Insights Care has curated a list of “The 10 Most Innovative Companies in Gene Therapy”, we are highlighting the stories of the leading companies that are catalyst to bring this revolutionary change.

1. Douglas Fambrough
Co-founder, President
& CEO
The Forefront of a Potential Revolution in Medicine
THE 10 MOST INNOVATIVE COMPANIES IN GENE THERAPY2019/Vol.9/Issue-1

4. September, 2019
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Co-designer Paul Belin
Art & Picture Editor Asha Bange
Art & Design Head Amol Kamble
Editor-in-Chief Pooja M. Bansal
Managing Editor Aishwarya Nawandhar
Senior Editor Anish Miller
Visualiser David King
Poonam MahajanArt & Design Assistant
Circulation Manager Tanaji
Research Analyst Eric Smith
Mark WilliamsBusiness Development Executive
Peter CollinsBusiness Development Manager
Sales Executives Sarah, Bill, John, Amy
SME-SMO Executives Manoj, Gemson
Digital Marketing Manager Alina Sege
Technical Consultants David, Robert
Technical Head Jacob Smile
Marketing Manager Marry D’Souza
Assistant Technical Head Pratiksha Patil
Assistant Digital Marketing Manager Prashant Chevale
Executive Editor Shambhavi Mhetre

5. Is Gene
Therapy the
Next Big Miracle
in Medicine?
Editor’s Column
rom the discovery of Penicillin to the use of numerous
Fantibiotics to help treat patients today, we have come a long
way. Back then, Penicillin was the miracle drug that treated
infections considered as fatal and had no probable cure before this
magic drug was invented. Recently, however, the stories of antibiotics
resistance, emergence of new, dangerous diseases, have hit the
headlines. Along with this, the story of the new-age miracle of
medicine- Gene Therapy, is also reaching a number of people. So, what
is gene therapy? Unlike the medicines that slow down the attack of
diseases on our bodies, gene therapy is a method of treating or
replacing the infected or non-functioning genes so as to end the disease
at its root. Although it was first discovered in the 1980s, it is still in its
infancy stage.
Even then, there have been a number of clinical trials conducted and
many success stories of not just treating, but curing some life-
threatening and chronic diseases, that were not treatable before this. A
toddler in UK, born with an inherited immune system disorder, under
went a pioneering treatment based on gene therapy. After this, he was
watched closely for a few years and as if his parents’ prayer was
answered, his immune system has been working normally. Apart from
this, in some parts of the world, gene therapy has been leveraged to
treat the inherited vision loss for the patients.
Another interesting story reported recently was of the two brothers
suffering from haemophilia, a genetic disease in which the body lacks
the ability to clot the blood, even from minor wounds. Although, the
treatment for it is available today, it reduces the quality of life, the
patient’s freedom to do things and also reduces life-expectancy. Both
the brothers enrolled for a clinical trial they heard of, which was related
to a gene replacement technique with a possibility to aid cure
haemophilia. The patient’s affected genes were removed and corrected
and then transported back in the body using AAV virus. However, one
of the brothers already had antibodies against AAV Virus in his body.
The second brother is, today, living a healthy life, with his body
producing the protein needed to clot the blood on its own.
There are a large number of such cases around the world where gene
therapy has come as an answer to several unmet medical conditions.
Although it shows a lot of potential for revolutionizing the future of
healthcare, there have been cases where the patients’ developed a side-
effect resulting in severe health conditions or even death. To exploit the
benefits that gene therapy has to offer rightly, more research and better
solutions need to be brought in. Also, the therapeutics based on this
novel technique should be made affordable and accessible to all for it to
bring a radical change in people’s lives!
Aishwarya Nawandhar

6. Article
24
Into the DNA
Gene Therapy:
Changing the DNA of
the Healthcare Space
Cover StoryCO
EN
NT
TS
TM
DicernaThe Forefront of a Potential Revolution in Medicine
08

7. 16
20
28
36
32
Atvio Biotech
Innovating and Developing Healthcare of Tomorrow
BioViva
Reversing the Cycle of Aging with Gene Therapy
Eukarÿs
The Future of Synthetic Gene Therapy
Gracell Biotechnologies
Conquering CAR-T Industry Hurdles with
Futuristic Solutions
VIROgene
Redefining the Future of Healthcare with Gene
Therapy

8. Augmenting Healthcare
with Novel Gene
Therapy Solutions
G
enes are what marks us different from every other person on this Earth. They contain DNA that defines the
form and functions of any human body and any kind of defect in this lead to diseases. Unlike the medicines,
gene therapy corrects these defects and stop the diseases once and for all. Additionally, medicines and
treatments till date work on a trial and error method, prescribed according to the symptoms a patient’s body shows.
However, every body type is dissimilar to one another and what may work for one person, doesn’t necessarily work
perfectly for the other. With gene therapy, personalized medicines, made specifically for the person affected by a
health condition, can be devised and this change from conventional healthcare to person-specific, will probably be the
greatest revolution the industry would witness.
In our issue of “The 10 Most Innovative Companies in Gene Therapy”, we are highlighting the stories of the
leading companies that are catalyst to bring this revolutionary change. They are not only dedicated to aid in creating
and offering therapeutics based on gene therapy, but also are working towards making it affordable for all.
TM
On the Cover, we have featured the inspiring story of Dicerna Pharmaceuticals. It is a biopharmaceutical
company using RNA interference, or RNAi, to create medicines that silence the genes that cause disease for patients
with rare genetic diseases and common disorders.
Apart from the cover story, the issue recognizes the contribution of the top companies that are transforming the
healthcare industry with their next-gen solutions. These include ATVIO Biotech, a specialty cell & gene therapy
(CTG) process development and innovation center; Gracell Biotechnologies, a clinical-stage biotech company with
mission to solve CAR-T industry hurdles; Eukarys, a French biotechnology company that provides novel therapeutic
solutions for the treatment of severe human disorders; VIROgene, a gene therapy startup that develops gene
therapies on viral technologies that are not based on mammalian pathogens, to offer alternatives with a higher level of
biosecurity; BioViva, a medical data analysis and research and development company that is leveraging gene therapy
to accelerate the development of products that will effectively, and cost-efficiently treat biological aging; Oxford
Biomedica; Puresyn; iCell Gene Therapeutics; and MeiraGTx.
As you flip the pages, you will also find an interesting article on gene therapy from our in-house editor.
Happy reading!

