KAER BioTeherapeutics: - the biopharmaceutical company of choice with first-in-class capability for the respiratory delivery of biotherapeutics

Kaer BioTherapeutics has several platform technologies, SUPRAER for high dose-rate delivery of biologics, a transfection carrier and a validated novel target site to treat inflammation in allergic asthma. In addtion, it has other physioological assays for evaluating the safety and efficacy of inhaled biologics.

SUPRAER Platform Technology

Biologics presently comprise 45% of the drug development pipeline. Aerosol delivery via the respiratory tract is the preferred mode of administration for many of these. However, the delivery of biotherapeutics by aerosol present formidable barriers not addressed by present devices. These include:-

• Inability to nebulize large macromolecules
• Shear-induced molecular degradation
• Long delivery times
• Unwanted excipients.

SUPRAER overcomes these issues by generating a large aqueous aerosol of the active agent. This aerosol is rapidly dried, concentrated and delivered on demand as a solid respirable aerosol of “pure” agent. SUPRAER is targeted at those conditions that require doses of 10 to 200 mg of medication in the clinic or at home.

Features of SUPRAER-drug combination for high dose-rate aerosol delivery:

•  Targets the drug directly to the diseased lungs

•  Enables home treatment

•  Reduces the total dose administered compared to oral or IV

•  Reduce the potential side-effects and complications with long term IV administration

•  Reduce the cost of the drug and its administration

•  Increases patient and physician acceptance and compliance

•  Produces an expected improvement in therapeutic outcome

Applications

Aerosol delivery of biotherapeutics:-

•  proteins such as alpha-1 Antitrypsin

•  Surfactant

•  siRNA

•  vaccines

•  viral vectors.

•  liposomes

AUGASOME Platform Technology

AUGASOME ™- An effective oligonucleotide transport and modification of gene expression carrier platform technology for clinical aerosol delivery.

AUGASOME is targeted to deliver sufficient physiologically active “genetic switches” with minimal toxicity in aerosol form in a rapid, safe and patient friendly manner. This will potentially enable pharmaceutical and biotechnology companies to access previously undruggable targets sites.

Advantages

• Enables aerosol route delivery, obviate the complexity of genetic materials delivery, such as siRNA, via the blood stream or directly injection into the target organs.
• Encapsulates genetic material, protect genetic material from physical and biochemical/enzymatic degradation
• improves endocytic activities for translocation and the residence time of the genetic materials in the lungs for uptake
• Minimizes interferon-like immunological responses
• preserves functional integrity with minimal toxicity to the cells
• economical to produce

siRNA targeted Allergic Asthma

Through utilization of proprietary cell lines together with proprietary assays, KAER has discovered a novel target site against atopic asthma. The oligo, KBT1000, encapsulated in our AUGASOME platform effectively blocks inflammatory-induced responses in our human bronchial genetically-engineered “asthmatic” cell line. This is a unique druggable target site in allergic asthma which conventional anti-asthma drugs fail to inhibit.

Discovery, Validation, and Safety Pharmacology Technologies for Drug Discovery and Development .

There is a lack of technologies to validate the druggable properties of the siRNA in terms of its efficiency, specificity and elicited physio-pharmacological responses. The company has developed

•  Proprietory Small Animal Exposure System, SARES, to optimize the SUPRAER-AUGASOME combination to achieve realistic pharmaco-kinetic (a high lung deposition, low oral availability, optimal lung residence time) and pharmaco-dynamics profiles (lung selectivity) well within the druggable range.

•  Proprietory cell based high content high throughput screening assays for physiological and pharmacological validation and based drug discovery

•  Proprietory cell based and tissue based physiological and pharmacological functional assays