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Company ReportsSpark Therapeutics

Spark Therapeutics(ONCE)

Spark Therapeutics, Inc. is a developer of gene therapy treatments, which treat debilitating genetic diseases. It is a subsidiary of Hoffmann-La Roche.

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Marketed Drugs

Therapeutic Areas:

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Classification:

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Include Generics:

Trade Name	Drug Name	Approved	Patent Expires	Indication
Luxturna	Voretigene neparvovec	2017-12-19		Retinitis pigmentosa , Leber congenital amaurosis

Clinical Trials

Therapeutic Areas:

All

Classification:

All

Approval:

All

Status:

All

Phases:

All

Drug Name

Phase 1

Phase 2

Phase 3

Phase 4

Indication

Voretigene neparvovec

retinal dystrophies

healthy volunteers/patients

,

uveal neoplasms

,

cutaneous malignant melanoma

,

,

huntington disease

,

,

urologic neoplasms

,

plasma cell neoplasms

,

chronic hepatitis c

,

mantle-cell lymphoma

,

biliary tract neoplasms

,

esophageal neoplasms

,

transitional cell carcinoma

,

choroidal neovascularization

,

,

acquired immunodeficiency syndrome

,

,

graft vs host disease

,

central nervous system neoplasms

,

unknown primary neoplasms

,

genomic instability

,

lobular carcinoma

,

invasive hydatidiform mole

,

,

coronary artery disease

,

myocardial ischemia

,

,

subarachnoid hemorrhage

,

,

aids-related complex

,

,

esophageal squamous cell carcinoma

,

idiopathic pulmonary fibrosis

,

cholangiocarcinoma

,

,

pulmonary emphysema

,

margins of excision

,

waldenstrom macroglobulinemia

,

aids-related lymphoma

,

hemolytic-uremic syndrome

,

thrombotic thrombocytopenic purpura

,

,

thrombocytopenic purpura

,

,

,

hereditary sensory and autonomic neuropathies

,

b-cell lymphoma marginal zone

,

uterine neoplasms

,

female genital neoplasms

,

sjogren's syndrome

,

nervous system neoplasms

,

burkitt lymphoma

,

precursor b-cell lymphoblastic leukemia-lymphoma

,

sex cord-gonadal stromal tumors

,

erythema multiforme

,

immunoblastic lymphadenopathy

,

,

,

,

large b-cell lymphoma diffuse

,

b-cell lymphoma

,

liver neoplasms

,

hepatocellular carcinoma

,

triple negative breast neoplasms

,

renal cell carcinoma

,

follicular lymphoma

,

rectal neoplasms

,

neoplasm metastasis

,

macular degeneration

,

,

,

,

,

multiple myeloma

,

,

pancreatic neoplasms

,

,

hematologic neoplasms

,

inflammatory breast neoplasms

,

,

type 2 diabetes mellitus

,

neoplasms by histologic type

,

neoplasms by site

,

brain neoplasms

,

colonic neoplasms

,

uterine cervical neoplasms

,

ulcerative colitis

,

head and neck neoplasms

,

thrombocytopenic purpura idiopathic

,

relapsing-remitting multiple sclerosis

,

,

,

mucinous adenocarcinoma

,

ductal carcinoma breast

,

urinary bladder neoplasms

,

lymphoproliferative disorders

,

ovarian epithelial carcinoma

,

malignant mesothelioma

,

diabetic retinopathy

,

bronchiolitis obliterans syndrome

,

,

,

,

circulating neoplastic cells

,

retinal diseases

,

vision disorders

,

,

bcr-abl positive chronic myelogenous leukemia

,

,

respiratory tract diseases

,

germ cell and embryonal neoplasms

,

small cell lung carcinoma

,

,

respiratory tract neoplasms

,

bronchogenic carcinoma

,

thoracic neoplasms

,

bronchial diseases

,

nerve tissue neoplasms

,

myeloid leukemia acute

,

guillain-barre syndrome

,

patient preference

,

large-cell lymphoma anaplastic

,

anaplastic thyroid carcinoma

,

papillary thyroid cancer

,

laryngeal neoplasms

,

erdheim-chester disease

,

prostatic neoplasms

,

salivary gland neoplasms

,

,

gallbladder neoplasms

,

diabetes mellitus

,

anti-neutrophil cytoplasmic antibody-associated vasculitis

,

membranous glomerulonephritis

,

,

disability evaluation

,

cognition disorders

,

hemolytic anemia autoimmune

,

nephrotic syndrome

,

focal segmental glomerulosclerosis

,

lipoid nephrosis

,

graft rejection

,

retinal neovascularization

,

type 3 von willebrand disease

,

breast neoplasms

,

colorectal neoplasms

,

non-small-cell lung carcinoma

,

,

rheumatoid arthritis

,

non-hodgkin lymphoma

,

b-cell chronic lymphocytic leukemia

,

,

,

ovarian neoplasms

,

,

stomach neoplasms

,

lymphoid leukemia

,

kidney transplantation

,

chronic hepatitis b

,

heart transplantation

,

chronic kidney failure

,

fallopian tube neoplasms

,

liver transplantation

,

kidney diseases

,

chronic renal insufficiency

,

granulomatosis with polyangiitis

,

renal insufficiency

,

peritoneal neoplasms

,

pulmonary fibrosis

,

microscopic polyangiitis

,

,

transplantation

,

immunosuppression therapy

,

,

polyomavirus infections

,

,

food hypersensitivity

,

liver cirrhosis

,

