Draft, not yet released. In stewardship review. Not indexed until the first approved release.

Key findings

Twelve findings carry the genomics baseline. They are grouped by coupling and never ranked. Each resolves to its source or, where Nexus analysis is involved, to its stated method.

7 min read1 figure0 tables24 notesNR-XTECH-GENOMICS-GLOBAL-2026-BASELINE

Contents
  1. ·Edition home
  2. ·Executive summary
  3. 1Genomics is data infrastructure and a therapeutic frontier
  4. 2Key findings
    1. 2.1 Headline finding
    2. 2.2 Health
    3. 2.3 Finance
    4. 2.4 Data and digital
    5. 2.5 Infrastructure
    6. 2.6 Energy
    7. 2.7 Trade
    8. 2.8 Governance
    9. 2.9 Systemic
  5. 3Systems and sectors at baseline
  6. 4Risks and capability
  7. 5Individualised editing outpaces delivery systems
  8. 6Readings from the other desks
  9. 7The 2027 agenda
  10. 8Systems in depth
  11. AMethodology
  12. BData appendix
  13. CReferences and record
  14. ·Complete edition, for print

2.1Headline finding

ReportedEQL 4NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-HLT-001

In a phase 3 study of exa-cel in severe sickle cell disease, 29 of 30 evaluable patients (97%) were free of severe vaso-occlusive crises for at least 12 consecutive months.

Figure 2.1Evaluable patients in the sickle cell study (count)
ItemBar, scaled to the largest valueValue
Evaluable for the primary end point30
Free of severe crises for 12 months or more29
Free of related hospitalisation for 12 months or more30

Source: Frangoul et al., NEJM 20242. 44 patients received exa-cel; 30 had sufficient follow-up.

Implication

The result supports reading ex vivo editing of blood stem cells as a working therapy for one disease. Median follow-up was 19.3 months, patients needed myeloablative conditioning, and no cancers occurred in the period reported. The FDA approved the product on 8 December 2023 for patients 12 and older12.

What would move it

Longer follow-up, treatment counts, price negotiations and cost-effectiveness studies would each move this finding67.

2.2Health

  1. ReportedEQL 4NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-HLT-001

    In a phase 3 study of exa-cel in severe sickle cell disease, 29 of 30 evaluable patients (97%) were free of severe vaso-occlusive crises for 12 months or more, and all 30 were free of related hospitalisation.2

  2. ReportedEQL 4NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-HLT-002

    In a phase 3 study of exa-cel in transfusion-dependent beta-thalassaemia, 32 of 35 evaluable patients (91%) reached transfusion independence for 12 months or more.3

  3. ReportedEQL 3NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-HLT-003

    A 2025 report describes one infant with carbamoyl-phosphate synthetase 1 deficiency who received a patient-specific base-editing therapy in two infusions; follow-up was 7 weeks after the first infusion and no serious adverse events occurred.45

  4. ReportedEQL 2NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-HLT-004

    In October 2025 a patient in a phase 3 trial of an in vivo editing therapy had a life-threatening liver event, and the FDA placed two trials on hold, according to trade press reporting company disclosures.939

2.3Finance

  1. ReportedEQL 3NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-FIN-005

    Casgevy revenue was USD 42.9 million in the first quarter of 2026 against USD 14.2 million a year earlier, and a cost-effectiveness analysis states that gene therapy access is limited by cost and manufacturing capacity.67

2.4Data and digital

  1. ReportedEQL 4NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-DAT-006

    The T2T-CHM13 sequence has 3.055 billion base pairs with nearly 200 million new, and the draft pangenome holds 47 phased diploid assemblies and adds 119 million base pairs of polymorphic sequence.1112

  2. ReportedEQL 4NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-DAT-007

    UK Biobank released whole genomes for 490,640 participants and All of Us released 245,388 genomes, of which 77% are from communities under-represented in research; literature finds most studies still rest on European ancestry.16171819

2.5Infrastructure

  1. ReportedEQL 2NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-INF-008

    Vendor pages claim more than 128 genomes per run on one sequencer and a USD 60 genome at list price on another; neither claim is independent evidence.141540

2.6Energy

  1. ReportedEQL 3NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-ENE-009

    A study of common analyses found biobank-scale association studies emit substantial carbon, and a software upgrade cut one tool footprint by 73%.2122

