NanoCell Therapeutics Investor Summary

Leader in Targeted Gene Therapy

Andreas M:+34 656 498 278 Value Proposition: Proprietary platform for efficient in-vivo cell engineering – eliminates
Eigenmann [email protected] need for viral transfection and electroporation and broadens clinical indications
Our cell-directed polymeric nanoparticle technology is able to deliver DNA plasmids and RNA to specific
Mridhu M: +91 97173 67027
Verma [email protected] cells of interest, such as T cells, upon IV administration. Our nanoparticles can also be directed to
monocytes, NK cells, B cells, etc. and subsets thereof, which broadens the spectrum of applicable clinical
indications beyond oncology (CAR-T; TCR), including autoimmune/inflammatory, infectious and beyond.
History:
The concept of polymeric nanoparticle- Valuation: ~$4.5 Billion Opportunity for every 1% Market Penetration in Solid Tumours
mediated in vivo cell engineering was
originally demonstrated by the Fred
Market Landscape: High unmet need in developing affordable and scalable gene therapies
Hutchinson Cancer Center - [Smith T.T.
et al. Nat Nanotechnol. 2017 August Despite tremendous promise, significant clinical and manufacturing challenges remain within gene
12(8): 813-820] therapies (CAR-T and TCR), such as high cost to develop, sub-optimal clinical performance, expensive to
scale, product inconsistency, limited DNA loading capacity and logistically complex to manufacture.
NanoCell Therapeutics recently acquired
the requisite patent-protected NanoCell addresses this high unmet need through its proprietary NanoCellTxTM technology platform.
technology components necessary for
this concept to be applicable in humans Commercial opportunities exist in Stable Genomic Editing [e.g. oncology, autoimmune / inflammatory /
and feasible for commercialization infectious diseases, haemophilia, etc] and Transient Gene Expression [e.g. CNS, cardiovascular and
metabolic diseases, regenerative medicines and vaccines].
Study Results:
Reference data use technology Product: Construct for NanoCell’s lead product candidate, NTI-1218, is ready to use
components that are suitable for animal
use but not for human application.
The NanoCellTxTM technology platform combines three, proven, proprietary technology platforms to
NanoCellTxTM uses similar technology create a New Biological Entity (NBE), not a cellular therapy:
components as Fred Hutchinson, but is 1.Cell-directed polymeric NanogelTM technology
suitable for human use 2.RBPS MinicircleTM plasmid DNA technology
3.Sleeping Beauty transposon / transposase technology
Efficacy: Tumors were eradicated in 7
out of 10 mice, while others showed
Success Factors: Positioned to be first to market with Intellectual Property rights in place
substantial regression along with an
average 58-day improvement in Offering a widely and highly- desired technology solution with a transformative platform:
survival; results were comparable with 1.Competitive landscape wide open - positioned to be first to market
treatment arm consisting of retrovirally
2.Nanocell holds unique composition of matter, methods and manufacturing IP
transduced CAR-T cells (5 million CAR+
cells)
3.Management team with decades of immuno-oncology R&D and operations experience

Safety: No treatment related macro- or R&D Milestones: Preclinical proof of concept already published
microscopic lesions; No abnormalities in
cell counts and blood chemistry profiles; Concept of polymeric nanoparticle-mediated in-vivo cell engineering was originally presented by the
< 1% of phagocytes in liver and spleen Fred Hutchinson Cancer Center in 2017, demonstrating efficacy and safety in the animal model.
expressed CAR, the signal of which NanoCell Therapeutics uses similar technology components, but suitable for human use.
decreased day by day, indicating no
expansion or proliferation in these cells NanoCell IP: Unique and essential composition of matter, methods and manufacturing IP
Management: 1.Cell-directed nanoparticle – New composition of matter IP
2.Gene transfer – New composition of matter IP and Freedom to Operate strategy
Maurits Geerlings, President & 3.CAR/TCR constructs – Proprietary IP optionally licensed
MD, MBA Chief Executive
Officer
Financing Needs – Seeking $7-10 million in financing (Seed / Series A)
Charles Nicolette, Chief Scientific
PhD Officer Value-driving milestones achievable within 12 - 15 months, including:
1.Preclinical Proof of Concept for in vivo CAR/TCR therapy
Steven Feder, Esq EVP, Strategic
Affairs, CFO & 2.Advancement of lead drug candidate to pre-GLP tox stage
General Counsel &
Secretary Next Steps – Significant value inflection expected for lead drug candidate, NCTI-1218
Vidal de la Cruz, VP, Preclinical R&D
PhD
1.In-vitro and in-vivo Proof of Concept [12 – 15 months post-financing] - Includes plasmid and
nanoparticle manufacturing, in-vitro/vivo PoC
Robert Hrubiec, VP, Intellectual 2.IND-approval-[Includes minicircle plasmid manufacturing scale up, QA/QC validation, GMP
J.D., PhD Property manufacturing and IND-enabling studies]
3.Phase-1/2a trial data -[Includes dose-escalating multicenter trial, product pipeline expansion]
Irina VP, Product
Tcherepanova, PhD Development
For further information contact ABE Strategy