Gene Therapy
Biotech Modality · Active
· Health Care
Latest report
2026-05-01 13:52
Biotech companies developing AAV-based or in-vivo gene therapy platforms targeting genetic disorders.
Pool
89
Industries
6
Cohort
28
Cohort MCap
43.0B
Layers
5
Topology Rationale
The gene therapy value chain is organized around companies that create and commercialize in-vivo genetic medicines, the specialized tools and services required to discover, test, manufacture, and deliver them, and the clinical/payer infrastructure that determines real-world adoption. Because the pool includes many broader pharma, medtech, and diagnostics names, layers separate true gene-therapy developers from enabling service providers, administration/diagnostic channels, reimbursement gatekeepers, and adjacent large-cap biopharma partners or acquirers.
Value-Chain Layers
1
In-vivo Gene Therapy Developers & Commercializing Platforms
Companies in this layer develop or commercialize gene therapies or closely related in-vivo genetic medicines for rare or genetic diseases.
8 companies
| Company | Role | Market Cap | Revenue ($M) | Cash & Equivalents ($M) | EPS Diluted (GAAP) | Total Assets ($M) | Total Liabilities ($M) |
|---|---|---|---|---|---|---|---|
|
Biomarin Pharmaceutical Inc
BMRN
FY2025
|
Commercialized gene therapy via Roctavian
Healthcare
|
10.5B | 3,175 | — | — | — | — |
|
Krystal Biotech Inc
KRYS
FY2025
|
Core genetic medicine developer with gene therapy focus
Healthcare
|
7.7B | 388 | 955 | — | — | — |
|
Sarepta Therapeutics Inc
SRPT
FY2025
|
Commercial gene therapy developer via Elevidys
Healthcare
|
2.2B | 2,198 | 801 | -7.13 | 3,350 | 2,209 |
|
Taysha Gene Therapies Inc
TSHA
FY2025
|
AAV gene therapy pipeline company
Healthcare
|
1.8B | 10 | 320 | -0.34 | 343 | 96 |
|
Precigen Inc
PGEN
FY2025
|
Gene therapy and genetic medicine programs
Healthcare
|
1.5B | 10 | 30 | -1.37 | 156 | 135 |
|
Uniqure NV
QURE
FY2025
|
AAV-based gene therapy platform
Healthcare
|
1.1B | 16 | 80 | -3.46 | 825 | 626 |
|
Vertex Pharmaceuticals Inc
VRTX
FY2025
|
Genetic medicine exposure including Casgevy
Healthcare
|
107.7B | 12,001 | 12,320 | 15.32 | 25,643 | — |
|
BioNTech SE
|
Advanced genetic medicines platform with cell/gene therapy activity
Healthcare
|
25.7B | — | — | — | — | — |
2
Life Sciences Tools, CROs & Bioprocessing Support
This layer supplies the research tools, analytics, development services, testing, and manufacturing support infrastructure needed to advance gene therapy programs from preclinical work through production.
