Date: 2016-05-19
Type of information: Granting of a patent
Product name: RhoNova™ (GT038)
Compound: adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing DNA encoding a rhodopsin gene
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Action mechanism: gene therapy. GT038 is an adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing a rhodopsin gene for the treatment of rhodopsin-linked retinitis pigmentosa.
Company: Genable Technologies (Ireland)
Disease: retinitis pigmentosa
Latest news: * On October 31, 2014, Genable Technologies announced that it has been granted ATMP classification from the European Medicines Agency (EMA) for, RhoNova™, its lead therapeutic. The Committee for Advanced Therapies have approved the classification of RhoNova™ as a gene therapy medicinal product. This classification enables the company to procure centralised scientific advice and guidance on the product's ongoing development.
* On December 13, 2012, the gene therapy product GT038, has been granted Orphan Drug Designation by the FDA for the treatment of retinitis pigmentosa. The product is currently undergoing formal pre-clinical assessments prior to commencing clinical studies in patients.
* On December 17, 2010, orphan designation was granted by the European Commission to Genable Technologies for adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin / adeno-associated viral vector containing a rhodopsin gene for the treatment of rhodopsin-linked retinitis pigmentosa.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2012-12-13
Orphan status UE: 2010-12-17
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
