Evidence generation and reproducibility in cell and gene therapy research: A call to action

Mohamed Abou-el-Enein; Aris Angelis; Frederick R. Appelbaum; Nancy C. Andrews; Susan E. Bates; Arlene S. Bierman; Malcolm K. Brenner; Marina Cavazzana; Michael A. Caligiuri; Hans Clevers; Emer Cooke; George Q. Daley; Victor J. Dzau; Lee M. Ellis; Harvey V. Fineberg; Lawrence S.B. Goldstein; Stephen Gottschalk; Margaret A. Hamburg; Donald E. Ingber; Donald B. Kohn; Adrian R. Krainer; Marcela V. Maus; Peter Marks; Christine L. Mummery; Roderic I. Pettigrew; Joni L. Rutter; Sarah A. Teichmann; Andre Terzic; Fyodor D. Urnov; David A. Williams; Jedd D. Wolchok; Mark Lawler; Cameron J. Turtle; Gerhard Bauer; John P.A. Ioannidis

doi:10.1016/j.omtm.2021.06.012

Evidence generation and reproducibility in cell and gene therapy research: A call to action

Mohamed Abou-el-Enein^*, Aris Angelis, Frederick R. Appelbaum, Nancy C. Andrews, Susan E. Bates, Arlene S. Bierman, Malcolm K. Brenner, Marina Cavazzana, Michael A. Caligiuri, Hans Clevers, Emer Cooke, George Q. Daley, Victor J. Dzau, Lee M. Ellis, Harvey V. Fineberg, Lawrence S.B. Goldstein, Stephen Gottschalk, Margaret A. Hamburg, Donald E. Ingber, Donald B. KohnAdrian R. Krainer, Marcela V. Maus, Peter Marks, Christine L. Mummery, Roderic I. Pettigrew, Joni L. Rutter, Sarah A. Teichmann, Andre Terzic, Fyodor D. Urnov, David A. Williams, Jedd D. Wolchok, Mark Lawler, Cameron J. Turtle, Gerhard Bauer, John P.A. Ioannidis

^*Corresponding author for this work

Research output: Contribution to journal › Editorial › Academic › peer-review

2 Citations (Scopus)

Original language	English
Pages (from-to)	11-14
Number of pages	4
Journal	Molecular Therapy - Methods and Clinical Development
Volume	22
DOIs	https://doi.org/10.1016/j.omtm.2021.06.012
Publication status	Published - 10 Sept 2021

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1-s2.0-S2329050121001145-mainFinal published version, 246 KBLicence: CC BY-NC-ND

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Abou-el-Enein, M., Angelis, A., Appelbaum, F. R., Andrews, N. C., Bates, S. E., Bierman, A. S., Brenner, M. K., Cavazzana, M., Caligiuri, M. A., Clevers, H., Cooke, E., Daley, G. Q., Dzau, V. J., Ellis, L. M., Fineberg, H. V., Goldstein, L. S. B., Gottschalk, S., Hamburg, M. A., Ingber, D. E., ... Ioannidis, J. P. A. (2021). Evidence generation and reproducibility in cell and gene therapy research: A call to action. Molecular Therapy - Methods and Clinical Development, 22, 11-14. https://doi.org/10.1016/j.omtm.2021.06.012

@article{50657fa574164a8e9287ab182d1cdf31,

title = "Evidence generation and reproducibility in cell and gene therapy research: A call to action",

author = "Mohamed Abou-el-Enein and Aris Angelis and Appelbaum, {Frederick R.} and Andrews, {Nancy C.} and Bates, {Susan E.} and Bierman, {Arlene S.} and Brenner, {Malcolm K.} and Marina Cavazzana and Caligiuri, {Michael A.} and Hans Clevers and Emer Cooke and Daley, {George Q.} and Dzau, {Victor J.} and Ellis, {Lee M.} and Fineberg, {Harvey V.} and Goldstein, {Lawrence S.B.} and Stephen Gottschalk and Hamburg, {Margaret A.} and Ingber, {Donald E.} and Kohn, {Donald B.} and Krainer, {Adrian R.} and Maus, {Marcela V.} and Peter Marks and Mummery, {Christine L.} and Pettigrew, {Roderic I.} and Rutter, {Joni L.} and Teichmann, {Sarah A.} and Andre Terzic and Urnov, {Fyodor D.} and Williams, {David A.} and Wolchok, {Jedd D.} and Mark Lawler and Turtle, {Cameron J.} and Gerhard Bauer and Ioannidis, {John P.A.}",

