Education for the translation of Advanced Therapy Medicinal Products

Adamo, Davide; Attico, Eustachio; Pellegrini, Graziella

doi:10.3389/fmed.2023.1125892

Cited by 3 publications

(2 citation statements)

References 37 publications

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“…As a result of acquired (chemical/thermal burns) or genetic conditions, these cells can be partially or entirely lost, resulting in a pathology known as Limbal Stem Cell Deficiency (LSCD), where corneal opacity and conjunctivalization lead to visual impairment and blood vessels migration, otherwise absent over the central cornea [2,3]. With a total prevalence of 1-5:10,000 individuals, LSCD received the orphan drug designation in 2008 (Orpha:171673) from the Committee for Orphan Medicinal Products [4][5][6].…”

Section: Introductionmentioning

confidence: 99%

Comparison between Cultivated Oral Mucosa and Ocular Surface Epithelia for COMET Patients Follow-Up

Attico,

Galaverni,

Torello

et al. 2023

IJMS

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Total bilateral Limbal Stem Cell Deficiency is a pathologic condition of the ocular surface due to the loss of corneal stem cells. Cultivated oral mucosa epithelial transplantation (COMET) is the only autologous successful treatment for this pathology in clinical application, although abnormal peripheric corneal vascularization often occurs. Properly characterizing the regenerated ocular surface is needed for a reliable follow-up. So far, the univocal identification of transplanted oral mucosa has been challenging. Previously proposed markers were shown to be co-expressed by different ocular surface epithelia in a homeostatic or perturbated environment. In this study, we compared the transcriptome profile of human oral mucosa, limbal and conjunctival cultured holoclones, identifying Paired Like Homeodomain 2 (PITX2) as a new marker that univocally distinguishes the transplanted oral tissue from the other epithelia. We validated PITX2 at RNA and protein levels to investigate 10-year follow-up corneal samples derived from a COMET-treated aniridic patient. Moreover, we found novel angiogenesis-related factors that were differentially expressed in the three epithelia and instrumental in explaining the neovascularization in COMET-treated patients. These results will support the follow-up analysis of patients transplanted with oral mucosa and provide new tools to understand the regeneration mechanism of transplanted corneas.

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Section: Introductionmentioning

confidence: 99%

Comparison between Cultivated Oral Mucosa and Ocular Surface Epithelia for COMET Patients Follow-Up

Attico,

Galaverni,

Torello

et al. 2023

IJMS

Self Cite

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“…More specifically, to overcome the bottleneck in the development and access to ATMPs for academic research in the field of transplantation, several strategies can be implemented [ 19 , 20 ]: i) Streamlining regulatory processes. One of the major barriers to the development and access of ATMPs is the complex and lengthy regulatory approval process.…”

mentioning

confidence: 99%

The Relevance of Advanced Therapy Medicinal Products in the Field of Transplantation and the Need for Academic Research Access: Overcoming Bottlenecks and Claiming a New Time

Piemonti,

Scholz,

de Jongh

et al. 2023

Transpl Int

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The field of transplantation has witnessed the emergence of Advanced Therapy Medicinal Products (ATMPs) as highly promising solutions to address the challenges associated with organ and tissue transplantation. ATMPs encompass gene therapy, cell therapy, and tissue-engineered products, hold immense potential for breakthroughs in overcoming the obstacles of rejection and the limited availability of donor organs. However, the development and academic research access to ATMPs face significant bottlenecks that hinder progress. This opinion paper emphasizes the importance of addressing bottlenecks in the development and academic research access to ATMPs by implementing several key strategies. These include the establishment of streamlined regulatory processes, securing increased funding for ATMP research, fostering collaborations and partnerships, setting up centralized ATMP facilities, and actively engaging with patient groups. Advocacy at the policy level is essential to provide support for the development and accessibility of ATMPs, thereby driving advancements in transplantation and enhancing patient outcomes. By adopting these strategies, the field of transplantation can pave the way for the introduction of innovative and efficacious ATMP therapies, while simultaneously fostering a nurturing environment for academic research.

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Comparison Between Cultivated Oral Mucosa and Ocular Surface Epithelia for COMET Patients Follow-up.

Attico,

Galaverni,

Torello

et al. 2023

Preprint

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Total bilateral Limbal Stem Cells Deficiency is a pathologic condition of the ocular surface due to the loss of corneal stem cells. Cultivated Oral Mucosa Epithelial Transplantation (COMET) is the only autologous successful treatment for this pathology in clinical application, although non-physiological peripheric corneal vascularization often occurs. Properly characterizing the regenerated ocular surface is needed for a reliable follow-up. So far, the univocal identification of transplanted oral mucosa had been challenging. Previously proposed markers were shown co-expressed by the different ocular surface epithelia in a homeostatic or perturbated environment. In this study, we compared the transcriptome profile of human oral mucosa, limbal and conjunctival cells, identifying PITX2 as a new marker that univocally distinguishes the transplanted oral tissue from the other epithelia. We validated PITX2 at RNA and protein levels to investigate 10-year follow-up cor-neal samples derived from a COMET-treated aniridic patient. Moreover, we found novel angiogenesis-related factors differentially expressed in the three epithelia and instrumental in explaining the neovascularization in COMET-treated patients. These results will support the follow-up analysis of patients transplanted with oral mucosa and provide new tools to understand the regeneration mechanism of the transplanted cornea.

show abstract

Education for the translation of Advanced Therapy Medicinal Products

Cited by 3 publications

References 37 publications

Comparison between Cultivated Oral Mucosa and Ocular Surface Epithelia for COMET Patients Follow-Up

Comparison between Cultivated Oral Mucosa and Ocular Surface Epithelia for COMET Patients Follow-Up

The Relevance of Advanced Therapy Medicinal Products in the Field of Transplantation and the Need for Academic Research Access: Overcoming Bottlenecks and Claiming a New Time

Comparison Between Cultivated Oral Mucosa and Ocular Surface Epithelia for COMET Patients Follow-up.

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