The gene therapy market is estimated to be worth $2.6 billion in 2023 and is expected to grow at compounded annual growth rate (CAGR) of 18.0% during the forecast period.

1. Tracking the healthcare innovation
Gene Therapy Market (5th Edition), 2022-2035
Distribution by Type of Therapy (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Delivery Method
Used (Ex Vivo and In Vivo), Type of Vector Used (Adeno-associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Retrovirus, Non-
Viral Vectors and Others), Target Therapeutic Areas (Cardiovascular Diseases, Dermatological Diseases, Genetic Diseases, Hematological
Diseases, Infectious Diseases, Metabolic Diseases, Muscle-related Diseases, Oncological Diseases, Ophthalmic Diseases and Others),
Route of Administration (Intraarticular, Intracerebral, Intracoronary, Intradermal, Intralesional, Intramuscular, Intrapleural, Intrathecal,
Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal, Topical and Others), and Key Geographical Regions (North America, Europe,
Asia-Pacific, and Rest of the World): Industry Trends and Global Forecasts
Free Insights

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2022 © Roots Analysis
High Target
Specificity
Gene delivery
vehicles, such as
viral vectors, are
used to target
specific cells
Estimated rate at
which the market is
likely to evolve till
2035
CAGR
~18%
Invested by industry
and non-industry
players in this
domain, since 2015
USD
Billion
36+
Engaged in the
development of
gene therapy
candidates
250+ Industry
Players
Gene therapies
have the potential
to increase /
restore function in
affected cells
Therapeutic
Potential
The corrected gene
is inherited by the
future generations,
with minimal / no
alterations
Long-Lasting
Effect
Filed in the last five
years for gene
therapies and gene
editing therapies
5,800+Patents
Presently being
conducted for the
evaluation of gene
therapies, worldwide
~300Clinical
Trials
1,150+Therapies
Being developed
for the treatment
of an array of
disease indications
Context
Current Industry
Landscape and
Upcoming Trends
Driven by the perpetual rise in the demand
for therapies offering a long-term effect
and supported by considerable
investments, the gene therapy market is
anticipated to grow at a considerable pace
in the foreseen future
Considering the potential of gene therapies to treat rare, inherited and acquired diseases, as well as those previously deemed
incurable, there is a growing interest in evaluating the potential of such products against rare disorders
Advantages
of Gene
Therapies
Massive advances in gene delivery and
gene editing platforms have expanded the
portfolio of tools available for genetic
manipulation; such novel technologies are
being used widely to enable the
development of improved candidates
Overview of the
Gene Therapy
Market
Given the lifelong therapeutic effect of
these pharmacological interventions for
the treatment of a wide range of
indications, players in this domain have
undertaken several research initiatives for
the advancement of gene therapies

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2022 © Roots Analysis
Over 1,150 gene therapy candidates are currently being investigated across different stages of development for
the treatment of a myriad of disease indications; till date, 11 gene therapies have been commercialized
Abbreviations: RMAT: Regenerative Medicine Advanced Therapy, PRIME: Priority medicines, AAV: Adeno-associated Virus, ARSA: Arylsulfatase A, HPV: Human papillomavirus, DMD: Duchenne muscular dystrophy, YoE: Year of Establishment, HQ: Headquarters, MENA: Middle East ant North Africa
Note 1: The pipeline also includes gene therapies for which information on phase of development was not disclosed by the developer
Note 2: Libmedly™ was approved by the European Commission in December 2020
Note 3: Industry players have been categorized based on company size (in terms of number of employees) as: Small: Less than 51 employees, Mid-sized: 51-500 employees, Large: 501-5,000 employees, Very Large: More than 5,000 employees
S. No. Drug Name
Phase of
Development
Developer(s) Target Indication Therapeutic Area Special Designation(s) Awarded
Type of
Vector Used
Gene /
Molecule
Type of Therapy
Delivery
Method
6 Libmeldy™ Approved2 Metachromatic
leukodystrophy
Genetic Diseases
Orphan drug, rare pediatric disease,
RMAT
Lentivirus ARSA Gene Augmentation Ex Vivo
13 BMN-270 Registration Hemophilia A
Hematological
Diseases
Orphan drug, PRIME, RMAT,
breakthrough therapy, priority review
AAV5 F8 Gene Augmentation In Vivo
25 ADXS-HPV Phase III Cervical cancer Oncological Diseases Orphan drug, fast track
Non-Viral
Vector
HPV
antigen
Oncolytic
Immunotherapy
In Vivo
49 SRP-9001 Phase III
Duchenne muscular
dystrophy
Muscle-related
Diseases
Orphan drug, fast track, rare
pediatric disease
AAVrh74 DMD Gene Augmentation In Vivo
103 AGTC 501 Phase II / III
X-linked retinitis
pigmentosa
Ophthalmic Diseases Orphan drug AAV2 RPGR Gene Augmentation In Vivo
218 INO-4700 Phase II
Middle east respiratory
syndrome
Infectious Diseases NA
Non-Viral
Vector
Spike Immunotherapy In Vivo
389 AGMG0201 Phase II Hypertension
Cardiovascular
Diseases
537 AT845 Phase I / II Pompe disease Metabolic Diseases
619 TSHA-118 Phase I Infantile batten disease Neurological Diseases
912 WYV-RA Preclinical Rheumatoid arthritis Autoimmune Diseases
1,154 KB107 Discovery
Junctional epidermolysis
bullosa
Dermatological
Diseases
Information on 790+ early stage products and 320+ clinical stage drugs is available in the detailed report
Gene Therapies: Marketed and Development Pipeline1
9%
10%
6%
11%
22%
42%
Pre-1990 1990-2000 2001-2005
2006-2010 2011-2015 Post-2015
Developer Landscape: Distribution by YoE, HQ and Company Size3
Content Illustrative
(details available upon request)
Example Highlights
55%
78% 73%
40%
33%
10% 27%
40%
10% 12%
20%
2%
Small Mid-sized Large Very Large
North America Europe Asia-Pacific MENA

