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01-09-2020 | Hypospadia | Topic Paper

Preclinical study for treatment of hypospadias by advanced therapy medicinal products

Authors: V. Sceberras, E. Attico, E. Bianchi, G. Galaverni, M. Melonari, F. Corradini, M. Fantacci, A. Ribbene, L. Losi, S. Balò, M. Lazzeri, C. Trombetta, M. Rizzo, R. Manfredini, G. Barbagli, G. Pellegrini

Published in: World Journal of Urology | Issue 9/2020

Abstract

Purpose

This paper explores the feasibility of a new therapy for the treatment of hypospadias patients. Hypospadias is a very common congenital malformation of male genitals, with very high rate of recurrences after surgery. The field of regenerative medicine, which offers innovative solutions for many pathologies, still does not offer reliable solution for this pathology. Here, we propose quality, safety, and clinical feasibility assessment for an oral mucosa advanced therapy medicinal product (ATMP) grown on a biocompatible scaffold for a clinical study on urethral reconstruction of hypospadias patients.

Methods

Urethral and oral mucosal epithelia from donor biopsies were cultivated between two fibrin layers, under clinical-grade conditions for cell and tissue characterization and comparison, aimed at tissue engineering. In addition, single-clone analyses were performed to analyze gene expression profiles of the two epithelia by microarray technology.

Results

Oral mucosa appeared suitable for urethral reconstruction. The resulting ATMP was proven to maintain stem cells and regenerative potency. The preclinical safety studies were performed on human tissues to assess abnormalities and tumorigenicity, and confirmed the safety of the ATMP. Finally, the patient selection and the clinical protocol for the upcoming clinical trial were defined.

Conclusions

Against this backdrop, in this paper, we are proposing a new reproducible and reliable ATMP for the treatment of hypospadias.

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Title: Preclinical study for treatment of hypospadias by advanced therapy medicinal products
Authors: V. Sceberras
E. Attico
E. Bianchi
G. Galaverni
M. Melonari
F. Corradini
M. Fantacci
A. Ribbene
L. Losi
S. Balò
M. Lazzeri
C. Trombetta
M. Rizzo
R. Manfredini
G. Barbagli
G. Pellegrini
Publication date: 01-09-2020
Publisher: Springer Berlin Heidelberg
Keywords: Hypospadia
Urethra Reconstruction
Published in: World Journal of Urology / Issue 9/2020
Print ISSN: 0724-4983
Electronic ISSN: 1433-8726
DOI: https://doi.org/10.1007/s00345-019-02864-x

At a glance: The STEP trials

Springer Medicine

Preclinical study for treatment of hypospadias by advanced therapy medicinal products

Abstract

Purpose

Methods

Results

Conclusions

At a glance: The STEP trials

Springer Medicine

Abstract

Purpose

Methods

Results

Conclusions

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