A Parallel Purification Strategy for Rapid Processing of Lentiviral Vector Suspension Feedstock Samp

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A Parallel Purification Strategy for Rapid Processing of Lentiviral Vector Suspension Feedstock Samples Sujeong Yang, Emma Burman, Joseph Fletcher, Beatriz Sant Mora, Daniella Steel, Ian Scanlon, Marc Hummersone Astrea Bioseparations Ltd, Horizon Park, Barton Road, Comberton, Cambridge, CB23 7AJ, UK

4

Figure 1 B

Minimal Affect of Optimized Clarification Step on Lentiviral Recovery

Figure 5 A

1

Adherent

Centrifugation at 300 x g, followed by filtration by 0.45um PES, no buffer exchange step

Suspension

Centrifugation at 1500 x g, followed by filtration by 0.45um PES then buffer exchanged with equilibrium buffer using 100kDa ultrafiltration (Condition 3).

Adherent and suspension feedstocks were processed according to the experimental plan in Figure 2. Purification was performed following the Nereus LentiHERO® NL100100 technical user guideline.

Figure 2

Harvest/Nuclease treatment

Adherent LV

A

Removal of impurities

HEK HCP

10

dsDNA µg

HCP µg

15 10

Figure 3

1

TU/ML (Crude Harvest 2.55E+07)

300

100

2

300

300

1.88E+07

3

1500

100

2.47E+07

4

1500

300

1.75E+07

5

4400

100

2.16E+07

6

4400

300

1.61E+07

Load

1.52E+07

Clarification was optimised by testing 6 conditions (Figure 3). Feed clarified by centrifugation at 1500 x g, followed by 100 kDa MWCO filtration (Condition #3) showed the highest TU titer, indicating minimal loss of functional LVV.

6 A

B

Total Time

Elution

Washing

Loading

20

25

Time (min) Elution

Equilibration

C

800

Load

0

Elution

M

1

2

3

98 62 49 38

4 2

3.2

kDA

28 0.67

Load

0.11

17

Elution

Total Time

6.0E+07

0.0E+00

2

3

B Transmission Electron Microscopy Images of LVV purified by LentiHERO®.

100 nm

28

LV-specific p24 around 24kDa molecular weight were identified in the eluted fractions (Figure 7A). TEM images revealed the typical healthy morphologies of LVVs from the eluates following LentiHERO® purification (Figure 7B).

p24

High Dynamic Binding Capacity of Nereus LentiHERO®

Figure 8 1

2

3

4

5

• Nereus LentiHERO® enables parallel purification of samples to scale out screening studies. • The high dynamic binding capacity of Nereus LentiHERO® demonstrates the significant potential of the composite nanofiber adsorbent at processing scale.

Dynamic binding capacity

60%

6

Condition

• Nereus LentiHERO® purification, used with an optimised clarification process, forms an efficient sample preparation workflow for suspension feedstock.

Any data or results provided are only examples and do not provide any guarantee of similar results in future. The products of Astrea Bioseparations may be covered by or for use under one or more patents: www.astreabioseparations.com/patents All trademarks, trade names, trade dress, product names and logos are the property of Astrea UK Services Ltd.

LV specific p24 Gag protein was detected using rabbit polyclonal anti-p24 IgG by Western blot analysis.

38

7

Key Findings

Search: Astrea Bioseparations

M:Marker, Lane 1: LV load Lane 2: Flow-through, Lane 3: LV Eluate

9.0E+07

Samples of suspension feedstock clarified from each of the above 6 conditions were purified using the Nereus LentiHERO® spin columns in a bench top centrifuge. This enabled 6 conditions to be prepared in less than 25 minutes (Figure 4A). Conditions 3 and 5 resulted in the highest TU recovery (Figure 4B).

