Cell & Gene Therapy Industry Survey

Question Title

* 1. Given evidence of decreasing donor activities at US blood centers, do you believe the CGT industry is likely to face bottleneck issues in future production due to the lack of available Source Material?

Yes

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* 2. Do you share these concerns related to your products?

Yes

n/a

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* 3. Do the products you are directly involved in developing require a large and/or ongoing Source Material donor base for adequate source material?

Yes

n/a

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* 4. Do you believe the CGT industry is likely to face bottleneck issues in future production because of a shortage of required Source Material either due to donor shortages, inability to identify/recruit 'rare' donors, or lack of donors in certain geographies?

Yes

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* 5. Do you believe some processing capabilities at Source Material collection facilities would be an advantage in the supply chain of CGT products?

Yes

Could be an advantage to some but not significant

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* 6. Do you believe the existence of vein-to-vein processing/manufacturing capabilities of CGT products at hospitals or health systems would help or hinder the evolution/expansion of CGT products?

Help

Hinder

Would have no material impact

Both help and hinder - explain

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* 7. Do you believe the future of CGT will be largely dominated by centralized manufacturing?

Yes

If yes, the primary exceptions will be

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* 8. Do you believe the future of CGT will be largely dominated by allogeneic CGT?

Yes

If yes, the primary exceptions will be

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* 9. Do you believe the future of allogeneic CGT will be largely dominated by centralized manufacturing?

Yes

If yes, the primary exceptions will be

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* 10. Do you believe the future of autologous CGT will be largely dominated by centralized manufacturing (choose best answer)?

Yes

Yes but less centralized than allogeneic CGT

Yes but less centralized than it is today and less than allogeneic CGT

No it will be dominated by decentralized (closed, automated, PoC or regional) manufacturing

No but it will be more decentralized than exists today

No it will be a fairly even mix of centralized and decentralized manufacturing

Question Title

* 11. Is or will your organization consider using cell expansion technologies such as any of the following (check all that apply):

TerumoBCT QuantumCES

Miltenyi CliniMACS Prodigy

Stir tank bioreactor

Rocker bioreactor

Rotary bioreactor

Plastic culture chambers

n/a

Other (please specify)

We do not do cell expansion

Question Title

* 12. In the future, to the extent that certain Source Material processing steps and/or CGT product manufacturing occurs in functionally closed systems, do you believe regulatory agencies will eventually allow these to be used in low-grade controlled environments (clean rooms) provided cGMP standards are maintained within and upstream/downstream of systems?

Yes

Not

Not sure - this is going to require significant regulatory evolution

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* 13. If technology were to prove capable of supporting safe and uniform/standardized manufacturing of CGT products in closed, automated, systems and they were commercially available, would your organization be more likely consider a decentralized manufacturing model?

Yes

n/a

Question Title

* 14. Do you believe there is (or will be in the near-term) a demand for Source Material provided in specific and uniform cell concentration levels such as total nucleated cell count (TNC) of 5 billion, 10 billion, 15 billion, etc?

Yes there will be a growing trend toward standardized Source Material

Yes but this will remain a small % of demand because most demand will still be for unique Source Material specifications customized to the product

Question Title

* 15. Does your organization use a blood center for the collection of any Source Material? (check all the apply)

Yes for use in product development

Yes as ingredients/reagents for clinical-grade production

Yes as starting material for the manufacture of a clinical product

Yes for use in diagnostic, device, equipment testing

n/a

Yes but for other uses (please specify)

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* 16. If no, are you personally aware of any CGT organization that uses a blood center for the collection of any Source Material?

Yes

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* 17. Identify which of the following organizations you are sufficiently familiar with to name one and/or explain what they do:

Independent blood centers

National blood collection organizations

National organization of independent blood centers

Commercial contract blood/cell collection companies

none of the above

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* 18. Does or will your organization require standardization among Source Material collection facilities of (check all that apply):

equipment

collection protocols

training

quality management systems (QMS)

deviation reporting and root cause investigations

document management systems

contracts

account management

none of the above

Other (please specify)

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* 19. Does or will your organization require a geographically broad infrastructure for Source Material collections for clinical trials and/or commercial distribution?

Yes

n/a

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* 20. Could your organization benefit from a single entity to assist in identifying, contracting with, and training all the collection sites your organization requires?

Yes

n/a

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* 21. Could your organization benefit from a single point of contact assisting with ongoing management, compliance and reporting from all the collection sites required?

