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Effective Process Development to Reduce HCP and Maximize LVV Yield with Innovative Nanofiber Resin

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Effective Process Development to Reduce Host Cell Proteins and Maximize Lentiviral Vector Yield with Innovative Nanofiber Adsorbent Sujeong Yang, Hanna Dreja, Caroline Daye, Emma Burman, Joe Fletcher, Adam Pinnock, Ben Wallis, Marc Hummersone, Ian Scanlon Astrea Bioseparations, Horizon Park, Barton Road, Comberton, Cambridge, CB23 7AJ, UK

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To perform bench-scale purification with innovative nanofiber adsorbent as a rapid screening tool to optimize clarification of suspension LV feed prior to LVV capture. To establish the LV purification workflow from benchscale to process development scale, incorporating nanofiber adsorbents that maximize the lentiviral recovery and impurities removal.

Novel nanofiber adsorbent with low shear flow path and high binding capacity for maximising LVV recovery Average mean flowpath diameter of 860 nm giving excellent flow characteristics for ATMP targets

Comparison of Infectious LVV titer post clarification Condition #1-3 LVV TU titer post clarification

Run detail

100%

1

Centrifugation, 0.45µm PES filtration

1.47E+08

1.5E+08

80%

2

Centrifugation, 0.45µm PES filtration, Diafiltration 100kDa, Buffer Ex 1xVOL

1.67E+08

3

Depth filtration CA

1.68E+08

1.2E+08

60%

9.0E+07 40%

6.0E+07

0.0E+00

The clarification steps overall gave less than 20% loss of infectious LVVs compared to the crude harvest. Condition 2 and 3 resulted in the most infectious LVVs (n=3).

0% Motherfeed Condition 1 Condition 2 Condition 3

TU/mL

5 Bed Volume 10 mL

Nereus LentiHERO® Spin column screening outcome from Condition #1-3 Infectious LVV recovery

3.0E+08

1

• Centrifugation and 0.45 µm PES filtration • Depth filtration (CA)

30%

20% 15% 1.0E+08

Centrifugation, 0.45µm PES filtration

Centrifugation, 0.45µm PES filtration

Depth filtration

• Condition 3: Depthfiltration (CA)

0.0E+00

0% Condition1

Condition2

0 HCP removal

Condition3 Recovery

Condition1

Nereus LentiHERO® bench scale purification

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Infectious LVV recovery 3.00E+07

100%

2.50E+07

80% 70%

2.00E+07

60% 40%

45%

50%

41%

1.50E+07

40% 30%

1.00E+07

Recovery (%)

80% Total TU

Recovery (%)

60%

20%

20%

5.00E+06

0% TU recovery

HCP

dsDNA

10%

0.00E+00

3x VOL

TU

Buffer to the feed volume

1x VOL 2x VOL 3x VOL

80

1500

60

1000

40

500 0

LV production: HEK 293T, Suspension cells TU assay: Jurkat cell, Flow cytometry GFP+ HCP: HEK HCP ELISA dsDNA: PicoGreen assay

0%

20 0

50

100

150

Chrom.1 UV mAU

METHOD

70%

120 100

2000

Post Nereus LentiHERO® purification

120%

2x VOL

140

% Recovery from the load

Infectious LVV and impurities level in the feed post buffer exchange

160

3000 2500

UV (mAU)

Optimization of buffer exchange volume for ultrafiltration

Viral genome recovery

3500

Conductivity (ms/cm)

Suspension LV load

1x VOL

Condition3

Process Development Scale Purification: Processing clarified suspension LV feedstock with LentiHERO® 1 capsule

LentiHERO® 1 purification

Before Buffer Ex

dsDNA removal Condition2

Buffer exchange* TFF or Ultrafiltration *buffer exchanged with EQ buffer, 0.02M Tris, 0.02M MgCl2, 0.1 M NaCl, pH7

2

Most HCPs were removed by the purification step post clarification. Condition 2 gave a high recovery yield with an efficient removal of dsDNA.