10. Douglas Fambrough
Co-founder, President
& CEO

11. C O V E R S T O R Y
The Forefront of a Potential Revolution in Medicine
We aim to build a fully
integrated company by
developing innovative
RNAi-based therapies
for high unmet medical
needs that reduce disease
burden for all patients.
“
“

12. F
TM
ounded in 2007 Dicerna Pharmaceuticals,
Inc. is a publicly traded (Nasdaq: DRNA) bio-
pharmaceutical company using RNA interference,
or RNAi, to create medicines that silence the genes that
cause disease for patients with rare genetic diseases and
common disorders. It is a leader in RNAi, harnessing the
potential of this powerful and complex process to
investigate therapeutics that can enhance health, improve
lives, and profoundly benefit patients with rare genetic
diseases and common disorders. Dicerna aims to use its
TM
proprietary GalXC RNAi technology platform to
develop therapies that are targeted, potent, and safe.
The Brain Behind Dicerna
Douglas Fambrough, the Co-founder, President, and
partner with Oxford Bioscience Partners, a Boston-based
venture capital firm. He co-created and served on the
Board of Directors in Sirna Therapeutics in 2003. On
behalf of Oxford, he invested in several ventures,
including Solexa, developer of the dominant ultra-high-
throughput DNA sequencing platform later acquired by
which Illumina, and Xencor, a leader in engineering
antibodies to improve their therapeutic properties.
How it Started
Following his scientific work, Douglas entered the VC
industry with a goal of converting genomic technology
and knowledge into useful medical advances. Pursuant to
that goal, in 2003, he and two other investors created
Sirna Therapeutics, a first-generation RNAi pioneer
Chief Executive Officer of Dicerna, is a genomic
scientist and venture capitalist and has been instrumental
in the evolution of the company from the very start. He
obtained his PhD in genetics from University of
California, Berkeley, and was a researcher at the
Whitehead/MIT Center for Genome Research (now
known as the Broad Institute). Throughout his career, he
has worked in genetics to better understand the human
genome and what causes disease. Douglas has led the
company effectively, from the development of its unique
GalXC platform, to Dicerna’s transformation into a
successful, publicly traded company.
Prior to joining Dicerna, Douglas specialized in financing
innovative life science technology companies as a general
which was eventually acquired by Merck in 2006.
Following the acquisition, Douglas decided to create
Dicerna Pharmaceuticals to continue his quest to develop
an RNAi therapy to address diseases with high unmet
medical needs. This aim to reshape the healthcare
industry by harnessing the power of genetic engineering
ignited the flame of Dicerna.
“Our vision is to build a fully integrated company that
delivers innovative RNAi-based therapies to address high
unmet medical needs. Ultimately, this will enable us to
reduce disease burden for all patients while broadly
capturing the value of our GalXC platform,” asserts
Douglas.
We believe all patients
deserve safe and effective
medicines. Urgent patient
needs drive our work to
create new medicines with
the potential to restore health.
“
“

13. Futuristic Solutions and Services
Dicerna has invented the RNAi technology platform
called GalXC, which is a proprietary technology platform
that advances the development of next-generation RNAi
based therapies designed to silence disease-driving genes
in a broad range of serious diseases, including rare
genetic diseases and common disorders. Dicerna is
advancing a growing pipeline of product candidates to
deliver transformative therapies to patients and currently
has three GalXC programs in clinical trials and several
more in early-stage development.
Dicerna’s three clinical stage programs are DCR-PHXC,
DCR-HBVS, and DCR-A1AT. DCR-PHXC is the only
investigational RNAi drug in development for the
communities, policymakers, patients, and patient
advocates. Dicerna has an open office plan in its current
Cambridge headquarters, its new Lexington headquarters
under construction, and its Boulder facility, that allows
for the free exchange of ideas between people and teams.
The Novel RNAi Technology Platform
Dicerna’s proprietary GalXC platform allows
development of RNAi therapies that are targeted, potent,
and safe. The Dicerna GalXC molecules are structured to
be processed by the enzyme Dicer, the initiation point for
RNAi in the human cell cytoplasm. Unlike earlier
generation RNAi molecules, which mimic the output
product of Dicer processing, Dicerna’s DsiRNAs,
including GalXC molecules, enter the RNAi pathway
treatment of all three genetic types of primary
hyperoxaluria (PH), a family of rare liver metabolic
disorders. DCR-HBVS is being developed for the
treatment of patients with chronic hepatitis B virus
(HBV) infection and DCR-A1AT is being developed for
patients with alpha-1 antitrypsin (A1AT) deficiency-
associated liver disease.
The collaborative culture of Dicerna sets it apart from its
contemporaries. It operates with a “no walls” philosophy.
The company breaks down the barriers that slow the
progress in rare diseases, like limited research, data,
knowledge, and treatments. Additionally, it is committed
to collaborating with the best and brightest, partnering
with employees, industry, the medical and research
prior to Dicer processing. By using the Dicer enzyme as
the entry point into the RNAi, Dicerna seeks to optimize
the activity of the RNAi pathway so that it operates in the
most specific and potent fashion.
Moreover, the GalXC RNAi platform does not involve
lipid nanoparticles (LNPs) or other formulation
components that facilitate drug delivery, simplifying the
platform and eliminating any requirement for functional
excipients. Instead, Dicerna’s GalXC molecules are
stabilized by chemical modifications and utilize a four
base sequence known as a tetraloop, where each base is
conjugated to a simple sugar, N-acetylgalactosamine
(GalNAc), that is specifically recognized by a receptor on
the surface of hepatocyte liver cells. The tetraloop
We strive to create medicines
using our GalXC RNAi
technology that silence the
genes that cause disease for
patients with rare genetic
diseases and common disorders.
“
“

