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Company ReportsIonis Pharmaceuticals

Ionis Pharmaceuticals(IONS)

Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics. The company has 3 commercially approved medicines: Spinraza (Nusinersen), Tegsedi (Inotersen), and Waylivra (Volanesorsen) and has 4 drugs in pivotal studies: tominersen for Huntington’s disease, tofersen for SOD1-ALS, AKCEA-APO(a)-LRx for cardiovascular disease, and AKCEA-TTR-LRx for all forms of TTR amyloidosis.

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Phases:

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Drug Name

Phase 1

Phase 2

Phase 3

Phase 4

Indication

cardiovascular diseases

,

,

,

acute lung injury

,

Antithrombin iii

primary myelofibrosis

,

healthy volunteers/patients

,

pancreatic neoplasms

,

uterine cervical neoplasms

,

autistic disorder

,

autism spectrum disorder

,

pervasive child development disorders

,

,

central nervous system neoplasms

,

nervous system neoplasms

,

non-small-cell lung carcinoma

,

breast neoplasms

,

,

,

,

colorectal neoplasms

,

alzheimer disease

,

ovarian neoplasms

,

b-cell lymphoma

,

small cell lung carcinoma

,

,

,

,

stomach neoplasms

,

,

ulcerative colitis

,

,

,

macular degeneration

,

,

hepatocellular carcinoma

,

renal cell carcinoma

,

triple negative breast neoplasms

,

ovarian epithelial carcinoma

,

large b-cell lymphoma diffuse

,

follicular lymphoma

,

lupus nephritis

,

,

,

non-hodgkin lymphoma

,

wet macular degeneration

,

,

,

liver neoplasms

,

urinary bladder neoplasms

,

peritoneal neoplasms

,

colonic neoplasms

,

head and neck neoplasms

,

b-cell chronic lymphocytic leukemia

,

lymphoid leukemia

,

multiple sclerosis

,

,

paroxysmal hemoglobinuria

,

,

,

,

squamous cell carcinoma

,

fallopian tube neoplasms

,

transitional cell carcinoma

,

neoplasm metastasis

,

kidney diseases

,

chronic progressive multiple sclerosis

,

retinal vein occlusion

,

,

,

hemolytic anemia

,

hemolytic-uremic syndrome

,

atypical hemolytic uremic syndrome

,

,

systemic lupus erythematosus

,

relapsing-remitting multiple sclerosis

,

nephrotic syndrome

,

,

lipoid nephrosis

,

,

ischemic stroke

,

,

,

esophageal squamous cell carcinoma

,

esophageal neoplasms

,

squamous cell carcinoma of head and neck

,

human influenza

,

prostatic neoplasms

,

castration-resistant prostatic neoplasms

,

urologic neoplasms

,

thyroid neoplasms

,

neuroendocrine carcinoma

,

papillary thyroid cancer

,

thyroid diseases

,

disease progression

,

,

chronic hepatitis b

,

,

chronic hepatitis

,

,

,

giant cell arteritis

,

polymyalgia rheumatica

,

membranous glomerulonephritis

,

respiratory tract diseases

,

germ cell and embryonal neoplasms

,

,

respiratory tract neoplasms

,

bronchogenic carcinoma

,

thoracic neoplasms

,

bronchial diseases

,

neoplasms by histologic type

,

neoplasms by site

,

nerve tissue neoplasms

,

ankylosing spondylitis

,

spondylarthritis

,

,

,

,

huntington disease

,

geographic atrophy

,

,

myasthenia gravis

,

muscle weakness

,

neuromyelitis optica

,

guillain-barre syndrome

,

autoimmune diseases of the nervous system

,

hashimoto disease

,

,

partial epilepsies

,

rectal neoplasms

,

mucinous adenocarcinoma

,

ductal carcinoma breast

,

inflammatory breast neoplasms

,

bcr-abl positive chronic myelogenous leukemia

,

disability evaluation

,

cognition disorders

,

