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industry accelerates, so will the demand for
best practices, and better tools and solutions.
For advanced therapies, the product and
patient journey is entirely different from
traditional trials. As such, biotech and pharma
companies will need to overcome challenges
and complexities from regulatory pathways and
patient recruitment to logistics and manufacturing.
CGT therapies also require specialty testing and lab
services, including molecular capabilities and immunohistochemistry testing. Adopting strategic partnerships with deep
CGT expertise with a broad spectrum of solutions and services is vital
to the success of a development program.

References
1. Cynober, Timothé. Why Are There Only 11 Cell and Gene Therapies
in Europe? LabIOTech.eu. Sept. 2020. https://www.labiotech.eu/
in-depth/atmp-cell-gene-therapy-ema/
2. Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter
Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation
and Research on new policies to advance development of safe and
effective cell and gene therapies. US Food and Drug Administration.
January 2019. https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-petermarks-md-phd-director-center-biologics
3. AveXis receives EC approval and activates " Day One " access
program for Zolgensma®, the only gene therapy for spinal muscular
atrophy (SMA). Novartis. May 2020. https://www.novartis.com/
news/media-releases/avexis-receives-ec-approval-and-activates-%22day-one%22-access-program-zolgensma-only-gene-therapy-spinal-muscular-atrophy-sma

4. FDA approves brexucabtagene autoleucel for relapsed or refractory
mantle cell lymphoma. US Food and Drug Administration. July
2020. https://www.fda.gov/drugs/fda-approves-brexucabtagene-autoleucel-relapsed-or-refractory-mantle-cell-lymphoma#:~:text=On%20July%2024%2C%202020%2C%20the,mantle%20
cell%20lymphoma%20(MCL).
5. Toby AuWerter, Jeff Smith, and Lydia The, " Biopharma
portfolio strategy in the era of cell and gene therapy, "
McKinsey & Company. April 2020. https://www.mckinsey.com/
industries/pharmaceuticals-and-medical-products/our-insights/
biopharma-portfolio-strategy-in-the-era-of-cell-and-gene-therapy
6. Innovation in the time of COVID-19: ARM Global Regenerative
Medicine & Advanced Therapy Sector Report. 2020. http://alliancerm.
org/wp-content/uploads/2020/08/ARM_1H-Report_-FINAL.pdf

ClinicalOMICs.com

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