Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi

Alessia Cavazza; Ayal Hendel; Rasmus O. Bak; Paula Rio; Marc Güell; Duško Lainšček; Virginia Arechavala-Gomeza; Ling Peng; Fatma Zehra Hapil; Joshua Harvey; Francisco G. Ortega; Coral Gonzalez-Martinez; Carsten W. Lederer; Kasper Mikkelsen; Giedrius Gasiunas; Nechama Kalter; Manuel A.F.V. Gonçalves; Julie Petersen; Alejandro Garanto; Lluis Montoliu; Marcello Maresca; Stefan E. Seemann; Jan Gorodkin; Loubna Mazini; Rosario Sanchez; Juan R. Rodriguez-Madoz; Noelia Maldonado-Pérez; Torella Laura; Michael Schmueck-Henneresse; Cristina Maccalli; Julian Grünewald; Gloria Carmona; Neli Kachamakova-Trojanowska; Annarita Miccio; Francisco Martin; Giandomenico Turchiano; Toni Cathomen; Yonglun Luo; Shengdar Q. Tsai; Karim Benabdellah

doi:10.1016/j.omtn.2023.102066

Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi

Alessia Cavazza
, Ayal Hendel
, Rasmus O. Bak
, Paula Rio
, Marc Güell
, Duško Lainšček
, Virginia Arechavala-Gomeza
, Ling Peng
, Fatma Zehra Hapil
, Joshua Harvey
, Francisco G. Ortega
, Coral Gonzalez-Martinez
, Carsten W. Lederer
, Kasper Mikkelsen
, Giedrius Gasiunas
, Nechama Kalter
, Manuel A.F.V. Gonçalves
, Julie Petersen
, Alejandro Garanto
, Lluis Montoliu

Marcello Maresca, Stefan E. Seemann, Jan Gorodkin, Loubna Mazini, Rosario Sanchez, Juan R. Rodriguez-Madoz, Noelia Maldonado-Pérez, Torella Laura, Michael Schmueck-Henneresse, Cristina Maccalli, Julian Grünewald, Gloria Carmona, Neli Kachamakova-Trojanowska, Annarita Miccio, Francisco Martin, Giandomenico Turchiano, Toni Cathomen, Yonglun Luo, Shengdar Q. Tsai, Karim Benabdellah

The Mina and Everard Goodman Faculty of Life Sciences - at Bar-Ilan University

Research output: Contribution to journal › Review article › peer-review

8 Scopus citations

Abstract

The European Cooperation in Science and Technology (COST) is an intergovernmental organization dedicated to funding and coordinating scientific and technological research in Europe, fostering collaboration among researchers and institutions across countries. Recently, COST Action funded the “Genome Editing to treat Human Diseases” (GenE-HumDi) network, uniting various stakeholders such as pharmaceutical companies, academic institutions, regulatory agencies, biotech firms, and patient advocacy groups. GenE-HumDi's primary objective is to expedite the application of genome editing for therapeutic purposes in treating human diseases. To achieve this goal, GenE-HumDi is organized in several working groups, each focusing on specific aspects. These groups aim to enhance genome editing technologies, assess delivery systems, address safety concerns, promote clinical translation, and develop regulatory guidelines. The network seeks to establish standard procedures and guidelines for these areas to standardize scientific practices and facilitate knowledge sharing. Furthermore, GenE-HumDi aims to communicate its findings to the public in accessible yet rigorous language, emphasizing genome editing's potential to revolutionize the treatment of many human diseases. The inaugural GenE-HumDi meeting, held in Granada, Spain, in March 2023, featured presentations from experts in the field, discussing recent breakthroughs in delivery methods, safety measures, clinical translation, and regulatory aspects related to gene editing.

