Preparation of IPSC for Cell Gene Editing for the Treatment of AATD
Launched by FONDAZIONE IRCCS POLICLINICO SAN MATTEO DI PAVIA · Mar 21, 2025
Trial Information
Current as of April 29, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
AAT is majorly produced in the hepatocytes therefore correction in the hepatocyte is a most promising approach. iHep cells as well as pluripotent stem cell or mesenchymal stem cell-derived hepatocyte-like cells provide potential cell sources for disease modeling, transplantation, and tissue engineering independent of donor organs.
However, all the studies reported in the literature, including the large drug library screening, are based on one or few AAT variant or at most few hiPSC cell lines derived from AATD patients. Moreover, only a subgroup of 10% of PI\*ZZ developed a clinically rele...
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • age ≥ 18 years at the time of signing the ICF
- • diagnosis of severe AATD (2 pathological variants)
- Exclusion Criteria:
- • incapability to give informed consent
- • subject under augmentation therapy
About Fondazione Irccs Policlinico San Matteo Di Pavia
Fondazione IRCCS Policlinico San Matteo di Pavia is a prestigious research hospital and clinical trial sponsor located in Italy, dedicated to advancing medical knowledge and improving patient care through innovative research. As a leading institution in the fields of clinical and translational medicine, the foundation actively conducts trials across various therapeutic areas, emphasizing rigorous scientific methodologies and adherence to ethical standards. With a commitment to fostering collaboration among healthcare professionals, researchers, and patients, Fondazione IRCCS Policlinico San Matteo serves as a pivotal contributor to the development of new treatments and healthcare solutions, ultimately enhancing patient outcomes and public health.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Pavia, , Italy
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported