Periodic Reporting for period 1 - DADA2GT (Development of gene therapy and genome editing strategies to treat adenosine deaminase 2 deficiency)
Okres sprawozdawczy: 2019-06-01 do 2021-05-31
- LV-ADA2 able to correct the enzymatic defect in HSPCs and macrophages of patients with DADA2;
- ADA2 over-expression did not impact the engraftment and multilineage potential of in healthy donors’ HSPCs;
- ADA2 reconstitution in patients’ macrophages prevents excessive inflammation.
These new findings supported the application for the new orphan drug designation for DADA2, which received approval by EMA in July 2021. The orphan drug designation for DADA2 is essential because it will stimulate research and development in the sector of orphan drugs and facilitate the clinical development of gene therapy for DADA2. The incentives assured by the EU authorities will be instrumental in attracting health and biotechnology industries to speed up future clinical studies.
The DADA2GT project also contributed to raise the overall awareness of DADA2 in many typologies of subjects, including academic researchers, clinicians, students, patients’ associations, no-profit organizations, and the general public in order to support early and accurate diagnosis.