Original language	English
Article number	102066
Number of pages	14
Journal	Molecular Therapy Nucleic Acids
Volume	34
DOIs	https://doi.org/10.1016/j.omtn.2023.102066
State	Published - 12 Dec 2023

Bibliographical note

Publisher Copyright:
© 2023 The Author(s)

Funding

This publication is based upon work from COST Action Gene Editing for the treatment of Human Diseases, CA21113 (https://www.genehumdi.eu) supported by COST (European Cooperation of Science and Technology). The study was also supported by the Consejería de Universidad, Investigación e Innovación under Plan Andaluz de Investigación, Desarrollo e Innovación (PAIDI 2020) (ProyExcel_00875) de la junta de Andalucía, and by Consejería de Salud y familia de la Junta de Andalucia/FEDER (PECART-0027-2020), K.B. held Nicolas Monardes contract from Consejería de Salud y Consumo de la Junta de Andalucía. A.C.: Conceptualization, data curation, Network administration, investigation, visualization, writing—original draft, writing—review and editing. A.H. R.O.B. P.R. M.G. D.L. V.A. L.P. F.Z.H. J.H. F.G.O. C.G.M. C.L. K.M. G.G. N.K. M.A.F.V.G.; A.G. L.M. M.M, J.G. M.L. R.S. J.R.R.M. N.M.P. L.T. M.S.H. C.M. J.G. G.C. A.M. F.M. G.T. T.C. Y.L. T.Q.S.: validation, review editing. K.B.: Conceptualization, funding acquisition, investigation, Network administration, resources, supervision, visualization, writing—review. P.R. has licensed the PGK:FANCAWpre∗ LV medicinal product and receives funding and equity from Rocket Pharmaceuticals, Inc. patents and royalties, research & consulting funding. D.L. is an inventor on a patent National Institute of Chemistry filed (WO/2021/032759 patent application, European patent application EP 3783104, China patent application CN 114269930 with National Phase entry EP2020756868). R.O.B. holds patents related to CRISPR-Cas genome editing and has equity in Graphite Bio and is consultant for UNIKUM Tx. G.G. holds patents related to CRISPR-Cas genome editing, is an employee of CasZyme, and has equity in CasZyme. S.Q.T. is a co-inventor on patents for GUIDE-seq, CHANGE-seq, and other genome editing technologies and a member of the scientific advisory boards of Prime Medicine and Ensoma. T.C. is a co-inventor on patents for CAST-seq, Abnoba-Seq, and other genome editing technologies, and a member of the scientific advisory boards of Cimeo Therapeutics, Excision BioTherapeutics, and GenCC. A.C. and G.T. are inventors on a patent for MEGA (WO/2023/079285), G.T. is also co-inventor on a patent for CAST-seq. This publication is based upon work from COST Action Gene Editing for the treatment of Human Diseases, CA21113 ( https://www.genehumdi.eu ) supported by COST (European Cooperation of Science and Technology). The study was also supported by the Consejería de Universidad, Investigación e Innovación under Plan Andaluz de Investigación, Desarrollo e Innovación ( PAIDI 2020 ) ( ProyExcel_00875 ) de la junta de Andalucía , and by Consejería de Salud y familia de la Junta de Andalucia /FEDER ( PECART-0027-2020 ), K.B. held Nicolas Monardes contract from Consejería de Salud y Consumo de la Junta de Andalucía.

Funders	Funder number
Abnoba-Seq
CHANGE-seq
Cimeo Therapeutics
Consejería de Universidad, Investigación e Innovación
Excision BioTherapeutics
GenCC	WO/2023/079285
National Institute of Chemistry filed	WO/2021/032759, EP2020756868
PAIDI 2020
Plan Andaluz de Investigación, Desarrollo e Innovación
European Cooperation in Science and Technology	CA21113
European Regional Development Fund	PECART-0027-2020
Consejería de Salud y Familias, Junta de Andalucía
Consejería de Salud y Consumo, Junta de Andalucía

Keywords

COST
European Cooperation in Science and Technology
GenE-HumDi
MT: RNA/DNA Editing
base editors
delivery systems
genome editing
regulatory guidelines

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

Access to Document

10.1016/j.omtn.2023.102066

Cite this

Cavazza, A., Hendel, A., Bak, R. O., Rio, P., Güell, M., Lainšček, D., Arechavala-Gomeza, V., Peng, L., Hapil, F. Z., Harvey, J., Ortega, F. G., Gonzalez-Martinez, C., Lederer, C. W., Mikkelsen, K., Gasiunas, G., Kalter, N., Gonçalves, M. A. F. V., Petersen, J., Garanto, A., ... Benabdellah, K. (2023). Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi. Molecular Therapy Nucleic Acids, 34, Article 102066. https://doi.org/10.1016/j.omtn.2023.102066