note = "Funding Information: The views expressed in this article are those of the authors, who are responsible for its content, and do not necessarily represent the views of the agencies or organizations with which the authors are affiliated. No statement in this report should be construed as an official position of the Agency for Healthcare Research and Quality (AHRQ) or the US Department of Health and Human Services. The work of J.D.W. is supported in part by US NIH/NCI Cancer Center Support grant P30 CA008748 , NIH/NCI grant R01 CA056821 , Swim Across America , the Ludwig Institute for Cancer Research , the Ludwig Center for Cancer Immunotherapy at Memorial Sloan Kettering , the Cancer Research Institute , and the Parker Institute for Cancer Immunotherapy . The work of M.A.C. is supported by US NIH grant R35CA210087 . M.A.H. is co-chair of the WHO Expert Advisory Group on Developing Global Standards for Governance and Oversight of Human Genome Editing. The work of A.T. is supported by US NIH grant R01 HL134664 , and the Marriott Family Foundation . M.L. is supported in part by grants to Health Data Research UK and DATA-CAN, the UK's Health Data Research Hub for Cancer. Funding Information: The views expressed in this article are those of the authors, who are responsible for its content, and do not necessarily represent the views of the agencies or organizations with which the authors are affiliated. No statement in this report should be construed as an official position of the Agency for Healthcare Research and Quality (AHRQ) or the US Department of Health and Human Services. The work of J.D.W. is supported in part by US NIH/NCI Cancer Center Support grant P30 CA008748, NIH/NCI grant R01 CA056821, Swim Across America, the Ludwig Institute for Cancer Research, the Ludwig Center for Cancer Immunotherapy at Memorial Sloan Kettering, the Cancer Research Institute, and the Parker Institute for Cancer Immunotherapy. The work of M.A.C. is supported by US NIH grant R35CA210087. M.A.H. is co-chair of the WHO Expert Advisory Group on Developing Global Standards for Governance and Oversight of Human Genome Editing. The work of A.T. is supported by US NIH grant R01 HL134664, and the Marriott Family Foundation. M.L. is supported in part by grants to Health Data Research UK and DATA-CAN, the UK's Health Data Research Hub for Cancer. N.C.A. is a member of the Boards of Directors for Novartis, Charles River Laboratories, and Maze Therapeutics as well as a member of the Scientific Advisory Board for Dyne Therapeutics. M.K.B. is on the advisory board of Bluebird Bio and Turnstone, founder member of Tessa Therapeutics and Marker Therapeutics, on the Scientific Advisory Board of Tessa Therapeutics, Marker Therapeutics, Allogene, Walking Fish, Memgen, KUUR, Bellicum Pharmaceuticals, Tscan, Poseida, and Abintus, and reports stock options for Marker Therapeutics, Allogene, and Walking Fish. M.A.C. is a co-founder of CytoImmune Therapeutics and Precision Technologies, a shareholder of Beigene, and chair or member of the scientific advisory boards of CBMG, OncoC4, Imugene, and Cytovia Therapeutics. H.C. is a member of the board of directors of Roche and Genentech, co-founder of Xilis and Surrozen, and SAB member of Volastra, Decibel, and Genentech. G.Q.D. holds patents related to pluripotent stem cells and hematopoietic derivatives and holds equity and receives consulting fees from 28/7 Therapeutics. L.M.E. is a consultant for Oncohost, the Data Safety Monitoring Committee for Fibrogen, and New Beta-Innovations. S.G. has patent applications in the field of T cell and gene-modified T cell therapy for cancer; is a consultant for Catamaran Bio, Nektar Therapeutics, and TESSA Therapeutics; is on the Scientific Advisory Board of Tidal; and is a DSMB member of Immatics. M.A.H. is a member of the Boards of Directors for Alnylam Pharmaceuticals. D.E.I. is a board member of Emulate Inc. Boa Biomedical Inc. and Pareto Bio Inc. chairs the scientific advisory boards of Emulate and Boa, and holds equity in Emulate, Boa, Pareto Bio, and FreeFlow Medical Devices Inc.; he also consults for Fulcrum Therapeutics and F. Hoffman La Roche. A.R.K. is a member of the Board of Directors of Stoke Therapeutics, a member of the Scientific Advisory Boards of Stoke Therapeutics, Skyhawk Therapeutics, Envisagenics Bioanalytics, Silagene, and Autoimmunity BioSolutions, and a consultant for Biogen. M.V.M. is an inventor on patents related to adoptive cell therapies held by Massachusetts General Hospital and University of Pennsylvania (some licensed to Novartis), holds equity in TCR2 and Century Therapeutics, and has served as a consultant for multiple companies involved in cell therapies. S.A.T. has received, in the past 3 years, remuneration for consulting and membership of Scientific Advisory Boards at Roche, Genentech, Biogen, GlaxoSmithKline, and ForeSite Labs. A.T. is co-founder of Rion LLC. F.D.U. is a scientific co-founder of Tune Therapeutics and an advisor to GSK. D.A.W. reports prior research funding from Bluebird Bio and Orchard Therapeutics; is on the Scientific Advisory Board of Geneception, Beam Therapeutics, and Orchard Therapeutics (SAB ended 5/20); is on the Insertion Site Advisory Board of Bluebird Bio and Biomarin; is co-founder of Alerion Biosciences (now licensed to Avro Bio) and Orchard Therapeutics; received payment in the past through a BCH institutional licensing agreement, with potential for future royalty/milestone income from Bluebird Bio, Orchard Therapeutics, and Alerion Biosciences and licensed certain IP relevant to hemoglobinopathies to Bluebird Bio. D.A.W. is on the steering Committee of Novartis ETB115E2201 (advisory fees donated to NAPAAC). J.D.W. is a Consultant for Amgen, Apricity, Ascentage Pharma, Arsenal IO, Astellas, AstraZeneca, Bayer, Bicara Therapeutics, Boehringer Ingelheim, Bristol Myers Squibb, Chugai, Daiichi Sankyo, Dragonfly, Eli Lilly, F Star, Georgiamune, Idera, Imvaq, Kyowa Hakko Kirin, Maverick Therapeutics, Merck, Neon Therapeutics, Psioxus, Recepta, Tizona, Trieza, Truvax, Trishula, Sellas, Surface Oncology, Syndax, Syntalogic, and Werewolf Therapeutics. J.D.W. reports grant/research support from Bristol Myers Squibb and Sephora and has Equity in Tizona Pharmaceuticals, Adaptive Biotechnologies, Imvaq, Beigene, Linneaus, Apricity, Arsenal IO, Georgiamune, Trieza and Maverik Therapeutics. M.L. has received an unrestricted educational grant from Pfizer for research unrelated to this work and honoraria from Pfizer, EMD Serono, Roche, and Carnall Farrar unrelated to this work. C.J.T. has served on the scientific advisory boards of ArsenalBio, Caribou Biosciences, Century Therapeutics, Eureka Therapeutics, Myeloid Therapeutics, Precision Biosciences, and T-CURX; has acted as an ad hoc consultant of Allogene, Amgen, AstraZeneca, Asher Bio, Nektar Therapeutics and PACT Pharma; has stock/options in ArsenalBio, Caribou Biosciences, Eureka Therapeutics, Myeloid Therapeutics, and Precision Biosciences; has received research funding from AstraZeneca, Juno Therapeutics/BMS, Nektar Therapeutics, and TCR2 Therapeutics; and has the right to receive royalties from Fred Hutch for a patent licensed to Juno Therapeutics/BMS. M.A. A.A. F.R.A. S.E.B. A.S.B. M.C. E.C. V.J.D. H.V.F. L.S.B.G. D.B.K. P.M. C.L.M. R.I.P. J.L.R. G.B. and J.P.A.I. declare no competing interests in relation to this work.",