hemolytic anemia autoimmune

,

endometrial neoplasms

,

iga glomerulonephritis

,

non-muscle invasive bladder neoplasms

,

choroidal neovascularization

,

,

retinal neovascularization

,

pathologic neovascularization

,

,

diabetic nephropathies

,

myocardial infarction

,

hypersensitivity

,

,

,

renal tubular acidosis

,

,

,

fetal growth retardation

,

placenta diseases

breast neoplasms

,

atopic dermatitis

,

,

,

disease susceptibility

,

healthy volunteers/patients

,

chronic obstructive pulmonary disease

,

,

obstructive lung diseases

,

myelodysplastic syndromes

,

,

,

glomerulonephritis

,

membranoproliferative glomerulonephritis

,

beta-thalassemia

,

healthy volunteers/patients

,

hypertriglyceridemia

,

,

hyperlipoproteinemia type i

,

cardiovascular diseases

,

atherosclerosis

healthy volunteers/patients

,

hereditary angioedemas

,

huntington disease

Aglatimagene besadenovec

castration-resistant prostatic neoplasms

,

,

prostatic neoplasms

ulcerative colitis

,

,

,

amyotrophic lateral sclerosis

,

motor neuron disease

,

parkinson disease

,

,

acquired immunodeficiency syndrome

,

bacterial infections

,

,

chronic hepatitis b

,

,

chronic hepatitis

,

immunologic tests

myeloid leukemia acute

,

ovarian neoplasms

,

ovarian epithelial carcinoma

,

prostatic neoplasms

,

castration-resistant prostatic neoplasms

,

,

angelman syndrome

,

huntington disease

,

developmental disabilities

,

mental disorders

,

neurodevelopmental disorders

,

autistic disorder

,

autism spectrum disorder

,

pervasive child development disorders

,

non-small-cell lung carcinoma

,

,

muscular atrophy

,

spinal muscular atrophy

,

,

multiple myeloma

,

plasma cell neoplasms

,

follicular lymphoma

,

diabetes mellitus

,

non-hodgkin lymphoma

,

alzheimer disease

,

sickle cell anemia

,

,

type 2 diabetes mellitus

,

paroxysmal hemoglobinuria

,

,

,

obsessive-compulsive disorder

,

compulsive personality disorder

,

brain neoplasms

,

unknown primary neoplasms

,

stomach neoplasms

,

small cell lung carcinoma

,

,

breast neoplasms

,

,

colorectal neoplasms

,

,

triple negative breast neoplasms

,

,

large b-cell lymphoma diffuse

,

b-cell lymphoma

,

multiple sclerosis

,

myeloid leukemia

,

rheumatoid arthritis

,

,

chronic renal insufficiency

,

kidney diseases

,

,

,

bcr-abl positive chronic myelogenous leukemia

,

,

renal cell carcinoma

,

,

,

,

,

,

,

systemic scleroderma

,

diffuse scleroderma

,

,

,

cerebrovascular disorders

,

giant cell arteritis

,

polymyalgia rheumatica

,

mucinous adenocarcinoma

,

ductal carcinoma breast

,

inflammatory breast neoplasms

,

chronic hepatitis

,

,

,

,

,

chronic hepatitis c

,

neoplasm metastasis

,

renal insufficiency

,

chronic kidney failure

,

chronic hepatitis b

,

iron-deficiency anemia

,

lymphatic diseases

,

iron deficiencies

,

hepatocellular carcinoma

,

liver neoplasms

,

,

,

idiopathic pulmonary fibrosis

,

pulmonary fibrosis

,

interstitial lung diseases

,

,

,

acquired immunodeficiency syndrome

,

liver cirrhosis

,

spinal muscular atrophies of childhood

healthy volunteers/patients

,

diabetes mellitus

,

,

,

myelodysplastic syndromes

,

myeloid leukemia acute

,

myeloid leukemia

,

,

carcinoid tumor

,

,

malignant carcinoid syndrome

,

serotonin syndrome

,

pancreatic neoplasms

,

neuroendocrine tumors

,

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