VALORIZZAZIONE DEI RISULTATI DELLA RICERCA ACCADEMICA E COLLABORAZIONE CON REALTA INDUSTRIALI FARMACEUTICHE BIOTECH

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1 VALORIZZAZIONE DEI RISULTATI DELLA RICERCA ACCADEMICA E COLLABORAZIONE CON REALTA INDUSTRIALI FARMACEUTICHE BIOTECH Lucia Faccio, Direttore Sviluppo Ricerca Fondazione Telethon

2 Why should academia transfer technologies to industry? i. It is their obligation as publicly funded organizations ii. It generates much needed funds to the university iii. Cooperation with industry may hold scientific advantages to academia

3 Conflict of interest University Industry Curiosity driven Knowledge Academic freedom Open Disclosure Management of Knowledge for Profit Confidentiality Limited Public disclosure

4 Technology transfer: bridge the gap Industry Academia

5 Number of Products R&D spending and output 160 R&D Spend ($ Billions) $ Total NDAs NMEs 131 $ $ $25 $20 $ $10 $ Year $0 Parexel, 2008/2009; Defined Health analysis; FDA website; Phrma.org website R&D Spending

6 Externally-Sourced Programs Drive Increasing Share of Pharma Revenue Percent of Pharma Sales Derived from External Discovery Source: Defined Health 2010

7 So a Longstanding Proportional Spending Error by Pharma Will Finally Be Corrected Self-originated vs. Licensed-in US R&D Spend % R&D Spend Uncategorized % R&D Spend on Licensed-In Projects % R&D Spend on Self- Originated Project Source: Defined Health 2010

8 Without Externally Discovered Programs Few Pharmas Would Have Late Stage Pipelines Today 100% Percent of New Molecule Entity Portfolio of Top 15 Large Pharma from External Origin (i.e. In-Licensing, Company or Product Acquisition) 80% 60% 40% 20% 0% Phase 1 Phase 2 Phase 3 Marketed Source: Defined Health 2010

9 years Licensing Why do we need to patent 13 1 product on the market (cost 1.6 bl $) Clinical Ph III/ Registration Pharma Preclinical & Clinical up to Ph II Biotech 0 Basic research University potential products

10 Technology transfer is stimulating communication between very different cultures; it only works if the intermediaries have a real understanding of both cultures Tim Cook, Director ISIS Innovation - Oxford

11 What academia scientists need to know: the dont s before patenting No publication/disclosure prior to filing e.g. no article, press release, conference presentation/abstract/poster/proceedings or blog entry/ students thesis/ laboratory web pages No Material Transfer to external laboratories No lecture or presentation prior to filing except under a confidentiality agreement (CDA) Seek OBT support and advice soon! File before others do! Patenting and Publication can move together!!!

12 Non Disclosure Agreement (NDA) It is a legal contract between at least two parties that outlines confidential material, knowledge, or information that the parties wish to share with one another for certain purposes. The parties agree not to disclose to third parties information covered by the agreement. An NDA creates a confidential relationship between the parties to protect any type of confidential and proprietary information or trade secrets. As such, an NDA protects nonpublic business information.

13 Material Transfer Agreements (MTA) It is a contract that governs the transfer of tangible research materials between two organizations, when the recipient intends to use it for his or her own research purposes. The MTA defines the rights of the provider and the recipient with respect to the materials and any derivatives. Biological materials, such as reagents, cell lines, plasmids, and vectors, are the most frequently transferred materials, but MTAs may also be used for other types of materials, such as chemical compounds and even some types of software.

14 Industry - Academia collaborations: possible scenarios Know How Industrial funding to academic research Patent Rights License

15 Industrial funding to academic research Must have in an industrial collaboration contract: SPONSORED RESEARCH Know How SERVICE TO INDUSTRY Definition of background IP and Know how Definition of ownership of future results Definition of access by Pharma to future results (i.e. option, license exclusive vs non exclusive etc) Management of joint IP Publication rights and evaluation of patentability Contract Termination : access by the parties to non patentable material/know how developed during the collaboration

16 Industry - Academia collaborations: possible scenarios Know How Industrial funding to academic research Patent Rights License

