Crossing the Valley of Death: The Translational Funding Crossing the Valley of Death: The Translational Funding and Management Gapand Management Gap

Moderator: Sharon Begley, Science Editor, Newsweek

Panelists:• Robi Blumenstein, President, CHDI Management/CHDI Foundation• Randall Carpenter, Co-Founder, President and Chief Executive Officer,

Seaside Therapeutics• Dennis Purcell, Managing Partner, Aisling Capital• Amy Comstock Rick, Chief Executive Officer, Parkinson's Action Network• Christian Loucq, PATH Malaria Vaccine Initiative

Brain Developmental

Disorders

Translating the mGluR Theory into Novel Therapeutics

Scientific Discovery

Druggable Targets

Efficacy in Model System

Candidate Drug

IND-enabling Research

Human Trials

Seaside Therapeutics Model

• Venture Philanthropy Seed Funding

• Core Competency in Translational Medicine

• Collaborative Business Model– Foundations – FRAXA, Autism Speaks, NFXF

– Merck & Co, Inc. – R&D Collaboration

– NIH UO1 Translational Research Cooperative Agreement• NIMH, NICHD, BPCA, NINDS, FRAXA & Autism Speaks

– Baylor Medical Center – Behavioral Research

– Vanderbilt University – Drug Discovery

– Correlagen Diagnostics – Companion Diagnostic

Basic Research

Prototype Design or Discovery

Preclinical Development

Clinical DevelopmentFDA Filing/ Approval &

Launch Preparation

The Development Pipeline and Valley of Death

Basic Research

[Mostly NIH Funded]

Prototype Design or Discovery

Preclinical Development

Clinical Development[Mostly Large Pharma]

FDA Filing/ Approval &

Launch Preparation

The Development Pipeline and Valley of Death

[Inconsistent Funding]

[Inconsistent Funding]

The Development Pipeline and Valley of DeathVarious Solutions

Prototype Design or Discovery

Preclinical Development

Clinical Development[Mostly Large Pharma]

FDA Filing/ Approval &

Launch Preparation

Private Foundation Research

NIH - SBIR

Venture Philanthropy

[Inconsistent Funding]

[Inconsistent Funding]

NIH – CTSA’s

TATRC

Basic Research

[Mostly NIH Funded]

TRND