Sjön Therapeutics: Executive Summary & Business Plan
Executive Summary:
Sjön Therapeutics (http://www.sjontherapeutics.com) is envisioned as a global biotechnology venture founded
by
Mr.
Somesh
R.
Doddi,
a
nobel
prize
in
medicine
candidate
(http://www.doddisnobelnomination.blogspot.com) on the same KEY technology pillars as his nobel research
work. Accordingly, the firm valuation (using nobel prize in medicine award proceeds as a benchmark) is at USA
$1,700,000. The KEY technology pillars, through ‘technology integration’ deliver KEY intellectual property
formulations using adeno-associated virus (aav) to treat via human gene therapy the conditions of cancer (lung,
liver), HIV/AIDS (integrase inhibitor), insulin resistant diabetes, and 'telomere extension' to slow human aging.
The entire aav product portfolio offering consists of approximately 20 unique products from one technology
platform- With the average product life cycle per product of 2 years, the cumulative company’s pipeline is
projected to be 40 years. This is the hallmark of a sustainable enterprise.
The global gene therapy industry is expected to reach USA $325 million by the end of 2015 (see attached .pdf
report).
The market structure is stratified, and the current conditions are ‘hot’ for entry of a large-sized market player
into the field of human gene therapy. Lonza is a prime large-sized ‘first market entrant’ (see attached .pdf
report).
The conclusion is that after human genome sequencing becomes commonplace and an accepted part of standard
clinical/medical practice, human gene therapy will grow by leaps and bounds. AAV technology has gained
respect in research and clinical trials, and will be here to stay for the beneficial future of mankind.
This will make Sjön Therapeutics a key player in the future of healthcare, mankind, and society.
Prepared exclusively by Mr. Somesh R. Doddi, Founder, Sjön Therapeutics
Page 1
Business Plan:
Valuation
The intellectual property is a direct byproduct of my work for the nobel prize in medicine
(http://www.doddinobelnomination.blospot.com) in KEY disease areas (cancer, HIV/AIDS, insulin resistant
diabetes, aging) so the KEY intellectual property is valued at the winning amount of the nobel prize in medicine
proceeds viz, USD $1,200,000. In addition, the same technology platform can yield ~≥15 more unique products
for human conditions and this intellectual property is valued at conservative USD $500,000. The total firm
value and selling price is ~USD $1,700,000. At present, I am offering a one-time (upto 30%) ownership
opportunity via a private equity offering of 10,000 shares valued at USD $50/share.
Product Portfolio
The intellectual property consists of the KEY human diseases/conditions of cancer (lung, liver), HIV/AIDS,
insulin resistant diabetes, and 'telomere extension' to slow human aging. This is not the end of the story though;
because the potential product portfolio and growth is diverse and enormous as outlined below:
Microgram Portfolio
Human conditions:
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Leber’s Congenital Amaurosis (eye), goi: RPE65 gene
Wet form of age related macular degeneration (eye) (brand name:, company: Avalanche
Biotechnologies), goi: PEDF
Dry form of age related macular degeneration (eye) (brand name: RST-001, company: Retrosense
Therapeutics), goi: CD59
Retinitis Pigmentosa (eye) (brand name: RST-001, company: Retrosense Therapeutics), goi: RHO gene
X-linked retinoschisis (eye), goi: RS-1 gene
Select Clinical Trials of Gene Therapy for Retinal Diseases
CLINICAL STUDY
RETINAL DISEASE
NAME/VECTOR
MECHANISM OF ACTION
SPONSOR/INSTITUTION
AMD (EXUDATIVE)
AAV2-SFLT01
EXPRESSES ANTI-VEGF PROTEIN
GENZYME/SANOFI
PHASE 1
CHORIODEMIA
rAAV2.REP1
ENCODES Rab-ESCORT PROTEIN
IMPERIAL COLLEGE OF LONDON
PHASE 1
(REP-1)
OXFORD UNIV/MOORFIELDS
PRODUCES RPE65 GENE
CHILDREN'S HOSPITAL OF
LEBER'S CONGENITAL
AAV2-hRPE65v2
AMAUROSIS
RETINITIS PIGMENTOSA (AR)
PHASE
PHASE III
PHILADELPHIA
rAAV2-VMD2-
PRODUCES MERTK
KING KHALED EYE HOSPITAL
PHASE I
EXPRESSES ABC4 GENE
OXFORD BIOSCEINCES/
PHASE I
hMERTK
STARGARDT'S
STARGEN
SANOFI
USHER'S SYNDROME (1B)
USHSTAT
PRODUCES MY07A PROTEIN
OXFORD BIOMEDICA
Prepared exclusively by Mr. Somesh R. Doddi, Founder, Sjön Therapeutics
PHASE I
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Milligram/Viral gm Portfolio (human clinical trial)
Human conditions:
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Beta thalassemia, goi: Beta-globin gene
Glioma, goi: herpes simplex virus thymidine kinase (HSV-tk)
Chronic heart failure, goi: FGF-4, FGF-5, VEGF
Cystic Fibrosis, goi: CFTR cDNA (4450 bp)
Hemophilia (B), goi: coagulant factor IX
Canavan’s disease, goi: ASPA gene
Parkinson’s disease, goi: tyrosine hydroxylase (th), L-amino acid decarboxylase (aadc), GDNF gene
Lung Cancer (quiescence), goi: p130/RBL2, rep: 2, cap: 5.
