- Genomics and Gene Editing 2026
- Executive summary
Executive summary
The edition on a few pages: the abstract, the key messages, the headline finding and what each steward reads in it.
Contents
- ·Edition home
- ·Executive summary
- 1Genomics is data infrastructure and a therapeutic frontier
- 2Key findings
- 3Systems and sectors at baseline
- 4Risks and capability
- 5Individualised editing outpaces delivery systems
- 6Readings from the other desks
- 7The 2027 agenda
- 8Systems in depth
- AMethodology
- BData appendix
- CReferences and record
- ·Complete edition, for print
S.1Abstract
Genomics and gene editing matter to the Nexus programme in two ways. They are a data and hardware infrastructure: sequencers, reference genomes, biobanks, compute and a workforce that reads results. They are also a therapeutic technology whose first products are reaching patients, with rules that differ by country. The therapy evidence is strong for one product family. In a phase 3 study of exagamglogene autotemcel in sickle cell disease, 29 of 30 evaluable patients (97%) were free of severe vaso-occlusive crises for 12 months or more2. In transfusion-dependent beta-thalassaemia, 32 of 35 evaluable patients (91%) reached transfusion independence3. The FDA approved the first CRISPR-based therapy on 8 December 20231. A 2025 report describes one infant treated with a patient-specific base editor4. The data base is large. The first complete human genome sequence has 3.055 billion base pairs11, UK Biobank has sequenced 490,640 participants16, and the All of Us programme released 245,388 genomes, 77% from communities under-represented in research17. The physical and legal base is less measured. Vendor pages claim sequencing prices of USD 60 or more per genome15, independent costs are not bound, and compute has a carbon footprint21. Trade controls shaped sequencer supply in 20253031. Heritable editing is judged unacceptable at this time by the Third Summit28. This baseline sets out what the evidence supports and where it is thin. It forecasts no clinical or market outcome and ranks no country or company.
S.2Key messages
Six readings that frame the genomics baseline, and the eight questions this edition answers, each with its status and source.
In two phase 3 studies most evaluable patients met the primary end point at short follow-up.23
Do editing therapies work?Reported
The FDA approved Casgevy on 8 December 2023 for patients 12 and older.1
Is the approval real?Reported
One infant received a patient-specific base editor in 2025; one case is not a trend.4
Can a therapy be made for one patient?Reported
No. A 2023 death after high-dose viral vector therapy and a 2025 liver event show open risks.910
Is the safety record settled?Reported
A complete genome of 3.055 billion base pairs and genome releases of 245,388 and 490,640 people.111617
How large are the data resources?Reported
Vendor pages claim list prices; independent cost data were not read.1415
What does a genome cost?Reported
The Third Summit judged it unacceptable at this time; national rules differ.2728
What are the rules on heritable editing?Reported
Whether delivery, payment and oversight can keep pace with individualised editing. SCN-XT05-DLV-01
What will 2027 test first?Expert input
S.3Headline 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.
| Item | Bar, scaled to the largest value | Value |
|---|---|---|
| Evaluable for the primary end point | 30 | |
| Free of severe crises for 12 months or more | 29 | |
| Free of related hospitalisation for 12 months or more | 30 |
Source: Frangoul et al., NEJM 20242. 44 patients received exa-cel; 30 had sufficient follow-up.
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.
S.4At a glance
S.5What each steward reads in it
GCRI · KEY T
Technical Truth
The evidence supports reading genomics as data infrastructure and as a therapeutic technology.
GRF · KEY M
Public Meaning
For most people the genomics story arrives as a cure headline, a data-privacy scare or a price.
GRA · KEY F
Capital Meaning
Readiness implications only. Revenue is small, access depends on cost and manufacturing capacity, and one-time prices raise payment questions.
NEXUS
Synthesis
Genomics is well described by single results and thinly measured as a system.
S.6What this edition is, and is not
What this edition is
A baseline: the 2026 state of genomics and gene editing as infrastructure and the couplings through which it carries risk into other systems. It synthesises published evidence and ranks no country, company or asset.
What it is not
Not a forecast of therapy uptake, a technology ranking, an endorsement of any vendor or an assessment of any programme. Clinical approval, data protection, trade controls and rules on heritable editing are decisions of competent authorities.
What comes next
National, regional and sector evidence feeds the 2027 genomics edition through the federation. Each edition reports what changed against this baseline: observations, source revisions, method changes, scenario updates and corrections.
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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 10Lek, A. et al., Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne Muscular Dystrophy, New England Journal of Medicine 2023 (abstract read through the Europe PMC record). Massachusetts Medical Society, Waltham. Observation year 2023. Accessed 2026-11-03.back
- 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
- 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
- 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