Yes

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* 22. Does or will your organization require a Source Material collection infrastructure capable of identifying donors with very specific characteristics (e.g., rare ethnicity, genetic trait, disease, etc)

Yes

n/a

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* 23. If yes, does or will your organization require (or prefer to have) multiple, ongoing collections from rare donors?

Yes

n/a

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* 24. In the future, do you believe all Source Material will eventually be cryopreserved (or stored long-term using other technology) or do you believe there will always be a need for fresh materials?

Most if not all will be cryopreserved (or otherwise stored long-term) in the future

The mix of fresh vs cryopreserved will likely stay relatively the same as it is now

Source Material shipped fresh to manufacturing will dominate

Other (please explain)

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* 25. Do you believe it could be advantageous to your organization or the future sales/delivery of your product, if the contract facility that collected your Source Material had a close working relationship with the hospital(s) which are involved in the delivery of your CGT products?

Yes

n/a

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* 26. Does or will your organization consider using Source Material as any of the following (check all that apply)

Feeder cells

Human Serum

Human Platelet Lysate

Other reagent (please specify)

None of the above

Cell & Gene Therapy Industry Survey

Survey Definitions and Questions

Question Title

* 1. Given evidence of decreasing donor activities at US blood centers, do you believe the CGT industry is likely to face bottleneck issues in future production due to the lack of available Source Material?

Question Title

* 2. Do you share these concerns related to your products?

Question Title

* 3. Do the products you are directly involved in developing require a large and/or ongoing Source Material donor base for adequate source material?

Question Title

* 4. Do you believe the CGT industry is likely to face bottleneck issues in future production because of a shortage of required Source Material either due to donor shortages, inability to identify/recruit 'rare' donors, or lack of donors in certain geographies?

Question Title

* 5. Do you believe some processing capabilities at Source Material collection facilities would be an advantage in the supply chain of CGT products?

Question Title

* 6. Do you believe the existence of vein-to-vein processing/manufacturing capabilities of CGT products at hospitals or health systems would help or hinder the evolution/expansion of CGT products?

Question Title

* 7. Do you believe the future of CGT will be largely dominated by centralized manufacturing?

Question Title

* 8. Do you believe the future of CGT will be largely dominated by allogeneic CGT?

Question Title

* 9. Do you believe the future of allogeneic CGT will be largely dominated by centralized manufacturing?

Question Title

* 10. Do you believe the future of autologous CGT will be largely dominated by centralized manufacturing (choose best answer)?

Question Title

* 11. Is or will your organization consider using cell expansion technologies such as any of the following (check all that apply):

Question Title

Question Title

* 13. If technology were to prove capable of supporting safe and uniform/standardized manufacturing of CGT products in closed, automated, systems and they were commercially available, would your organization be more likely consider a decentralized manufacturing model?

Question Title

* 14. Do you believe there is (or will be in the near-term) a demand for Source Material provided in specific and uniform cell concentration levels such as total nucleated cell count (TNC) of 5 billion, 10 billion, 15 billion, etc?

Question Title

* 15. Does your organization use a blood center for the collection of any Source Material? (check all the apply)

Question Title

* 16. If no, are you personally aware of any CGT organization that uses a blood center for the collection of any Source Material?

Question Title

* 17. Identify which of the following organizations you are sufficiently familiar with to name one and/or explain what they do:

Question Title

* 18. Does or will your organization require standardization among Source Material collection facilities of (check all that apply):

Question Title

* 19. Does or will your organization require a geographically broad infrastructure for Source Material collections for clinical trials and/or commercial distribution?

Question Title

* 20. Could your organization benefit from a single entity to assist in identifying, contracting with, and training all the collection sites your organization requires?

Question Title

* 21. Could your organization benefit from a single point of contact assisting with ongoing management, compliance and reporting from all the collection sites required?

Question Title

* 22. Does or will your organization require a Source Material collection infrastructure capable of identifying donors with very specific characteristics (e.g., rare ethnicity, genetic trait, disease, etc)

Question Title

* 23. If yes, does or will your organization require (or prefer to have) multiple, ongoing collections from rare donors?

Question Title

* 24. In the future, do you believe all Source Material will eventually be cryopreserved (or stored long-term using other technology) or do you believe there will always be a need for fresh materials?

Question Title

* 25. Do you believe it could be advantageous to your organization or the future sales/delivery of your product, if the contract facility that collected your Source Material had a close working relationship with the hospital(s) which are involved in the delivery of your CGT products?

Question Title

* 26. Does or will your organization consider using Source Material as any of the following (check all that apply)