Orphan designation: Autologous CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector encoding the interferon alpha-2 gene Treatment of glioma, 20/06/2023 Positive

Orphan designation: Autologous dendritic cells pulsed with allogeneic tumour cell lysate Treatment of pancreatic cancer, 20/06/2023 Positive

Orphan designation: exenatide Treatment of moderate and severe closed traumatic brain injury, 20/06/2023 Positive

ACT EU Training for non-commercial sponsors: Transitioning trials to the CTR (CTIS) , Online, European Medicines Agency, Amsterdam, the Netherlands, Broadcast, from 9 February 2024, 10:00 (CET) to 9 February 2024, 13:00 (CET)

Working instructions for Notified Bodies on the application of Article 54 of Regulation (EU) 2017/745 on medical devices and Article 48 of Regulation (EU) 2017/746 on in vitro diagnostic medical devices

EU-Innovation Network Workplan 2024

Orphan designation: Adeno-associated virus vector serotype 9 containing the PKP2 gene Treatment of arrhythmogenic right ventricular cardiomyopathy due to plakophilin-2 gene mutations, 16/08/2023 Positive

Orphan designation: 2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting PLP1 pre-mRNA Treatment of Pelizaeus-Merzbacher disease, 16/08/2023 Positive

Orphan designation: Herpes simplex virus-1, derived from strain F, with deletions on genes gamma (1) 34.5 and UL39 Treatment of glioma, 16/08/2023 Positive

Orphan designation: Virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene Treatment of Huntington’s disease, 16/08/2023 Positive