Tides Final Event Guide 2

1 Registration EUROTIDES DAY 1: TUESDAY 6TH NOVEMBER. MANUFACTURING OF OLIGONUCLEOTIDE THERAPEUTICS AT CMO/CRO: PROCESS ECONOMY, PROCESS DEVELOPMENT AND PROCESS UPSCALING. Room 11, 12 & 14. Chairperson's Opening Remarks Thomas Rupp, 3[RIV . 4 VMRGMTEP, Thomas Rupp Consulting, Germany GMP Oligonucleotide Manufacturing- Do It Yourself or Use A CMO? Hagen Cramer, 7 IRMSV (MVIGXSV SJ 1 ERYJEGXYVMRK, WAVE Life Sciences Key Aspects of Upscaling Your Process: Process Development In-House or At The CMO? Thomas Rupp, 3[RIV . 4 VMRGMTEP, Thomas Rupp Consulting, Germany Lunch How to Foster A Successful Client - CMO/CRO Relationship?)]]

2 Stefan Vonhoff, :MGI 4 VIWMHIRX '1', NOXXON Pharma AG, Germany Afternoon Coffee Break Considerations for Establishing a Sustainable Supply Chain for API. Supply for a Commercial Oligonucleotide Mike Webb, 'SRWYPXERX MikeWebbPharma Ltd. UK. Closing Discussion Session End of Workshop 17. Registration EUROTIDES DAY 2: WEDNESDAY 7TH NOVEMBER. OPENING PLENARY: Room 6 - 8. 09:00 Chairperson's Opening Remarks 09:10 RNAi Therapeutics Delivered: From Principles to Patients Muthiah (Mano) Manoharan, 7 IRMSV :MGI 4 VIWMHIRX SJ (VYK (MWGSZIV] Alnylam Pharmaceuticals, Inc. 09:50 LNA, Antisense, Pre-Clinical Updates Troels Koch, , :MGI 4 VIWMHIRX ERH ,IEH SJ 6 IWIEVGL 62% 8 LIVETIYXMGW Roche pRED, Roche Innovation Center Copenhagen 10:30 Morning Coffee and Networking TRACK 1: Delivery of Oligonucleotides and R&D Strategies TRACK 2: Manufacturing and Scale Up Strategies Room 6- 7 Room 8.))

3 Chairperson's Opening Remarks Chairperson's Opening Remarks Bo Rode Hansen, 4 VIWMHIRX '73 ,IEH SJ 6. ( Yogesh Sanghvi, 4 VIWMHIRX Rasayan, USA. Genevant Sciences 11:15 From Stereopurity To Precision Medicine: 11:15 Launching Clinical Antisense Oligonucleotide Optimising The Properties Of Antisense Nucleic Acid Manufacturing Capability in a Biologics Company Therapeutics Sheron Branham, %WWSMGEXI (MVIGXSV Biogen Chandra Vargeese, 7 IRMSV :4 ,IEH SJ (VYK . (MWGSZIV] WAVE Life Sciences 2I[ (MWGSZIVMIW [MXLMR 7 XIVSHI RIH 11:45 Considerations of Process Design for the Solid Phosphorothioate LNA Phase Oligonucleotide Synthesis Erik Daa Funder, 7 IRMSV 7 GMIRXMWX 6 SGLI 4 LEVQE Jale Muslehiddinoglu, 7 IRMSV 4 VMRGMTEP )RKMRIIV.))))]

4 6 IWIEVGL ERH )EVP] (IZIPSTQIRX Roche Innovation Janssen Pharmaceuticals of Johnson & Johnson, Centre Copenhagen A/S Belgium 9 RHIVWXERHMRK %73 8 VEJ GOMRK 12:15 Insights into the Sequence Fidelity of Emily Linnane, 4 SWXHSGXSVEP 6 IWIEVGLIV Chemically Synthesised Oligonucleotides AstraZeneca, UK H seyin Ayg n, '73 BioSpring GmbH. 12:45 Lunch and Networking 13:45 Oligonucleotide Therapeutics Now on Target: 13:45 Developments In The Manufacturing Of Activity of Oligonucleotide Antibody Conjugates Tricyclo-DNA Oligonucleotides Arthur A. Levin, )\IGYXMZI :MGI 4 VIWMHIRX 6 IWIEVGL Branislav Dugovic, '83 Synthena AG, Switzerland ERH (IZIPSTQIRX Avidity NanoMedicines 14:15 Ligand Conjugates for Targeting of Viral and 14:15 A Systematic Approach Towards Development Endogenous Gene Targets of a Robust Process for Oligonucleotide Kieu Lam, (MVIGXSV 8 IGLRSPSK] (IZIPSTQIRX Manufacturing Genevant Sciences Corp.))

