Clinical and TranslationalScience Institute / CTSIat the University of California, San Francisco

CTSI SLGNovember 7, 2011Early Translational Research

June Lee, MD

Translational research

Academia

Start up company/ biotech

Large pharma/company

Academia

Start up company/

biotechLarge

pharma/company

Early (T1) Late (T2)

Bench Bedside Community

Outcomes/ ImpactOutcomes/ Impact BetterHealth

T1 Translational Catalyst Award

• Identify the best projects– Diagnostics, devices, or therapeutics

• Bring in missing pieces– Panels include people from industry and academics– Panels review and provide individualized feedback

• Leverage limited resources– Identify partnerships if appropriate

• Feedback– Pilot grants helped advance programs– Customized consulting service very useful

T1 Catalyst Award program assessment

• Status– In our 4th cycle of funding– Number of proposals submitted increased

substantially– Changed the review process to include multiple

specialized tracks• Therapeutics (13 proposals)• Diagnostics (8 proposals)• Devices (4 proposals)• mHealth (8 proposals)• Orphan/ rare diseases (TBA)

T1 Catalyst Panels/ Consultations

• Consultation panel comprised of:– Faculty– Industry experts (Approximately 80% volunteers)

• Drug/Device/Diagnostics development experts• IP attorneys• Venture capitalists• Experienced bioentrepreneurs

– OTM representative– QB3 representative

Consultation awards

• Offered to approximately 50% of applicants

• Average award value $2K-$5K

• Pilot award and 100K award recipients will be determined in February

Other key activities in T1 Catalyst Program

• Deploying the consultation service to UCSF investigators beyond the T1 Catalyst Award participants– In collaboration with OTM

• Enhance the consultant panel

• Identifying additional sources of funding– Pharma partners– Entrepreneurs/VCs

•

T1 translational catalyst program

Identify & Enable

ScienceInvention

InventionProduct

Gap: Science to Invention, Invention to Product

Translational research in drug development

•Unmet medical

•need/potential clinical

indication(s)

•~18 months

•DISCOVERY •ESR •LSR •ED •ECD (Ph I/II)

•IND submission

and clearance

Early phase drug development activities• Discovery/Early stage research/ Late stage research

– Target selection/validation– Proof of activity– Lead molecule optimization– Evaluation of unmet need

– Define target candidate profile (TCP)

• Late stage research/ Pre-early development– Select lead molecule– Indication (s) selection– Define target product profile (TPP)

• Early Development– Clinical manufacturing– IND enabling toxicology study– Regulatory filing strategy– Overall clinical development strategy

Innovative partnerships

• Critical to extending the T1 translational capacity in academia

• Non-traditional models and re-defining the boundaries of private/public partnerships– Pharma/academia– Early stage investors (e.g. Venture capital)– Foundations and venture philanthropy– Others