Miamibioinnovation 2

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4th ANNUAL BIO-MANUFACTURING SUMMIT June 7th - 8th 2017 Miami, Florida BRIDGING THE GAP BETWEEN BUSINESS, TECHNOLOGY AND SCIENCE TO OPTIMIZE BIOPROCESSING ​ Sarfaraz K. Niazi, Ph.D. Adjunct Professor, University of Illinois

Bioinnovation: Cost and Time Optimized Development of Biosimilars 2

Niazi, largest solo biotech inventor

Cost & Time Drivers for Biosimilars • New Reality • Commercial scale (GMP) lots for testing • Structural variance within reference variance • Side by side stability testing with reference

• Drivers • DOE • Facility dedication • Scale up

Downstream

Capture

Filtration

• Float column, resin puck, modular • Multiple resins • Lossless

• Nonspecific, In situ • Protein, metabolites • Customized

• Non-blocking • Air-scrubbed • Built-in

Upstream

Mixing

Contamination

• Unstirred, convective • Horizontal, bed sparge • Noninvasive Monitor

• Unstirred • Levitating • Gravitational

• Single-pass HVAC • Virus Removal • Filter-free Exhaust

Source: www.worldwide.escapenet.com Approximately 127 results found in the Worldwide database for txt = sarfaraz and txt = niazi 3

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Key Invention Categories • Base:

• Horizontal, single-use, free-standing pillow, orbital motion, short dwell time, DOE

• ISO 9 operable:

• Patented ports, filter-free exhaust, recirculation, burn off

Winner: Innovation of the Year

• Convective heat transfer:

• Cell shock, instant cycling, improved PTMs

• Harvesting first:

• No centrifugation and filtration

• Continuous and Perfusion:

One of the fastest patents ever issued by USPTO

• Non-blocking filter, gravity driven, point of expession capture

• Scale up and tech transfer:

• Pooling, geometric mixing, no comparability protocol

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More Than a Bioreactor

• • • • • • • • •

Pillow, un-walled Convective heat Capture first Perfusion, continuous ISO9 Full bed gassing Short-dwell, PTMs DOE Tech transfer

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Managing At Scale Requirement

Perfusion

• Closed system • Gravity drive • No strain on cells and protein—no pump • Continuous or end of cycle • Cell or cell and protein separation • No contamination

• Modular upstream: CFR 21 compliant. • One size validated—no scaling up • Combining multiple sources of upstream without using larger container, geometric dilution, contamination-proof system

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Continuous Bioreactor

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Single Container Manufacturing • Removes three major steps—time and cost • Improves yield • Direct capture, washing, elution • Improves quality of product • Conserves resin cost

• Expansion-production • Gravity-cell strain • Perpetual cycle • Continuous harvest • Universal cell lines

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Single Use Downstream Purification

Non-blocking Filtration

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• Air/liquid drive scrubbing of filter surface • Solid ceramic filters permanently installed • Eliminates depth filtration, paper filter and use of pressure filtration • Disposed and sterilized inside bioreactor • Global use of slurry treatment

• Stacked discs reclaim resin • Multiple columns combined in one • Custom configurations • High yield • No column packing • All parts single use • Float Column

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Facility

Non-invasive Monitoring • No sensors in contact with media • No interference due to chemical reaction • Cuvette in liquid concept • Allows expensive sensors—not disposable

• • • •

• Cost effective • Readily validated

Single-pass low loss HVAC Maximum energy recovery Each area individually classified Viral clearance: air scrubbing

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Cost & Time Drivers Managed

Exhaust Control • Recycle exhaust, replenish nutrition gas • Condenser reflux, venture effect • Bunsen burner with added oxygen • Connected to outside • One-way outlet for pressure changes • Positive pressure in container

• Create one small size scale: multiply rather than increase size • Operate in a ISO9 facility • Use horizontal bioreactor for PTM and variation management • Use same bioreactor for DOE and Commercial manufacturing • Develop multiple products simultaneously • Eliminate cell culture separation and reduce depth filtration volume • Single-use upstream and downstream • Enclosed perfusion and continuous manufacturing • Minimize Comparability Protocol constraints 17

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New Tech New Challenges Considerations

Examples Recombinant Proteins Organs Fine Chemicals Energy Sources

New Products

• • • •

New Expression Systems

• Bacteria, Mammalian Cells • Plant and insect Cells, Yeast • Multiple Expressions Lines

Regulatory Compliance Commercial Manufacturing

• Cleaning Validation, CrossContamination, Viral Clearance • Regulatory Expectations • Scale-Up, Portability, point of use • Technology Transfer • Continuous Yield, Single container Utilities, Ceiling Height ISO9, Foot-Print, Size Qualification, COGS Continuous yield Hardware

Capital Cost

• • • • •

Development Cost

• Fast to the market possibility

Key Questions Is there a universal platform for all products?

Is there a universal bioreactor for all expression systems? Is there a safe, contamination-resistant bioreactor? Is there a scale-up and tech-transfer-friendly platform?

Is there a cost-effective bioreactor suitable for all business type?

Is there a bioreactor for fast-to-market capability? 19

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