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6

0

14

3.0E+07 15

8

1200

Elution

17

Total TU

Loading Washing

10

Load

Silver stained SDS-PAGE gel

LV p24 detection

1

49

1.2E+08

5

TU

Lentiviral Structure is Well Preserved

kDA

1.5E+08

Equilibration

0

Suspension LV

dsDNA

10

1388.6

1600

400

0

62

% Breakthrough (C/C0)

A

Adherent LV

HEK HCP

0.67

Elution

Figure 7

Infectious LV recovery Post clarification & LentiHERO® purification

Parallel operation time

0

Suspension feedstock post clarification contained high levels of HCP. Nereus LentiHERO® purification significantly reduced HCP and process related dsDNA contamination from both adherent and suspension feed (Figure 6A & B). HCP was significantly reduced, with barely detectable level of HCP in the eluate (Figure 6C).

LentiHERO® - A Highly Efficient Screening Tool

Figure 4

4 2

98

3

10

Host cell proteins and dsDNA were measured by HEK HCP ELISA or PicoGreen assay, respectively. n=4 /feed

Conditions for Clarification

Condition

20

HCP 99.7 ± 0.3% dsDNA 83.6 ± 0.8%

2000

6.82

6

1.3

0

Ultrafiltration MWCO

dsDNA

8

5

Centrifugation speed (x g)

30

Removal of impurities

24.6

25 20

2

40

Suspension LV

B

HCP 94.8 ± 0.7% dsDNA 90.1 ± 0.7%

30

LentiHERO® Purification

50.07

50

High HCP in Clarified Suspension Feed Removed by LentiHERO® Purification

Figure 6

Clarification Centrifuge/0.45 µm PES filtration Buffer exchange TFF or Ultrafiltration MWCO

Clarification Centrifuge/0.45 µm PES filtration

59.07

59.20

P24

Suspension Feedstock

Adherent Feedstock

63.33

60

Suspension feedstock processed with Condition 3 and processed adherent feedstock were purified with LentiHERO®. LVV recoveries were similar for both physical and infectious titers (Figure 5A & B). Range of values for both feedstocks: P24 recovery, 3-4E+10; TU recovery, 1-3E+8. n=4/feed.

5

Experimental Plan

LVV recovery

80 70

Clarification Conditions

While suspension culture systems are often used for large scale processing due to ease of scaling up and reduced costs, sample preparations can be lengthy due to the high levels of contaminants, such as host cell proteins (HCP). Therefore, we developed a process for LVV purification from suspension feedstock using a novel spin column, the Nereus LentiHERO® (Figure 1A). This device contains a functionalized high-flow, high-capacity composite nanofiber adsorbent, AstreAdept® (Figure 1B), previously shown to rapidly deliver high LVV yields from adherent production systems. After optimization of the suspension feedstock clarification step, we compared the performance of the Nereus LentiHERO® purification step from both types of LVV feedstocks.

B

Feedstock System

% recovery to Load

A

dsDNA µg

Bottlenecks caused by sample preparation during the development of lentiviral vector (LVV) therapeutic assets are common, both in upstream and downstream process development, and negatively impact pipeline milestones.

HCP µg

Abstract

FT 5

50% 40%

FT 3

FT 2

FT 4

30%

Fraction

Accumulated Volume (mL)

PP/mL

FT1

4

1.34E+08

6%

FT2

8

5.23E+08

23%

FT3

12

6.41E+08

28%

FT4

16

6.53E+08

28%

FT5

20

8.02E+08

35%

C/C0

20%

* Load concentration is 2.30E+09 PP/mL

FT 1

10% 0% 0

5

10

15

20

Table 1

Accumulated Volume (mL) DBC15%BT/Device is 1.5E+10 Single Nereus LentiHERO® unit loaded at 20mV/min, analysed by P24 ELISA to calculate % of physical LVVs break through. C0: titer of Load, C: titer of flow-through (FT).

P24 recovery

TU recovery

n=4

Load

Elution

Load

Adherent LV

6.52E+10

4.10E+10

4.84E+08

2.88E+08

Suspension LV

5.08E+10

2.56E+10

1.75E+08

9.73E+07

Elution

Nereus LentiHERO® purification of suspension feedstock processed with the optimised clarification conditions demonstrated high dynamic binding capacity (DBC, Figure 8), with recovery of physical particles (PP) and infectious particles (Table 1). When extrapolated to bioprocessing size this interpolates to a DBC of 1.25E+11 PP/mL, indicating Nereus LentiHERO® is a powerful purification tool for the development of downstream processing conditions.


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