@article{0977bc8884244335ad23ed6bf27c0631,

title = "Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi",

abstract = "The European Cooperation in Science and Technology (COST) is an intergovernmental organization dedicated to funding and coordinating scientific and technological research in Europe, fostering collaboration among researchers and institutions across countries. Recently, COST Action funded the “Genome Editing to treat Human Diseases” (GenE-HumDi) network, uniting various stakeholders such as pharmaceutical companies, academic institutions, regulatory agencies, biotech firms, and patient advocacy groups. GenE-HumDi's primary objective is to expedite the application of genome editing for therapeutic purposes in treating human diseases. To achieve this goal, GenE-HumDi is organized in several working groups, each focusing on specific aspects. These groups aim to enhance genome editing technologies, assess delivery systems, address safety concerns, promote clinical translation, and develop regulatory guidelines. The network seeks to establish standard procedures and guidelines for these areas to standardize scientific practices and facilitate knowledge sharing. Furthermore, GenE-HumDi aims to communicate its findings to the public in accessible yet rigorous language, emphasizing genome editing's potential to revolutionize the treatment of many human diseases. The inaugural GenE-HumDi meeting, held in Granada, Spain, in March 2023, featured presentations from experts in the field, discussing recent breakthroughs in delivery methods, safety measures, clinical translation, and regulatory aspects related to gene editing.",

keywords = "COST, European Cooperation in Science and Technology, GenE-HumDi, MT: RNA/DNA Editing, base editors, delivery systems, genome editing, regulatory guidelines",

author = "Alessia Cavazza and Ayal Hendel and Bak, \{Rasmus O.\} and Paula Rio and Marc G{\"u}ell and Du{\v s}ko Lain{\v s}{\v c}ek and Virginia Arechavala-Gomeza and Ling Peng and Hapil, \{Fatma Zehra\} and Joshua Harvey and Ortega, \{Francisco G.\} and Coral Gonzalez-Martinez and Lederer, \{Carsten W.\} and Kasper Mikkelsen and Giedrius Gasiunas and Nechama Kalter and Gon{\c c}alves, \{Manuel A.F.V.\} and Julie Petersen and Alejandro Garanto and Lluis Montoliu and Marcello Maresca and Seemann, \{Stefan E.\} and Jan Gorodkin and Loubna Mazini and Rosario Sanchez and Rodriguez-Madoz, \{Juan R.\} and Noelia Maldonado-P{\'e}rez and Torella Laura and Michael Schmueck-Henneresse and Cristina Maccalli and Julian Gr{\"u}newald and Gloria Carmona and Neli Kachamakova-Trojanowska and Annarita Miccio and Francisco Martin and Giandomenico Turchiano and Toni Cathomen and Yonglun Luo and Tsai, \{Shengdar Q.\} and Karim Benabdellah",

note = "Publisher Copyright: {\textcopyright} 2023 The Author(s)",

year = "2023",

month = dec,

day = "12",

doi = "10.1016/j.omtn.2023.102066",

language = "אנגלית",

volume = "34",

journal = "Molecular Therapy Nucleic Acids",

issn = "2162-2531",

publisher = "Cell Press",

}

Cavazza, A, Hendel, A, Bak, RO, Rio, P, Güell, M, Lainšček, D, Arechavala-Gomeza, V, Peng, L, Hapil, FZ, Harvey, J, Ortega, FG, Gonzalez-Martinez, C, Lederer, CW, Mikkelsen, K, Gasiunas, G, Kalter, N, Gonçalves, MAFV, Petersen, J, Garanto, A, Montoliu, L, Maresca, M, Seemann, SE, Gorodkin, J, Mazini, L, Sanchez, R, Rodriguez-Madoz, JR, Maldonado-Pérez, N, Laura, T, Schmueck-Henneresse, M, Maccalli, C, Grünewald, J, Carmona, G, Kachamakova-Trojanowska, N, Miccio, A, Martin, F, Turchiano, G, Cathomen, T, Luo, Y, Tsai, SQ & Benabdellah, K 2023, 'Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi', Molecular Therapy Nucleic Acids, vol. 34, 102066. https://doi.org/10.1016/j.omtn.2023.102066

TY - JOUR

T1 - Progress and harmonization of gene editing to treat human diseases

T2 - Proceeding of COST Action CA21113 GenE-HumDi

AU - Cavazza, Alessia

AU - Hendel, Ayal

AU - Bak, Rasmus O.

