Future Perspectives of Oral Delivery of Next Generation Therapies for Treatment of Skin Diseases

Gene therapies have conspicuously bloomed in recent years as evidenced by the increasing number of cell-, gene-, and oligo-based approved therapies. These therapies hold great promise for dermatological disorders with high unmet need, for example, epidermolysis bullosa or pachyonychia congenita. Fur...

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Main Authors: Pia Pernille Søgaard, Marianne Lind, Chatpakorn Rassemeena Christiansen, Karsten Petersson, Adam Clauss, Ester Caffarel-Salvador
Format: Article
Language:English
Published: MDPI AG 2021-10-01
Series:Pharmaceutics
Subjects:
Online Access:https://www.mdpi.com/1999-4923/13/10/1722
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author Pia Pernille Søgaard
Marianne Lind
Chatpakorn Rassemeena Christiansen
Karsten Petersson
Adam Clauss
Ester Caffarel-Salvador
author_facet Pia Pernille Søgaard
Marianne Lind
Chatpakorn Rassemeena Christiansen
Karsten Petersson
Adam Clauss
Ester Caffarel-Salvador
author_sort Pia Pernille Søgaard
collection DOAJ
description Gene therapies have conspicuously bloomed in recent years as evidenced by the increasing number of cell-, gene-, and oligo-based approved therapies. These therapies hold great promise for dermatological disorders with high unmet need, for example, epidermolysis bullosa or pachyonychia congenita. Furthermore, the recent clinical success of clustered regularly interspaced short palindromic repeats (CRISPR) for genome editing in humans will undoubtedly contribute to defining a new wave of therapies. Like biologics, naked nucleic acids are denatured inside the gastrointestinal tract and need to be administered via injections. For a treatment to be effective, a sufficient amount of a given regimen needs to reach systemic circulation. Multiple companies are racing to develop novel oral drug delivery approaches to circumvent the proteolytic and acidic milieu of the gastrointestinal tract. In this review, we provide an overview of the evolution of the gene therapy landscape, with a deep focus on gene and oligonucleotide therapies in clinical trials aimed at treating skin diseases. We then examine the progress made in drug delivery, with particular attention on the peptide field and drug-device combinations that deliver macromolecules into the gastrointestinal tract. Such novel devices could potentially be applied to administer other therapeutics including genes and CRISPR-based systems.
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spelling doaj.art-91ea12f4b5d540a39d93d281321846ea2023-11-22T19:40:23ZengMDPI AGPharmaceutics1999-49232021-10-011310172210.3390/pharmaceutics13101722Future Perspectives of Oral Delivery of Next Generation Therapies for Treatment of Skin DiseasesPia Pernille Søgaard0Marianne Lind1Chatpakorn Rassemeena Christiansen2Karsten Petersson3Adam Clauss4Ester Caffarel-Salvador5Regenerative Medicine Department, LEO Pharma A/S, Industriparken 55, 2750 Ballerup, DenmarkExplorative Formulation and Technologies, LEO Pharma A/S, Industriparken 55, 2750 Ballerup, DenmarkRegenerative Medicine Department, LEO Pharma A/S, Industriparken 55, 2750 Ballerup, DenmarkExplorative Formulation and Technologies, LEO Pharma A/S, Industriparken 55, 2750 Ballerup, DenmarkRegenerative Medicine Department, LEO Pharma A/S, Industriparken 55, 2750 Ballerup, DenmarkRegenerative Medicine Department, LEO Pharma A/S, Industriparken 55, 2750 Ballerup, DenmarkGene therapies have conspicuously bloomed in recent years as evidenced by the increasing number of cell-, gene-, and oligo-based approved therapies. These therapies hold great promise for dermatological disorders with high unmet need, for example, epidermolysis bullosa or pachyonychia congenita. Furthermore, the recent clinical success of clustered regularly interspaced short palindromic repeats (CRISPR) for genome editing in humans will undoubtedly contribute to defining a new wave of therapies. Like biologics, naked nucleic acids are denatured inside the gastrointestinal tract and need to be administered via injections. For a treatment to be effective, a sufficient amount of a given regimen needs to reach systemic circulation. Multiple companies are racing to develop novel oral drug delivery approaches to circumvent the proteolytic and acidic milieu of the gastrointestinal tract. In this review, we provide an overview of the evolution of the gene therapy landscape, with a deep focus on gene and oligonucleotide therapies in clinical trials aimed at treating skin diseases. We then examine the progress made in drug delivery, with particular attention on the peptide field and drug-device combinations that deliver macromolecules into the gastrointestinal tract. Such novel devices could potentially be applied to administer other therapeutics including genes and CRISPR-based systems.https://www.mdpi.com/1999-4923/13/10/1722skin diseasesoral biologicsgene therapyoligonucleotidesCRISPRoral devices
spellingShingle Pia Pernille Søgaard
Marianne Lind
Chatpakorn Rassemeena Christiansen
Karsten Petersson
Adam Clauss
Ester Caffarel-Salvador
Future Perspectives of Oral Delivery of Next Generation Therapies for Treatment of Skin Diseases
Pharmaceutics
skin diseases
oral biologics
gene therapy
oligonucleotides
CRISPR
oral devices
title Future Perspectives of Oral Delivery of Next Generation Therapies for Treatment of Skin Diseases
title_full Future Perspectives of Oral Delivery of Next Generation Therapies for Treatment of Skin Diseases
title_fullStr Future Perspectives of Oral Delivery of Next Generation Therapies for Treatment of Skin Diseases
title_full_unstemmed Future Perspectives of Oral Delivery of Next Generation Therapies for Treatment of Skin Diseases
title_short Future Perspectives of Oral Delivery of Next Generation Therapies for Treatment of Skin Diseases
title_sort future perspectives of oral delivery of next generation therapies for treatment of skin diseases
topic skin diseases
oral biologics
gene therapy
oligonucleotides
CRISPR
oral devices
url https://www.mdpi.com/1999-4923/13/10/1722
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