Notification report

Full notification file

General information

Notification Number

Member State to which the notification was sent

Date of acknowledgement from the Member State Competent Authority

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?
Germany; France; United Kingdom; Italy;

Has the same GMO been notified elsewhere by the same notifier?

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:
18/12/2019 00:00:00