2.7Trade

  1. ReportedEQL 3NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-TRD-010

    China listed Illumina as an unreliable entity in February 2025, banned its sequencer imports in March 2025 and lifted the ban on 10 November 2025; the BIOSECURE Act was enacted on 18 December 2025.3031

2.8Governance

  1. ReportedEQL 5NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-GOV-011

    The FDA approved Casgevy and Lyfgenia on 8 December 2023, and the Third Summit organising committee judged heritable genome editing unacceptable at this time on 8 March 2023.12728

2.9Systemic

  1. Expert inputEQL 2NRF-XTECH-GENOMICS-GLOBAL-2026-BASELINE-SYS-012

    The leading compound pathway for 2027 testing is individualised editing moving faster than the payment, manufacturing and oversight systems around it. § scenario

Notes

Numbers are the edition’s reference numbers, so a note reads the same in every chapter. The full list, with every source and its licence, is in Annex C. Annex C, references

  1. 2Frangoul, H. et al., Exagamglogene Autotemcel for Severe Sickle Cell Disease, New England Journal of Medicine 2024 (abstract read through the Europe PMC record; 44 treated, 30 evaluable, 29 free of crises for 12 months or more). Massachusetts Medical Society, Waltham. Observation year 2024. Accessed 2026-11-03.back
  2. 1US Food and Drug Administration (ed.), FDA approves first gene therapies to treat patients with sickle cell disease (press announcement, 8 December 2023; Casgevy uses CRISPR/Cas9, Lyfgenia a lentiviral vector; patients 12 years and older). FDA, Silver Spring. Observation year 2023. Accessed 2026-11-03.back
  3. 6Vertex Pharmaceuticals (ed.), Vertex Reports First Quarter 2026 Financial Results (company press release read through a wire page; Casgevy revenue USD 42.9 million against USD 14.2 million; pediatric submission completed; company statement, weak evidence). Vertex, Boston. Observation year 2026. Accessed 2026-11-03.back
  4. 7Chetlapalli, K. et al., Haploidentical transplant, gene therapy, and standard care in sickle cell disease: a cost-effectiveness analysis, Blood 2026 (abstract read through the Europe PMC record; gene therapy access limited by cost and manufacturing capacity). American Society of Hematology, Washington. Observation year 2026. Accessed 2026-11-03.back
  5. 3Locatelli, F. et al., Exagamglogene Autotemcel for Transfusion-Dependent beta-Thalassemia, New England Journal of Medicine 2024 (abstract read through the Europe PMC record; 52 treated, 35 evaluable, 32 transfusion independent). Massachusetts Medical Society, Waltham. Observation year 2024. Accessed 2026-11-03.back
  6. 4Musunuru, K. et al., Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease, New England Journal of Medicine 2025 (abstract read through the Europe PMC record; one infant, two infusions at about 7 and 8 months of age). Massachusetts Medical Society, Waltham. Observation year 2025. Accessed 2026-11-03.back
  7. 5Clinical Trials Arena (ed.), FDA unveils new pathway to usher bespoke therapies to market (trade press, 13 November 2025; describes the plausible mechanism pathway announced in a journal article by the FDA Commissioner and the CBER Director; secondary report, weak evidence). Clinical Trials Arena, London. Observation year 2025. Accessed 2026-11-03.back
  8. 9BioSpace (ed.), Life-threatening liver toxicity triggers FDA freeze on two Intellia gene therapy studies (trade press, 30 October 2025; relays company disclosures; secondary report, weak evidence). BioSpace, Boston. Observation year 2025. Accessed 2026-11-03.back
  9. 39UNVERIFIED, not opened: Intellia Therapeutics and trade press on the lifting of the 2025 clinical holds on the MAGNITUDE trials, taken from search-result summaries that differ on dates. Observation year 2026. Accessed 2026-11-03.back
  10. 11Nurk, S. et al., The complete sequence of a human genome, Science 2022 (abstract read through the Europe PMC record; 3.055 billion base pairs; nearly 200 million base pairs of new sequence). American Association for the Advancement of Science, Washington. Observation year 2022. Accessed 2026-11-03.back