14. configuration, which is unique to Dicerna’s GalXC
compounds, interfaces effectively with the RNAi
machinery, allowing flexible and efficient conjugation to
the targeting ligands, and stabilizing the RNAi duplex to
enable effective delivery of Dicerna’s GalXC RNAi-
inducing molecules directly to the liver.
Why RNAi?
Since the inventers of RNAi, Dr. Craig Mello and
Andrew Fire won the Nobel Prize in 2006, Dicerna’s
scientists have been studying RNAi and working to
optimize the technology in humans. As gene therapies
and gene editing become better understood in the
industry, the company is learning more and more about
RNAi technology and how to develop innovative
therapies. The scientists at Dicerna are experts in their
respective fields, from biology to chemistry to
experimental science, and more. Many of them present
and participate in leading scientific and medical
conferences and publish papers on RNAi and recent
advancements in the space. Specifically using the RNAi
process to treat disease, Dicerna leverages its several
important advantages over other approaches to drug
development:
· RNAi does its work earlier in the disease-causing
process. While some conventional therapies block the
activity of proteins responsible for disease, RNAi
therapies prevent these proteins from being created in
the first place.
· Unlike therapies that are too large to reach desired
targets, RNAi can reach any target, including some of
the most well-validated disease targets that are only
expressed inside cells.
· RNAi therapies can be considered a safer option than
permanently editing the genes in your DNA. Directly
editing the genes that cause disease can cause
unintended effects to the genes needed to maintain good
“
“
Dedication, Innovation,
Communication, Excellence,
Respect, Needs based,
Accountability- These are
the values that reect the
soul of Dicerna.

15. health. RNAi addresses this risk because it is reversible,
preventing protein building instead of editing the genes
themselves. RNAi therapies have the potential for fewer
side effects compared to small molecules and
antibodies.
· RNAi therapies also have the potential to reduce the
treatment burden for patients. Their long duration of
effect means they can be administered via an infrequent
injection under the skin, also known as a subcutaneous
injection.
Pivotal Benchmarks in its Journey
In its long and challenging journey of more than a
decade, Dicerna has seen and leveraged various
opportunities and overcome number of hurdles. On its
way to success, there have been some major milestones:
· November 2007 – Dicerna secured exclusive
worldwide license for Dicer substrate short interfering
RNA (DsiRNA), the inspiration for Dicerna's GalXC
RNAi platform.
· December 2011 – Dicerna received a U.S. patent for its
Dicer Substrate™ technology, a precursor to its current
GalXC platform.
· June 2016 – Dicerna introduces GalXC, its proprietary,
next-generation, RNAi technology platform.
· November 2017 – Boehringer Ingelheim and Dicerna
announce research collaboration and license agreement
to develop novel GalXC RNAi therapies for chronic
liver diseases, beginning with non-alcoholic
steatohepatitis (NASH).
· May 2018 – Dicerna initiates PHYOX™ Phase 1
clinical trial of DCR-PHXC in patients with primary
hyperoxaluria type 1 (PH1) and type 2 (Ph2).
· September 2018 – Dicerna presents successful proof-
of-concept data for DCR-PHXC for the treatment of
primary hyperoxaluria.
· October 2018 – Eli Lilly and Dicerna announce a global
licensing and research collaboration focused on creating
new medicines in cardiometabolic disease,
neurodegeneration, and pain. Alexion Pharmaceuticals
and Dicerna announce a collaboration to create RNAi
therapies for complement-mediated diseases.
· January 2019 – Dicerna initiates first dosing in Phase 1
clinical trial of DCR-HBVS for the treatment of
hepatitis B virus infection.
· July 2019 – Dicerna submits a clinical trial
authorization application to conduct a first-in-human
Phase 1/2 study of DCR-A1AT for the treatment of
alpha-1 antitrypsin (A1AT) deficiency-associated liver
disease.
· July 2019 – Dicerna doses the first patient in the
PHYOX3 clinical trial.
Valuable Collaborations
Dicerna has active collaborations with three major
biopharmaceutical companies: Eli Lilly and Company,
Alexion Pharmaceuticals, and Boehringer Ingelheim
International GmbH. All three companies are
collaborating with Dicerna to apply the GalXC
technology to therapeutic target genes to create new
potential therapies for a variety of disease conditions. In
total, more than $250 million in upfront payments,
milestone payments, and equity investments have been
made by these collaborators to Dicerna.
Looking into the Future
Dicerna is pursuing its vision with a clear and focused
dual strategy. First, the company will pursue
opportunities that address high unmet medical needs and
that it foresees yielding both clinical and commercial
success. The company’s clinical pipeline currently
centers on two rare diseases: primary hyperoxaluria and
alpha-1 antitrypsin deficiency-associated liver disease.
The company plans to drive development and
commercialization, either wholly or largely on its own,
for both of these rare diseases. The other program is for a
prevalent disease, chronic hepatitis B virus infection, for
which it is seeking a development and commercialization
partner once it has the Phase 1 proof-of-concept data.
Secondly, Dicerna aims to realize the potential of its
technology against all remaining targets through
collaboration and discovery stage licensing agreements
with therapeutic area leaders like Eli Lilly, Alexion, and
Boehringer Ingelheim.
Dicerna plans to own certain assets in the rare disease
space that fit within the company’s strategy and will work
with leading pharmaceutical partners to collaborate on
assets for larger disease populations.

18. ATVIO BIOTECHInnovating and Developing Healthcare of Tomorrow
Advanced cell and gene therapy is the future of
medicine. It brings new hope and solutions for
some severe diseases where conventional medicine
fail to provide answers. Not just that, it has the potential to
change the entire process of treatment in healthcare and for
better. But the complexity, cost, and sensitivity involved in
its production restrict its medical use. Overcoming these
challenges will be a revolutionary breakthrough in the field
of medicine. Turning this imagination into reality, Atvio
Biotech Ltd. is redefining the future of healthcare.
Atvio is a specialty cell & gene therapy (CTG) process
development and innovation center. The company is a
unique blend of scientists, biotech engineers, GMP experts,
and engineering expertise, aiming to bridge the gap
between science and technology. It believes that CTG is the
future of medicine and without new and unique
innovations, which will enable bringing the science to the
patients, the industry will never deliver to its potential.
Atvio is working towards making this a reality by
combining its expertise with the therapy developers and
innovating in the manufacturing space.
The Multifaceted Persona
Dr. Ohad Karnieli is the Founder and President of Atvio.
He is a big believer of CTG and its potential for helping
patients and has been in this industry for over two decades.
In addition to being a scientist, Dr. Karnieli always had an
attraction towards engineering and headed a medical device
division in an Israeli company. This experience changed his
perspective on what can be done with good engineers and
tools. He headed technology and manufacturing at a
prominent company where he got the chance to design a
state-of-the-art facility and technology with highly talented
team members. Like others, Dr. Karnieli identified the gap
translating the science to the clinic. The difference was that
he decided to take action and jump into the gap and build
the bridge that would enable CTG companies to cross
safely and bring their therapies to the patients.
One of his biggest achievements is “infecting” a group of
talented people with the vision of thinking differently and
innovating to make therapies accessible to patients. The
vision and spirit of helping bring lifesaving innovative
therapies to patients, every day, drives him and the whole
team of Atvio.
The Journey of Atvio
As the realization came to him, Dr. Karnieli set to influence
the industry and help get treatment to the patients by
extending his expertise not only to one company but to the
industry, holistically. He then left the position of VP at his
16 | September 2019 |

