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Disease Reports

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

Clinical Trials

Therapeutic Areas:

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Status:

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Phases:

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Drug Name

Phase 1

Phase 2

Phase 3

Phase 4

Indication

Voretigene neparvovec

leber congenital amaurosis

,

retinal dystrophies

,

,

retinitis pigmentosa

healthy volunteers/patients

,

central nervous system neoplasms

,

uterine cervical neoplasms

,

pancreatic ductal carcinoma

,

cutaneous malignant melanoma

,

ureteral neoplasms

,

,

hepatic insufficiency

,

chronic kidney failure

,

renal insufficiency

,

sezary syndrome

,

mycosis fungoides

,

t-cell lymphoma cutaneous

,

cholangiocarcinoma

,

large b-cell lymphoma diffuse

,

,

,

prostatic neoplasms

,

endometrial neoplasms

,

squamous cell carcinoma of head and neck

,

,

,

renal cell carcinoma

,

,

small cell carcinoma

,

mantle-cell lymphoma

,

,

medulloblastoma

,

squamous cell carcinoma

,

squamous cell neoplasms

,

uterine neoplasms

,

biliary tract neoplasms

,

esophageal neoplasms

,

b-cell lymphoma marginal zone

,

triple negative breast neoplasms

,

ovarian epithelial carcinoma

,

malignant mesothelioma

,

brain neoplasms

,

laryngeal diseases

,

,

,

,

,

,

rhabdomyosarcoma

,

female genital neoplasms

,

carcinoid tumor

,

,

follicular lymphoma

,

waldenstrom macroglobulinemia

,

castration-resistant prostatic neoplasms

,

adenocarcinoma of lung

,

bronchiolo-alveolar adenocarcinoma

,

malignant pleural effusion

,

thyroid diseases

,

multiple myeloma

,

mucinous adenocarcinoma

,

,

synovial sarcoma

,

,

diffuse intrinsic pontine glioma

,

desmoplastic small round cell tumor

,

urogenital neoplasms

,

cervical intraepithelial neoplasia

,

uterine cervical dysplasia

,

hydatidiform mole

,

choriocarcinoma

,

trophoblastic neoplasms

,

male breast neoplasms

,

ductal carcinoma

,

pleural neoplasms

,

,

neuroendocrine tumors

,

islet cell carcinoma

,

,

,

hypercholesterolemia

,

interdigitating dendritic cell sarcoma

,

gaucher disease

,

,

thymus neoplasms

,

oropharyngeal neoplasms

,

adolescent development

,

dental implants

,

,

,

esophageal squamous cell carcinoma

,

binge-eating disorder

,

reactive arthritis

,

,

breast neoplasms

,

colorectal neoplasms

,

,

neoplasm metastasis

,

ovarian neoplasms

,

,

head and neck neoplasms

,

stomach neoplasms

,

pancreatic neoplasms

,

rectal neoplasms

,

liver neoplasms

,

colonic neoplasms

,

small cell lung carcinoma

,

urinary bladder neoplasms

,

hepatocellular carcinoma

,

non-hodgkin lymphoma

,

fallopian tube neoplasms

,

,

,

b-cell chronic lymphocytic leukemia

,

peritoneal neoplasms

,

ischemic stroke

,

,

chronic renal insufficiency

,

urologic neoplasms

,

,

neoplasms by site

,

gastrointestinal neoplasms

,

,

glucose metabolism disorders

,

endocrine system diseases

,

,

urethral neoplasms

,

transitional cell carcinoma

,

ankylosing spondylitis

,

non-alcoholic fatty liver disease

,

,

,

,

pituitary dwarfism

,

cardiovascular diseases

,

,

digestive system neoplasms

,

b-cell lymphoma

,

lymphoid leukemia

,

hodgkin disease

,

,

carotid stenosis

,

pathologic constriction

,

juvenile arthritis

,

,

spondylarthritis

,

,

spinal fractures

,

myelodysplastic syndromes

,

myeloid leukemia acute

,

congenital abnormalities

,

megakaryoblastic leukemia acute

,

monocytic leukemia acute

,

