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Spark 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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Therapeutic Areas:
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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
Bevifimod
healthy volunteers/patients
,
hepatic insufficiency
,
esophageal neoplasms
,
hiv infections
,
neuroblastoma
,
small cell lung carcinoma
,
pancreatic ductal carcinoma
,
multiple myeloma
,
plasma cell neoplasms
,
bronchiolitis obliterans syndrome
,
congenital abnormalities
,
chromosome aberrations
,
brain stem neoplasms
,
chronic hepatitis c
,
chronic hepatitis
,
typhoid fever
,
infertility
,
medulloblastoma
,
rhabdoid tumor
,
ependymoma
,
choroid plexus neoplasms
,
primitive neuroectodermal tumors
,
ganglioglioma
,
pinealoma
,
neuroepithelial neoplasms
,
ganglioneuroblastoma
,
neuroectodermal tumors
,
phenylketonurias
,
precursor t-cell lymphoblastic leukemia-lymphoma
,
nasopharyngeal carcinoma
,
ureteral neoplasms
,
pelvic neoplasms
,
adenoid cystic carcinoma
,
metabolic syndrome
,
follicular lymphoma
,
adenocarcinoma
,
pancreatic neoplasms
,
kidney neoplasms
,
recurrence
,
prostatic neoplasms
,
syndrome
,
glioma
,
head and neck neoplasms
,
neurofibromatoses
,
neurofibroma
,
liver neoplasms
,
sarcoma
,
liver diseases
,
neurofibromatosis 1
,
plexiform neurofibroma
,
systemic scleroderma
,
diffuse scleroderma
,
b-cell chronic lymphocytic leukemia
,
meningioma
,
hematologic neoplasms
,
endometrial neoplasms
,
neuroendocrine carcinoma
,
central nervous system neoplasms
,
ovarian neoplasms
,
mantle-cell lymphoma
,
urinary bladder neoplasms
,
squamous cell carcinoma
,
uterine cervical neoplasms
,
myelodysplastic syndromes
,
non-hodgkin lymphoma
,
nervous system neoplasms
,
hypereosinophilic syndrome
,
systemic mastocytosis
,
mastocytosis
,
uveal neoplasms
,
squamous cell carcinoma of head and neck
,
t-cell lymphoma
,
transitional cell carcinoma
,
primary myelofibrosis
,
metaplasia
,
graft vs host disease
,
adenocarcinoma of lung
,
diffuse intrinsic pontine glioma
,
disease progression
,
oligodendroglioma
,
neurofibromatosis 2
,
acoustic neuroma
,
neurilemmoma
,
hodgkin disease
,
islet cell carcinoma
,
hematologic diseases
,
myeloid leukemia accelerated phase
,
preleukemia
,
mast-cell leukemia
,
aggression
,
germ cell and embryonal neoplasms
,
tuberculosis
,
anaplastic thyroid carcinoma
,
t-cell lymphoma peripheral
,
waldenstrom macroglobulinemia
,
castration-resistant prostatic neoplasms
,
gliosarcoma
,
cholangiocarcinoma
,
chronic disease
,
large-cell lymphoma anaplastic
,
plasma cell granuloma
,
diarrhea
,
skin neoplasms
,
neuroma
,
postmenopause
,
gouty arthritis
,
endocrine gland neoplasms
,
virus diseases
,
urogenital neoplasms
,
male genital diseases
,
prostatic diseases
,
abdominal neoplasms
,
male genital neoplasms
,
genital diseases
,
sjogren's syndrome
,
retinitis pigmentosa
,
retinitis
,
synovitis
,
giant cell tumors
,
pigmented villonodular synovitis
,
giant cell tumor of tendon sheath
,
choroidal neovascularization
,
pathologic neovascularization
,
hemangioblastoma
,
von hippel-lindau disease
,
inflammatory breast neoplasms
,
invasive hydatidiform mole
,
multidrug-resistant tuberculosis
,
local neoplasm recurrence
,
t-cell lymphoma cutaneous
,
peutz-jeghers syndrome