19. previous company and started to dream of building a
venture that would provide tools and development services
to make therapies accessible to all. That is when Atvio was
born!
Believing that CTG is the future and providing a unique
blend of scientific and development expertise with
engineering skills is critical, he built a team of experts.
However, getting the funds to start the company was not so
easy. Dr. Karnieli overcame this challenge but was
introduced to another one. Being based in Israel, Atvio
realized that the industry for cell and gene therapy lied
outside; in the US, the EU, and Asia. But Israel also brought
with it an advantage of being the best place in the world to
innovate with the best ecosystem and talent with a world
winning record. Converting all its challenges to
opportunities, Atvio has today emerged to be an excelling
company in its field.
The major breakthrough for Atvio was the presentation of
ADVA CART device, which has been developed by Atvio
for ADVA biotechnology. This device is a great example of
the company’s unique offering. It will revolutionize the
CART industry and will enable the therapy to patients all
over the world at high availability and low price. Such a
game-changing device could only be developed with the
unique amalgamation of people and expertise Atvio offers.
Hereafter, there was no looking back.
Futuristic Products and Services
Atvio allows cell therapy developers to focus on their
scientific and medical challenges and utilize the expertise,
tools, and knowledge it has, to get their product
manufactured at quality, scale, and price. It is difficult for
the companies to find all the needed experience and
expertise in such a complicated evolving industry and this
is where Atvio comes to their aid. It does everything, from
gap analysis via simple process improvements to
custom-made enabling solutions, such as the ADVA device
or tailor-made bioreactors to fit the specific need.
ADVA, a revolutionary product by Atvio, has brought to
reality the vision of a device where you insert blood from
one side and get CART from the other in a hospital. The
company leverages its potential to overcome the bug
challenges the industry faces.
What makes the company stand out is its constant
innovation, the unique blend of expertise, and the best and
We are here to
bridge science with the
technologies, by
turning great ideas
to revolutionary
cell therapy solutions
‘‘
‘‘
Dr. Ohad Karnieli
Founder & President
| September 2019 | 17

20. most dedicated team one can find. Adding to that is the innovative and scientific ecosystem in Israel. Talking about his team,
Dr. Karnieli asserts, “It’s all about the team! I have great team members who are dedicated, talented, and motivated to get the
cell therapy to the patients. This blend of talent makes us unique and offers something different and better than anyone
around.”
A Long Way to Go
Atvio’s latest focus is to build solutions such as the ADVA, which enable cost-effective, high-quality manufacturing at the
point of care. The company always looks for and adopts new technologies and dares to try something new and better, every
day. When asked about the future of Atvio, Dr. Karnieli says, “I see the company becoming stronger, validating our model
and solutions, and starting to integrate unique tools and solutions with our partners on their therapies. I do believe our
unique offering will be more prominent in the next few years, once people see the solutions we provide our partners.”
Atvio believes that good science is the base of all therapies but without innovative manufacturing solutions and good
development, patients will not be able to access them. This is exactly what the company is set to do.
18 | September 2019 |

22. OUR VISION
IS A WORLD
IN WHICH
INNOVATION
AND SAFETY
COME FIRST
AND THROUGH
FAST ACTION
WE CREATE
A WORLD
WITH LESS
SUFFERING
“
“
Elizabeth Parrish
Founder & CEO
20 | September 2019 |

23. BioViva, a trail-blazing medical data analysis and
research and development company, was started to
change the face of healthcare treatments by
bringing enhanced and definitive solutions for some of the
most perilous diseases. The inspiration came from a life-
changing incident when the founder’s son was diagnosed
with Type 1 Diabetes. On the path to find the most adequate
treatment for him, she was confronted with the reality of
children suffering from genetic diseases. She also
discovered that, although laboratory animals have been
successfully treated for many of these diseases, those
therapies haven’t yet been made available to humans,
because of the medical profession’s exaggerated risk
aversion and the FDA’s precautionary attitude. This meant
that despite the discovery of appropriate therapies, the kids
die because these therapies have not yet been through a
lengthy and extremely expensive chain of paperwork and
clinical trials. Determined to change this condition, she
established BioViva.
Today, BioViva analyzes biomarkers from trials and studies
involving patients referred to the clinics by its partner
company, Integrated Health Systems. The company is
focused on speeding up the number of medical innovations
by cooperating with companies that offer consenting
patients pioneering therapies that they cannot get in the
United States. Using medical researchers to oversee the
patients, it beats the gold standard of the US FDA and
creates exponential technology for the future. BioViva is
the first company in the world to look at the data of
regenerative gene therapies in humans.
The Multi-faceted Kingpin
Elizabeth Parrish, the Founder and CEO of BioViva, is a
humanitarian, entrepreneur, innovator, author, podcaster,
and a leading voice for genetic cures. As a strong proponent
of progress and education for the advancement of
regenerative medicine modalities, she serves as a
motivational speaker to the public at large for the life
sciences. She is actively involved in international
educational media outreach. Dedicated to the cause of
improving and safeguarding more and more lives, Elizabeth
asserts, “We are focused on saving as many lives as
possible by making tomorrow’s therapies available. This is
about Life and Death. Risk aversion and the delaying the
approval of therapies that have been successful in research
simply kills.”
Revolutionary Products
BioViva provides gene testing kits that allow customers to
learn information about their own genome from various
perspectives, such as their biological age, risk for various
diseases, etc. It is the only company in the world that has
access to data from patients who are taking regenerative
gene therapies. Additionally, the company is launching a
multi-omics repository for clients to upload their data,
regardless of what company they obtained it from, allowing
the customer to have all their health data in one place.
BioViva works in partnership with Integrated Health
Systems to help people access advanced therapeutics.
Concentrating on research and development, it is designing
Reversing the Cycle of Aging with Gene Therapy
| September 2019 | 21