year = "2021",

month = sep,

day = "10",

doi = "10.1016/j.omtm.2021.06.012",

language = "English",

volume = "22",

pages = "11--14",

}

Abou-el-Enein, M, Angelis, A, Appelbaum, FR, Andrews, NC, Bates, SE, Bierman, AS, Brenner, MK, Cavazzana, M, Caligiuri, MA, Clevers, H, Cooke, E, Daley, GQ, Dzau, VJ, Ellis, LM, Fineberg, HV, Goldstein, LSB, Gottschalk, S, Hamburg, MA, Ingber, DE, Kohn, DB, Krainer, AR, Maus, MV, Marks, P, Mummery, CL, Pettigrew, RI, Rutter, JL, Teichmann, SA, Terzic, A, Urnov, FD, Williams, DA, Wolchok, JD, Lawler, M, Turtle, CJ, Bauer, G & Ioannidis, JPA 2021, 'Evidence generation and reproducibility in cell and gene therapy research: A call to action', Molecular Therapy - Methods and Clinical Development, vol. 22, pp. 11-14. https://doi.org/10.1016/j.omtm.2021.06.012

TY - JOUR

T1 - Evidence generation and reproducibility in cell and gene therapy research

T2 - A call to action

AU - Abou-el-Enein, Mohamed

AU - Angelis, Aris

AU - Appelbaum, Frederick R.

AU - Andrews, Nancy C.

AU - Bates, Susan E.

AU - Bierman, Arlene S.

AU - Brenner, Malcolm K.