myelomonocytic leukemia acute

,

refractory anemia with excess of blasts

,

erythroblastic leukemia acute

,

,

nutrition disorders

,

obstructive sleep apnea

,

sleep apnea syndromes

,

,

rectal diseases

,

gastrointestinal diseases

,

intestinal neoplasms

,

digestive system diseases

,

colonic diseases

,

intestinal diseases

,

prediabetic state

,

,

intracranial hemorrhages

,

,

,

hematologic diseases

,

femoral fractures

,

unknown primary neoplasms

,

respiratory tract diseases

,

,

respiratory tract neoplasms

,

bronchogenic carcinoma

,

thoracic neoplasms

,

bronchial neoplasms

,

neoplastic processes

,

pathologic processes

,

ulcerative colitis

,

type 2 diabetes mellitus

,

non-small-cell lung carcinoma

,

diabetes mellitus

,

type 1 diabetes mellitus

,

,

,

,

postmenopausal osteoporosis

,

insulin resistance

,

rheumatoid arthritis

,

,

myocardial infarction

,

diabetic neuropathies

,

psoriatic arthritis

,

metabolic diseases

,

,

,

,

metabolic syndrome

,

knee osteoarthritis

,

metabolic bone diseases

,

,

,

gestational diabetes

,

inferior wall myocardial infarction

,

,

,

short bowel syndrome

,

,

coronary artery disease

,

glucose intolerance

,

,

osteogenesis imperfecta

,

,

,

pseudotumor cerebri

,

hypopituitarism

,

,

hyperlipidemias

,

memory disorders

,

cognitive dysfunction

Globulin, immune

,

multiple myeloma

,

plasma cell neoplasms

,

non-small-cell lung carcinoma

,

knee osteoarthritis

,

,

hidradenitis suppurativa

,

,

,

,

,

,

renal cell carcinoma

,

neoplasm metastasis

,

,

respiratory syncytial virus infections

,

psoriatic arthritis

,

juvenile arthritis

,

,

ulcerative colitis

,

,

,

,

congenital heart defects

,

premature birth

,

bronchopulmonary dysplasia

,

inflammatory bowel diseases

,

,

acquired immunodeficiency syndrome

,

,

,

giant cell arteritis

,

behcet syndrome

,

interstitial cystitis

,

rheumatoid arthritis

,

,

,

,

ankylosing spondylitis

,

spondylarthritis

,

coronary artery disease

,

obstructive sleep apnea

,

sleep apnea syndromes

,

,

cardiovascular diseases

,

,

,

respiratory tract infections

,

communicable diseases

,

,

respiratory syncytial viruses

,

,

cognitive dysfunction

,

type 1 diabetes mellitus

,

parkinson disease

,

autism spectrum disorder

,

substance-related disorders

,

,

,

,

,

,

,

pulmonary edema

,

,

,

cardiogenic shock

,

cluster headache

,

,

,

pulmonary arterial hypertension

,

familial primary pulmonary hypertension

,

,

,

hyperbilirubinemia

,

triple negative breast neoplasms

,

otitis media with effusion

,

extracorporeal membrane oxygenation

,

transfusion reaction

,

,

mental disorders

,

,

alzheimer disease

,

,

,

,

cigarette smoking

,

,

neoplasm metastasis

,

memory disorders

,

ocular motility disorders

,

critical illness

,

tobacco use disorder

,

,

marijuana abuse

,

acute kidney injury

,

kidney neoplasms

,

,

cholangiocarcinoma

,

,

,

primary progressive aphasia

,

cerebrovascular disorders

,

esophageal and gastric varices

,

nutrition disorders

,

infratentorial neoplasms

,

video-assisted thoracic surgery

,

antineoplastic combined chemotherapy protocols

,

,

,

keratoconjunctivitis

,

,

,

psychological sexual dysfunctions

,

,

,

oropharyngeal neoplasms

,

marijuana smoking

,

,

,

,

,

,

,

,

anxiety disorders

,

,

,

,

chronic obstructive pulmonary disease

,

,

,

,

type 2 diabetes mellitus

,

,

smoking cessation

,

,

blood coagulation disorders

,

obstructive lung diseases

,

post-traumatic stress disorders

,