  11. 12Liao, W.-W. et al. (Human Pangenome Reference Consortium), A draft human pangenome reference, Nature 2023 (abstract read through the Europe PMC record; 47 phased diploid assemblies; 119 million base pairs added). Springer Nature, London. Observation year 2023. Accessed 2026-11-03.back
  12. 16UK Biobank Whole-Genome Sequencing Consortium, Whole-genome sequencing of 490,640 UK Biobank participants, Nature 2025 (abstract read through the Europe PMC record). Springer Nature, London. Observation year 2025. Accessed 2026-11-03.back
  13. 17All of Us Research Program Genomics Investigators, Genomic data in the All of Us Research Program, Nature 2024 (abstract read through the Europe PMC record; 245,388 genomes; 77% from communities under-represented in research). Springer Nature, London. Observation year 2024. Accessed 2026-11-03.back
  14. 18Fatumo, S. et al., A roadmap to increase diversity in genomic studies, Nature Medicine 2022 (abstract read through the Europe PMC record). Springer Nature, London. Observation year 2022. Accessed 2026-11-03.back
  15. 19Sirugo, G., Williams, S.M. and Tishkoff, S.A., The Missing Diversity in Human Genetic Studies, Cell 2019 (abstract read through the Europe PMC record). Cell Press, Cambridge. Observation year 2019. Accessed 2026-11-03.back
  16. 14Illumina (ed.), NovaSeq X Series product page (vendor claims: 16 to 21 Tb per run for NovaSeq X Plus, more than 128 genomes per run, run time about 14 to 48 hours; vendor evidence, weak). Illumina, San Diego. Observation year 2026. Accessed 2026-11-03.back
  17. 15Ultima Genomics (ed.), germline genetics product page (vendor claim of a USD 60 genome at list price over 4 wafers on the UG200 series; the browser title reads USD 80; vendor evidence, weak). Ultima Genomics, Newark. Observation year 2026. Accessed 2026-11-03.back
  18. 40UNVERIFIED, not opened: Ultima Genomics (ed.), UG 100 Solaris launch announcement of 24 February 2025 (USD 80 genome at list price), taken from search-result summaries; vendor evidence, weak. Ultima Genomics, Newark. Observation year 2025. Accessed 2026-11-03.back
  19. 21Grealey, J. et al., The Carbon Footprint of Bioinformatics, Molecular Biology and Evolution 2022 (abstract read through the Europe PMC record; a software upgrade cut one tool footprint by 73%). Oxford University Press, Oxford. Observation year 2022. Accessed 2026-11-03.back
  20. 22Lannelongue, L., Grealey, J. and Inouye, M., Green Algorithms: Quantifying the Carbon Footprint of Computation, Advanced Science 2021 (abstract read through the Europe PMC record). Wiley, Weinheim. Observation year 2021. Accessed 2026-11-03.back
  21. 30Latham and Watkins (ed.), BIOSECURE Act becomes law, limiting grants with biotechnology companies of concern (law-firm summary; enacted 18 December 2025 in the FY2026 NDAA; OMB list due by 18 December 2026; secondary source of a statute). Latham and Watkins, Los Angeles. Observation year 2025. Accessed 2026-11-03.back
  22. 31MedTech Dive (ed.), China to lift Illumina export ban (trade press, November 2025; listing in February 2025, import ban in March 2025, lift on 10 November 2025, instrument sales in China down 54% in the third quarter; secondary report, weak evidence). Industry Dive, Washington. Observation year 2025. Accessed 2026-11-03.back
  23. 27World Health Organization (ed.), Human genome editing: a framework for governance (technical document, 12 July 2021; companion recommendations published separately and not read). WHO, Geneva. Observation year 2021. Accessed 2026-11-03.back
  24. 28Royal Society (ed.), Statement from the Organising Committee of the Third International Summit on Human Genome Editing (8 March 2023; convened by the Royal Society, the Academy of Medical Sciences, the US National Academies and The World Academy of Sciences). Royal Society, London. Observation year 2023. Accessed 2026-11-03.back

Cite this chapter

Valadbaygi, S. (Ed.). (2026). Chapter 2: Key findings. In Exponential Technology Report 2026: Genomics and Gene Editing (Baseline Edition 2026, Report No. NR-XTECH-GENOMICS-GLOBAL-2026-BASELINE) [Draft under review]. The Global Centre for Risk and Innovation (GCRI).

Every citation form of the whole report, BibTeX and RIS included, is on the edition home. Cite this report

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