Transcript New Light Path TM Services

New Light Path TM Services
Light PathTM: streamlined custom material
supply for discovery to early development
drug
discovery
preclinical
development
phase I
kg
mg
non-GMP
GMP

Leverage Lonza’s proven technology platforms & expertise to meet
your exact early development needs in a lean, cost effective and
speedy manner

Complete gene to GMP production service offerings for both
microbial & mammalian technology

Partner with Lonza early and be on the road to a marketable
product
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Meet all your discovery, pre-clinical &
phase 1 material needs with Light PathTM
Drug
Discovery
Light Path™
Discovery
Light Path™
Development
Preclinical
Development
Phase I
1mg – 500mg, non GMP
in 6 to 10 weeks
1g – 1000 g, non GMP
in 6 to 10 weeks
1 - 100 grams, non-GMP
in 4 to 8 months
1g - 1000 grams, GMP
in 3 to 6 additional months
* starting from gene sequence, timeline guidance includes gene synthesis.
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Mammalian Light Path TM Services
Mammalian Light PathTM Discovery for
custom milligram material supply (non-GMP)
Vector Construction
Product
Expression
Culture
Preparation
Affinity
Chromatography
Material Supply
Gene
synthesis
Vector
construction
DNA
amplification
Transient
expression
or
clarified
supernatant
up to 10L
Protein A or
similar affinity step
Delivery of
up to 500mg of
product
Transfectant
pool

Product expression using Lonza’s proprietary GS Gene Expression System™

Option to perform vector construction suitable for future stable cell line development

Option to apply your specific affinity chromatography step

Option to monitor endotoxin levels and include additional purification steps

Material supply for multiple product candidates
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Mammalian Light PathTM Development for
pre-clinical through to your IND
Starting from a gene sequence:
Cell Line
Construction
Streamlined
PD
non-GMP Pilot
Supply Option
(1-100g)
Pre-production
Activities
GMP
Production
(10g – 1000g)
option for mg
supply
via Light Path™
Discovery
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Compare Mammalian Light PathTM Development
to Lonza’s standard GMP programs
Service
Standard
Options for development of unclonal or clonal
cell lines
 Vector construction, transfection and multiple
rounds of cell line screening where applicable
 Cell line stability study on three candidate cell
lines
 Supply of up to 5 mg of Protein A purified
product from top 9 selected cell lines

Cell line
Construction
Application and evaluation of Lonza’s platform
upstream process
 Performance of 2x10L scale and material
supply for purification process development
 Material supply of up to 5mg Protein A purified
product for animal efficacy studies

Upstream
Development
Light Path
Options for development of transfectant pool,
unclonal or clonal cell line
 Vector construction, transfection and limited
rounds of cell line screening where applicable
 No cell line stability study unless requested
 Supply of up to 5mg of Protein A purified product
from top 9 selected cell lines

Application of Lonza’s platform upstream process
only
 Performance of 2x10L lab scale for purpose of
material supply for purification evaluation only

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Compare Mammalian Light PathTM Development
to Lonza’s standard GMP programs - continued
Service
Standard
Application and evaluation of all steps of
Lonza’s platform downstream process
 Intermediate product stability study

Downstream
Development
Light Path

Assumes product is a monoclonal antibody of
subclass IgG1, 2 or 4 with pI>8 and aggregate
levels of <10
Evaluation of limited steps of Lonza’s
platform downstream process to a “good
enough” level


No intermediate product stability study
Analytical
Development

Evaluation of Lonza’s Protein A HPLC
and all batch release platform assays

Evaluation of limited subset of platform assays
Pilot
Production

Non-GMP runs at 130L, 250L or 400L
scale (tox material supply)

No non-GMP pilot run unless requested

Any scale

200L, 500L, 800L, 1000L
GMP
Production
*only size available for transfectant pool
option
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Decide if Mammalian Light PathTM
Development is right for you

Take the Light Path™ when your focus is strictly on filing an IND

If certain budgetary restrictions would require scope limitations, then Lonza will
focus on only necessary activities

Light Path™ requires an in-depth discussion with Lonza to agree in partnership
on how to best design you program. Lonza will highlight areas where potential
challenges may arise that could require scope change.

