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Landscape In… » Gene Therapy Companies « LANDSCAPE IN… » GENE THERAPY COMPANIES ©BioPharmAnalyses UPDATED MAY 2018 All material published in this study is protected by copyright. No text or part thereof may be reproduced in any way without prior written consent of the publisher. Publisher: BioPharmAnalyses 40, rue du petit bois, 78370 Plaisir France. Phone: 33 (0)686 683 220. Contact: [email protected]/ CONTENTS Companies Mentionned……………………………………………………..p3 Pathologies mentionned……………………………………………………..p6 Investors in Gene Therapy mentionned……………………………….......p9 (including, Federal or National Agencies) AAV-based Gene Therapy Companies…………………………………..p12 LV-based Gene Therapy Companies ……………………………………p13 RNA-based Gene Therapy Strategies …………………………………..p14 Contract, Development & Manufacturing Organization (CDMO)……...p15 Miscalleneous (including Hubs and Non-Viral Gene Transfer.………..p16 Detailed presentation of Companies……………………………………..p17 Companies Mentionned 4D Molecular Therapeutics Beat BioTherapeutics Abeona Therapeutics Benitec Biopharma Adverum Biotechnologies BioCancell Therapeutics Aevitas Therapeutics (see Fortress Biogen Biotech) BioMarin Pharmaceuticals Agilis Biotherapeutics BioNTech AG Applied Genetic Technologies Corp Bio-Path Holdings Akcea Therapeutics BioReliance Akouos Bioverativ Allergan Biovian Oy Alnylam Pharmaceuticals BioViva Amarna Therapeutics Bluebird Bio American Gene Technologies BrainVectis Amylon Therapeutics Brammer Bio AnGes Caribou Biosciences Angionetics Casebia Therapeutics Antisense Therapeutics Cell & Gene Therapy Catapult Apceth Biopharma CellGenix GmbH Apic Bio Celsion Applied Viromics CEPiA Atlantic Healthcare CEVEC Pharmaceuticals Arbutus BioPharma Clearside Biomedical Arcturus Therapeutics Cobra Biologics Arrowhead Pharmaceuticals Copernicus Therapeutics Arthrogen CRISPR Therapeutics Asklepios BioPharmaceutical CureVac Astellas Cyprium Therapeutics (see Fortress Audentes Therapeutics Biotech) AveXis Dicerna Pharmaceuticals AvroBio Dimension Therapeutics Batavia Biosciences Editas Medicine 4 Eloxx Pharmaceuticals Juventas Therapeutics Errant Gene Therapeutics Klogene Therapeutics ERS Genomics Esteve Laboratories Krystal Biotech (Laboratorios del Dr Esteve) Lentigen, a Miltenyi Biotec Company Excision BioTherapeutics Locus Biosciences Exicure LogicBio Therapeutics Expression Therapeutics Lonza Eyevensys Lysogene Fibrocell Science Marina Biotech Fight for Sight MaxCyte FinVector Vision Therapies Oy MeiraGTx Fortress Biotech Milo Biotechnology FUJIFILM Diosynth Biotechologies Mina Therapeutics GE Healthcare Miragen Gene Signal Moderna Therapeutics GeneCure MolMed Genenta Sciences Myonexus Generation Bio NanoCor Therapeutics Genethon NightstaRx Therapeutics Genezen Laboratories Novartis GenIBET Biopharmaceuticals Novasep GenSight Biologics Odylia Therapeutics Global BioTherapeutics Ophthotech GlyGenix Therapeutics Opko Health GlaxoSmithKline (see Orchard Orchard Therapeutics Therapeutics) Oxford Biomedica Herantis Pharma Oyj Oxford Genetics Homology Medicines Paragon Biosciences Horama PCI Biotech (see RXI Horizon Discovery Pharmaceuticals) Ichor Medical Systems Pfizer Idera Pharmaceuticals Phoenix Nest Immusoft Plasmid Factory Intellia Therapeutics Poseida Therapeutcs inteRNA Technologies Precision Biosciences Ionis Pharmaceuticals Prevail Therapeutics Isarna Therapeutics Preveceutical Medical iVeena Delivery Systems ProQR Therapeutics 5 PTC Therapeutics Synpromics Quark Pharmaceuticals System Bioscience RegenXBio Tamid Bio (see Fortress Biotech) Regulus Theragene Pharmaceuticals Renova Therapeutics Thera Neuropharma Rocket Pharmaceuticals Touchlight Genetics RXI Pharmaceuticals Translate Bio Sangamo Therapeutics Treeway Sanofi Ultragenyx Sarepta Therapeutics Uniqure Selecta Biosciences VBL Therapeutics Shire Vectalys Signagen Vertex Pharmaceuticals Silence Therapeutics Vibalogics Sirion Biotech Vigene Biosciences Sirnaomics Viromed/VM Biopharma Solid Biosciences Vive Biotech Somagenics Vivet Therapeuics Spark Therapeutics Voyager Therapeutics Stride Bio Wave Life Sciences Sylentis Yposkesi (see Genethon) 6 Pathologies mentionned Achondroplasia Carbapenem-Resistant Achromatopsia Enterobacteriaceae (CRE) infections Acromegaly Catecholaminergic polymorphic Acute angle closure glaucoma ventricular tachycardia Acute myeloid leukemia Cerebral adrenoleukodystrophy Age-related macular degeneration Chronic obstructive pulmonary disease (AMD) (COPD) Acute non-arteritic ischemic optic Chikungunya neuropathy (NAION) Choroidemia Adenosine deaminase severe Chronic granulomatous disease combined immunodeficiency (ADA- Chronic myeloid leukemia SCID) Chronic wound healing Adrenoleukodystrophy Citrullinemia type I Alpha-1 antitrypsin deficiency CLN2 disease/Batten’s disease Alpha-sarcoglycanopathy Clostridium