RH Acquisition LLC Technology Description

1
RH Acquisition LLC Technology Description

• Michael D. Scott, Principal
• MScottEsq_at_Yahoo.com
• 317.598.1054

2
Background

• RH Acquisition LLC was founded to acquire and
  commercialize gold-standard, next generation
  inductible gene regulation technology.

3
Strategy

• Exploit the technology for bio-therapeutic
  manufacturing business.
• Quickly cash-flow positive
• Highly profitable.
• Commercialize the technology for other
  applications through strategic relationships and
  licenses.
• Drug discovery
• Proteomics
• Immunotherapeutics
• Human gene therapy

4
The Technology

• Precisely controls expression of human transgenes
  in eukaryotic cell systems.
• Both level and timing of expression are
  effectively controlled.
• Level based on concentration of inducer ligand.
• Timing based on presence of inducer ligand

5
The Technology

• Control over level of expression.
• Avoids exceeding post-translation modification
  capability of host cell.
• Optimizes fidelity of glycolsylation and folding.
  
• Optimizes bioactive yield.

6
The Technology

• Control over timing of expression.
• No leakage
• No expression in absence of inducer ligand.
• Allows expression of cytotoxic proteins not
  capable with current generation technology.

7
The Technology

• Ecdysone-based receptor system.
• Licensed from Stanford (Hogness, et al.)
• Extensive class of inducer ligands.
• Comprehensive mammalian toxicity data set
  non-toxic to mammals.
• 3000 compounds.
• Composition and use patents.

8
The Technology

• Advantages over current generation induction
  systems.
• Can modify both inducer and receptor to optimize
  for broad range of applications
• Inducer ligands chemically diverse,
  nonsteroidal, non-toxic, non-teratogenic.
• Receptors biological diversity, specificity,
  tight regulation.
• Host system optimization - broad host range
  (eukaryotic cell systems) broad performance
  range.
• Potential for simultaneous regulation of multiple
  genes.

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Technology Applications -Drug Discovery

• Discovery and validation of novel targets
• Technical Advantages
• Reduce false positives, false negatives
• Mimic activity of drug by down- or up-regulation
• Express cytotoxic proteins
• Identify targets in common class or pathway
• Significantly reduce development cost
• Competitive Advantages
• Physiologically relevant expression
• Minimal pleiotropic effects

10
Technology Applications Proteomics

• Technical Advantages
• Improvement over knockouts or other
  irreversible genetic modifications
• Competitive Advantage
• Enables interactive, user-directed, temporal
  modulation of one or more transgenes in living
  cells

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Technology Applications Immunotherapeutics

• Unique combination of ultra-sensitive mass
  spectrometry and immunology for protein discovery
• Identify naturally processed peptide epitopes
  associated with class I and II MHC
• Identify proteins processed through class I and
  II MHC pathways
• Generate a pipeline of peptides and proteins
  relevant for cancer and infectious disease
• HLA-A2 transgenic mouse system for in vivo
  vaccine analysis

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Technology Applications Human Gene Therapy

• Technology Advantage
• Solves 3 of 4 major problems
• Delivery of therapeutic.
• At the right time.
• To the right place.
• At the right dose.
• Competitive Advantage
• Safety, efficacy and precise dosing.
• Proprietary inducer drug is value capture
  mechanism for gene therapy provider

13
Technology Applications Biotherapeutic
Manufacture

• Technology Advantages
• Manufacture of cytotoxics at commercial scale.
• Improved post translation fidelity.
• Increased yield.
• Reduced cycle time.
• Competitive Advantage
• Improved process economics
• Increased manufacturing capacity

14
Technology Applications Biotherapeutic
Manufacture

• Substantial commercial potential.
• Comprehensive business plan projections
• Minimal capital investment requirement.
• Cash-flow positive quickly
• Projected revenues of 78M and net income of
  26M in Year 5.