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2022 © Roots Analysis
Gene Therapy: Market Landscape
Distribution by Type of Therapy2
1%
0.5%
2%
0.5%
7%
11%
8%
43%
27%
Marketed Registration Phase III
Phase II / III Phase II Phase I / II
Phase I Preclinical Discovery
More than 70% of the therapies are currently in early stages of development; close to 80% therapies being
evaluated in late stage studies (phase II and above) use viral vectors for delivery of the target gene
Gene Therapy: Market Landscape
Distribution by Phase of Development1
Gene Therapy: Market Landscape
Distribution by Type of Vector Used3, 4, 5
Abbreviations: AAV: Adeno-associated Virus, HSV: Herpes Simplex Virus
Note 1: Gene therapies for which information on phase of development was available have been included in this representation
Note 2: Gene therapies for which information on type of therapy was available have been included in this representation
Note 3: Gene therapies for which information on type of vector used was available have been included in this representation
Note 4: Gene therapies using more than one type of vector have been counted multiple times in this representation
Note 5: Other viral vectors category includes alphavirus vectors, anellovirus vectors, gammaretroviral vectors, seneca valley virus vectors, simian virus vectors, sindbis viral vectors, vaccinia virus vectors and venezuelan equine encephalitis virus vectors
Gene Augmentation Oncolytic Immunotherapy
Gene Editing Immunotherapy
Gene Regulation
65% 18%
6%
2%
9%
53%
19%
10%
8%
3%
2%
5%
AAV Vectors
Non-Viral
Vectors
Adenovirus
Vectors
Lentivirus
Vectors
HSV Vectors
Retrovirus
Vectors
Other Viral
Vectors
73%
Gene therapies
based on gene
augmentation utilize
AAV vectors for
delivery of the
transgene
Example Highlights
82%
Marketed gene
therapies involve the
addition of correct
version of the faulty
gene in the patients

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2022 © Roots Analysis
Patent
Value
Area under the Rectangle: Number of Patents
1%
739
1,534
2,575
3,828
5,227 5,371
2017 2018 2019 2020 2021 2022
54% 14% 2% 30%
North America Europe Asia-Pacific WIPO
Patent Analysis
Cumulative Distribution by Year of Publication1
Patent Analysis
Cumulative Distribution by Type of Player1, 2
Over the years, the intellectual capital related to gene therapies and gene editing has grown at a commendable
pace; several patents have been filed by both industry stakeholders and academic players
Abbreviation: WIPO: World Intellectual Property Organization
Note 1: For 2022, data is updated as of month when the analysis / research was conducted
Note 2: For the purpose of this analysis, patents filed by more than one type of players have been counted multiple times in this representation
Note 3: This analysis includes patents for which information on relevant parameters was available
Note 4: The value of intangible assets was calculated by considering the following parameters: [A] type of patent, [B] patent age, [C] number of citations and [D] jurisdiction
Example Highlights
Granted Patents (32%) Patent Applications (68%)
Distribution by Type of Patent
Distribution by Region
Distribution by Patent Valuation3, 4
Patent analysis on gene
editing is available in the
report titled Gene Therapy
Market (5th edition), 2022-
2035
368
798
1,298
1,888
2,609 2,678
440 894
1,513
2,307
3,129 3,219
2017 2018 2019 2020 2021 2022
Non-Industry Players
Industry Players

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43% 32% 14%
3%
1%
7%
Venture (Series A) Venture (Series B)
Venture (Series C) Venture (Series D)
Venture (Series E) Venture (Series Unknown)
5%
3%
12%
30%
7%
21%
11%
11%
Seed Debt
Grant Venture Capital
IPO Secondary Offerings
Private Equity Other Equity
61
115
167
236
316
438
551
562
2.4
4.2
6.8
12.1
17.8
26.4
35.9
2015 2016 2017 2018 2019 2020 2021 2022
Cumulative Count of Investments
Cumulative Amount Invested (USD
Billion)
36.3
72%
22%
4% 2%
71%
1%
7%
6%
2%2%
1%
1%
1%
1%
1%
2%
1%
0.7%
0.3%
2%
Funding and Investments
Cumulative Distribution of Instances1, 2
Funding and Investments
Regional Distribution of Funding Instances
Abbreviations: IPO: Initial Public Offering, MENA: Middle East and North Africa
Note 1: For 2022, data is updated as of month when the analysis / research was conducted
Note 2: Funding and investment instances for which information on year of funding was available have been included in this representation
Funding and Investments
Distribution of Instances by Type of Funding
Foreseeing lucrative returns, many public and private investors have made investments worth USD 36.4 billion,
across more than 560 instances, since 2015; the funding activity is well distributed across geographical regions
Example Highlights
~ USD 9.5 Billion
Invested in 2021
North America
US
Canada
Europe Asia-Pacific
MENA
UK
France
Italy
Netherland
China
Japan
South Korea
Singapore
Israel
Germany
Norway
Sweden
Switzerland
Spain

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2022 © Roots Analysis
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