24. a new gene therapy delivery method to solve the very
important problem of cell delivery of genes.
The company also has an explicit focus on aging as a
disease, because, according to BioViva, aging is a disease
that underlies many others. “Time is your most valuable
asset, we want you to have more of it. Creating a world with
longer healthier lifespans gives us time to solve climate
change and to come together as a species to solve social
impact and reduce racism,” says Elizabeth.
Shrewd Insight on Gene Therapy
BioViva believes that gene therapy offers a permanent cure
for a variety of diseases; it is the answer to monogenic
diseases, such as progeria, and cystic fibrosis, as well to
hemophilia b and sickle cell anemia. However according to
the experts at BioViva, it also offers promise in the case of
more complex diseases involving multiple genes.
Addressing the same, Elizabeth says, “I regard aging as the
master disease in this respect. By changing a set of genes
such as the gene coding for the enzyme telomerase or the
protein inhibitor follistatin, you can alter a person’s
biological age, and thereby make him or her healthier,
because youth is health, and old age is sickness. Aging is
cellular degeneration over time, with gene therapy we can
make the cell regenerate faster than it degenerates. I
therefore regard gene therapy as the future, because it will
address the scourge of aging with increased success, and
we are working to offer first generation therapies to people
who need them now.”
Into a Bright Future
BioViva has currently funded the Rutger’s University to
work on developing a better vector for future gene therapy
than the existing solution. It foresees that this project will
be almost ready for future use in next five years.
Additionally, the company hopes to have amassed the data
that shows how different gene therapies, at least ten of them
by 2024, have worked in humans, in the next five years.
22 | September 2019 |

26. ENE
THERAPYCHANGING THE DNA OF THE HEALTHCARE SPACE
Into the DNA
24 | September 2019 |

27. Many questions arise on
how amazing science is
and how complicated
nature can be! The human anatomy is
created in a way that is very
complex. There are around 7.7
billion people on the earth today and
every single person is different from
one another. Each individual has
approximately 3 billion pairs of
nucleotides and about 25,000 genes
per cell. The base of any tissue is
cells and the building blocks of all
the cells are the nucleotides.
Interestingly, the genes contain DNA
that basically controls all the
functions of a person. From the
formation of various organs to the
functioning and regulation of
different parts of the body is possible
due to the proper functioning of these
small molecules. The genes are
inherited by both the parents that
make us stand out of the crowd. The
uniqueness of an individual is
reflected in all the aspects like
physical traits including eye, skin,
and hair color.
Along with these traits, DNA is the
main reason for a person’s behavior.
There is an interesting question about
how similar is human DNA? The
answer to this is, 99.9% of the DNA
composition of the human race is the
same. However, what distinguishes
| September 2019 | 25

28. us from each other is that 0.1%. Each
person has a particular DNA
sequence which is never similar to
any other creature on this planet.
There are many diseases and
disorders caused mainly due to
variations in the genes. Even a single
mutation or change in the whole
genome sequence can lead to some
disorder. For a long time, researchers
have been trying to find a technique
that can help change these variations.
An upcoming technique has given
some hopes for solving this age-old
problem. Gene therapy is an
experimental technique that uses
particular genes to treat or prevent
the disease. Though gene therapy is
at its infancy stage, it has been
showing some promising results in
various clinical trials. The treatment
of inherited diseases or physical
conditions can be a reality in the
coming future with the help of gene
therapy. This technique is used to add
a new gene into the patient’s cell that
is said to replace the missing or
malfunctioning gene with a healthy
copy of the gene. It may also be
useful in inactivating a particular
gene that has been causing a
problem.
There are mainly two types of gene
therapy currently known that are
being used in clinical trials all around
the globe:
· Somatic Gene Therapy:
In somatic gene therapy, usually, a
piece or a section of DNA is
transferred to the desired cell that is
not involved in the production of
eggs or sperm. The section of DNA is
directly inserted in the body cells like
the bone marrow. In this type of gene
therapy, the effects of the process are
not passed to the next generation.
· Germline Gene Therapy
Germline Gene Therapy uses a
section of DNA that is transferred to
the cells which are involved in the
production of eggs or sperm. The
effects of this therapy are also seen in
future generations.
Gene therapy is practiced by three
main techniques. Here are three
techniques of gene therapy that are
used:
· Gene Augmentation Therapy:
Various diseases that are caused by
mutations are in the process of being
treated by the gene augmentation
therapy. In this, a gene is added to a
cell that has a non-functioning gene.
After the addition of this new gene,
the cell functions normally,
thereafter. A common example of
gene augmentation therapy being
used is for the treatment of cystic
fibrosis.
· Gene Inhibition Therapy:
There are a few infectious diseases
that are caused by the malfunctioning
of a particular gene. Gene inhibition
therapy is then used by introducing a
new gene that blocks the faulty gene
that leads to the proper functioning
of the cell and ultimately treating the
disease. A very good example is the
treatment of oncogene that is the
leading cancer-causing gene.
· Killing Specific Gene Therapy:
This technique is rather different. As
the name suggests, the technique
kills a specific gene that is causing
trouble. There are two ways in which
a diseased cell can be destroyed.
· In this type, a suicide gene is
inserted into the cell that ultimately
produces a toxic product leading to
the death of the cell.
· While in the second type, a marker
gene is added so those marker
proteins are exposed on the surface.
The immune system then attacks
the marker proteins that will
destroy the cell later.
It is said that diseases like cancer can
be cured by this method. Ultimately,
the cells die that are causing such
diseases and disorders.
This is a promising technique,
though it has many challenges during
the process. The main challenge is
placing the gene in the right place.
The crucial step after placing the
gene correctly is switching it on. If
the gene is placed in a wrong
position in the cell, the gene will be
ineffective and may cause severe
health issues. While most of the time
cells perform normally, it may
happen that cell shuts down the
genes. Sometimes, the new genes are
treated as a potential harmful
intruder. If the genes are misplaced,
then they interfere with the
functioning of the other genes.
Although, there are many challenges,
there is a wide range of potential
applications of gene therapy in the
treatment of various diseases.
Diseases like cancer, cystic fibrosis,
heart diseases, AIDS, hemophilia,
diabetes, etc can be treated by this
method.
- Shambhavi Mhetre, Editor
26 | September 2019 |

