Table of Contents List of Tables List of Figures Introduction Global Markets Direct Report Coverage Huntingtin (Huntington Disease Protein or HTT) - Overview Huntingtin (Huntington Disease Protein or HTT) - Therapeutics Development Products under Development by Stage of Development Products under Development by Therapy Area Products under Development by Indication Products under Development by Companies Products under Development by Universities/Institutes Huntingtin (Huntington Disease Protein or HTT) - Therapeutics Assessment Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Huntingtin (Huntington Disease Protein or HTT) - Companies Involved in Therapeutics Development AFFiRiS AG F. Hoffmann-La Roche Ltd Neurimmune Holding AG nLife Therapeutics SL PTC Therapeutics Inc reMYND NV Shire Plc UniQure NV Voyager Therapeutics Inc Vybion Inc WAVE Life Sciences Ltd Huntingtin (Huntington Disease Protein or HTT) - Drug Profiles AMT-130 - Drug Profile Product Description Mechanism Of Action R&D Progress Antisense Oligonucleotide to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile Product Description Mechanism Of Action R&D Progress Antisense Oligonucleotides to Inhibit HTT for Huntington's Disease - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy to Inhibit Huntingtin Protein for Huntington’s Disease - Drug Profile Product Description Mechanism Of Action R&D Progress INT-41 - Drug Profile Product Description Mechanism Of Action R&D Progress IONIS-HTTRx - Drug Profile Product Description Mechanism Of Action R&D Progress NI-302 - Drug Profile Product Description Mechanism Of Action R&D Progress NLF-HD XXXX - Drug Profile Product Description Mechanism Of Action R&D Progress P-301905 - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules to Inhibit Huntingtin Protein for Huntington’s Disease - Drug Profile Product Description Mechanism Of Action R&D Progress Synthetic Peptide to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile Product Description Mechanism Of Action R&D Progress Vaccine to Target Huntingtin for Huntington's Disease - Drug Profile Product Description Mechanism Of Action R&D Progress VYHTT-01 - Drug Profile Product Description Mechanism Of Action R&D Progress WVE-120101 - Drug Profile Product Description Mechanism Of Action R&D Progress WVE-120102 - Drug Profile Product Description Mechanism Of Action R&D Progress Huntingtin (Huntington Disease Protein or HTT) - Dormant Products Huntingtin (Huntington Disease Protein or HTT) - Product Development Milestones Featured News & Press Releases Dec 13, 2017: New drug could halt progression of Huntingtons disease Oct 18, 2017: uniQure Presents New Preclinical Data on AMT-130 in Huntington’s Disease at the ESGCT 25th Anniversary Congress in Berlin Oct 06, 2017: UniQure Announces FDA Orphan Drug Designation for AMT-130 in Huntington’s disease Jul 17, 2017: Wave Life Sciences Initiates Phase 1b/2a Clinical Trial: PRECISION-HD1 in Patients with Huntington's Disease Jul 17, 2017: Wave Life Sciences Announces Initiation of Phase 1b/2a Clinical Trial: PRECISION-HD2 in Patients with Huntington's Disease Jun 22, 2017: Enrollment in Phase 1/2a Study of IONIS-HTT Rx in Patients with Huntingtons Disease Completed and Open-Label Extension Study to Open in 2H 2017 Jun 01, 2017: Voyager Therapeutics Selects Lead Clinical Candidate for Huntington's Disease Apr 26, 2017: uniQure Presents New Preclinical Data on AMT-130 in Huntington's Disease at CHDI's 12th Annual Huntington's Disease Therapeutics Conference Apr 24, 2017: uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy Apr 04, 2017: uniQure Publishes Data Demonstrating Wide Distribution of AAV5 in the Central Nervous System Jan 30, 2017: Vybion Huntington's Disease Drug Patent receives Track I Status Jan 06, 2017: WAVE Life Sciences Provides Update on Lead Huntington’s Disease Program WVE-120101 Jan 06, 2017: WAVE Life Sciences Provides Update on Lead Huntington’s Disease Program WVE-120102 Aug 24, 2016: Novel Gene Therapy Drug for Huntington Disease Jun 21, 2016: WAVE Life Sciences Receives Orphan Drug Designation from FDA for its Lead Candidate Designed to Treat Huntington’s Disease Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer

List of Tables Number of Products under Development by Stage of Development, H1 2018 Number of Products under Development by Therapy Areas, H1 2018 Number of Products under Development by Indication, H1 2018 Number of Products under Development by Companies, H1 2018 Products under Development by Companies, H1 2018 Number of Products under Investigation by Universities/Institutes, H1 2018 Products under Investigation by Universities/Institutes, H1 2018 Number of Products by Stage and Mechanism of Actions, H1 2018 Number of Products by Stage and Route of Administration, H1 2018 Number of Products by Stage and Molecule Type, H1 2018 Pipeline by AFFiRiS AG, H1 2018 Pipeline by F. Hoffmann-La Roche Ltd, H1 2018 Pipeline by Neurimmune Holding AG, H1 2018 Pipeline by nLife Therapeutics SL, H1 2018 Pipeline by PTC Therapeutics Inc, H1 2018 Pipeline by reMYND NV, H1 2018 Pipeline by Shire Plc, H1 2018 Pipeline by UniQure NV, H1 2018 Pipeline by Voyager Therapeutics Inc, H1 2018 Pipeline by Vybion Inc, H1 2018 Pipeline by WAVE Life Sciences Ltd, H1 2018 Dormant Projects, H1 2018List of Figures Number of Products under Development by Stage of Development, H1 2018 Number of Products under Development by Therapy Areas, H1 2018 Number of Products under Development by Indications, H1 2018 Number of Products by Mechanism of Actions, H1 2018 Number of Products by Stage and Mechanism of Actions, H1 2018 Number of Products by Routes of Administration, H1 2018 Number of Products by Stage and Routes of Administration, H1 2018 Number of Products by Molecule Types, H1 2018 Number of Products by Stage and Molecule Types, H1 2018