Milano, September 7 th 2016

Transcription

1 Milano, September 7 th 2016 Dear IRDiRC Consortium Assembly, I would be delighted to share with the IRDIRC Therapeutic Scientific Committee my knowledge and expertise in both fields of regulatory affairs and translational medicine for rare diseases. I have a strong background built in a large pharmaceutical company, where I worked in the R&D Clinical Development and CMC Regulatory Affairs areas supporting new chemical entity (NCE) projects from the translational stage up to registration. In January 2011, I joined Fondazione Telethon and focused my activities on gene therapies for rare genetic diseases. In particular, in my role as Head of Alliance Management and Regulatory Affairs Manager, I have contributed to the completion of the Strimvelis development up to registration and market access. My key accountability is acting as primary point of contact and coordinating activities and priorities among the three members of the strategic alliance established in 2010 to the aim of developing gene therapies for rare diseases and making them accessible to patients: the charity Fondazione Telethon and the academic hospital Ospedale San Raffaele (which jointly own the San Raffaele- Telethon Institute for Gene Therapy in Milan) and the pharmaceutical company GSK. Since 2011, three out of the seven ex- vivo gene therapy programs included in the alliance have been licensed to GSK and one of them (addressing the genetic diseases ADA- SCID) has reached the registration in EU with the commercial name of Strimvelis and is currently completing the market access process. The registration process for the other two licensed programs (addressing metachromatic leukodystrophy and Wiskott- Aldrich syndrome, respectively) is underway. My competency in Orphan Drug Designation, Paediatric Investigational Plan and Scientific Advice/Protocol Assistance at EMA has been key to advancing these activities effectively. With more than 15 years experience in the pharmaceutical industries and in academia/charity organizations, I have faced all the road blocks of drug development (spanning from NCE up to Advanced Therapy Medicinal Products) and I hope I may share my experience within the IRDIRC Therapeutic Scientific Committee to the benefit of the Rare Disease Community. Thank you for considering my request. With best regards, Head of Alliance Management & Regulatory Affairs Manager Fondazione Telethon

2 Curriculum Vitae European Format ( September 2016 update) Personal Information - Surname/First name Gabaldo Michela - Place and Date of Birth - Gender/Marital status - Address/Contacts Arzignano (VI), Italy on 20 th June 1972 Female, married with 2 children Via Domenico Turazza, 21/G Verona (VR), Italy Mobile: / gabaldo.michela@libero.it / mgabaldo@telethon.it - Nationality Italian (hold Italian passport) - Driving licence Full clean EU car driving licence Professional Experience Current Position July 2015 Present: Head, Alliance Management & Regulatory Affairs Manager within SR-TIGET Telethon Institute for Gene Therapy Milan, Italy. The SR-TIGET (San Raffaele-Telethon Institute for Gene Therapy) is a jointventure between the Ospedale San Raffaele and the Telethon Foundation committed to perform innovative research in the area of gene transfer and cell transplantation and to translate the results into successful clinical application of gene and cell therapy for genetic diseases. On October 2010, Telethon and Ospedale San Raffaele enters a strategic alliance with GlaxoSmithKline (GSK), 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). I m the Telethon/San Raffaele Alliance Manager responsible for the execution of this first strategic Alliance with GSK managing projects at a different stage of development ranging from preclinical, to clinical, to EMA Marketing Registration & market access stage. Additionally, I m also Head, Alliance Management supporting and training Junior managers dealing with other strategic alliances established by SR-TIGET to progress their promising research for a different indication. Alliance Management Expertise and responsibilities: - Being responsible for developing and maintaining broad and deep external partner relations with company partners in all solutions areas; - Being knowledgeable of the partner s propositions, their markets, business challenges and customer opportunities; - Being accountable for ensuring partner account of the vision, strategies and capabilities of the alliance (different vision/priorities between multinational giant and Academia/No-profit organization); Pag 1 Curriculum vitae of - Ensure good communication between pharmaceutical giant and Academia/No-

3 profit organization; - Building multi-level relationships with partners including those in the executive level; - Coordinating Alliance Joint Steering Committees, Joint Development Team, Joint Project Clinical Team, Joint Operational Team and other ad-hoc Meetings; - Developing and executing attainable and strategic plans with the market needs and the potential ODD Market Exclusivity; - Coordinating internal discussions (with both Telethon Foundation and San Raffaele Hospital) and constant engagements with the external Partners in terms of Market Access strategy (Price & Reimbursement) for ex-vivo Gene Therapy - Being knowledgeable of Translational Medicine from Drug Discovery to Clinical Translation, to Marketing Registration and Market Access for gene therapy - Actively Monitoring the services provided by the GMP CMO (i.e., Contract Manufacturign Organisation MolMed S.p.A.) which manufactures under GMP the Investigational Medicinal Products (IMPs) for our clinical trials, in terms of contracts, orders, capacity, logistics, quality, changes, compliance. - Being accountable for finalizing service contracts/collaboration agreement with external Partners in conjunction with Technology Transfer, Contract and Legal Offices (i.e., for the conduction of GLP/R&D preclinical studies; for the conduction of sponsored clinical trials; others); - Training and Managing Junior employees working on other strategic alliances established by SR-TIGET; - Contributing to the implementation and improvement of the Quality Management System (i.e., active player in the set-up and certification of the first Academic GLP Certified Test Facility ); Regulatory Affairs expertise and responsibilities: - Being responsible for EU and US Orphan Drug Designation, EU Scientific Advice/Protocol Assistance and EU PIP (i.e., Paediatric Investigational Plan) applications for internal and external researchers; - Being active player in the EMA GCP Inspection for Strimvelis registration in July 2015; - Supervising clinical trial submissions to AIFA (ISS) and Ethical Committee for ex-vivo gene therapy - Supervising requests for Compassionate use and Hospital Exemption according to Italian Regulatory Authorities laws for our gene therapy - Being knowledgeable of and facilitate/promote internal awareness of any new regulation/regulatory Agency initiatives (i.e., New EU Clinical Trial Regulation, Adaptive Licenses/Pathways, FDA Breakthrough products, EMA PRIME, etc) Previous Experiences December 2014 June 2015: Senior Alliance Manager & Regulatory Affairs Manager within SR-TIGET Telethon Institute for Gene Therapy Milan, Italy. January 2011 November 2014: Alliance Manager within SR-TIGET Telethon Institute for Gene Therapy Milan, Italy. October 2010 January 2011: Senior Medical Writer within Global Clinical R&D department at Novartis Vaccines and Diagnostics s.r.l. Siena, Italy Pag 2 Curriculum vitae of

