These slides discusses on cellular and gene therapy: the use of cells and genes to treat disease. These therapies can be effective on a wide range of previously untreated diseases, such as hematological, ocular, neurodegenerative diseases, and several types of cancers.

1. Cellular & Gene Therapy
Rapid Development for Evolving Healthcare
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2. What is cellular & Gene TherapY?
Cellular and gene therapy is
the use of cells and genes to
treat disease.
Research & development in the
United States continue to grow
at a fast rate, with a number of
products advancing in clinical
development.
Gene therapy
modifies a
person’s genes
to treat or cure
disease.
Source: https://www.fda.gov/biologicsbloodvaccines/cellulargenetherapyproducts/default.htm
Cell therapy
uses cells from
either the
patient or a
donor to treat
diseases.

3. Cellular Therapy
Cellular therapy (CT) is the
transplantation of human
cells to replace or repair
damaged tissue and/or cells.
Some of the cells that may
be used include various
types of stem cells,
lymphocytes, dendritic cells,
and pancreatic islet cells.
Source: http://www.aabb.org/aabbcct/therapyfacts/Pages/default.aspx
Potential applications include:
• Cellular immunotherapies
• Cancer treatment & vaccines
• Infectious disease
• Rebuild cartilage in joints
• Repair spinal cord injury
• Improve weakened immune system

4. Cellular Therapy Process
Source: https://medicalxpress.com/news/2018-08-stem-cell-transplants-crohn-disease.html
Harvest
Cells are harvested
from patient
themselves or donor.
Isolation
Depending on
therapeutic need,
harvested cells are
sorted to isolate
desired cells.
Growth &
Manufacture
Isolated cells are
grown by cell/tissue
culture and can be
upscaled for
production.
Infusion
Once the desired
result is achieved,
cells are introduced
back into the patient
by drip, injection, or
grafting.

5. Gene Therapy
Human gene therapy aims to modify or
manipulate the expression of a gene or
to alter the biological properties of living
cells for therapeutic use.
It can work by:
• Editing – Replacing a disease-causing gene
with a healthy copy of the gene
• Silencing – Inactivating a disease-causing
gene that is not functioning properly
• Addition – Introducing a new or modified
gene into the body to help treat a disease
Source: https://www.nature.com/articles/d41573-019-00014-x
Gene therapy products are being studied
to treat diseases including cancer,
genetic diseases, and infectious diseases.

6. Gene Therapy Products
Source: https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm573960.htm
Plasmid DNA
Circular DNA
molecules that are
genetically
engineered to carry
therapeutic genes
into human cells.
Viral Vectors
Modified to remove their ability to
cause infectious disease, these
modified viruses are used as vectors
(vehicles) to carry therapeutic genes
into human cells.
Human Gene
Editing
Disrupt harmful
genes or to repair
mutated genes.
Bacterial
Vectors
Modified and used
as vectors to carry
therapeutic genes
into human tissues.
Patient-derived
cellular gene
therapy
Cells removed from
patient are
genetically modified
and then reinfused.

7. Gene Therapy Methods
Source: https://www.researchgate.net/figure/Strategies-of-in-vivo-gene-therapy-
and-ex-vivo-gene-therapy-In-vivo-gene-therapy-on-the_fig1_322970469
In Vivo
*within the living
The desired
therapeutic gene
is packaged into a
vector such as a
modified virus or
bacteria, and then
infused into the
patient.
Ex Vivo
*out of the living
Stem cells are
isolated from the
patient, the
desired gene is
packaged and
transduced into
the cells.
Transduced cells
are then infused
into the patient.

8. The Center for Biologics Evaluation & Research
CBER regulates cellular
therapy products, human
gene therapy products,
and certain devices
related to cell and gene
therapy. It is one of 6 main
centers under FDA.
CBER has approved both
cellular and gene therapy
products.
Source: CDER Report on 2018 New Drug Approvals

9. Recent Hpcs Approval
8 of 16 approved
therapies are
HPC, Cord Blood 50%
Source: FDA Approved Cellular & Gene Therapy Products
HEMACORD by New York
Blood Center
HPC Cord Blood
ALLOCORD by SSM Cardinal
Glennon Children's Medical
HPC Cord Blood
CLEVECORD by Cleveland
Cord Blood Center
HPC Cord Blood
HPCs transplants are used for hematopoietic and immunologic
reconstitution in patients with blood disorders.

10. hematopoietic progenitor CELLS
Hematopoietic progenitor cells
(HPCs) or hematopoietic stem cells
(HSCs) are cells found in the blood
and bone marrow.
HPCs are capable of forming
mature blood cells, such as red
blood cells (that carry oxygen),
platelets (that help stop bleeding)
and white blood cells (that fight
infections).
Source: http://www.aabb.org/aabbcct/therapyfacts/Pages/hsc.aspx
Stem cells are cells that have the ability
to develop into a variety of different
specialist cells within the body.