difficile infection Alport’s disease Congestive heart failure Alzheimer’s disease Corneal graft rejection Amyotrophic lateral sclerosis (ALS) Corneal neovascularisation Aromatic L-amino acid decarboxylase Crigler-Najjar syndrome (AADC) deficiency Critical Limb Ischemia (CLI) Arthritis Crohn's disease Atherosclerosis Cunningham virus Atopic dermatitis Cystic Fibrosis ATTR amyloidosis Cystinosis Atypical hemolytic uremic syndrome Cytomegalovirus infection Becker muscular dystrophy Delayed graft function (DGF) Best disease following kidney transplantation Beta-sarcoglycanopathy Diabetic foot ulcer Beta thalassemia Diabetic macular oedema Biallelic RPE65-mediated inherited Diabetic peripheral neuropathy retinal disease Dry eye syndrome Bladder cancer Duchenne Muscular Disease Blood cancers including cutaneous T- Dystrophic Epidermolysis Bullosa cell lymphoma (DEB) Breast cancer Epidermolysis bullosa simplex Fabry disease 7 Familial adenomatous polyposis Homozygous familial Familial amyloid polyneuropathy hypercholesterolemia Familial chylomicronemia Human metapneumovirus Familial dysautonomia and parainfluenza virus type 3 infection Familial intrahepatic cholestasis Huntington’s disease Fanconi anemia Hypercholesterolemia Fibrotic conditions including Hypertension cutaneous fibrosis Hypertriglyceridemia Friedreich’s ataxia Hypertrophic scars Frontotemporal dementia Inclusion body myositis Frontotemporal lobar degeneration Infantile malignant osteopetrosis Gamma-sarcoglycanopathy Inflammatory bowel disease Gaucher disease Influenza Glaucoma Ischemia Glioblastoma Ischemic central retinal vein occlusion Globoid cell leukodystrophy Ischemic heart failure Glycogen storage disease Ia KRAS cancer Glycogen storage disease type III Lambert–Eaton myasthenic syndrome GM1 gangliosidosis Lamellar ichthyosis Graft vs host disease (Transglutaminase-1 deficient HBV infection autosomal recessive congenital Head and neck cancer ichthyosis) Head and neck squamous cell Late infantile neuronal ceroid carcinoma lipofuscinosis Heart failure Leber congenital amaurosis type 10 Hemoglobinopathy Leber hereditary optic neuropathy Hemophilia A (LHON) Hemophilia B Leukocyte adhesion deficiency-I Hepatitis B Limb-girdle muscular dystrophy Hereditary angioedema Lung cancer Hereditary cerebral hemorrhage Lymphoma Hereditary cerebral hemorrhage Macular degeneration with amyloidosis of the Dutch type Melanoma (HCHWA-D) Menkes disease Herpes simplex type 1 Metabolic syndrome Herpes simplex type 2 Metachromatic leukodystrophy HIV Methylmalonic acidemia Mucopolysaccharidosis type I 8 Mucopolysaccharidosis type II Propionic acidemia Mucopolysaccharidosis type III Prostate cancer (Sanfilippo syndrome) Psoriasis Mucopolysaccharidosis type IIIB Pulmonary hypertension Mucopolysaccharidosis Type IVA Pyruvate kinase deficiency Mucopolysaccahridosis Type VI Radiosensitive severe combined Multiple myeloma immunodeficiency Multiple sclerosis (RS-SCID Artemis gene mutation) Myocardial ischemia Renal cell carcinoma Myotubular myopathy Retinal scarring Neovascular diseases of the retina Retinal vein occlusion including AMD Retinitis pigmentosa Non alcoholic fatty liver disease Scleroderma (NAFLD) Severe combined immuno- deficiency Non alcoholic steatotic hepatitis (SCID) (NASH) Sickle cell disease Non infectious uveitis Spinal muscular atrophy (SMA) Non-small-cell lung carcinoma Stargardt’s disease Obesity Systemic atrophy Ocular allergies Tauopathies Oculopharyngeal muscular dystrophy Thyroid cancer Ornithine transcarbamylase (OTC) Transthyretin amyloidosis deficiency Type 1 diabetes Ovarian cancer Type 2 diabetes P. aeruginosa infection Ulcerative colitis Pain Usher syndrome IB Pancreatic cancer Usher syndrome IIa Parkinson's disease Wet age-related macular degeneration Paroxysmal nocturnal hemoglobinuria (Wet-AMD) Peripheral arterial disease Wilson’s disease Phenylketonuria Wiskott Aldrich syndrome Polyendocrinopathy X-linked syndrome Xerostomia Pompe disease X-linked myotubular myopathy Pouchitis X-linked retinoschisis Primary hyperoxaluria Zika virus 9 Investors in Gene Therapy mentionned (including, Federal or National Agencies) 600 Mile Challenge Fund (USA) Boulder Ventures (USA) 4BIO Capital (UK), Boxer Capital (Bahamas) 5AM Ventures (USA) BPi France (FR) Abingworth Bioventures (UK) Brace Pharma Capital (USA) Abstract Ventures (USA Brookside Capital (USA) Abu Dhabi Investment Authority (ADIA) BVF Partners (USA) Adage Capital Management (USA) Calculus Capital (UK) Agent Capital (USA) California Institute for Quantitative Aglaia Biomedical Ventures (NL) Biosciences (USA) Aisling Capital (USA) California Institute for Regenerative Alexandria Venture Investments (USA) Medicine (USA) Alexion Pharmaceuticals (USA Camden Partners (USA) Alumni Venture Group (USA) CapDecisif Management
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