This document discusses the significance and applications of RNA technologies in life sciences, particularly highlighting their roles in vaccines, therapeutics, and agriculture. It covers the history of mRNA, the impact of COVID-19 mRNA vaccines, and advancements in RNA therapies for various diseases. Additionally, it introduces innovative RNA-based solutions for crop biocontrol with minimal environmental impact.

1. Potential Applications in Vaccines, Therapeutics & Agriculture
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RNA Technologies in Life Sciences
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2. RNA Technologies IN LIFE SCIENCES
Contents Overview
• RNA & 3 Major Types
• Discovery to Applications
• COVID-19: Rise to New Class
of Drugs
• mRNA in Vaccines
• mRNA in Therapeutics
• Challenges in Agriculture
• RNA-based Biocontrol in
Agriculture
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3. What is RNA?
Source: https://www.genome.gov/genetics-glossary/RNA-Ribonucleic-Acid
Ribonucleic acid (RNA) is a
molecule that is similar to
deoxyribonucleic acid (DNA).
While DNA is double-stranded,
RNA is single-stranded. Its
backbone is made of alternating
sugar (ribose) and phosphate
groups.
Attached to each sugar is one of
four bases: adenine (A), uracil (U),
cytosine (C), or guanine (G).
Credit: Khan Academy
3 July 2021. © Xeraya Capital.

4. 3 Major Types of RNA: Protein Synthesis
Source: https://www.britannica.com/science/RNA
4
tRNA
• Transfer RNAs
• Molecules that carry amino
acids (building-blocks) to
ribosomes in order to
synthesize proteins
mRNA
• Messenger RNAs
• Strand that carry specific
codes from the DNA to the
ribosomes (sites of protein
synthesis)
rRNA
• Ribosomal RNAs
• Molecules that are part of
the ribosome (sub-units)
which translates mRNA into
designated protein
Credit: Encyclopaedia Britannica
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5. Other Types of RNA
Source: https://www.news-medical.net/life-sciences/-Types-of-RNA-mRNA-rRNA-and-tRNA.aspx
5 July 2021. © Xeraya Capital.
Several varieties of RNA exist that are involved in post-transcriptional modification,
DNA replication, and gene regulation.
Small Nuclear
RNA
Regulatory RNAs
Transfer-
messenger
RNA
RNA Enzymes
Double-
stranded RNA
snRNA:
involved in
processing of
pre-mRNA into
mature mRNA.
miRNA:
Micro RNA,
acts through
interference.
Blocks or
breaks down
paired mRNA.
siRNA:
Small
interfering RNA
often produced
by breakdown
of viral RNA,
there are also
endogenous
sources.
asRNA:
Antisense RNA,
also known as
antisense
oligonucleotide
(ASO)
regulates gene
expression
tmRNA:
Tag proteins
encoded by
mRNAs lacking
stop codons for
degradation,
prevent
ribosome from
stalling
Ribozymes:
exhibit features
of a classical
enzyme: an
active site, a
binding site for
substrate and
binding site for
cofactor
dsRNA:
has 2 strands
bound
together.
dsRNA forms
the genetic
material of
some viruses
150 nt
(nucleotides)
21-22 nt 20-25 nt <200 nt (short)
>200 nt (long
325-400 nt Varies Ideal 60 bp
(base pairs)

6. mRNA: From Discovery to Applications
6
Sydney Brenner & colleagues
discovered an unstable
intermediate molecule that
copies encoded DNA and
directs protein synthesis: RNA.
1961
1969
Jerry B. Lingrel & Lockard
provided 1st evidence of
in-vitro translation; mRNA
was translated in the lab.
Paul A. Krieg & Melton provided 1st
evidence of in-vitro transcription;
mRNA was synthesized in the lab.
SP6 RNA polymerase eventually
commercialized.
1984
Source: https://the-dna-universe.com/2021/04/15/the-history-of-mrna-applications/
1990
Jon A. Wolff laid foundation
concept of mRNA as therapeutic
agent, injected naked RNA into
mice (direct gene transfer in-
vivo).
Scripps Research Institute used
mRNA to transiently reverse
diabetes insipidus in Brattleboro
rats that do not produce the
hormone vasopressin
1992
1995
Robert M. Conry & team
designed 1st mRNA
vaccine that encoded
cancer antigens.
2009
1st clinical trial of cancer
immunotherapy using
mRNA-based vaccines.
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7. COVID-19 mRNA Vaccines
Turning Point Giving Rise to New Class of Drugs
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8. 3 Keys to Life
8
Source: https://www.wbur.org/commonhealth/2021/01/11/modified-mrna-future-treatments
Credit: Art of the Cell
“DNA makes mRNA makes protein
makes life”
- Derrick Rossi, co-founder of Moderna
Moderna and Pfizer’s coronavirus vaccines were
the first medical products to use mRNA as a
technology, and they certainly won’t be the last.
Being able to control mRNA would allow
scientists to manipulate life in astonishing new
ways.
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9. Significance of RNA Technology
DNA is often described as the
building block of life but is
essentially a database of
information cells need to create
proteins.
RNA are the ones that does the work of
creating proteins. They are the pivotal
‘molecule of life’, involved in almost all
aspects of cell biology.
Source: https://www.wbur.org/commonhealth/2021/01/11/modified-mrna-future-treatments
Credit: Pearson Education
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10. MRNA Vaccine Highlights
Source: https://www.nytimes.com/interactive/2021/world/covid-vaccinations-tracker.html
10
Comirnaty by Pfizer & BioNTech was the 1st
coronavirus vaccine to be granted Emergency
Use Authorization. With an efficacy rate of 95%, it
is currently used 107 countries.
95%
Efficacy
Moderna’s mRNA-1273 was the 2nd coronavirus
vaccine granted EUA by the FDA. It is currently
used in 56 countries in the global vaccination
effort against COVID-19.
94.1%
Efficacy
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11. mRNA Therapeutics
Harnessing RNA for use in Medicine
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12. 3 Broad Categories of RNA Therapies
Source: https://www.nature.com/articles/d41586-019-03068-4
12 July 2021. © Xeraya Capital.
Most RNA therapies can be
sorted into one of 3 broad
categories:
1. Those that target nucleic acids
(either DNA or RNA)
2. Those that target proteins
3. Those that encode proteins
There are emerging hybrid
approaches that combine several
RNA-based mechanisms into a
single package.
Credit: Pharmacological Reviews

