Statements (62)
| Predicate | Object |
|---|---|
| gptkbp:instance_of |
gptkb:gene_therapy
|
| gptkbp:advocacy |
critical for treatment access
support awareness and funding |
| gptkbp:affects |
still being studied
|
| gptkbp:approves |
gptkb:United_States
gptkb:U._S._Food_and_Drug_Administration |
| gptkbp:biotechnology_field |
represents advancements in gene therapy.
|
| gptkbp:business_model |
discussed in medical ethics
|
| gptkbp:clinical_trial |
Phase 3
published in medical journals multiple sites globally completed Phase 1 and 2 |
| gptkbp:collaborations |
academic institutions
with other biotech firms |
| gptkbp:developed_by |
gptkb:Bluebird_Bio
gptkb:bluebird_bio |
| gptkbp:duration |
single treatment
|
| gptkbp:effective_date |
gptkb:2022
September 2022 |
| gptkbp:eligibility |
ages 4 to 17
|
| gptkbp:events |
long-term monitoring required
|
| gptkbp:feedback |
generally positive
|
| gptkbp:financial_support |
available for families
|
| gptkbp:funding |
various grants and investments
|
| gptkbp:future_prospects |
focused on broader applications of gene therapy
|
| gptkbp:gene_delivery_method |
lentiviral vector
|
| gptkbp:healthcare |
limited in some regions
|
| https://www.w3.org/2000/01/rdf-schema#label |
Skysona
|
| gptkbp:indication |
gptkb:X-linked_adrenoleukodystrophy
gptkb:cerebral_adrenoleukodystrophy |
| gptkbp:manufacturing_process |
complex and regulated
|
| gptkbp:market_launch |
gptkb:2023
|
| gptkbp:marketed_as |
gptkb:Skysona
|
| gptkbp:mechanism_of_action |
genetic modification of hematopoietic stem cells
corrects ABCD1 gene mutation |
| gptkbp:patient_education |
provided by healthcare teams
|
| gptkbp:patient_population |
children with ALD
|
| gptkbp:price |
approximately $3 million
|
| gptkbp:production_company |
gptkb:Bluebird_Bio
|
| gptkbp:provides_guidance_on |
developed for administration
|
| gptkbp:public_awareness |
increased through campaigns
|
| gptkbp:publication |
numerous studies conducted
|
| gptkbp:recruitment |
based on genetic testing
|
| gptkbp:regulatory_compliance |
FDA approved
European Medicines Agency pending navigated during approval process |
| gptkbp:research |
conducted prior to clinical trials
|
| gptkbp:research_focus |
gene therapy advancements
|
| gptkbp:route_of_administration |
intravenous infusion
|
| gptkbp:safety_measures |
ongoing post-marketing surveillance
|
| gptkbp:serves |
gptkb:cerebral_adrenoleukodystrophy
|
| gptkbp:side_effect |
potential for serious adverse reactions
|
| gptkbp:target_audience |
pediatric patients
|
| gptkbp:training |
required for administration
|
| gptkbp:treatment |
gptkb:cerebral_adrenoleukodystrophy
monitored in clinical settings assessed through clinical trials improved neurological function stabilize disease progression |
| gptkbp:type_of_insurance |
varies by provider
|
| gptkbp:bfsParent |
gptkb:Bluebird_Bio
|
| gptkbp:bfsLayer |
6
|