Leverage of Lonza’s proven GS expression system™ and processes
development platforms to generate an IND enabling
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Microbial Light PathTM Services
Microbial Light PathTM Discovery for custom
milligram material supply (non-GMP)
Strain Acquisition
Expression of tagged
proteins
Affinity
Chrom.
Tag removal or downstream “scouting”
w/ parallel expression of untagged
protein
Delivery
option 1
Final purification run
Delivery
option 2
4x500mL Fermentation
Client
Strains
XS Strain
Development
or
Empty
Vector
Strain 1
Strain 2
Strain 3
IMA
C
Tag removal
or
Tag removal
& DS Dev
Fermentation
Purification

Option to start with a minimal XS E. coli strain development to explore expression feasibility

Default service assumes His-tagged protein, option to have His-clipped version

Option to have a minimal DSP development performed to purify untagged native product

Deliverable is 1-500 mg target protein at >70% purity.


18-Jul-15
Delivery
option 3
Actual quantities delivered depend on expression levels and DSP yields achieved
Light path™ Discovery XS strain can be used as the starting point for Light Path™
development service
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Microbial Light PathTM Development for
pre-clinical through to your IND
Starting from a gene sequence:
XS Strain
Development
Streamlined
PD
non-GMP Pilot
Supply Option
(1-100g)
Pre-production
Activities
GMP
Production
(10g – 1000g)
option for mg
supply
via Light Path™
Discovery
Drop-in an existing process:
Customer
Strain &
Process
Transfer / Adapt /
Minimal Development
non-GMP Pilot
Supply Option
(1-100g)
Pre-production
Activities
GMP
Production
(10g – 1000g)
option for mg
supply
via Light Path™
Discovery
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Compare Microbial Light PathTM Development
to Lonza’s standard GMP programs
Service
Strain
Development
Fermentation
Development
Standard

Evaluate a variety of expression systems
and hosts (E. coli, pichia, bacillus)
 Full fermentation screening via 4 x 4 1L
DASGIP runs


Perform 18 x 10L fermentations
 Screen up to 8 parameters to establish
a robust process
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Develop 3-4 chromatography steps
 Screen 6 resins per chromotography step
 Develop 2 TFF steps

Perform up to 4 consistency runs (10L)
Multiple pilot runs at 20L, 40L or 200L
scale (can supply tox needs)


Downstream
Development
Lab Consistency
& Pilot Runs


Lay down Master & Working Cell Banks
1 to 2 engineering runs before initial GMP
batch
 Any GMP scale


GMP Production
Light Path
Explore most attractive options only
 Reduced scope of fermentation screening
(only 2 x 4 1L DASGIP runs)
Perform only 3 x 4 1L DASGIP runs PLUS
4 x 10L fermentations
 Screen only 3 parameters to establish a
“good enough” process
Assumes 2 or 3 chromatography steps are sufficient
 Screen 3 resins per chromotography step
 Assumes 1 TFF step is sufficient

Perform only 2 consistency runs (10L)
Encourage at least one pilot run, but not
always necessary
Lay down only Master Cell Bank
 First and only engineering run can be
released as GMP if successful
 GMP scales limited to 20L, 150L & 800L

(total volumes)*
*Lonza will not transfer into GMP with significant risk of failure. Additional scope change may be needed to establish an adequate process.
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Decide if Microbial Light PathTM
Development is right for you

Take the Light Path™ when your focus is strictly on filing an IND

If certain budgetary restrictions would require scope limitations, then Lonza will
focus on only necessary activities

Light Path™ requires an in-depth discussion with Lonza to agree in partnership
on how to best design you program. Lonza will highlight areas where potential
challenges may arise that could require scope change.

In lieu of costly non-GMP engineering runs at scale, Lonza will establish your
IND-enabling GMP process in a cost-efficient pilot laboratory environment

Lonza will not proceed into GMP without sufficient data to indicate that the
process has a high likely hood to initially succeed at scale on the first run
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