Lung Cancer (apoptosis), goi: E1-A adenoviral protein w/radioactive thymidine, rep: 2, cap: 5.
Liver Cancer (quiescence), goi: p130/RBL2, rep: 2, cap: 3.
Liver Cancer (apoptosis), goi: E1-A adenoviral protein w/radioactive thymidine, rep: 2, cap: 3.
HIV/AIDS, goi: 2,5-diketo-D-gluconic acid reductase A, rep: 2, cap: 2.
Insulin resistant (type 2) diabetes, goi: Acrp30, rep: 2, cap: 2.
Slow aging, goi: hTERT, rep: 2, cap: 9.
Duchenne’s muscular dystrophy, goi: minidystrophin genes
Pancreatitis (brand name: Glybera, company: Uniqure), goi: LPL gene
Market Analysis
The current state of affairs is that there are small, mid-size, and large players in the industry.
The small players due to their restricted funding and capital structure have decided to focus on production of
small, purified aav virus for application in a variety of human eye conditions/disease. The production process is
inexpensive although labor intensive, but small quantities of product can be sold at premium prices.
The mid-size players have a ‘fill and finish’ attitude wherein they will produce the product on ‘fee for service’
basis. Pay them, and they will produce the product.
The large players have currently not gotten into the human gene therapy market, with the exception of Lonza.
By large players, I am referring to companies the size of Novartis and Sanofi-Aventis.
Prepared exclusively by Mr. Somesh R. Doddi, Founder, Sjön Therapeutics
Page 3
Strategy
With adequate capital funding, manufacturing and purification can be performed in-house. Alternatively, the
company intends to outsource it's manufacturing component through joint venture/partnership agreements in
which the external company will finance the manufacturing of the products. So far there is one potential Swiss
company that might buy my proposition; all others have indicated they will manufacture on 'a fee for service'
basis.
The future management may have to invest significant due diligence in seeking suitable joint venture
candidates/partners. Mr. Somesh R. Doddi has attempted to seek entry into a key strategic alliance which will
serve as the industry benchmark: The Virovek/Avalanche Biotechnologies/Lonza adeno-associated virus (aav)
deal in which Virovek will license it’s aav production technology to Avalanche Biotechnologies, and an
agreement will be signed between Avalanche Biotechnologies and Lonza.
Deliverables
Upon completion of sale, the following deliverables will be assigned and delivered to the client.
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Webmaster and web-editing access (with password) to http://www.sjontherapeutics.com
Technical notes: Small scale (ug) production of virus using roller bottle technology.
Technical notes: Large scale (mg/vg) production of virus using bioreactor.
Raw materials: An itemised list of vendors for purchase/sourcing of raw materials.
Financials
INCOME STATEMENT: JAN 1, 2012- DEC 31,2012
Debits
Credits
Revenues
23andMe DNA spit kit
Proprietary product pipeline
Human genome service(s)
Research products
Expenses
Corporate website hosting (annual)
0
0
0
0
22
Net Income
-22
BALANCE SHEET: DEC 31,2012
Debits
Assets
Securities Issued based on valuation
Credits
Liabilities
0
17,00,000
Owner's Equity
17,00,000
NOTE: All currency is in USA dollars.
Future Engagement:
Mr. Somesh R. Doddi has expressed interest in being an ongoing Board Member of Sjön Therapeutics; handling
managerial or scientific advisory responsibilities. In return for his service, he asks for an annual and recurring
compensation package equivalent to: USD $30,000 + stock ownership (private or public) in Sjön Therapeutics.
Prepared exclusively by Mr. Somesh R. Doddi, Founder, Sjön Therapeutics
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