17 Nine things to remember in licensing 1. Universities should reserve the right to practice licensed inventions and to allow other non-profit and governmental organizations to do so 2. Exclusive licenses should be structured in a manner that encourages technology development and use 3. Strive to minimize the licensing of future improvements 4. Universities should anticipate and help to manage technology transfer related conflicts of interest 5. Ensure broad access to research tools 6. Enforcement action should be carefully considered 7. Be mindful of export regulations 8. Be mindful of the implications of working with patent aggregators 9. Consider including provisions that address unmet needs, such as those of neglected patient populations or geographic areas, giving particular attention to improved therapeutics, diagnostics and agricultural technologies for the developing world From

18 PEER REVIEW 1. TELETHON S MISSION AND ACTIONS INTRAMURAL RESEARCH: 13.3M TIGEM Institute: genetics and medicine (Naples) HSR-TIGET Institute: gene therapy (Milan) Dulbecco Telethon Institute: career program EXTERNAL RESEARCH: 15,2 M Research projects Biobanks Collaborative programs Diagnostic, observational and palliative studies RESEARCH INVESTMENT yearly competitive call 1-3-yr funding 5-yr grant renewal (site visits/audits) PERCENT FUNDING DISTRIBUTION, % DISEASE 9% 48% 35% funding to therapeutic approaches/clinical trials 18 projects in the clinical pipeline 9% 10% Studies on mechanism Gene identification 16% CURE Clinical trials ADA-SCID In vivo therapeutic approaches In vitro therapeutic approaches MISSION TO ADVANCE BIOMEDICAL RESEARCH TOWARDS CURES FOR MUSCULAR DYSTROPHY AND OTHER GENETIC DISEASES BACKGROUND Major Italian biomedical charity focused on genetic diseases Founded in 1990 out of the wish of a group of patients Support through fundraising FIGURES M research investment 2351 research grants 1461 principal investigators 459 genetic diseases studied 7964 scientific publications AVERAGE CITES/PAPER Telethon:15.8 USA: 9.7 Italy: 7.8 EU 27: 7.3

19 STAKEHOLDERS 2. TELETHON S VISION AND STRATEGIES COLLABORATIVE STAKEHOLDER MODEL OUR VISION FOR 2015 WE WANT TO CONVERT THE RESULTS OF EXCELLENT, SELECTED AND SUSTAINED RESEARCH INTO AVAILABLE THERAPIES Research Development Delivery GENETIC DISEASES Basic research Pre-clinical studies Pre-clinical development Clinical Trials Commerc. & post market surveillance Public domain Patients A B Telethon C Pharma Co/Biotech D TELETHON S STRATEGIC PLAN CONTINUE SUPPORTING THE BEST RESEARCH ON GENETIC DISEASES BY Applying the rigorous merit-based selection developed at Telethon Seeking additional support through public funding (A) Establishing co-financing activities with other charities/ foundations/ patient organizations (B) DEVELOP THE TRANSLATIONAL DRIVE OF TELETHON RESEARCH BY: Promoting networking activities across Europe and abroad (A) Promoting involvement of patients in data / biomaterial collection and sharing (B) Potentiating IP and technology transfer activities (including results of external research) (C) Liaising with regulatory bodies for the clinical development of preclinical studies (D)

20 GSK, Fondazione Telethon and Fondazione San Raffaele to collaborate on gene therapy for rare diseases London UK 18 October 2010 GlaxoSmithKline PLC (GSK), Fondazione Telethon and Fondazione San Raffaele today announced a new strategic alliance to research and develop novel treatments to address rare genetic disorders, using gene therapy carried out on stem cells taken from the patient s bone marrow (ex vivo). The alliance capitalises on research performed at the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), a joint venture between Fondazione Telethon and Fondazione San Raffaele established since Under the terms of the agreement, GSK will gain an exclusive licence to develop and commercialise an investigational gene therapy, for ADA Severe Combined Immune Deficiency (ADA-SCID) - a rare and life-threatening immune deficiency, which affects approximately 350 children worldwide. Phase I/II studies have demonstrated the potential of this treatment option to restore long-term immune function and protect against severe infections in children with ADA deficiency. i In addition, GSK will co-develop with Fondazione Telethon and Fondazione San Raffaele, six further applications of ex vivo stem cell therapy, using a new gene transfer technology developed by HSR- TIGET scientists, with the potential to treat a range of rare disorders. This first of these will be metachromatic leukodystrophy (MLD) and Wiskott-Aldrich Syndrome (WAS). Others include; betathalassemia, mucopolysaccharoidosis type I (MPS); globoid leukodystrophy (GLD); and chronic granulomatous disorder (CGD). Clinical trials for WAS and MLD were initiated at HSR-TIGET last spring and are currently recruiting patients.

21 Thank you!