4 July 2010 September 2010: Regulatory Executive at Aptuit s.r.l (former GlaxoSmitkline S.p.A.), Verona, Italy April 2008 June 2010: Principal Regulatory Executive within Corporate CMC (Chemistry Manufacturing and Control) Global Regulatory Affairs, Pre-Approval at GlaxoSmitkKline S.p.A. Verona, Italy, with line-manager based in the UK. October 2005 March 2008: Senior Regulatory Executive permanent position within Corporate CMC Global Regulatory Affairs, New Submissions at GlaxoSmitkKline S.p.A. Verona, Italy, but with line-manager based in the UK. August 2003 September 2005: Junior Regulatory Executive within Corporate CMC Global Regulatory Affairs, New Submissions at GlaxoSmitkKline S.p.A. Verona, Italy, but with line-manager based in the UK. April 2002 July 2003: Junior Research Scientist within Clinical Science and Study Operations group at GlaxoSmitkKline S.p.A. October 2001 March 2002: Junior Research Technician within Clinical Pharmacology Unit at GlaxoSmitkKline S.p.A. Secondment Experiences September December 2005: on secondment at GlaxoSmithKline, Corporate CMC Global Regulatory Affairs, Pre-Approval, Ware & Harlow, UK to gain experience of late phase and Major Filings (NDA/MAA) Dossiers for NCEs November 2009 July 2010: on secondment within GlaxoSmithKline, Corporate BioPharm Global Regulatory Affairs, Pre-Approval, Harlow, UK (based in Verona, but working for UK) to gain experience of filing Dossiers for Biopharmaceutical products (focus on monoclonal antibodies) Educational Experience 2010: 2 nd Level Master in Regulatory Affairs and Market Access for NCE and Biopharmaceutical Drugs at the University of Novara, Italy 2002: Granted Pharmacist Professional Licence 2001: Pharmaceutical Chemistry and Technology Degree from Pharmacy School of University of Padua, Italy. Final score: 105/ : High school graduation from Liceo Classico Ginnasio Statale A. Pigafetta, Vicenza, Italy Member - Member of TOPRA, The Organisation of Professionals in Regulatory Affairs, UK - Member of AFI, Italian Society of Pharmaceutical Industries (scientific society) Participation in meetings/workshops - Panel discussant: Avicenna A strategy for In-silico Clinical Trial, Event 4, Brussels, Belgium, February ARM, Alliance for Regenerative Medicines Europe Advanced Therapies Summit, Paris, France, 12 March 2015 Pag 3 Curriculum vitae of

5 - Phacilitate Cell & Gene Therapy Europe 2015, Barcelona, Spain September Invited speaker: School of Italian Pharmaceutical Industry - Rome, Italy 1,2 e 3 December 2015, Gene Therapy (Quality/Safety/Efficacy) - Panel discussant: FADOI National Congress on Research from No-Profit Sponsor, Rome, Italy, 8-9 March Panel discussant: multi-stakeholder meeting on Advanced therapy medicines: exploring solutions to foster development and expand patient access in Europe, EMA office, London, 27 May Panel discussant: TopRA Workshop Regulatory Challenges of Successfully Developing ATMPs, London, 15 Jun 2016 Upcoming: - Invited expert to the AD-HOC MEETING on BIOLOGICALS International Nonproprietary Names, WHO, September 2016, Geneva, Switzerland Leadership Skills Analytical and strategic thinking, problem solving skills, excellent organizational attitudes judgment skills and good reliability confirmed by several recognition awards received for the high commitment and dedication to the projects; Result orientation and sense of continuous improvement, concern for quality, efficiency and accuracy, adaptability, self-management and development, organizational alignment; committed team worker; Integrity, loyalty and conscience, discretion, independence and confidence, respect for diversity; Able to work on different projects and with different roles; Able to guide and to advise colleagues in different project related activities and at different company level; Having very good communications skills. Excellent in building and maintaining relationships at any company level; Very disciplined in working with people as well as culturally sensitive in dealing with them. Languages IT Italian: mother tongue; English: Excellent (written/spoken). Advance knowledge of PC and MAC main programs (I authorize the use of my data - Dlgs 196/2003) Pag 4 Curriculum vitae of