,
acute coronary syndrome
,
st elevation myocardial infarction
,
malignant mesothelioma
,
mesothelioma
,
type 1 diabetes mellitus
,
hypoglycemia
,
intestinal neoplasms
,
testicular neoplasms
,
osteosarcoma
,
brain stem infarctions
,
epilepsy
,
sturge-weber syndrome
,
klippel-trenaunay-weber syndrome
,
neurofibrosarcoma
,
nerve sheath neoplasms
,
carcinoma in situ
,
breast diseases
,
multiple hamartoma syndrome
,
hamartoma
,
urologic neoplasms
,
fallopian tube neoplasms
,
t-cell leukemia
,
localized scleroderma
,
myelomonocytic leukemia chronic
,
myelomonocytic leukemia acute
,
myelomonocytic leukemia juvenile
,
ameloblastoma
,
adamantinoma
,
ovarian epithelial carcinoma
,
sickle cell anemia
,
streptococcus agalactiae
,
colorectal neoplasms
,
myeloid leukemia acute
,
lymphoma
,
glioblastoma
,
neoplasm metastasis
,
brain neoplasms
,
hepatocellular carcinoma
,
astrocytoma
,
thyroid neoplasms
,
thyroid diseases
,
chronic urticaria
,
urticaria
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
blast crisis
,
triple negative breast neoplasms
,
hypertension
,
colonic neoplasms
,
arthritis
,
carcinoid tumor
,
pulmonary arterial hypertension
,
familial primary pulmonary hypertension
,
fibrosis
,
gastrointestinal neoplasms
,
stomach neoplasms
,
rectal neoplasms
,
meningitis
,
meningococcal meningitis
,
meningococcal infections
,
rheumatoid arthritis
,
organ transplantation
,
graft rejection
,
digestive system neoplasms
,
cutaneous malignant melanoma
,
stomatitis
,
lymphangioleiomyomatosis
,
lupus nephritis
,
nephritis
,
hypersensitivity
,
peanut hypersensitivity
,
multiple sclerosis
,
relapsing-remitting multiple sclerosis
,
interstitial lung diseases
,
seizures
,
lung transplantation
,
malignant carcinoid syndrome
,
serotonin syndrome
,
food hypersensitivity
,
psoriatic arthritis
,
psoriasis
,
muscular atrophy
,
spinal muscular atrophy
,
atrophy
,
mucositis
,
lung diseases
,
idiopathic pulmonary fibrosis
,
pulmonary fibrosis
,
covid-19
,
chronic inducible urticaria
,
infectious skin diseases
,
bacterial skin diseases
,
skin diseases
,
leukemia
,
myeloid leukemia
,
neoplasms
,
bcr-abl positive chronic myelogenous leukemia
,
breast neoplasms
,
carcinoma
,
lung neoplasms
,
non-small-cell lung carcinoma
,
renal cell carcinoma
,
melanoma
,
kidney transplantation
,
philadelphia chromosome
,
lymphoid leukemia
,
neuroendocrine tumors
,
gastrointestinal stromal tumors
,
precursor cell lymphoblastic leukemia-lymphoma
,
sclerosis
,
myeloid leukemia chronic-phase
,
liver transplantation
,
renal insufficiency
,
tuberous sclerosis
,
chronic kidney failure
,
heart transplantation
,
kidney diseases
,
islet cell adenoma
,
infections
,
angiomyolipoma
,
macular degeneration
,
hepatitis c
,
hepatitis
,
hepatitis a
,
autosomal dominant polycystic kidney
,
communicable diseases
,
polycystic kidney diseases
,
arthrogryposis
,
diabetes mellitus
,
tick-borne encephalitis
,
encephalitis
,
immunosuppression therapy
,
cytomegalovirus infections
,
rare diseases
,
large cell carcinoma
,
vascular diseases
,
macular edema
,
edema
,
vision disorders
,
low vision
,
type 2 diabetes mellitus
,
cross-sectional studies
,
viremia
,
polyomavirus infections
,
cryopyrin-associated periodic syndromes
,
polycythemia vera
,
polycythemia
,
heart failure
,
systolic heart failure
,
myocardial infarction
,
inflammation
