Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)

Jennifer E Adair^{1

2}, Lindsay Androski³, Lois Bayigga⁴, Deus Bazira⁵, Eugene Brandon⁶, Lynda Dee^{7

8

9

10}, Steven Deeks¹¹, Mohamed Draz¹², Karine Dubé¹³, Mark Dybul⁵, Umut Gurkan¹², Evelyn Harlow¹⁴, Cissy Kityo⁴, Michael Louella¹⁵, Punam Malik¹⁶, Vikram Mathews¹⁷, Adrian McKemey⁶, Henry Mugerwa⁴, Daniel Muyanja⁴, Olabimpe Olayiwola¹², Rimas J Orentas^{15

18

19}, Alex Popovski³, Jeff Sheehy¹⁸, Francis Ssali⁴, Moses Supercharger Nsubuga⁴, John F Tisdale²⁰, Els Verhoeyen^{21

22}, Boro Dropulić²³

Affiliations

¹ Fred Hutchinson Cancer Research Center, Seattle, WA, USA. jadair@fredhutch.org.
² University of Washington, Seattle, WA, USA. jadair@fredhutch.org.
³ Roivant Social Ventures, New York, NY, USA.
⁴ Joint Clinical Research Centre, Kampala, Uganda.
⁵ Georgetown University Medical Center, Washington, District of Columbia, USA.
⁶ IQVIA, Durham, NC, USA.
⁷ AIDS Action Baltimore, Baltimore, MD, USA.
⁸ amFAR Institute for HIV Cure Community Advisory Board, New York, NY, USA.
⁹ Delaney AIDS Research Enterprise Community Advisory Board, San Francisco, CA, USA.
¹⁰ Martin Delaney Collaboratory Community Advisory Board, Bethesda, MD, USA.
¹¹ University of California at San Francisco, San Francisco, CA, USA.
¹² Case Western Reserve University, Cleveland, OH, USA.
¹³ University of North Carolina, Chapel Hill, NC, USA.
¹⁴ Ministry of Health, Kampala, Uganda.
¹⁵ University of Washington, Seattle, WA, USA.
¹⁶ Cincinnati Children's Medical Hospital, Cincinnati, OH, USA.
¹⁷ Christian Medical College, Vellore, India.
¹⁸ Caring Cross, Gaithersburg, MD, USA.
¹⁹ Seattle Children's Research Institute, Seattle, WA, USA.
²⁰ National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
²¹ Université Côte d'Azur, INSERM, C3M, Nice, France.
²² CIRI, INSERM, Université Lyon, CNRS, ENS de Lyon, Lyon, France.
²³ Caring Cross, Gaithersburg, MD, USA. boro.dropulic@caringcross.org.

PMID: 34493840
PMCID: PMC10113145
DOI: 10.1038/s41434-021-00284-4

Review

Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)

Jennifer E Adair et al. Gene Ther. 2023 Apr.

. 2023 Apr;30(3-4):216-221.

doi: 10.1038/s41434-021-00284-4. Epub 2021 Sep 8.

Authors

Affiliations

¹ Fred Hutchinson Cancer Research Center, Seattle, WA, USA. jadair@fredhutch.org.
² University of Washington, Seattle, WA, USA. jadair@fredhutch.org.
³ Roivant Social Ventures, New York, NY, USA.
⁴ Joint Clinical Research Centre, Kampala, Uganda.
⁵ Georgetown University Medical Center, Washington, District of Columbia, USA.
⁶ IQVIA, Durham, NC, USA.
⁷ AIDS Action Baltimore, Baltimore, MD, USA.
⁸ amFAR Institute for HIV Cure Community Advisory Board, New York, NY, USA.
⁹ Delaney AIDS Research Enterprise Community Advisory Board, San Francisco, CA, USA.
¹⁰ Martin Delaney Collaboratory Community Advisory Board, Bethesda, MD, USA.
¹¹ University of California at San Francisco, San Francisco, CA, USA.
¹² Case Western Reserve University, Cleveland, OH, USA.
¹³ University of North Carolina, Chapel Hill, NC, USA.
¹⁴ Ministry of Health, Kampala, Uganda.
¹⁵ University of Washington, Seattle, WA, USA.
¹⁶ Cincinnati Children's Medical Hospital, Cincinnati, OH, USA.
¹⁷ Christian Medical College, Vellore, India.
¹⁸ Caring Cross, Gaithersburg, MD, USA.
¹⁹ Seattle Children's Research Institute, Seattle, WA, USA.
²⁰ National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
²¹ Université Côte d'Azur, INSERM, C3M, Nice, France.
²² CIRI, INSERM, Université Lyon, CNRS, ENS de Lyon, Lyon, France.
²³ Caring Cross, Gaithersburg, MD, USA. boro.dropulic@caringcross.org.