10 companies
| Company | Role | Market Cap | Revenue ($M) | Operating Cash Flow ($M) | Capex ($M) | Free Cash Flow ($M) | Net Income (GAAP) ($M) | Gross Margin (%) |
|---|---|---|---|---|---|---|---|---|
|
Charles River Laboratories
CRL
Q4-2025
|
Preclinical and research services for therapy developers
Healthcare
|
8.1B | — | — | — | — | — | — |
|
Thermo Fisher Scientific Inc
TMO
Q1-2026
|
Biopharma services and life sciences tools supplier
Healthcare
|
173.3B | 11,005 | 1,192 | 376 | 825 | 1,651 | 40.7% |
|
Danaher Corporation
DHR
Q1-2026
|
Bioprocess and life sciences instrumentation exposure
Healthcare
|
126.5B | 5,951 | 1,322 | 237 | 1,085 | 1,029 | 60.4% |
|
Agilent Technologies Inc
A
Q1-2026
|
Analytical and life sciences workflow tools
Healthcare
|
31.5B | 1,798 | 268 | 93 | 175 | 305 | — |
|
Waters Corporation
WAT
FY2025
|
Analytical instrumentation for biologics characterization
Healthcare
|
28.7B | 3,165 | 653 | 113 | — | 643 | — |
|
IQVIA Holdings Inc
IQV
FY2025
|
R&D solutions and clinical services provider
Healthcare
|
26.3B | 16,310 | 2,654 | 603 | 2,051 | 1,360 | — |
|
Illumina Inc
ILMN
FY2025
|
Sequencing platform supports genetic medicine development
Healthcare
|
18.3B | 4,343 | 1,079 | 148 | 931 | 850 | — |
|
Medpace Holdings Inc
MEDP
Q1-2026
|
CRO supporting clinical development
Healthcare
|
11.8B | 707 | 152 | 7 | — | 124 | — |
|
STERIS plc
STE
Q3-FY2026
|
Sterilization and life sciences support services
Healthcare
|
20.9B | 1,496 | 1,006 | 279 | 738 | 193 | — |
|
Becton Dickinson and Company
BDX
Q1-2026
|
Biopharma systems and life sciences consumables
Healthcare
|
41.3B | 5,252 | 657 | 108 | 549 | 382 | 45.9% |
3
Delivery, Administration & Patient Identification Infrastructure
These companies provide the devices, diagnostics, lab services, and care pathways used to identify eligible patients, support treatment administration, and monitor outcomes.
7 companies
| Company | Role | Market Cap | Revenue ($M) | Operating Income (GAAP) ($M) | Operating Margin (GAAP) (%) | Operating Cash Flow ($M) | Capex ($M) |
|---|---|---|---|---|---|---|---|
|
Abbott Laboratories
ABT
Q1-2026
|
Diagnostics capabilities support patient testing workflows
Healthcare
|
158.7B | 11,164 | 1,345 | 12.0% | — | — |
|
Natera Inc
NTRA
FY2025
|
Genetic testing helps identify eligible patients
Healthcare
|
27.8B | 2,300 | — | — | — | — |
|
Laboratory Corporation of America Holdings
LH
Q1-2026
|
Central lab and diagnostic services support therapy programs
Healthcare
|
21.2B | 3,538 | 381 | 10.8% | 192 | 121 |
|
Quest Diagnostics Incorporated
DGX
Q1-2026
|
Diagnostics network supports screening and monitoring
Healthcare
|
21.0B | 2,895 | 399 | 13.8% | 278 | 114 |
|
EXACT Sciences Corporation
EXAS
FY2025
|
Precision diagnostics infrastructure relevant to patient selection
Healthcare
|
20.0B | 3,247 | -206 | — | 491 | 135 |
|
Fresenius Medical Care Corporation
|
Specialty care delivery sites can administer complex therapies
Healthcare
|
12.1B | — | — | — | — | — |
|
Insulet Corporation
PODD
FY2025
|
Drug-delivery expertise adjacent to administration infrastructure
Healthcare
|
11.1B | 2,708 | 474 | 17.5% | 569 | 192 |
4
Large Biopharma Partners, Acquirers & Rare-Disease Commercialization Channels
This layer includes broader biopharma companies that can partner with, acquire, co-develop, finance, or commercialize gene therapy assets as programs mature.