29. | September 2019 | 27

30. 28 | September 2019 |

31. Headquartered at the Evry Genopole, Eukarÿs SAS
(eukaryotic universal expression system) is a
French biotechnology company. It was created to
develop products and applications from the C3P3
expression system, including a radically novel therapeutic
approach named synthetic gene therapy, and the
development of C3P3 cellular systems to increase protein
and virus bio-production yields.
Eukarÿs developed the first ever non-viral eukaryotic
expression system named C3P3 (chimeric cytoplasmic
capping-prone phage polymerase) from the scratch. This
artificial system generated by synthetic biology allows the
autonomous production of messenger RNA (mRNA) in at
high yields, and therefore the proteins of interest. The use
the C3P3 system is decisive for the approach of synthetic
gene therapy as it determines the effectiveness.
Eukarÿs' synthetic gene therapy is based on a proprietary
synthetic DNA production system and the C3P3 system,
which determines its safety, efficacy, and good tolerance, as
well as its production at a reasonable cost. In addition, the
structure of the synthetic DNA used in synthetic gene
therapy allows its use not only for the rare genetic diseases,
but also the most frequent multifactorial diseases for which
multiple genes or pathways should be targeted.
Based on this generic synthetic gene therapy technology,
Eukarÿs is developing a pipeline of treatments for severe
diseases without therapeutic solutions. Its first treatments
are targeted on liver diseases. The company's EUK-LPR,
the first pro-regenerative treatment, which is the most
advanced gene therapy treatment. It makes it possible to
induce the regeneration of this organ before liver resection
surgery. The first treatment trial of this on animals
demonstrated its excellent efficacy and Eukarÿs has
launched two new programs, one for liver diseases and the
other for cancer therapy, which will be announced soon.
The Leading Light of Eukarÿs
Philippe JAÏS is the President and Scientific Director of
Eukarÿs. He has a rich academic background with the
degrees of MD, PhD, and MSc in physiology, molecular
genetics, and biostatistics, respectively. He is a Hepato-
Gastroenterologist and has specialized in molecular
genetics of cancers as a PhD student and positional cloning
as a post-doctoral fellow. He has served at various positions
in a plethora of companies in the biotechnology industry,
pharma companies in research, genetics,
pharmacogenomics, translational medicine, genomic
biomarkers, and early clinical development.
Philippe has designed and realized the early clinical
development and biomarker strategy of many small
chemical molecules and therapeutic proteins. He also
invented an approach for large-scale identification of human
monogenic pathologies treatable by synthetic chemical
ligands of nuclear receptors. In 2009, Philippe invented the
C3P3 technology that he developed until its proof of
concept, before cofounding Eukarÿs. He has published
more than fifty articles, books and abstracts, and is the
inventor of several patents including all those of the
company.
“
“
| September 2019 | 29

32. The Ingenious Solution
Eukarÿs has developed a unique non-viral, non-infectious,
and non-integrative named synthetic gene therapy
approach. This synthetic gene therapy exploits the C3P3
system and determines the effectiveness. In addition, the
artificial DNA used for the synthetic gene therapy by
Eukarÿs explains its safety, good tolerance, as well as its
production at a reasonable cost. Moreover, the modular
structure of these treatments makes them usable with
minimal adaptation for the majority of human diseases,
including monogenic and multifactorial disorders, which is
virtually impossible for most existing gene therapy
methods.
Although usable for the treatment of many organ diseases,
the initial goal of the company is the creation of a pipeline
of synthetic gene therapy treatments for severe liver
diseases and diseases related to deficiencies in secreted
proteins, with significant unmet medical needs. They have
now extended this pipeline to oncology for the treatment of
very frequent cancer.
In a business prospective, Eukarÿs develops its own
pipeline, but is open to partnerships with Pharma and
Biotech companies. The most interesting factor of the
company which differentiates it from the rest is that all its
technologies were developed from scratch and are under
constant evolution.
Why Synthetic Gene Therapy?
“Today, gene therapy can only be used for some ultra-rare
and ultra-severe diseases in some ultra-rich countries. Our
ambition is to make synthetic gene therapy accessible to a
large number of patients for a large number of diseases
today without a therapeutic solution,” says Philippe.
Eukarÿs' synthetic gene therapy approach excels all others.
Gene therapy needs to be safe: The risk of integration of
virus leading to cancer limits the use of viral gene therapy
to ultra-severe disorders for which the risk is acceptable.
Eukarÿs synthetic gene therapy is devoid of this risk.
Gene therapy needs to be well-tolerated: Viral gene
therapies, as well as the synthetic RNA are usually poorly
tolerated, which is a significant drawback, especially for
chronic disorders. Eukarÿs gene therapy uses a specific
design of the synthetic DNA, which explains the good
tolerance of synthetic gene therapy and allows its long-term
uses.
Gene therapy has to be efficient: The classical non-viral
gene therapy and the synthetic RNA are notably inefficient
because the amount of target mRNA produced or uptaken
by the cells with these technologies is drastically low. The
synthetic DNA exploits the performances of the C3P3
expression system, which is a proficient system to produce
high amounts of target mRNA in the cells.
Gene therapy should economically accessible: Today, the
cost of production of a single dose for patient of
recombinant virus is ranging from €200,000-€600,000! In
addition, due to the immunogenicity and/or the episomal
nature of the viral gene therapy, re administration of viral
gene therapy is probably needed every few years, which
increases its costs further. In contrast, the synthetic DNA
used for Eukarÿs' synthetic gene therapy can be produced at
reasonable cost.
Gene therapy should be used for a broad number of
diseases and not some ultra-rare monogenic disorders: This
is not only due to cost of viral gene therapy, but also due to
technical limitations, since only one small genes can be
used for viral vector constructions. In contrast, the synthetic
DNA used by Eukarÿs can assemble several genes with no
length limitations. Therefore, Eukarÿs' synthetic gene
therapy can be used not only for the rare genetic diseases,
but also the most frequent multifactorial diseases.
Awards and Accolades
Eukarÿs has won several awards through the years. This
includes, French Competition “Concours de Création
d'Entreprises de Technologie Innovantes” in 2009 and a
second time in 2011 in the Creation category, which is the
highest possible reward, Techninov award, and Ubistart
award. Additionally, the company was also labeled as Young
Innovative Company by the French Ministry of Research
and Innovative Biocluster Enterprise by Polinvest's from
the Ile-de-France Medicen Competitiveness Cluster.
What's Ahead?
Eukarÿs aims to demonstrate the efficacy of the synthetic
gene therapy in phase I/IIa clinical study, in 2023. It
anticipates that this will introduce the company on the
public market. It, then, plans to extend its therapeutic
approach to many other therapeutic fields. In 2022-2023,
Eukarÿs also anticipates of creating a subsidiary or a branch
of Eukarÿs dedicated to protein and virus bio-production
using the C3P3 technology, possibly in the USA.
30 | September 2019 |