,
infarction
,
inferior wall myocardial infarction
Coagulation factor ix human
hemophilia a
,
hemophilia b
Demand
chronic urticaria
,
urticaria
,
sickle cell anemia
,
arthritis
,
gouty arthritis
,
gout
,
macular degeneration
Pcc
healthy volunteers/patients
,
post-acute covid-19 syndrome
,
transfusion reaction
,
thromboembolism
,
hip fractures
,
bone fractures
,
disease
,
esophageal neoplasms
,
carcinoma
,
squamous cell carcinoma of head and neck
,
squamous cell carcinoma
,
blood transfusion
,
postoperative complications
,
congenital heart defects
,
acute lung injury
,
lung injury
,
transfusion-related acute lung injury
,
venous thromboembolism
,
embolism
,
embolism and thrombosis
,
human influenza
,
transsexualism
,
fatigue
,
heart diseases
,
hemorrhagic shock
,
substance-related disorders
,
coagulation protein disorders
,
ovarian neoplasms
,
ovarian epithelial carcinoma
,
thrombocytopenia
,
hematologic diseases
,
hemorrhagic disorders
,
heart failure
,
traumatic brain injuries
,
cardiotoxicity
,
dental plaque
,
hemophilia a
,
hemophilia b
,
hemorrhage
,
blood coagulation disorders
,
hemostatic disorders
,
hiv infections
,
intracranial hemorrhages
,
wounds and injuries
,
thrombosis
,
cerebral hemorrhage
,
stroke
,
liver cirrhosis
,
surgical blood loss
,
cerebrovascular disorders
,
postpartum hemorrhage
,
poisoning
,
fibrosis
,
cardiac surgical procedures
,
cardiovascular surgical procedures
,
traumatic brain hemorrhage
,
pheochromocytoma
,
knee osteoarthritis
,
prostatic neoplasms
,
hip osteoarthritis
,
spinal dysraphism
,
heart valve diseases
,
liver diseases
,
cerebral intraventricular hemorrhage
,
depression
,
covid-19
,
hypersensitivity
,
hypertension
,
cardiovascular diseases
,
maternal death
,
depressive disorder
,
breast neoplasms
,
tuberculosis
,
hepatitis c
,
hepatitis
,
hepatitis a
,
post-traumatic stress disorders
,
dyslipidemias
,
obesity
,
postoperative pain
,
factor vii deficiency
,
cardiopulmonary bypass
,
diabetes mellitus
,
vascular diseases
,
central nervous system diseases
,
brain diseases
,
nervous system diseases
,
lung transplantation
,
primary graft dysfunction
,
disseminated intravascular coagulation
,
afibrinogenemia
,
traumatic intracranial hemorrhage
,
chronic disease
,
gastrointestinal diseases
,
endocrine system diseases
,
digestive system diseases
,
gonorrhea
,
chlamydia infections
,
emergencies
,
obstetric labor complications
,
chronic pain
,
adenoma
,
psychological stress
,
hearing loss
,
deafness
,
colorectal neoplasms
,
uterine cervical neoplasms
,
perioperative care
,
palliative care
,
dementia
,
osteoporosis
,
respiratory tract diseases
,
respiration disorders
,
chronic fatigue syndrome
,
encephalomyelitis
,
multidrug-resistant tuberculosis
,
coronary artery disease
,
myocardial ischemia
,
chagas disease
,
hiv
,
hepatitis b
,
schistosomiasis
,
strongyloidiasis
,
infections
,
pneumonia
,
sinusitis
,
communicable diseases
,
infectious skin diseases
,
cellulitis
,
parent-child relations
,
adolescent development
,
psychopathology
,
anxiety
,
traumatic stress disorders
,
feasibility studies
,
patient acceptance of health care
,
injection site reaction
,
graves disease
,
syndrome
,
neoplasms
,
oncogenes
,
hypertensive intracranial hemorrhage
,
sleep initiation and maintenance disorders
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