38 companies
| Company | Role | Market Cap | Revenue ($M) | EPS Diluted (GAAP) | Net Income (GAAP) ($M) | Cash & Equivalents ($M) | Total Assets ($M) |
|---|---|---|---|---|---|---|---|
|
Eli Lilly and Company
LLY
Q1-2026
|
Large-cap pharma partner and acquirer potential
Healthcare
|
760.4B | 19,799 | 8.26 | 7,396 | — | — |
|
Johnson & Johnson
JNJ
Q1-2026
|
Global pharma platform for partnering and commercialization
Healthcare
|
547.3B | 24,062 | 2.14 | 5,235 | — | — |
|
AbbVie Inc
ABBV
Q1-2026
|
Large pharma capital and commercial infrastructure
Healthcare
|
360.6B | 15,002 | 0.39 | — | — | — |
|
AstraZeneca PLC
|
Global pharma collaborator and business development buyer
Healthcare
|
287.1B | — | — | — | — | — |
|
Merck & Company Inc
MRK
Q1-2026
|
Large pharma balance sheet and commercialization reach
Healthcare
|
274.0B | 16,286 | -1.72 | -4,240 | — | — |
|
Novartis AG ADR
|
Has meaningful gene/cell therapy adjacency
Healthcare
|
272.7B | — | — | — | — | — |
|
Amgen Inc
AMGN
Q1-2026
|
Biotech scale player with partnering capacity
Healthcare
|
182.2B | 8,618 | 3.34 | 1,819 | 12,038 | 92,504 |
|
Novo Nordisk A/S
|
Large biopharma capital provider though not core gene therapy
Healthcare
|
178.7B | — | — | — | — | — |
|
Gilead Sciences Inc
GILD
FY2025
|
Advanced therapy experience and partnering relevance
Healthcare
|
159.9B | 29,443 | 6.78 | 8,510 | 10,605 | 59,023 |
|
Pfizer Inc
PFE
FY2025
|
Large pharma with rare disease and deal capability
Healthcare
|
149.4B | 62,579 | 1.36 | 7,771 | — | — |
|
Bristol-Myers Squibb Company
BMY
Q1-2026
|
Cell therapy adjacency and commercialization capabilities
Healthcare
|
123.7B | 11,489 | 1.31 | 2,677 | — | — |
|
Sanofi ADR
|
Rare disease and specialty pharma partner relevance
Healthcare
|
110.3B | — | — | — | — | — |
|
GlaxoSmithKline PLC ADR
|
Pharma scale partner for advanced medicines
Healthcare
|
102.6B | — | — | — | — | — |
|
Regeneron Pharmaceuticals Inc
REGN
Q1-2026
|
Innovative biotech/pharma peer with partnering relevance
Healthcare
|
72.0B | — | — | — | — | — |
|
Takeda Pharmaceutical Co Ltd ADR
|
Rare disease pharma with partnership utility
Healthcare
|
51.5B | — | — | — | — | — |
|
argenx NV ADR
|
Specialty biotech adjacent in rare disease markets
Healthcare
|
48.5B | — | — | — | — | — |
|
Teva Pharma Industries Ltd ADR
TEVA
Q1-2026
|
Pharma channel access but not core gene therapy
Healthcare
|
41.2B | 3,982 | 0.31 | 369 | 3,741 | 40,040 |
|
Alnylam Pharmaceuticals Inc
ALNY
FY2025
|
Genetic medicines adjacency in rare disease
Healthcare
|
40.2B | 2,987 | — | — | — | — |
|
BeiGene, Ltd.
ONC
FY2025
|
Innovative biotech but not direct gene therapy core
Healthcare
|
32.3B | 5,343 | — | — | — | — |
|
Revolution Medicines Inc
RVMD
FY2025
|
Biotech capital markets comparator only
Healthcare
|
29.9B | — | -5.95 | -1,131 | 2,026 | 2,355 |
|
Insmed Inc
INSM
FY2025
|
Rare disease biotech adjacent to specialty commercialization
Healthcare
|
29.4B | 606 | -6.42 | — | 510 | 2,265 |
|
Royalty Pharma Plc
RPRX
FY2025
|
Provides non-dilutive capital to biopharma assets
Healthcare
|
29.3B | — | — | 1,324 | 619 | 19,621 |
|
Biogen Inc
BIIB
Q1-2026
|
Neuro-focused biotech with rare disease partnering relevance
Healthcare
|
28.5B | — | — | — | — | — |
|
United Therapeutics Corporation
UTHR
FY2025
|
Specialty biotech adjacent in orphan disease markets
Healthcare
|
25.0B | 3,183 | 27.86 | 1,335 | — | 7,880 |