5 Ramesh Rajeswaran, 7 IRMSV +VSYT 0 IEHIV Syngene International Ltd, India 18. EUROTIDES DAY 2: WEDNESDAY 7TH NOVEMBER. 14:45 CriPec Nanomedicines: rationally designed 14:45 Enzyme Catalysed Assembly Of oligonucleotide therapeutics for superior tumour Oligonucleotides treatments David Tew, 4 VSNIGX 0 IEHIV . +7/ 7 IRMSV *IPPS[ . Jimmy Weterings, PhD, 4 VMRGMTEP 7 GMIRXMWX 'LIQMWXV] &MSPSKMGEP 8 IGLRSPSKMIW 6( 1 EREKIQIRX . Cristal Therapeutics, The Netherlands GlaxoSmithKline, UK. 15:15 Advances in RNA delivery using the Viromer 15:15 Enzymatic DNA Synthesis, a Novel Approach Technology for Therapeutic Nucleic Acid Synthesis Steffen Panzner, 1 EREKMRK (MVIGXSV Lipocalyx GmbH Elise Champion, :4 6 IWIEVGL DNA Script, France 15:30 AJIPHASE Track Record in Europe: Recent Developments and Scale Up Experience at Ajinomoto OmniChem Geert Schelkens, 6.))

6 ( 1 EREKIV 4 LEVQEGIYXMGEP *MRI . 'LIQMGEPW Aji Bio Pharma Services 16:00 Afternoon Coffee and Networking 16:30 Next Generation R&D Strategies and 16:30 High Purity Liquid Phase Oligonucleotide -RRSZEXMZI 'LIQMWXVMIW ERH 1 SHM GEXMSRW Synthesis with Nanostar Sieving Michael E. stergaard, 6 IWIEVGL *IPPS[ Jack Cordrey, 4L( 7 XYHIRX Imperial College Ionis Pharmaceuticals 17:00 Lipophilic LNA-based Antisense 17:00 Is Solution API Right For You Practical and Oligonucleotides Strategic Considerations Jesper Wengel, 4 VSJIWWSV 'IRXIV (MVIGXSV &MS2)' % Robert Simler, , %WWSGMEXI (MVIGXSV )RKMRIIVMRK . :/6 'IRXIV SJ )\GIPPIRGI, FKF- University of Southen ERH 8 IGLRSPSK] Biogen Denmark 17:30 Overview of Next-generation Approches for 17:30 GMP Manufacturing of RNA/DNA Containing Rapid Production of Nanoparticles for Use in Gene 0 MTSWSQEP *SVQYPEXMSRW *VSQ 1 MGVS YMHMGW XS.)

7 And Cell Therapies Large-Scale Samuel Clarke, (MVIGXSV SJ 6. ( Charalampos Koutsoulas, PhD, ,IEH SJ 0 MTSWSQI . Precision NanoSystems, Inc. 6. ( 4SP]QYR 7 GMIRXM G +QF, 18:00 End of Day 2 & Networking Evening Reception in the Exhibiton Hall 19. Registration EUROTIDES DAY 3: THURSDAY 8TH NOVEMBER. OPENING PLENARY: Room: 6 - 8. 09:00 Chairperson's Opening Remarks Bill Marshall, ')3, MiRagen Therapeutics, USA. 09:10 DCR-PHXC: Successful Translation of RNAi from Bench to Clinic Ralf Rosskamp, MD, 'LMIJ 1 IHMGEP 3J GIV Dicerna Pharmaceuticals Bob Brown, '73 ERH 7:4 6 IWIEVGL . (IZIPSTQIRX Dicerna Pharmaceuticals 09:50 RNA Interference for Mass Markets: Groundbreaking Therapy for Cardiovascular Disease Brings Manufacturing Challenges Clive Meanwell, 'LMIJ )\IGYXMZI 3J GIV The Medicines Company 10:30 Morning Coffee and Networking TRACK 1: Non-Clinical, Preclinical and Clinical Development TRACK 2: CMC and Analytical Method Development Strategies Room 6- 7 Room 8.))

