Programme Workshop Drug Discovery DDD innova(on: Financing & Re- purposing

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1 Programme Workshop Drug Discovery DDD innova(on: Financing & Re- purposing : : : : Welcome and introduc/on (Dr. Cecilia Nilsson, UU Innova/on and Dr. Vendela Parrow, SciLifeLab DDD) The SciLifeLab DDD Pla>orm (Prof. Per Arvidsson, Execu/ve Director SciLifeLab DDD) Re- purposing case study New and Old drugs for Cerebral Cavernous Malforma/on (Prof. ElisabeBa Dejana, UU) Intellectual property considera/ons on re- purposing (Dr. Mark Didmon & Simon CurGs, PoNer Clarkson, UK) LUNCH : : : take Funding mechanisms for early drug discovery projects the private capital take (Dr. Nanna Luneborg, Novo Seeds) Funding mechanisms for early drug discovery projects the public capital take (Dr. Maria Kaaman, VINNOVA) Funding mechanisms for early drug discovery projects the crowd funding capital (Prof. Magnus Essand, UU) : Separate Workshop with the Innova/on system (Trippelrummet)

2 Science for Life Laboratory A na/onal infrastructure for life science Develop and use large- scale technologies for life science

3 Science for Life Laboratory Develop and use large- scale technologies for molecular biosciences with a focus on health and environment Joint Uppsala Stockholm center with two nodes Hosted by four universiges Official start, July 1, 2013 ~1000 researchers Infrastructure for molecular bioscience

4 SciLifeLab mission Technology pla>orms for na/onal use Service Technology Development Analy/cal support Courses and workshops Generate a strong research environment Strong interdisciplinary community SciLifeLab Fellows Program Na/onal projects Interna/onal collabora/ons

5 600 SciLifeLab publicagons Publications JIP 9 JIP <25 6 JIP <9 JIP < Year

6 SciLifeLab focus translagonal medical research Define poten/al drug targets using omics and systems biology Mapping all human genes and proteins using omics Development of poten/al drugs (chemicals and biologicals) Clinical genomics for rou/ne pa/ent diagnos/cs Personalized medicine (stra/fica/on of pa/ents) Early detec/on of diseases and side- effects of drugs Science, January 23, 2015

7 Drug Discovery and Development pla]orm Per I Arvidsson Execu/ve Pla>orm Director

8 Pla]orm mission and descripgon The global pharmaceu/cal industry is currently undergoing a paradigm shib where more of early drug research is done in collabora/on with academia and through public- private partnerships. The SciLifeLab Drug Discovery & Development Pla>orm was established through special funds from the Swedish government to be a na(onal pladorm offering Swedish researchers access to industry standard infrastructure and exper/se for early stage drug discovery. The mission of SciLifeLab DDD is to help translate basic research to early drug development programs and build an environment for scien/fic collabora/ons of highest interna/onal standard, competence, and advanced infrastructure. Opera/onal since Q2-2014

9 SciLifeLab Drug Discovery & Development Pla]orm Industry standard drug discovery infrastructure and expertise for the Swedish academic community Pla]orm SM Ab Proposal EvaluaGon & Priority Screen Cmd handling Project Exit Preclin PoC Researcher ConGnuously improving biological understanding Researcher retains project & IP ownership

10 Scope of platform in Drug D&D process/value chain Target IdenGficaGon - OMICS & Academia SciLifeLab DDD pla]orm AddiGonal Funding* Med Chem op/miza/on Chemical & Biological TherapeuGcs Transla/onal package Candidate Drug Biologics characteriza/on *Spin- out Risk sharing

11 DistribuGon of pla]orm users 2014 SU 4% IniGal project discussions LiU 2% GU 7% UmU 14% LU 27% Swedish Research Council projects funded in Medicine & Health 2014 CTH 0% SU 1% GU 15% KTH LiU 0% 4% SLU 1% UmU 6% LU 17% UU 16% KI 30% UU 12% KI 44% 56 total

12 EducaGon and outreach Drug discovery workshops twice every year Biophysical methods in DD, Uppsala Re- purposing and funding in DD, Uppsala, How are drugs born (joint with Swedish chemical society), Solna Innova/ve Medicines Ini/a/ves u/lizing DDD infrastructure Enable, UU coordinated ULTRA- DD, SGC coordinated Extensive par/cipa/on in the Vinnova sponsored research school Mul/ple ar/cles in Swedish media during 2014, interna/onal media coverage ongoing Presenta/ons at na/onal and interna/onal conferences NewsleNer to be started Q2-2015

13 Project Application Process 1 2 PI: Introduce project and goal DDDP: Review drug discovery potential, introduce working model Confidential second opinion Tentative Project PI: Background description using template DDDP: Assessment, database searches, IP evaluation, written comment. Continuously - monthly turnaround Technical" feasibility" PI"group" Unmet"medical" need" 6" 5" 4" 3" 2" 1" 0" Therapeu8c" concept" Compe88veness" 3 4 Preclinical" feasibility" Clinical" feasibility" Intellectual" property" Small project: Evaluation by leadership team monthly. PI: Sign agreement including project plan and cost estimates. DDD Project: Evaluation by steering board bi-annually. Detailed project plan Alternative routes forward? DDDP: Initiate work on the project Project start Bi-annual re-prioritization Academic customers - Personnel & Infrastructure included, user pay for consumables

14 Visions and Future plans SciLifeLab Drug Discovery & Development Turning Discoveries into Innova(ons SciLifeLab DDD is recognized as Northern Europe s leading academic drug discovery centre. SciLifeLab DDD is an essen/al part of the Swedish innova/on infrastructure. Swedish scien/sts & research consor/a use the infrastructure and exper/se provided by DDD to engage in both na/onal & interna/onal public- private partnerships. Projects progressed through the pla>orm are considered high quality drug discovery projects, and are globally compe//ve for funding and clinical development. U- READ & Swetox

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