|
Incyte Corporation
INCY
Q1-2026
|
Innovative biotech but outside direct gene therapy core
Healthcare
|
19.8B | 1,273 | 1.47 | 303 | — | 7,339 |
|
Roivant Sciences Ltd
ROIV
Q2-2026
|
Drug development platform and financing adjacency
Healthcare
|
19.7B | — | — | — | — | — |
|
Moderna Inc
MRNA
Q1-2026
|
Genetic medicines platform adjacent to gene therapy
Healthcare
|
18.1B | 389 | -3.4 | — | 1,908 | 11,488 |
|
Viatris Inc
VTRS
FY2025
|
Commercial pharma channels but not direct value-chain core
Healthcare
|
17.4B | 14,300 | -3 | -3,515 | 1,322 | 37,193 |
|
Summit Therapeutics PLC
SMMT
Q4-2025
|
Innovative biotech comparator rather than direct gene therapy
Healthcare
|
16.2B | — | — | — | 710 | — |
|
Genmab AS
|
Biologics-focused biotech adjacent through partnering model
Healthcare
|
16.0B | — | — | — | — | — |
|
BridgeBio Pharma Inc
BBIO
Q4-2025
|
Rare disease biotech adjacent to genetic disorder markets
Healthcare
|
13.8B | 362 | — | — | 588 | — |
|
Ascendis Pharma AS
|
Rare disease biotech adjacent, not gene therapy core
Healthcare
|
13.6B | — | — | — | — | — |
|
Neurocrine Biosciences Inc
NBIX
FY2025
|
Specialty neuroscience biotech adjacent only
Healthcare
|
13.2B | 2,860 | 4.67 | 479 | 2,543 | — |
|
Jazz Pharmaceuticals PLC
JAZZ
FY2025
|
Specialty pharma commercial platform adjacency
Healthcare
|
12.5B | 4,268 | -5.84 | -356 | 1,392 | 11,659 |
|
Ionis Pharmaceuticals Inc
IONS
Q1-2026
|
RNA therapeutics genetic medicine adjacency
Healthcare
|
12.4B | 246 | -0.56 | -93 | — | 3,450 |
|
Madrigal Pharmaceuticals Inc
MDGL
FY2025
|
Biotech comparator outside direct gene therapy chain
Healthcare
|
12.0B | 958 | — | — | 989 | — |
|
Dr. Reddy’s Laboratories Ltd ADR
|
Global pharma distribution adjacency only
Healthcare
|
11.5B | — | — | — | — | — |
|
Exelixis Inc
EXEL
FY2025
|
Oncology biotech comparator rather than gene therapy player
Healthcare
|
11.3B | 2,320 | 2.78 | 783 | — | — |
5
Payers, Benefit Managers & Reimbursement Gatekeepers
These organizations determine coverage, reimbursement design, utilization management, and outcomes-based contracting that are critical for adoption of very high-cost gene therapies.
6 companies
| Company | Role | Market Cap | Revenue ($M) | Net Income (GAAP) ($M) | Operating Cash Flow ($M) | Capex ($M) | Long-Term Debt ($M) | Medical Costs ($M) | Premiums ($M) |
|---|---|---|---|---|---|---|---|---|---|
|
UnitedHealth Group Incorporated
UNH
Q1-2026
|
Major insurer and care platform setting coverage terms
Healthcare
|
336.7B | 111,721 | 6,280 | 8,912 | 763 | 71,440 | 73,489 | — |
|
CVS Health Corp
CVS
FY2025
|
PBM and health benefits platform influences access
Healthcare
|
107.5B | 402,067 | 1,768 | 10,639 | 2,832 | 60,502 | — | — |
|
Elevance Health Inc
ELV
Q1-2026
|
Large managed care organization shaping reimbursement
Healthcare
|
81.8B | 49,494 | 1,764 | 4,332 | 235 | 30,768 | — | 41,024 |
|
Cigna Corp
CI
Q1-2026
|
Health plan and specialty services gatekeeper
Healthcare
|
77.1B | 68,494 | 1,654 | — | — | — | — | 9,812 |
|
Humana Inc
HUM
Q1-2026
|
Insurer with care delivery and reimbursement control
Healthcare
|
29.2B | — | — | — | — | — | — | — |
|
Centene Corp
CNC
Q1-2026
|
Medicaid-heavy payer relevant for rare disease access
Healthcare
|
26.5B | 49,944 | 1,541 | 4,366 | 200 | — | 38,303 | — |
Report History
| Generated | Pool | Layers |
|---|---|---|
| 2026-05-01 13:52 | 89 | 5 |