8 Chairperson's Opening Remarks Chairperson's Opening Remarks Professor Cy Stein, (ITEVXQIRX SJ 1 IHMGEP Stefan Vonhoff, :MGI 4 VIWMHIRX '1' NOXXON. 3 RGSPSK] ERH )\TIVMQIRXEP 8 LIVETIYXMGW City of Pharma AG, Germany Hope Medical Center, USA. 11:30 The Dose Makes All the Difference: Impact of 11:30 Future State of Collaborations for Targeted Delivery on Appearance and Therapeutic Oligonucleotides Margins for the Typical 2'-MOE ASO Class Effects. Rachel Orr, 7 IRMSV 7 GMIRXMWX GlaxoSmithKline R&D. Scott Henry, PhD, , :MGI 4 VIWMHIRX SJ 2 SRGPMRMGEP . (IZIPSTQIRX Ionis Pharmaceuticals, Inc. 12:00 In Vitro And In Vitro Assessment Of Human -QTYVMX] UYEPM GEXMSR WXVEXIK] JSV ERXMWIRWI.)

9 7 TIGM G 3 PMKSRYGPISXMHIW *SV (YGLIRRI 1 YWGYPEV oligonucleotides Dystrophy Yannick Fillon, 7 IRMSV 7 GMIRXMWX Biogen Pietro Spitali, %WWMWXERX 4 VSJIWWSV Leiden University Medical Center 12:30 Update from Sirnaomics RNAi Platform and 12:30 Screening for Success- Selectivity Drives Therapeutic Pipeline Method Development David Evans PhD, '73 Sirnaomics Inc., Gaithersburg, Brian M Kile, 4 VMRGMTEP 7 GMIRXMWX Nitto Avecia Inc. MD, USA. 13:00 Lunch and Networking 20. EUROTIDES DAY 3: THURSDAY 8TH NOVEMBER. 14:15 Preclinical and Clinical Updates on 3 PMKSRYGPISXMHIW EFWSPYXI UYERXM GEXMSR ERH . Oligonucleotides P=O/P=S ratio in a single ICP-MS/MS run Bill Marshall, ')3 MiRagen Therapeutics, USA Phillipe De Raeve, 7 GMIRXM G (MVIGXSV.)

10 Quality Assistance 14:45 Childhood Blindness due to a Photoreceptor 'SRXVSP ERH 5 YEPM GEXMSR SJ -QTYVMXMIW JSV . Cilium Defect Treated with an Intravitreal Antisense Oligonucleotides Oligonucleotide (QR-110) Nadim Akhtar, 4 VMRGMTEP 7 GMIRXMWX AstraZeneca Pete Adamson, 7V :4 3 TLXLEPQSPSK] . ProQR Therapeutics 15:15 Asymmetric siRNA Targeting Fibrotic and 15:15 Oligonucleotide Characterisation by NMR and Ocular Disorders Consideration of Orthogonal Techniques Sun Woo Hong, (MVIGXSV OliX Pharmaceuticals Thomas Brown, 7 IRMSV 7 GMIRXMWX 3 PMKSRYGPISXMHIW . Intertek Pharmaceutical Services, UK. 15:45 Afternoon Coffee and Networking 16:15 Spherical Necleic Acids and Their Use in 16:15 Evaluating the impact of sterilication on Treatment of Disease oligonucleotide based drug products David Giljohann, ')3 Exicure Erik van der Hage, PhD, (MVIGXSV %REP]XMGEP.)