AU - Cavazzana, Marina

AU - Caligiuri, Michael A.

AU - Clevers, Hans

AU - Cooke, Emer

AU - Daley, George Q.

AU - Dzau, Victor J.

AU - Ellis, Lee M.

AU - Fineberg, Harvey V.

AU - Goldstein, Lawrence S.B.

AU - Gottschalk, Stephen

AU - Hamburg, Margaret A.

AU - Ingber, Donald E.

AU - Kohn, Donald B.

AU - Krainer, Adrian R.

AU - Maus, Marcela V.

AU - Marks, Peter

AU - Mummery, Christine L.

AU - Pettigrew, Roderic I.

AU - Rutter, Joni L.

AU - Teichmann, Sarah A.

AU - Terzic, Andre

AU - Urnov, Fyodor D.

AU - Williams, David A.

AU - Wolchok, Jedd D.

AU - Lawler, Mark

AU - Turtle, Cameron J.

AU - Bauer, Gerhard

AU - Ioannidis, John P.A.

N1 - Funding Information: The views expressed in this article are those of the authors, who are responsible for its content, and do not necessarily represent the views of the agencies or organizations with which the authors are affiliated. No statement in this report should be construed as an official position of the Agency for Healthcare Research and Quality (AHRQ) or the US Department of Health and Human Services. The work of J.D.W. is supported in part by US NIH/NCI Cancer Center Support grant P30 CA008748 , NIH/NCI grant R01 CA056821 , Swim Across America , the Ludwig Institute for Cancer Research , the Ludwig Center for Cancer Immunotherapy at Memorial Sloan Kettering , the Cancer Research Institute , and the Parker Institute for Cancer Immunotherapy . The work of M.A.C. is supported by US NIH grant R35CA210087 . M.A.H. is co-chair of the WHO Expert Advisory Group on Developing Global Standards for Governance and Oversight of Human Genome Editing. The work of A.T. is supported by US NIH grant R01 HL134664 , and the Marriott Family Foundation . M.L. is supported in part by grants to Health Data Research UK and DATA-CAN, the UK's Health Data Research Hub for Cancer. Funding Information: The views expressed in this article are those of the authors, who are responsible for its content, and do not necessarily represent the views of the agencies or organizations with which the authors are affiliated. No statement in this report should be construed as an official position of the Agency for Healthcare Research and Quality (AHRQ) or the US Department of Health and Human Services. The work of J.D.W. is supported in part by US NIH/NCI Cancer Center Support grant P30 CA008748, NIH/NCI grant R01 CA056821, Swim Across America, the Ludwig Institute for Cancer Research, the Ludwig Center for Cancer Immunotherapy at Memorial Sloan Kettering, the Cancer Research Institute, and the Parker Institute for Cancer Immunotherapy. The work of M.A.C. is supported by US NIH grant R35CA210087. M.A.H. is co-chair of the WHO Expert Advisory Group on Developing Global Standards for Governance and Oversight of Human Genome Editing. The work of A.T. is supported by US NIH grant R01 HL134664, and the Marriott Family Foundation. M.L. is supported in part by grants to Health Data Research UK and DATA-CAN, the UK's Health Data Research Hub for Cancer. N.C.A. is a member of the Boards of Directors for Novartis, Charles River Laboratories, and Maze Therapeutics as well as a member of the Scientific Advisory Board for Dyne Therapeutics. M.K.B. is on the advisory board of Bluebird Bio and Turnstone, founder member of Tessa Therapeutics and Marker Therapeutics, on the Scientific Advisory Board of Tessa Therapeutics, Marker Therapeutics, Allogene, Walking Fish, Memgen, KUUR, Bellicum Pharmaceuticals, Tscan, Poseida, and Abintus, and reports stock options for Marker Therapeutics, Allogene, and Walking Fish. M.A.C. is a co-founder of CytoImmune Therapeutics and Precision Technologies, a shareholder of Beigene, and chair or member of the scientific advisory boards of CBMG, OncoC4, Imugene, and Cytovia Therapeutics. H.C. is a member of the board of directors of Roche and Genentech, co-founder of Xilis and Surrozen, and SAB member of Volastra, Decibel, and Genentech. G.Q.D. holds patents related to pluripotent stem cells and hematopoietic derivatives and holds equity and receives consulting fees from 28/7 Therapeutics. L.M.E. is a consultant for Oncohost, the Data Safety Monitoring Committee for Fibrogen, and New Beta-Innovations. S.G. has patent applications in the field of T cell and gene-modified T cell therapy for cancer; is