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Non For Profit Modelfor Rare Disease Therapy

Development

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Rare Disease Therapy Development - Historically

• Therapy Development Process– Historically big business– Large investment– Time to Trial 6 to 8 years– Rare disease economically not attractive

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Typical Rare Disease “Proactive” Advocacy Groups

• Foster collaborations• Liaisons between Basic Research and Biotech

companies• Deliver resources to fill funding gaps in the

development process• “Managing science”

–resources/timeframes/direction/goals• Therapy focus – move what is known today forward• Political Involvement

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Standard Therapeutic Options

• Enzyme Therapy

• Gene Transfer

• Stem Cell Therapy

• Small Molecule Pharmaceuticals– High Through-put Screening

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Therapy Development Process

• Proof of Concept• Investigational New Drug Application (IND)

– Protocol Development– Markers and Endpoints– Toxicology

• Short Term Studies• Long Term Studies

– Pharmacology– Chemistry/Manufacturing– Dosage and Delivery Studies

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Therapy Development Process (cont.)

• Institutional Review Board (IRB)– Informed Consent Forms– Approvals

• Recombinant Advisory Committee (RAC)

• Food and Drug Administration (FDA)

• Clinical Trial Initiation

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Rare Disease Therapy Development Process Commonality

Disease

AB C

• Proof of Concept X X X

• IND X X X

• IRB X X X

• RAC X X X

• FDA X X X

• Clinical Trial Initiation X X X

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Rare Disease Synergies

• Regardless of the therapy we all face the same procedural development issues.

• Uniting empowers rare diseases

• What is good for one of us is good for all

• How do we leverage the commonality?

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Non-For-Profit Therapy Development Model

• Non-For-Profit foundation sponsors the therapy development

• Academic society is the Principle Investigator

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Non-For-Profit Therapy Development Model Benefits

• Goal is to develop therapies verses commercialization of a drug– Quickly take proof of concept from the lab to the clinic– NPF assume risk and responsibility– Leveraging infrastructure and regulatory knowledge– Provide data to companies for future clinical phases for long

term solutions– Funding sources: foundations, companies, or NIH– Cost savings in therapy development for companies to prove

concept before large scale investment– Business issues not a concern to achieve the NFP goals

regarding intellectual properties or patents

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Advancing Rare Diseases to the Clinic in a United Approach

• Rare diseases have a platform to move to a clinical program

• Together we have stronger voice to regulatory committees

• Quicker to clinic

• Develop NFP models for each type of therapy

• We all face the same issues