13. RNA Therapies For Rare Diseases
Source: https://www.nature.com/articles/d41586-019-03068-4
13 July 2021. © Xeraya Capital.
Inotersen Nusinersen Eteplirsen Patisiran Pegaptanib
Amyloidosis drug
for treatment of
nerve damage in
adults
Treatment of
spinal muscular
atrophy
Treatment of
Duchenne
muscular
dystrophy
Amyloidosis drug
for treatment of
polyneuropathy
(damage or disease
affecting peripheral
nerves)
Treatment for a
form of age-
related macular
degeneration
(blood vessels
penetrate retina)
FDA Approval: 2018 FDA Approval: 2016 FDA Approval: 2016 FDA Approval: 2018 FDA Approval: 2004
Single-stranded
antisense
oligonucleotides
(ASOs), tags mRNA
for degradation
ASO that alters
splicing (sculpting
process of precursor
mRNA into its
mature form)
ASO blocks mutated
portion of target
gene, resulting in
only functional
proteins
Small interfering
RNAs (siRNAs) or
microRNAs, to
degrade mRNA
(preventing
translation)
Use of RNA aptamer
that binds to and
block a specific
protein to block
vascular endothelial
growth

14. Potential RNA Therapies
In recent decades, ongoing
research includes:
- Use of RNAi to target a sequence
in the hepatitis C virus for
destruction
- Use of RNAi to suppress HIV
replication in macrophages
- Use of RNAi-mediated gene
silencing in humans is reported in
a phase I trial in people with the
skin cancer melanoma
Source: https://www.nature.com/articles/d41586-019-03068-4
Credit: C. Goldsmith
14 July 2021. © Xeraya Capital.

15. RNA IN Agriculture
Targeted Biocontrol with Minimal Impact
15 July 2021. © Xeraya Capital.

16. Global Food Challenge
Source: https://www.cabi.org/projects/global-burden-of-crop-loss/
16 July 2021. © Xeraya Capital.
Worldwide, an estimated
20-40% of crop yield is lost to
pests and diseases every
year, hindering progress in
several of UN’s Sustainable
Development Goals.
Many destructive insects, weeds,
viruses and fungi have developed
resistance to pesticides. Farmers
have fewer options for pest
control.
Credit: International Rice Research Institute

17. GreenLight Biosciences: Targeted Biocontrol
An RNA-based solution can
be applied to a plant and can
silence a specific gene in the
pest that is critical to its
growth (this is done without
any genetic modifications).
The pest within the immediate
area is eliminated, and the plant
stays healthy without any negative
impact on the environment.
Source: https://www.nature.com/articles/d41586-019-03068-4
The Colorado Potato Beetle. Credit: Phys.org
17 July 2021. © Xeraya Capital.
Greenlight Biosciences has developed a dsRNA
(double-stranded RNAs) product that can
target a specific pest via RNA interference. It
has been tested in over 20 crop fields.

18. • RNA was first discovered as an intermediary molecule
between DNA and cellular proteins. While DNA is essentially a
database of information cells need to create proteins, RNA
are the pivotal ‘molecules of life’, involved in almost all
aspects of cell biology.
• The COVID-19 pandemic and the recently approved mRNA
vaccines have brought RNA technology into focus, with
potential applications in more vaccines and therapeutics
against other diseases (rare or common).
• Recent discoveries have led to the use of RNA as an effective
biocontrol for crops, with minimal impact on the environment.
Conclusion
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19. By xeraya capital
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