Bluebird Bio

我
佬美
逗目
是股 BLUE:NASDAQ
肱
股 Bluebird Bio, Inc.
神
About The Company
Bluebird bio, Inc. (BLUE) is a clinical-stage biotechnology company. The
Company is focused on developing gene therapies for severe diseases and cancer.
With its lentiviral-based gene therapy and gene editing capabilities, it has built an
integrated product platform with various applications in these areas.
The Company's clinical programs in severe genetic diseases include its

LentiGlobin product candidate to treat transfusion-dependent ß-thalassemia and
to treat severe sickle cell disease (SCD) and its Lenti-D product candidate to treat
cerebral adrenoleukodystrophy (CALD).
Its programs in oncology focuses on developing T cell-based immunotherapies,

including chimeric antigen receptor (CAR) and T cell receptor (TCR) T cell
therapies. bb2121, its lead product candidate in oncology, is a CAR T cell product
candidate for the treatment of multiple myeloma. It also has discovery research
programs utilizing megaTALs/homing endonuclease gene editing technologies for
use across its pipeline.
02 《我老逗是股神》- 智慧型投资佳所
WHAT IS GENE THERAPY?
Gene therapy is an experimental technique that uses genes to treat or prevent
disease. In the future, this technique may allow doctors to treat a disorder by
inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers
are testing several approaches to gene therapy, including:
- Replacing a mutated gene that causes disease with a healthy copy of the gene.
- Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
- Introducing a new gene into the body to help fight a disease.
Although gene therapy is a promising treatment option for a number of diseases

(including inherited disorders, some types of cancer, and certain viral infections),
the technique remains risky and is still under study to make sure that it will be safe
and effective. Gene therapy is currently being tested only for diseases that have no
other cures.
How Are Genes Added ?
Gene addition uses a delivery system, called a vector, to insert the new genes
directly into cells. Vectors can be selected parts of viruses that have been
genetically modified so they can deliver the new genes into the cells without
causing an infectious disease. Viruses are used because they have a natural ability
to deliver genetic material into cells.
Source: Company
Bluebird Bio
Pipeline
Bluebird Bio
Pipeline
The world first gene therapy approved for
transfusion-dependent β-thalassemia (TDT).
Source: Company
Source: Company
Financial
Performance
Bluebird Bio
From 10-Q
For the
quarterly
period ended
September 30,
2019
Bluebird Bio
From 10-K
For the fiscal
year ended
December 31,
2018
Future
Development
Industry Outlook
Global Cell and Gene Therapy Market to Reach $11.96 Billion by 2025
August 6, 2019 by PR Newswire
According to a new market intelligence report by BIS Research titled

"Global Cell and Gene Therapy Market- Analysis and Forecast, 2019-2025",
the global cell and gene therapy market was valued at $1.07 billion in 2018
and is projected to grow over $8.95 billion by 2025.
The global cell and gene therapy market is expected to grow at a double-
digit compound annual growth (CAGR) rate of 36.52% during the forecast
period 2019-2025. The growth of the market propelled primarily due to the
impressive growth of the underlying service market of cell and gene therapy.
Source: Company
The price tag on this blockbuster treatment
Zynteglo
will cost about
usd1,800,000
per patient
Is $1.8 million ridiculous?

Bluebird had come a plan that it can be paid in equal installments over five
years, and if the drug stops working before then, payers won't have to keep
making their payments.
This value-based approach makes a lot of sense. Payers only pay if Zynteglo
works; and if it works, then payers could save hundreds of thousands of dollars
per year from year five on because it can cost between $300,000 to $500,000 per
year to treat people with rare diseases such as beta-thalassemia.
Conclusion
- The BLA submission for ZYNTEGLO to the FDA is likely to conclude by the first
half of 2020 but there is no guarantee that the FDA will approve ZYNTEGLO for
marketing approval in the United States. If it get approved it would boost its
addressable market by another 1,400 eligible patients.
- The first commercial patient to be dosed with ZYNTEGLO for transfusion-

dependent beta thalassemia in the European Union is expected in early 2020.
- Bluebird is also developing other drugs, such as CALD. So far, the only known
effective way to treat CALD is with a stem cell transplant. Bluebird's candidate
treatment is currently in phase 2/3 testing.
- Bluebird is also developing LentiGlobin, a drug to treat sickle cell disease (SCD).
Bluebird recently reported encouraging study results for this drug. SCD is typically
treated with regular blood transfusions. It's estimated that about 100,000 people in
the U.S. have sickle cell disease and millions more have the illness in other parts of
the world.
Bluebird bio is advancing a series of innovative pipeline to tackle several therapeutics areas
with significant unmet clinical needs. With potentially 4 approved products in the market
with either best in class or first in class potential, bluebird is transitioning from a clinical
stage toward to a blockbuster commercial stage biotechnology company.
Biotechnology is back to life by the end of 2019. The Nasdaq Biotechnology Index rose 20%
between October and December, surpassing the 9% increase in the S&P 500 Index.
According to some analysts, this growth momentum will continue into 2020.
Invest in biotech companies which are still at R&D stage is a very high risk investment.
When the clinical result announces, it share price could go up or go down by 40%-70% in a
day. But if you still interested in a cancer immunotherapy company and are afraid that its
stock price will be too volatile, you can consider this ETF: CNCR
CNCR tracks the Loncar Cancer Immunotherapy Index, which is made up of 25

pharmaceutical or biotechnology companies having a high strategic focus on the
development of immunotherapy. You can view their holding company at
https://loncarfunds.com/cncr-holdings (BLUE also in)
Technical
Analysis
Disclaimer
This report is purely for knowledge sharing and data analysis,
and does not contain any buying and selling suggestions. There
are certain risks to investing in stocks. Before making any
investment, please ask your financial planner or investment
broker for details. The team is not responsible for any loss of
money in stock trading.
Thank
谢谢阅读。
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The Company's clinical programs in severe genetic diseases include its

Its programs in oncology focuses on developing T cell-based immunotherapies,

Although gene therapy is a promising treatment option for a number of diseases

According to a new market intelligence report by BIS Research titled

Is $1.8 million ridiculous?

- The first commercial patient to be dosed with ZYNTEGLO for transfusion-

CNCR tracks the Loncar Cancer Immunotherapy Index, which is made up of 25

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