40

20

5%

Total TU

• Ultrafiltration with 100kDa or 300kDa MWCO • TFF (*cross flow filtration 100kDa)

60

10%

5.0E+07

Clarification with a buffer exchange

• Condition 2: CF at 1500 x g, TFF 100kDa with EQ buffer change

80 25%

1.5E+08

Fresh LV harvest

100

29%

24% 2.0E+08 Total TU

Primary clarification

Conditions 2 and 3 gave the highest recovery of TU titer after Nereus LentiHERO purification. n=2

Post clarification & Nereus LentiHERO® purification

35%

31%

2.5E+08

Study Overview: LVV purification workflow using nanofiber adsorbents at bench and PD scales

RESULT

Impurities removal

Post clarification & Nereus LentiHERO® purification

Removal %

Bed Volume 1 mL

Recovery (%)

Recovery %

Bench-scale

(Crude Harvest 1.75E+08)

RESULT

20%

3.0E+07

Nanofiber adsorbents in spin column and capsule formats for bench-scale and PD scale processing

TU/ML

Condition

1.8E+08

Recovery (%)

Aim

The significant presence of host cell proteins in suspension lentiviral (LV) feedstock highlights the need to optimize clarification in order to maximize downstream processing lentiviral vector (LVV) recovery and reduction of impurities. Effective process development tools are required to rapidly purify LVV while minimising LVV loss and damage.

TU/mL

Challenge

0

200

Chrom.1 Cond mS/cm

100

METHOD

100

• HEK 293 T, Suspension cells

69.38

80

LentiHERO® 1 capsule

60

• 200 mL of harvest clarified centrifugation and 0.45 µm filtration

40 20

• Diafiltration through 100 kDa filter

0

Load

Elution

• Concentration to <150 mL to enable loading via superloop

Result: Functional LVV recovery was 53% (measured by GFP integration into Jurkat cells).

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High recovery and high purity with optimized workflow using LentiHERO® 1 Process Development scaled Capsule

Recovery

Result: 1× volume (VOL) buffer exchange to feed gave the highest infectious titer, with >40% removal of HCPs and

LOAD Physical Particles*/mL adsorbent

LOAD TU** /mL adsorbent

RECOVERY TU** (600 mM NaCl)

Adherent LV 242 mL load

4.5E+11

4.7E+09

2.6E+09 (54%)

Suspension LV 200 mL harvest load

1.75E+12

2.5E+09

LV

dsDNA. These feeds were submitted for the purification step, performed with Nereus LentiHERO®. 1× VOL to the feed resulted in the highest infectious titer, with 70% TU recovery to the load and was adopted for the UF/DF step.

1.3E+09 (53%)

Impurities level from LVV post LentiHERO® 1 purification Adherent LV feed

3

100%

Optimization of Centrifugation and Ultrafiltration

The effects of centrifugation speeds and MWCO UF filter on fresh harvest were examined. Conditions for clarification Condition 1

TU/ML

RESULT

(Crude Harvest 2.55E+07)

300

100

2

300

300

1.88E+07

3

1500

100

2.47E+07

1.52E+07

4

1500

300

1.75E+07

5

4400

100

2.16E+07

6

4400

300

1.61E+07

Infectious LVV recovery

Post clarification & Nereus LentiHERO® purification

1.5E+08 1.2E+08

Total TU

Equilibration Loading Washing Elution

9.0E+07 6.0E+07 3.0E+07

0

5

10

15

20

25

Time (min) Elution

Washing

Loading

Equilibration

0.0E+00

1

Total time

2

3

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: astreabioseparations.com/patents All trademarks, trade names, trade dress, product names and logos are the property of Astrea UK Services Ltd.

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Condition

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100%

HEK HCP

hcDNA

100% 77%

80% 70% 60% 50%

39%

30%

Ultrafiltration MWCO

Parallel operation time

100%

90%

40%

Centrifugation speed (x g)

Total time

100%

Suspension LV feed

5

6

• Minimizing processing time is essential to maintain infectivity potential, and Nereus LentiHERO® enabled six conditions to be screened within 25 minutes. • UF gave optimal conditions when considering the final recovery under low shear conditions. • Condition 3 was adopted as the clarification step in subsequent workflow with a LentiHERO® 1 chromatography capsule.

20%

18%

15%

10%

3%

2%

0%

Harvest

Elution

Harvest

Post TFF

Elution

* Zeptometrix P24 ELISA ** Jurkat transduction

Key Findings • Nereus LentiHERO® purification forms an efficient sample preparation workflow for suspension feedstock including clarification methods. • Minimizing processing time is essential to maintain infectivity potential of LVVs, and Nereus LentiHERO® enabled parallel purification of samples within 25 minutes. • The high DBC of LentiHERO® 1 PD scaled capsule demonstrates the significant potential of this innovative adsorbent for bioprocessing at scale. • LentiHERO® 1 purification achieved the high purity LVVs with >98% removal of HCPs and >85% removal of dsDNA from the harvest.


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