11. HPCs are used in the treatment
of many malignant (leukemia,
lymphoma) and non-malignant
(sickle cell) diseases to replace or
rebuild a patient's hematopoietic
system*.
This treatment is called bone
marrow or stem cell transplant.
HPCs also have been used in
clinical trials for the treatment of
autoimmune diseases, genetic
diseases, and others.
hematopoietic progenitor CELL Therapy
Source: http://www.aabb.org/aabbcct/therapyfacts/Pages/hsc.aspx
*Haematopoiesis
is the formation
of blood cellular
components.
All cellular blood
components are
derived from
haematopoietic
stem cells.

12. Gene therapy for Hematologic Diseases
Hematologic diseases, disorders
of the blood and blood-forming
organs, affect millions of
Americans.
One of the more devastating and
difficult to treat is Acute
Myelogenous Leukemia (AML) –
cancer of the blood and bone
marrow. Epigenetic-based
therapies offer the possibility of
treating AML by targeting only
mutated cells.
Source: http://www.imagobio.com/research/hematologic-neoplasia/
2017:
Approximately
21,000 new cases
of AML were
reported in the US.
Treatments for
AML have changed
very little in the
past 30 years.

13. CBER Highlights: Cancer
IMLYGIC
by BioVex (Amgen)
Treatment of unresectable (cannot be
removed via surgery) cutaneous,
subcutaneous, and nodal lesions in patients
with melanoma (skin cancer).
YESCARTA
by Kite Pharma
Treatment of Large
B-Cell Lymphoma
(blood cancer in
the lymph nodes).
KYMRIAH
by Novartis
Treatment of B-cell
acute lymphoblastic
leukemia, cancer in
which the bone
marrow makes too
many white blood
cells (lymphocytes).
PROVENGE
by Dandreon
Corp.
Treatment of
prostate cancer.
Source: FDA Approved Cellular & Gene Therapy Products

14. GINTUIT
by Organogenesis
Treatment of
mucogingival
conditions in
adults. It is a type
of cellular sheet to
predictably
generate oral soft
tissue.
CBER Highlights: Other Therapies
MACI
by Vericel Corp.
Repair of full-
thickness
cartilage defects
of the knee.
LUXTURNA
by Spark Therapeutics
Virus vector-based gene therapy for the
treatment of mutation-associated
retinal dystrophy.
Source: FDA Approved Cellular & Gene Therapy Products

15. outlook for cellular & gene Therapy
Globally, the cellular & gene
therapy market was valued
at $6.02 billion in 2017.
Market players are actively
investing in research for
developing new and
improved therapies. These
products will address critical
unmet needs of patients.
Source: https://www.globenewswire.com/news-release/2019/02/05/1710761/0/en/Global-
Cell-and-Gene-Therapy-Market-to-Surpass-US-35-4-Billion-by-2026.html
Global cell therapy market size valued at
$6.02 billion in 2017. Global gene therapy
market valued at $584 million in 2016.

16. Market Trends & Projection
Market
Projection:
The cellular &
gene therapy
market is
projected to
surpass $35.4b
by 2026.
CAGR of 21.9%
from 2018-2026.
Source: https://www.globenewswire.com/news-release/2019/02/05/1710761/0/en/Global-
Cell-and-Gene-Therapy-Market-to-Surpass-US-35-4-Billion-by-2026.html
Research &
Development
Increasing R&D
activities of cell
and gene therapy.
Increasing
Prevalence
Cardiovascular
disease, cancer,
and genetic
disorders.
Increased Support
Introduction of effective
guidelines, rising funding
by governments and
organizations.

17. RadioPharmacy: Nuclear Medicine
Radiopharmacy employs radioactive
materials to diagnose & treat
specific diseases.
It consists of a radioactive isotope
and a pharmaceutical. The
accumulating signals form an image.
The global radiopharmaceutical
market was valued at $5.8 billion in
2016 and is estimated to reach $10.5
billion by 2023.
Source: https://www.marketwatch.com/press-release/radiopharmaceutical-market-2019-
global-sharetrendsegmentation-and-forecast-to-2023-2019-04-08
The rising incidence of chronic diseases
translates into the demand for diagnostic
tests such as SPECT and PET scans.

18. • Cellular & Gene therapy is the use of cells and genes to treat
disease. Cell therapy uses cells that are taken either from the
patient themselves or a donor to treat diseases, whereas gene
therapy employs genetic material to treat genetic diseases.
• These therapies are applicable to a broad range of diseases.
Given enough research and funding, they can radically treat the
causes of the diseases instead of only relieving the symptoms.
• Initial research show that such therapies can be effective on a
wide range of previously untreated diseases, such as
hematological, ocular, neurodegenerative diseases, and several
types of cancers.
Conclusion
Source: Allied Market Research

19. By xeraya capital
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www.xeraya.com