acquired immunodeficiency syndrome

,

hereditary angioedemas

,

,

von willebrand diseases

,

,

opioid-related disorders

,

prostatic neoplasms

,

,

caregiver burden

,

,

,

,

,

psychological distress

,

afibrinogenemia

,

pain management

,

hemorrhagic disorders

,

multiple myeloma

,

,

medication adherence

,

coagulation protein disorders

,

,

chronic urticaria

,

,

ureterolithiasis

,

sickle cell anemia

,

peptic esophagitis

,

female genital neoplasms

,

,

hereditary angioedema types i and ii

,

feeding behavior

,

,

obstructive sleep apnea

,

,

gouty arthritis

,

,

inborn genetic diseases

,

,

plasma cell neoplasms

,

type 3 von willebrand disease

,

ureteral calculi

,

factor vii deficiency

,

,

thrombotic thrombocytopenic purpura

,

child development

,

male circumcision

,

treatment adherence and compliance

,

hereditary angioedema type iii

,

sleep apnea syndromes

,

nociceptive pain

,

vision disorders

,

wet macular degeneration

,

x-linked genetic diseases

,

inherited blood coagulation disorders

,

breakthrough pain

,

subarachnoid hemorrhage

,

intraoperative complications

,

,

,

sjogren's syndrome

,

,

anesthesia and analgesia

,

highly active antiretroviral therapy

,

,

growth disorders

,

hereditary autoinflammatory diseases

,

familial mediterranean fever

,

,

,

aplastic anemia

,

intractable pain

,

bone marrow failure disorders

,

,

severe combined immunodeficiency

,

immune system diseases

,

thrombocytopenic purpura idiopathic

,

blood platelet disorders

,

autoimmune diseases

,

thrombocytopenic purpura

,

,

skin manifestations

,

,

immunoglobulin light-chain amyloidosis

,

factor x deficiency

,

,

erectile dysfunction

,

,

postoperative pain

,

hepatocellular carcinoma

,

,

diabetes mellitus

,

liver neoplasms

,

healthy volunteers/patients

,

breast neoplasms

,

,

wounds and injuries

,

gastroesophageal reflux

,

premature birth

,

premature ejaculation

,

,

,

hemostatic disorders

,

spinal cord injuries

,

,

respiratory insufficiency

,

,

,

hematologic diseases

,

,

,

attention deficit disorder with hyperactivity

,

replacement arthroplasty knee

,

myocardial ischemia

,

,

local anesthesia

,

conduction anesthesia

,

respiratory aspiration

,

physiological sexual dysfunction

,

ankylosing spondylitis

,

,

esophageal neoplasms

,

patient-controlled analgesia

,

,

allergic rhinitis

,

,

,

spondylarthritis

,

,

knee osteoarthritis

,

sexually transmitted diseases

,

patient satisfaction

,

,

,

,

,

,

dry eye syndromes

,

,

insulin resistance

,

,

macular degeneration

,

postoperative nausea and vomiting

,

,

spinal anesthesia

,

,

neuromuscular blockade

,

postoperative period

,

,

retinal vein occlusion

,

keratoconjunctivitis sicca

,

non-erosive reflux disease

,

,

,

brain neoplasms

,

irritable bowel syndrome

,

renal insufficiency

,

,

,

chlamydia infections

,

,

general anesthesia

,

,

,

,

bariatric surgery

,

orthopedic procedures

,

,

atrophic gastritis

,

attention deficit and disruptive behavior disorders

,

impulse control disruptive and conduct disorders

,

oppositional defiant disorder

,

allergic rhinitis perennial

,

supratentorial neoplasms

,

periodontal diseases

,

furcation defects

,

,

muscle relaxation

,

pancreatic fistula

,

,

surgical wound infection

,

,

bacterial sexually transmitted diseases

,

depressive disorder

,

,

,

,

psychotic disorders

,

,

,

coronary artery disease

,

colorectal neoplasms

,

,

major depressive disorder

,

traumatic brain injuries

,