AU - Rio, Paula

AU - Güell, Marc

AU - Lainšček, Duško

AU - Arechavala-Gomeza, Virginia

AU - Peng, Ling

AU - Hapil, Fatma Zehra

AU - Harvey, Joshua

AU - Ortega, Francisco G.

AU - Gonzalez-Martinez, Coral

AU - Lederer, Carsten W.

AU - Mikkelsen, Kasper

AU - Gasiunas, Giedrius

AU - Kalter, Nechama

AU - Gonçalves, Manuel A.F.V.

AU - Petersen, Julie

AU - Garanto, Alejandro

AU - Montoliu, Lluis

AU - Maresca, Marcello

AU - Seemann, Stefan E.

AU - Gorodkin, Jan

AU - Mazini, Loubna

AU - Sanchez, Rosario

AU - Rodriguez-Madoz, Juan R.

AU - Maldonado-Pérez, Noelia

AU - Laura, Torella

AU - Schmueck-Henneresse, Michael

AU - Maccalli, Cristina

AU - Grünewald, Julian

AU - Carmona, Gloria

AU - Kachamakova-Trojanowska, Neli

AU - Miccio, Annarita

AU - Martin, Francisco

AU - Turchiano, Giandomenico

AU - Cathomen, Toni

AU - Luo, Yonglun

AU - Tsai, Shengdar Q.

AU - Benabdellah, Karim

PY - 2023/12/12

Y1 - 2023/12/12

N2 - The European Cooperation in Science and Technology (COST) is an intergovernmental organization dedicated to funding and coordinating scientific and technological research in Europe, fostering collaboration among researchers and institutions across countries. Recently, COST Action funded the “Genome Editing to treat Human Diseases” (GenE-HumDi) network, uniting various stakeholders such as pharmaceutical companies, academic institutions, regulatory agencies, biotech firms, and patient advocacy groups. GenE-HumDi's primary objective is to expedite the application of genome editing for therapeutic purposes in treating human diseases. To achieve this goal, GenE-HumDi is organized in several working groups, each focusing on specific aspects. These groups aim to enhance genome editing technologies, assess delivery systems, address safety concerns, promote clinical translation, and develop regulatory guidelines. The network seeks to establish standard procedures and guidelines for these areas to standardize scientific practices and facilitate knowledge sharing. Furthermore, GenE-HumDi aims to communicate its findings to the public in accessible yet rigorous language, emphasizing genome editing's potential to revolutionize the treatment of many human diseases. The inaugural GenE-HumDi meeting, held in Granada, Spain, in March 2023, featured presentations from experts in the field, discussing recent breakthroughs in delivery methods, safety measures, clinical translation, and regulatory aspects related to gene editing.

AB - The European Cooperation in Science and Technology (COST) is an intergovernmental organization dedicated to funding and coordinating scientific and technological research in Europe, fostering collaboration among researchers and institutions across countries. Recently, COST Action funded the “Genome Editing to treat Human Diseases” (GenE-HumDi) network, uniting various stakeholders such as pharmaceutical companies, academic institutions, regulatory agencies, biotech firms, and patient advocacy groups. GenE-HumDi's primary objective is to expedite the application of genome editing for therapeutic purposes in treating human diseases. To achieve this goal, GenE-HumDi is organized in several working groups, each focusing on specific aspects. These groups aim to enhance genome editing technologies, assess delivery systems, address safety concerns, promote clinical translation, and develop regulatory guidelines. The network seeks to establish standard procedures and guidelines for these areas to standardize scientific practices and facilitate knowledge sharing. Furthermore, GenE-HumDi aims to communicate its findings to the public in accessible yet rigorous language, emphasizing genome editing's potential to revolutionize the treatment of many human diseases. The inaugural GenE-HumDi meeting, held in Granada, Spain, in March 2023, featured presentations from experts in the field, discussing recent breakthroughs in delivery methods, safety measures, clinical translation, and regulatory aspects related to gene editing.

KW - COST

KW - European Cooperation in Science and Technology

KW - GenE-HumDi

KW - MT: RNA/DNA Editing

KW - base editors

KW - delivery systems

KW - genome editing

KW - regulatory guidelines

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AN - SCOPUS:85176456320

SN - 2162-2531

VL - 34

JO - Molecular Therapy Nucleic Acids

JF - Molecular Therapy Nucleic Acids

M1 - 102066

ER -

Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi

Abstract

Bibliographical note

Funding

Keywords

UN SDGs

Access to Document

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