34. GRACELL
BiotechnologiesCONQUERING CAR-T INDUSTRY HURDLES WITH FUTURISTIC SOLUTIONS
The healthcare industry needs to bring in more
effective solutions at affordable prices for all its
unsolved questions. Cancer treatment is one of
the major focus areas in this. There are various
techniques and treatment solutions being devised in this
area and a lot more are still under the research stage.
CAR-T is one such technique that has shown a lot of
potential to combat cancer. Although, it has already been
developed, owing to CAR-T’s high costs and complex
development procedure, it has not reached all of the
population. To rectify this and to find better solutions
against cancer, Gracell Biotechnologies is relentlessly
working and innovating its way to a highly advanced and
better future.
Gracell Biotechnologies is a clinical-stage biotech
company with mission to solve CAR-T industry hurdles.
The aim to solve the problems like high cost of goods,
lengthy and ineffective manufacturing process, lack of
off-the-shelf products, and high relapse rate even with
the achievement of complete remission for
hematological malignancies. Being committed to
research and development, it is bringing novel cell
therapies with highly cost-effectiveness from the lab to
the bedside. Gracell aims to bring best-in-class and
affordable cellular medicines to the cancer patients.
Founded in 2017, Gracell has today built a state-of-the-
art 44,000 sq. ft. R&D center in Shanghai and 63,000 sq.
ft. GMP facility in Suzhou with an additional plant
reserved for future commercial needs and advanced
multiple IND filings. Currently, the company has over 90
talented FTEs.
A Numero Uno
Wei (William) CAO is the Founder, Chairman, and
CEO of Gracell. He is a seasoned front runner with over
three decades of experience in biotech and R&D. Before
formulating his ideas into Gracell today, he co-founded
and was the former CEO of Nasdaq listed CBMG;
served at the former senior scientific management
positions at Chiron and Affymetrix. William also has
over 50 issued patents and applications for advanced cell
therapies. He has built Gracell from scratch and has
shaped its success with his rich experience. Talking
about the company, he says, “Gracell’s mission is to
make drugs accessible to ordinary patients. Many of the
patients we see now in our clinical studies are not well-
off. This mission is what keeps us going through the ups
and downs of biotech entrepreneurship.”
The Problem
Chimeric antigen receptor or CAR-T cells are genetically
engineered T cells, redirected to bind and kill tumor
cells. These T cells, taken from patients’ own
(autologous) or healthy donor’s (allogenic) blood, with
activation, modification, and expansion, are administered
to the patients. Preparation of conventional CAR-T cells
can take weeks of processing, with high cost of goods.
32 | September 2019 |

35. For patients at late-stage, with very limited life
expectancy, long manufacturing time may compromise
the medical benefits.
Gracell’s Solutions
Gracell is developing next generation CAR-T for
advanced hematological malignancies, including
leukemia, lymphoma and myeloma. It has multiple
platform technologies and product candidates currently
under clinical evaluations.
FasT CAR
FasT CAR is proprietary and patented technology of
Gracell. Unlike conventional CAR, FasT CAR
production only takes overnight for cell culturing. This
results in affordable and highly cost-effective solution for
patients. GC007F (anti-CD19), Gracell’s one of the lead
program, is redirected to target B cell acute
lymphoblastic leukemia (B-ALL). This product of the
company has multiple advantages, including younger
phenotype, higher proliferation capabilities, less
exhaustion, more potent eliminating leukemia
malignancies, capability to infiltrate into bone marrow,
etc. With all these features, clinical study has further
demonstrated its remarkable outcome. Proving the
th
effectiveness of GC007F, on 25 May 2019, 13/13
patients under test achieved complete remission under at
least 28 days follow-up, with only mild side effects.
Concluding, GC007F has the potential to be best-in-class
anti-CD19 CAR-T profile.
3
UCAR
Lymphocytes from certain portion of patients cannot be
adequately expanded to generate sufficient quality
CAR-T cells. With this hurdle in the mind, Gracell is
developing its own solution that would provide much
better cost economics, mass production per lot, and
followed by one-shot release, not specific to each
3
individual patient. UCAR will be purely for off-the-shelf
use.
3
UCAR technology of Gracell is based on lymphocytes
obtained from healthy donors and modified with gene
We want to make
drugs accessible
to all by resolving
the major challenges
of cellular gene
therapy industry
“
“
Wei (William) CAO
Founder, Chairman, and CEO
| September 2019 | 33

36. editing to avoid GvHD as well as rejection. Genetic
modification also helps protect from host versus graft
3
(HvG) reaction and to enhance UCAR T cell persistence
and proliferation. The company’s lead program is
focused on treatment for T cell malignancies, and it is to
enroll patients soon.
3
In addition to FasT CAR and UCAR , Gracell has been
developing Enhanced CAR-T and Dual CAR-T
platform technologies. These are currently under clinical
evaluations.
What’s Different?
Among number of other competitors, Gracell is making
its own mark and making its presence evident. Gracell
has set up strong alliance with top tier hospitals; a
network of 18 3A hospitals nationwide and a total
50,000-beds capacity. Additionally, with advances in
cellular immunotherapy therapeutics, Gracell has
obtained a strong support from the government, with
subsidy, tax incentives, and perspective commercial land
issuance.
The most important aspect that sets Gracell apart is its
capability to expedite product development. This
significantly relies on its well-built and highly efficient
team, in regard to process and assay development,
regulatory compliance, facility engineering, and clinical
functions. Moreover, the company does not just rely on
in-house R&D activities, it also believes that external
partnership can enhance and extend its capabilities and
strengths. Gracell has a strong interest in the areas of
immuno-oncology and cellular and gene-edited
therapeutics that drives clinical unmet needs and is open
to co-development and licensing opportunity.
Further Roadmap
Speaking of the CAR-T industry’s problems, William
asserts, “The CAR-T industry is still in its infancy and I
realized there are still many imperfections with the
technology.” He continues, “As a drug developer, we
shall be compensating as well as be responsible for those
patients who cannot obtain clinical benefits from current
standard of care.” Gracell is planning to submit its first
filing by the 3rd quarter of 2019 and its new GMP
facility will be open to operation by the first quarter of
2020. Additionally, it plans to start IPO within next two-
three years. Gracell envisions to become the global
leader in cellular cancer immunotherapy.
34 | September 2019 |