musculoskeletal diseases

,

psychological stress

,

postpartum depression

,

multiple sclerosis

,

cardiovascular diseases

,

ischemic stroke

,

bipolar disorder

,

attitudes health knowledge practice

,

psychological burnout

,

alcohol drinking

,

,

colonic neoplasms

,

pancreatic neoplasms

,

professional burnout

,

inflammatory bowel diseases

,

health behavior

,

cesarean section

,

,

,

sleep initiation and maintenance disorders

,

occupational stress

,

,

,

head and neck neoplasms

,

traumatic stress disorders

,

cognition disorders

,

sedentary behavior

,

psychological well-being

,

,

,

,

drug-related side effects and adverse reactions

,

replacement arthroplasty hip

,

adenomatous polyps

,

,

rheumatoid arthritis

,

metabolic syndrome

,

musculoskeletal pain

,

,

,

surgical amputation

,

autistic disorder

,

physical fitness

,

developmental disabilities

,

respiratory distress syndrome

,

,

,

,

,

occupational diseases

,

cerebral hemorrhage

,

ulcerative colitis

,

patient compliance

,

,

,

,

prediabetic state

,

,

tobacco use cessation

,

coronary disease

,

,

deglutition disorders

,

,

neurologic gait disorders

,

intestinal diseases

,

,

myocardial infarction

,

,

perinatal death

,

,

hypercholesterolemia

,

neurodevelopmental disorders

,

,

chronic renal insufficiency

,

kidney diseases

,

disease progression

,

stomach neoplasms

,

,

newborn respiratory distress syndrome

,

,

,

anterior cruciate ligament injuries

,

communicable diseases

,

lupus nephritis

,

systemic lupus erythematosus

,

interstitial lung diseases

,

,

family characteristics

,

,

pediatric obesity

,

essential tremor

,

,

,

suicidal ideation

,

emotional regulation

,

,

obsessive-compulsive disorder

,

sleep wake disorders

,

,

atrial fibrillation

,

bites and stings

,

,

,

,

renal cell carcinoma

,

learning disabilities

,

,

,

,

,

,

,

postpartum hemorrhage

,

somatoform disorders

,

,

neuromuscular diseases

,

tobacco smoking

,

,

,

non-small-cell lung carcinoma

,

personal satisfaction

,

,

artificial respiration

,

,

help-seeking behavior

,

,

,

problem behavior

,

social isolation

,

pervasive child development disorders

,

,

,

uterine cervical neoplasms

,

,

liver cirrhosis

,

hypersensitivity

,

,

,

speech intelligibility

,

attitude to health

,

coronavirus infections

,

,

,

consumer behavior

,

,

rectal neoplasms

,

,

,

multitasking behavior

,

acute coronary syndrome

,

,

,

wasting syndrome

,

,

self-injurious behavior

,

,

,

,

,

,

ankle fractures

,

,

,

body weight changes

,

,

,

maternal behavior

,

squamous cell carcinoma

,

squamous cell neoplasms

,

kidney transplantation

,

postoperative complications

,

respiratory tract infections

,

chronic disease

,

,

heart valve diseases

,

,

motion sickness

,

,

laparoscopic cholecystectomy

,

ovarian neoplasms

,

,

compulsive personality disorder

,

sleep deprivation

,

,

feeding and eating disorders

,

,

athletic injuries

,

,

patient participation

,

mouth neoplasms

,

gastrointestinal diseases

,

digestive system diseases

,

adolescent behavior

,

addictive behavior

,

suicide prevention

,

,

short-term memory

,

neurocognitive disorders

,

brain concussion

,

schizophrenia spectrum and other psychotic disorders

,

cerebral small vessel diseases

,

,

,

heat stress disorders

,

,

adolescent psychiatry

,

,

cystic fibrosis

,

pregnancy complications

,

physician-patient relations

,

central serous chorioretinopathy

,

ultrasonography

,

abdominal neoplasms

