Statements (98)
| Predicate | Object |
|---|---|
| gptkbp:instance_of |
gptkb:gene_therapy
|
| gptkbp:administered_by |
subretinal injection
|
| gptkbp:advocacy |
required before treatment
required prior to treatment |
| gptkbp:affects |
not fully known
|
| gptkbp:approves |
gptkb:European_Union
gptkb:FDA gptkb:United_States gene therapy for inherited retinal disease |
| gptkbp:associated_with |
retinal specialists
|
| gptkbp:availability |
expanding beyond the US
|
| gptkbp:available_in |
gptkb:European_Union
gptkb:United_States |
| gptkbp:awards |
various medical awards
|
| gptkbp:biologic_license_application |
submitted to FDA
|
| gptkbp:breakthrough_therapy_designation |
granted by FDA
|
| gptkbp:clinical_trial |
completed
Phase 3 published in peer-reviewed journals completed successfully Phase 3 trial Phase 1/2 trial RESCUE trial RESTORE trial |
| gptkbp:collaboration |
gptkb:University_of_Pennsylvania
|
| gptkbp:delivers |
RPE65 gene
|
| gptkbp:developed_by |
gptkb:Spark_Therapeutics
|
| gptkbp:duration |
long-term effect
|
| gptkbp:educational_resources |
available for patients and families
|
| gptkbp:effective_date |
gptkb:2017
December 19, 2017 |
| gptkbp:eligibility |
ages 12 and older
age 12 months and older |
| gptkbp:events |
recommended after treatment
at least 5 years ongoing vision assessments |
| gptkbp:feedback |
generally positive
|
| gptkbp:financial_support |
available through Spark Therapeutics
|
| gptkbp:first_approved_gene_therapy |
for an inherited retinal disease
|
| gptkbp:funding |
gptkb:National_Institutes_of_Health
philanthropic contributions |
| gptkbp:future_prospects |
gene therapy innovations
|
| gptkbp:gene_delivery_method |
AAV vector
|
| gptkbp:healthcare |
granted by FDA
limited by cost |
| https://www.w3.org/2000/01/rdf-schema#label |
Luxturna
|
| gptkbp:impact_on_vision |
positive outcomes reported
|
| gptkbp:indication |
vision loss
vision loss due to RPE65 mutation |
| gptkbp:influenced_by |
patient advocacy groups
|
| gptkbp:ingredients |
voretigene neparvovec
|
| gptkbp:involves |
multidisciplinary teams
|
| gptkbp:is_effective_against |
improves vision
|
| gptkbp:manufacturer |
gptkb:Spark_Therapeutics
|
| gptkbp:market |
ongoing
|
| gptkbp:market_launch |
January 2018
|
| gptkbp:marketed_as |
gptkb:Luxturna
|
| gptkbp:mechanism_of_action |
gene replacement therapy
replaces defective RPE65 gene |
| gptkbp:part_of |
orphan drug program
|
| gptkbp:partnerships |
with healthcare providers
|
| gptkbp:patient_education |
provided by healthcare providers
|
| gptkbp:patient_population |
inherited retinal disease patients
estimated 1,000 patients in the US |
| gptkbp:post_treatment_monitoring |
necessary
necessary for safety |
| gptkbp:price |
approximately $850,000
|
| gptkbp:provides_guidance_on |
recommended for eligible patients
|
| gptkbp:publication |
peer-reviewed journals
|
| gptkbp:recruitment |
based on genetic testing
|
| gptkbp:regulatory_compliance |
approved
approved in Europe approved in the US |
| gptkbp:requires |
genetic testing
specialized administration |
| gptkbp:research_continues |
for other genetic disorders
|
| gptkbp:research_focus |
gene therapy advancements
|
| gptkbp:route_of_administration |
single dose
subretinal injection |
| gptkbp:safety_features |
generally well tolerated
|
| gptkbp:serves |
gptkb:Leber_congenital_amaurosis
retinal dystrophy |
| gptkbp:service_frequency |
once per eye
|
| gptkbp:side_effect |
mild to moderate
significant improvement inflammation in the eye |
| gptkbp:specialties |
performed by retinal specialists
|
| gptkbp:success_rate |
high in clinical trials
|
| gptkbp:supply_chain |
managed by Spark Therapeutics
|
| gptkbp:target_audience |
patients with RPE65 mutation
patients with RPE65 mutations |
| gptkbp:treatment |
sustained vision improvement
improved visual function sustained improvement in vision |
| gptkbp:type_of_insurance |
varies by plan
|
| gptkbp:vision_assessment |
using multi-luminance mobility test
|
| gptkbp:bfsParent |
gptkb:Spark_Therapeutics
|
| gptkbp:bfsLayer |
5
|