adenocarcinoma of lung

,

,

,

,

,

retinal vein occlusion

,

,

acute kidney injury

,

,

endometrial neoplasms

,

uterine hemorrhage

,

,

hematopoietic stem cell transplantation

,

,

spinal muscular atrophy

,

spinal muscular atrophies of childhood

,

alzheimer disease

,

frontotemporal dementia

,

progressive supranuclear palsy

,

pick disease of the brain

,

hodgkin disease

Globulin, immune

healthy volunteers/patients

,

myeloid leukemia

,

head and neck neoplasms

,

myelodysplastic syndromes

,

,

uveal neoplasms

,

cutaneous malignant melanoma

,

,

endometrial neoplasms

,

,

,

multiple myeloma

,

mantle-cell lymphoma

,

,

esophageal neoplasms

,

,

,

biliary tract neoplasms

,

plasma cell neoplasms

,

choroidal neovascularization

,

,

waldenstrom macroglobulinemia

,

b-cell lymphoma marginal zone

,

aids-related lymphoma

,

bronchiolitis obliterans syndrome

,

unknown primary neoplasms

,

genomic instability

,

type 1 diabetes mellitus

,

cholangiocarcinoma

,

meningeal carcinomatosis

,

coronary disease

,

coronary artery disease

,

myocardial ischemia

,

,

vascular graft occlusion

,

margins of excision

,

,

hemolytic-uremic syndrome

,

thrombotic thrombocytopenic purpura

,

thrombocytopenic purpura

,

,

,

behcet syndrome

,

extranodal nk-t-cell lymphoma

,

hereditary sensory and autonomic neuropathies

,

graft vs host disease

,

castleman disease

,

,

systemic lupus erythematosus

,

sjogren's syndrome

,

diabetic nephropathies

,

,

renal hypertension

,

diabetes mellitus

,

nasopharyngeal carcinoma

,

nasopharyngeal neoplasms

,

central nervous system neoplasms

,

nervous system neoplasms

,

pathologic neovascularization

,

burkitt lymphoma

,

precursor b-cell lymphoblastic leukemia-lymphoma

,

sex cord-gonadal stromal tumors

,

interstitial lung diseases

,

erythema multiforme

,

reactive arthritis

,

immunoblastic lymphadenopathy

,

,

myasthenia gravis

,

neuroendocrine carcinoma

,

,

large b-cell lymphoma diffuse

,

b-cell lymphoma

,

liver neoplasms

,

follicular lymphoma

,

hepatocellular carcinoma

,

rectal neoplasms

,

triple negative breast neoplasms

,

,

renal cell carcinoma

,

neoplasm metastasis

,

macular degeneration

,

,

,

pancreatic neoplasms

,

,

colonic neoplasms

,

,

small cell lung carcinoma

,

ovarian epithelial carcinoma

,

urinary bladder neoplasms

,

,

esophageal squamous cell carcinoma

,

uterine cervical neoplasms

,

urologic neoplasms

,

transitional cell carcinoma

,

inflammatory breast neoplasms

,

ulcerative colitis

,

anti-neutrophil cytoplasmic antibody-associated vasculitis

,

brain neoplasms

,

myeloid leukemia acute

,

prostatic neoplasms

,

thrombocytopenic purpura idiopathic

,

,

circulating neoplastic cells

,

malignant mesothelioma

,

diabetic retinopathy

,

multiple sclerosis

,

mucinous adenocarcinoma

,

ductal carcinoma breast

,

hematologic neoplasms

,

,

,

iga glomerulonephritis

,

squamous cell carcinoma of head and neck

,

squamous cell carcinoma

,

castration-resistant prostatic neoplasms

,

retinal diseases

,

vision disorders

,

,

,

chronic progressive multiple sclerosis

,

relapsing-remitting multiple sclerosis

,

neuromyelitis optica

,

myelin oligodendrocyte glycoprotein antibody-associated disease

,

guillain-barre syndrome

,

patient preference

,

lymphoproliferative disorders

,

neoplasms by histologic type

,

neoplasms by site

,

salivary gland neoplasms

,

,

gallbladder neoplasms

,

,

systemic vasculitis

,

disability evaluation

,

cognition disorders

,

,

hemolytic anemia autoimmune

,

malignant pleural effusion

,

nephrotic syndrome

,

focal segmental glomerulosclerosis

,

lipoid nephrosis

,

ankylosing spondylitis

,

retinal neovascularization

,

membranous glomerulonephritis

,

breast neoplasms

,

colorectal neoplasms

,

non-small-cell lung carcinoma

,

rheumatoid arthritis

,

,

non-hodgkin lymphoma

,

b-cell chronic lymphocytic leukemia

,

,

lymphoid leukemia

,

ovarian neoplasms

,

stomach neoplasms

,

kidney transplantation

,

chronic kidney failure

,

granulomatosis with polyangiitis

,

,

microscopic polyangiitis

,

fallopian tube neoplasms

,

heart transplantation

,

kidney diseases

,

peritoneal neoplasms

,

pulmonary fibrosis

,

immunosuppression therapy

,

graft rejection

,

,

transplantation

,

renal insufficiency

,

food hypersensitivity

,

,

,

,

,

adenocarcinoma of lung

,

,

retinal vein occlusion

,

,

antiphospholipid syndrome

,

,

,

severe acute respiratory syndrome

,

communicable diseases

,

,

,

acquired immunodeficiency syndrome

,

neoadjuvant therapy

,

molecular targeted therapy

,

liver cirrhosis

,

atopic dermatitis

erectile dysfunction

,

physiological sexual dysfunction

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