,

cutaneous fistula

,

,

obstetric labor

,

,

hip osteoarthritis

,

brachial plexus block

,

colonic diseases

,

,

,

liver transplantation

,

immunologic deficiency syndromes

,

antibiotic prophylaxis

,

precursor cell lymphoblastic leukemia-lymphoma

,

lymphoid leukemia

,

systemic scleroderma

,

,

auditory perception

,

rotator cuff injuries

,

femoracetabular impingement

,

joint dislocations

,

diabetes complications

,

,

peripheral nervous system diseases

,

,

,

,

decision support techniques

,

myeloid leukemia acute

,

barrett esophagus

,

,

abdominal injuries

,

drug interactions

,

pharmacological phenomena

,

rotator cuff tear arthropathy

,

,

executive function

,

amniotic fluid embolism

,

psoriatic arthritis

,

,

,

acute cholecystitis

,

,

acalculous cholecystitis

,

obstructive jaundice

,

,

stomatognathic diseases

,

postoperative care

,

male breast neoplasms

,

,

hemorrhagic stroke

,

rheumatic diseases

,

collagen diseases

,

mobility limitation

,

amyotrophic lateral sclerosis

,

motor neuron disease

,

,

aspiration pneumonia

,

axial spondyloarthritis

,

student dropouts

,

nervous system diseases

,

,

physiologic monitoring

,

,

endoscopic retrograde cholangiopancreatography

,

anesthesia recovery period

,

,

thoracic injuries

,

olfaction disorders

,

,

,

mesenteric ischemia

,

intraabdominal infections

,

sensitivity training groups

,

professional-patient relations

,

accidental injuries

,

,

bilateral hearing loss

,

hospitalization

,

,

,

treatment-resistant schizophrenia

,

pregnancy in adolescence

,

transient ischemic attack

,

secondary prevention

,

obstetric labor complications

,

infant mortality

,

,

communication disorders

,

status epilepticus

,

,

,

psychomotor agitation

,

transcutaneous electric nerve stimulation

,

thyroid diseases

,

shoulder impingement syndrome

,

,

iron-deficiency anemia

,

iron deficiencies

,

postpartum period

,

respiration disorders

,

muscular dystrophies

,

craniofacial abnormalities

,

,

,

circulating neoplastic cells

,

behavior and behavior mechanisms

,

,

,

edentulous mouth

,

psychology biofeedback

,

neuroendocrine tumors

,

,

patient acceptance of health care

,

peroneal neuropathies

,

,

,

,

,

fragile x syndrome

,

cerebellar ataxia

,

,

acute lung injury

,

feasibility studies

,

inhalation administration

,

drug delivery systems

,

,

venous insufficiency

,

,

noise-induced hearing loss

,

food preferences

,

,

attempted suicide

,

,

magnetic resonance imaging

,

cognitive aging

,

,

staphylococcal infections

,

staphylococcus aureus

,

,

,

generalized anxiety disorder

,

risk reduction behavior

,

hematologic neoplasms

,

post-cardiac arrest syndrome

,

water supply sanitary surveys

,

congenital hip dislocation

,

hip dislocation

,

developmental dysplasia of the hip

,

soft tissue injuries

,

,

diverticular diseases

,

,

colonic diverticulitis

,

,

patient discharge

,

maxillofacial injuries

,

cholecystectomy

,

hereditary nonpolyposis colorectal neoplasms

,

hereditary breast and ovarian cancer syndrome

,

,

bronchopulmonary dysplasia

,

cardiorespiratory fitness

,

bipolar and related disorders

,

aortic valve stenosis

,

pathologic constriction

,

muscle weakness

,

patient reported outcome measures

,

neglected diseases

,

psychological dependency

,

,

borderline personality disorder

,

personality disorders

,

,

,

,

color perception

,

post-exercise hypotension

,

perinatal mortality

,

spinocerebellar degenerations

,

spinocerebellar ataxias