38. Aprominent healthcare organization walking arm in
arm with the latest developments in technology is
VIROgene, a gene therapy startup based in
Buenos Aires. The company was established in 2017 by a
group of biotechnology and medicine professionals having
enriching experience in genetic engineering, molecular
engineering, synthetic biology and medicine.
With its multidisciplinary team of renowned academics,
developers, and clinicians, VIROgene is uniquely
positioned to translate the technology into veterinary and
human therapeutics. It designs innovative therapies using
genetic engineering and synthetic biology applied to a
broad range of viruses, and optimizes the development of
therapies to solve health issues.
About the Leading Light
Martin Williams, a biotechnology professional, is the
CEO and Founder of VIROgene. With his unwavering
passion and effort, he raised and built this revolutionary
leading Gene Therapy Startup. His core purpose behind the
inception of the company was to explore and build
technological paths with a differential imprint to those
already existing in the field of frontier molecular medicine.
He believes, “Genes can be very effective therapeutic drugs
when other strategies do not offer adequate solutions. Also,
it is an incredibly exciting time to be a part of the Gene
Therapy Revolution”.
An Inspiring Journey
It all started when Martin was pursuing his biotechnology
studies and decided to take his degree thesis in gene
therapy. Having a deep passion for genetic engineering and
synthetic biology as well as in human and animal diseases,
he always wished to start a company based on his
knowledge in these fields. During the time, he looked for a
lab to make his thesis and found the Genetic Engineering
Laboratory of Universidad Nacional de Quilmes in Buenos
Aires. After finishing his thesis, Martin asked his thesis
partner and his Director, Dr. Mariano Belaich, to build a
gene therapy startup company. He then started to participate
in contests and events to look for funding. The central focus
was on veterinary medicine in the first place to reach the
market in less time, and then to focus on human medicine.
State-of-the-art Services
Gene and immune therapies are nowadays the most
promising treatments for a great number of genetic diseases
including cancer, diabetes, and hemophilia. VIROgene’s
prototype, V5, is a gene and immune therapy for
mammalian tumor treatment. This unique therapy combines
the high transduction efficiency and high gene expression of
viruses, with the design of novel logic gates that trigger
cellular and immunological responses. Although, many
veterinary and human therapies exist to fight oncologic
processes (chemotherapy, immunotherapy, radiotherapy and
extractive surgery), some of them might compromise the
patient’s physiological homeostasis, in different grades,
making the entire organism susceptible to opportunist
pathologies or even worse, creating new ones.
VIROgene is more focused on developing diabetes and
hemophilia gene therapies as well, and vaccines using Virus
Like Particles (VLPs) to trigger immune response against a
broad range of pathogens. To bring in more innovation to its
work, VIROgene develops gene therapies on viral
technologies that are not based on mammalian pathogens,
to offer alternatives with a higher level of biosecurity.
Setting Itself Apart
VIROgene is pioneering the development of life-changing
treatments for severe diseases with engineered viruses that
harbor therapeutic genes to cure diseases at the molecular
VIROgeneRedefining the Future of Healthcare with Gene Therapy
36 | September 2019 |

39. and cellular level. This novel platform is built on
the discovery that viruses can instruct the body’s
cellular machinery to produce nearly any protein of
interest, from native proteins to antibodies and other
entirely novel protein constructions that can have
therapeutic activity inside and outside of cells
giving the human body the capabilities to develop
targeted lifesaving therapeutics. This approach also
allows the intracellular production of therapeutic
RNA.
The startup has multidisciplinary group of
professionals that are continuously developing new
technologies to be at top of the ladder.
Implementing synthetic biology, they are capable of
creating complex gene circuits that allow them to
adjust cellular activity in a right way.
On the top of all these characteristics, VIROgene
uses non-infective virus, making its therapies highly
safe. Besides, getting into the molecular level, the
company’s engineered genetic circuits are based on
the design of logic gates that assure the appropriate
response to a certain cellular activity. All these
features make its therapeutics very attractive to
markets.
Our vision is to
build a global
leading and integrated
company that
exploits its technology
to deliver transformative
gene therapy
products to patients
(domestic animals
and humans) with
severe medical
conditions
“
“
Martin Williams
CEO & Founder
| September 2019 | 37

40. Moving ahead with Evolving Science
VIROgene is moving very close to the revolution in which great therapeutics will help thousands of lives. “Science is really
tricky, and there are many uncontrolled factors in this environment that must be adjusted. Not everything is going to go
perfectly and smoothly, but there is an endless effort underway that inspires everyone involved. Part of the diseases we work
on, are ones that profoundly affect lives, and that´s why we are committed with that, and this has a significant impact in the
development of human and animals’therapeutics,” says Martin.
The company believes that everything it develops starts with the science and leads to the medicine and that there are many
“firsts” along this journey that get repeated. It believes that ‘We have to be disruptors for change in a system that does not
know how to deal with disruption.’
In 2017, the Argentinian Science and Technology ministry recognized VIROgene as ‘One of the Most Innovative StartUps in
Argentina’ for developing a novel cancer gene therapy prototype. Also, in 2018, they were selected as ‘One of the Most
Disruptive StartUps in Argentina’.
Future Endeavour
Today, there are a few gene therapies that are in clinical trial phase III for genetic diseases such as cancer, diabetes,
hemophilia, Duchenne´s Muscular Dystrophy, and from those therapies, few are being developed for animals (Canine, Feline,
Equine, and other companion animals). Hence, VIROgene ensures to stay at pace with the latest developments through every-
day brainstorming and research, and using technology that allows to develop novel and biosafe therapeutics.
VIROgene strives to generate solutions using disruptive technology in the human and veterinary medicine and to offer novel
and effective alternatives to conventional treatments, enabling precise and corrective therapeutics. The company also plans to
increment its developments for a diverse range of genetic and non-genetic diseases and also to develop novel-engineered viral
systems that lend greater effectiveness to those available. “We see VIROgene as a leading global company, developing novel
and disruptive treatments for severe human and animal conditions,” says Martin.
38 | September 2019 |