a consultant for Catamaran Bio, Nektar Therapeutics, and TESSA Therapeutics; is on the Scientific Advisory Board of Tidal; and is a DSMB member of Immatics. M.A.H. is a member of the Boards of Directors for Alnylam Pharmaceuticals. D.E.I. is a board member of Emulate Inc. Boa Biomedical Inc. and Pareto Bio Inc. chairs the scientific advisory boards of Emulate and Boa, and holds equity in Emulate, Boa, Pareto Bio, and FreeFlow Medical Devices Inc.; he also consults for Fulcrum Therapeutics and F. Hoffman La Roche. A.R.K. is a member of the Board of Directors of Stoke Therapeutics, a member of the Scientific Advisory Boards of Stoke Therapeutics, Skyhawk Therapeutics, Envisagenics Bioanalytics, Silagene, and Autoimmunity BioSolutions, and a consultant for Biogen. M.V.M. is an inventor on patents related to adoptive cell therapies held by Massachusetts General Hospital and University of Pennsylvania (some licensed to Novartis), holds equity in TCR2 and Century Therapeutics, and has served as a consultant for multiple companies involved in cell therapies. S.A.T. has received, in the past 3 years, remuneration for consulting and membership of Scientific Advisory Boards at Roche, Genentech, Biogen, GlaxoSmithKline, and ForeSite Labs. A.T. is co-founder of Rion LLC. F.D.U. is a scientific co-founder of Tune Therapeutics and an advisor to GSK. D.A.W. reports prior research funding from Bluebird Bio and Orchard Therapeutics; is on the Scientific Advisory Board of Geneception, Beam Therapeutics, and Orchard Therapeutics (SAB ended 5/20); is on the Insertion Site Advisory Board of Bluebird Bio and Biomarin; is co-founder of Alerion Biosciences (now licensed to Avro Bio) and Orchard Therapeutics; received payment in the past through a BCH institutional licensing agreement, with potential for future royalty/milestone income from Bluebird Bio, Orchard Therapeutics, and Alerion Biosciences and licensed certain IP relevant to hemoglobinopathies to Bluebird Bio. D.A.W. is on the steering Committee of Novartis ETB115E2201 (advisory fees donated to NAPAAC). J.D.W. is a Consultant for Amgen, Apricity, Ascentage Pharma, Arsenal IO, Astellas, AstraZeneca, Bayer, Bicara Therapeutics, Boehringer Ingelheim, Bristol Myers Squibb, Chugai, Daiichi Sankyo, Dragonfly, Eli Lilly, F Star, Georgiamune, Idera, Imvaq, Kyowa Hakko Kirin, Maverick Therapeutics, Merck, Neon Therapeutics, Psioxus, Recepta, Tizona, Trieza, Truvax, Trishula, Sellas, Surface Oncology, Syndax, Syntalogic, and Werewolf Therapeutics. J.D.W. reports grant/research support from Bristol Myers Squibb and Sephora and has Equity in Tizona Pharmaceuticals, Adaptive Biotechnologies, Imvaq, Beigene, Linneaus, Apricity, Arsenal IO, Georgiamune, Trieza and Maverik Therapeutics. M.L. has received an unrestricted educational grant from Pfizer for research unrelated to this work and honoraria from Pfizer, EMD Serono, Roche, and Carnall Farrar unrelated to this work. C.J.T. has served on the scientific advisory boards of ArsenalBio, Caribou Biosciences, Century Therapeutics, Eureka Therapeutics, Myeloid Therapeutics, Precision Biosciences, and T-CURX; has acted as an ad hoc consultant of Allogene, Amgen, AstraZeneca, Asher Bio, Nektar Therapeutics and PACT Pharma; has stock/options in ArsenalBio, Caribou Biosciences, Eureka Therapeutics, Myeloid Therapeutics, and Precision Biosciences; has received research funding from AstraZeneca, Juno Therapeutics/BMS, Nektar Therapeutics, and TCR2 Therapeutics; and has the right to receive royalties from Fred Hutch for a patent licensed to Juno Therapeutics/BMS. M.A. A.A. F.R.A. S.E.B. A.S.B. M.C. E.C. V.J.D. H.V.F. L.S.B.G. D.B.K. P.M. C.L.M. R.I.P. J.L.R. G.B. and J.P.A.I. declare no competing interests in relation to this work.

PY - 2021/9/10

Y1 - 2021/9/10

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DO - 10.1016/j.omtm.2021.06.012

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AN - SCOPUS:85110629051

SN - 2329-0501

VL - 22

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EP - 14

JO - Molecular Therapy - Methods and Clinical Development

JF - Molecular Therapy - Methods and Clinical Development

ER -

Evidence generation and reproducibility in cell and gene therapy research: A call to action

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