 technologies (NINDS)• Novel Dopamine D2 Receptor Antagonists

• Small molecule• D2 specificity minimizes off-target side effects• Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias

Issued US patent (9,550,742); pending EP application

• Wirelessly Powered MRI Signal Amplification System• Implantable or ingestible• Issued US Patents (9,864,026; 10,203,382); pending EP application

• Research reagents• Cell lines• Antibodies

• Visit www.ott.nih.gov/opportunities for NIH licensing opportunities

• Contact us to discuss collaborations and other partnering opportunities• Provide 1 page summary of opportunity

Page 14: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);

National Institutes of HealthNational Institute of Neurological Disorders and Stroke

Neuroscience@NIH

[email protected]

301-435-5515

[email protected]

[email protected]

Neuroscience.nih.gov

Partnering Opportunities:

Collaborations

Clinical Trials

Research Materials

Patents

Licenses

Page 15: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);
Page 16: Thank You - MAVA•Small molecule •D2 specificity minimizes off-target side effects •Schizophrenia and psychotic syndromes, diabetes, cardiac arrhythmias Issued US patent (9,550,742);