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Notification report


Full notification file


General information

Notification Number
B/NL/19/016

Member State to which the notification was sent
Netherlands

Date of acknowledgement from the Member State Competent Authority
23/07/2019

Title of the Project
Gene therapy clinical development program (CDP) comprising clinical trials using the
advanced therapy medicinal product (ATMP) SPK-3006, a recombinant adeno-associated viral (AAV) vector with a bioengineered capsid and a codon-optimized expression cassette
encoding a secretable human acid alpha glucosidase (GAA) gene for gene transfer in individuals with Pompe disease.


Proposed period of release:
01/01/2020 to 31/12/2030

Name of the Institute(s) or Company(ies)
Spark Therapeutics Ireland Limited, ;


3. Is the same GMO release planned elsewhere in the Community?
Yes:
Germany; France; United Kingdom; Italy;

Has the same GMO been notified elsewhere by the same notifier?
No

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Order: Parvoviridae
Genus: Dependoparvovirus
Species: Adeno-associated virus (AAV)
Strain: the GMO is a pseudotyped AAV vector
ITRs present in the vector genome are derived from wild type AAV
The capsid is derived from bioengineering the capsid of a wild-type nonhuman
primate (NHP) AAV serotype.
Common name: SPK-3006


Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
SPK-3006ParvoviridaeDependoparvovirusAdeno-associated Virus-SPK-3006

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known