,

,

intellectual disability

,

ovarian epithelial carcinoma

,

plantar fasciitis

,

cooperative behavior

,

,

cocaine-related disorders

,

congenital abnormalities

,

bundle-branch block

,

,

microvascular angina

,

squamous cell carcinoma of head and neck

,

urinary tract infections

,

,

,

,

,

,

,

gastroenteritis

,

sexual behavior

,

intestinal perforation

,

abdominal aortic aneurysm

,

aortic aneurysm

,

aortic diseases

,

voice disorders

,

,

,

infantile diarrhea

,

urinary retention

,

,

,

spondylolisthesis

,

procedural pain

,

multidrug-resistant tuberculosis

,

,

morphine dependence

,

,

,

,

psychotic affective disorders

,

social psychology

,

,

,

,

postcholecystectomy syndrome

,

biliary tract diseases

,

arterial occlusive diseases

,

extracorporeal circulation

,

stroke rehabilitation

,

,

autologous blood transfusion

,

acute necrotizing pancreatitis

,

,

,

high-energy shock waves

,

neurogenic urinary bladder

,

psychosocial functioning

,

,

,

,

,

excoriation disorder

,

esophageal achalasia

,

,

,

physical therapy modalities

,

,

,

precancerous conditions

,

,

gastrointestinal endoscopy

,

immediate hypersensitivity

,

,

physiological stress

,

cross infection

,

activities of daily living

,

treatment outcome

,

occupational therapy

,

,

,

,

reproductive behavior

,

contraception behavior

,

,

joint instability

,

shoulder dislocation

,

therapeutic alliance

,

nurse-patient relations

,

,

hypoventilation

,

inferior wall myocardial infarction

,

thoracic neoplasms

,

,

,

parent-child relations

,

osteochondrosis

,

deep brain stimulation

,

forensic psychiatry

,

,

malignant hyperthermia

,

,

,

,

muscle spasticity

,

movement disorders

,

aortic valve disease

,

mitral valve insufficiency

,

,

internet addiction disorder

,

prolonged grief disorder

,

,

femoral neck fractures

,

,

huntington disease

,

,

mediterranean diet

,

,

health education

,

,

,

,

,

war-related injuries

,

urinary incontinence

,

stress urinary incontinence

,

,

cumulative trauma disorders

,

medically unexplained symptoms

,

chronic kidney disease-mineral and bone disorder

,

plyometric exercise

,

post-concussion syndrome

,

clinical decision rules

,

procrastination

,

mother-child relations

,

recreational drug use

,

social marginalization

,

basal cell carcinoma

,

basal cell neoplasms

,

pulmonary fibrosis

,

trauma and stressor related disorders

,

progressive bulbar palsy

,

prostheses and implants

,

heart disease risk factors

,

,

,

body temperature regulation

,

social behavior

,

smoking reduction

,

spontaneous abortion

,

neurodegenerative diseases

,

contraceptive effectiveness

,

,

,

central nervous system sensitization

,

alcohol-related disorders

,

digestive system neoplasms

,

,

glandular and epithelial neoplasms

,

occlusal splints

,

pregnancy-induced hypertension

,

muscle strength

,

exercise therapy

,

postural balance

,

,

,

,

separation anxiety

,

,

,

,

diabetic retinopathy

,

antisocial personality disorder

,

,

,

,

choledocholithiasis

,

preoperative care

,

elective surgical procedures

,

smoldering multiple myeloma

,

endometrial neoplasms

,

,

uterine hemorrhage

,

adjustment disorders

,

uterine prolapse

,

,

univentricular heart

,

visual perception

,

,

psychomotor performance

,

lactose intolerance

,

ventilator-associated pneumonia

,

chronic brain injury

,

,

,

,

,

decision making

,

post-acute covid-19 syndrome

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