To enlighten investors on the impact of these breakthroughs and the opportunities they should create, we began publishing Big Ideas in 2017. This annual research report seeks to highlight


Cell and Gene Therapy: Generation 2



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ARK–Invest BigIdeas 2021

Cell and Gene Therapy: Generation 2
To date, the FDA has approved only 10 gene therapies. As of year-end 2020, 712 active gene therapy clinical trials were underway, 
238 of them initiated in 2020. Adjusting for the typical trial fail-rate, roughly 170 gene therapies are likely to be approved and 
commercialized
1
during the next decade.
Gene Therapy And Editing Trials Have Increased Five-Fold Since 2010
Phase I
323
Phase II
351
Phase III
37
Phase IV
1
Expected
170
Approved Today
10
Approved Drugs
Active Trials
Expected Commercializations
Gene Therapies: Stages of Advancement



Forecasts are inherently limited and cannot be relied upon. 
Source: ARK Investment Management 2021, BCC Research Blood Cancer Therapeutics, https://www.bccresearch.com/market-research/pharmaceuticals/blood-cancer-therapeutics-markets-report.html, 
Global Solid Tumor Therapeutics Market Size 2020, https://www.marketgrowthreports.com/global-solid-tumor-therapeutics-market-16188814 
111
Cell and Gene Therapy: Generation 2
Liquid and Solid Tumor Therapies: Total Addressable Market
So
lid
 T
um
or
s
Li
qu
id
 T
um
or
s
Late Stage
Early Stage
$70bn
Potentially addressable with
allogeneic advances.
$150bn
Potentially addressable
with allogeneic advances
and innovations in cellular
immunotherapies.
$13bn
Currently 
addressable
by cell 
therapy.
$30bn
Potentially 
addressable 
with
innovations
in cellular
immune-
therapies.
S I Z I N G T H E O P P O R T U N I T Y

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