PMID: 34493840
PMCID: PMC10113145
DOI: 10.1038/s41434-021-00284-4

Erratum in

Correction: Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI).
Adair JE, Androski L, Bayigga L, Bazira D, Brandon E, Dee L, Deeks S, Draz M, Dubé K, Dybul M, Gurkan U, Harlow E, Kityo C, Louella M, Malik P, Mathews V, McKemey A, Mugerwa H, Muyanja D, Olayiwola O, Orentas RJ, Popovski A, Sheehy J, Ssali F, Nsubuga MS, Tisdale JF, Verhoeyen E, Dropulić B. Adair JE, et al. Gene Ther. 2023 Apr;30(3-4):405. doi: 10.1038/s41434-021-00293-3. Gene Ther. 2023. PMID: 34584245 Free PMC article. No abstract available.

Abstract

The gene and cell therapy field saw its first approved treatments in Europe in 2012 and the United States in 2017 and is projected to be at least a $10B USD industry by 2025. Despite this success, a massive gap exists between the companies, clinics, and researchers developing these therapeutic approaches, and their availability to the patients who need them. The unacceptable reality is a geographic exclusion of low-and middle-income countries (LMIC) in gene therapy development and ultimately the provision of gene therapies to patients in LMIC. This is particularly relevant for gene therapies to treat human immunodeficiency virus infection and hemoglobinopathies, global health crises impacting tens of millions of people primarily located in LMIC. Bridging this divide will require research, clinical and regulatory infrastructural development, capacity-building, training, an approval pathway and community adoption for success and sustainable affordability. In 2020, the Global Gene Therapy Initiative was formed to tackle the barriers to LMIC inclusion in gene therapy development. This working group includes diverse stakeholders from all sectors and has set a goal of introducing two gene therapy Phase I clinical trials in two LMIC, Uganda and India, by 2024. Here we report on progress to date for this initiative.

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Conflict of interest statement

The content and perspectives expressed in this publication are solely the responsibility of the authors and do not necessarily represent the official views of the National Institutes of Health. JEA receives intellectual property licensing revenue from Rocket Pharmaceuticals and Orchard Therapeutics, and serves on the Scientific Advisory Board for Caring Cross. RJO is a cofounder of Caring Cross and receives research support from Lentigen, a Miltenyi Biotec company, and consulting fees from Umoja Biopharma. EV receives research support and consulting fees from Sana and serves on the Scientific Advisory Board for Caring Cross. CK serves on the Scientific Advisory Board of Caring Cross. VM is supported by the India Alliance Wellcome DBT senior fellowship grant (IA/CPHS/18/1/503930). KD provides socio-behavioral and ethics support to the California Institute of Regenerative Medicine (CIRM)/City of Hope, the defeat HIV Collaboratory (UM1 AI126623) and the BEAT-HIV Collaboratory (UM1AI126620), and has ongoing socio-behavioral sciences and ethics collaborations with the Delaney AIDS Research Enterprise (UM1AI126611). UG and Case Western Reserve University have financial interests in BioChip Labs Inc., Xatek Inc., Hemex Health Inc. Umut Gurkan has financial interests in DxNow Inc. LA and AP have financial interests in Roivant Sciences Ltd., and RSV receives support from Roivant Sciences Inc. PM receives royalties from Aruvant Sciences and CSL Behring, and is a Scientific Advisor to Aruvant Sciences. All other authors declare no conflict of interest.

Figures

Fig. 1. The highest prevalence of HIV/AIDS, sickle cell disease and thalassemia, and thus the populations who could most benefit from one-time curative treatments, are low- and middle-income countries.
Each dot represents a single country. For each disease indication (y-axis), the prevalence of the disease is represented as patient count percentile (pct; rate metric per 100,000), as measured by the Institute for Health Metrics & Evaluation’s 2019 Global Health Data Exchange, and is plotted against the gross national income (GNI) per capita for the same country as reported by the World Bank 2021 classifications (x-axis).

See this image and copyright information in PMC

References

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1. Alliance for Regenerative Medicine. 2020: Growth & Resilience in Regenerative Medicine Annual Report2021. Available from: http://alliancerm.org/wp-content/uploads/2021/03/ARM_AR2020_FINAL-PDF.pdf.
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Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)

Affiliations

Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)

Authors

Affiliations

Erratum in

Abstract

Conflict of interest statement

Figures

References

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