ABSTRACT The clinical translation of regenerative-medicine products, including cell therapies, therapeutic tissue engineering products, and human cell and tissue products, remains limited

because of the so-called ‘valley of death’, that is, the lack of resources necessary to move a product from early preclinical to clinical development. To advance more regenerative-medicine

products into the clinic, academic researchers may benefit greatly from insights into the commercialization process, in particular, through academic startups. In this Review, we discuss key

commercialization aspects, that is, protecting intellectual property, navigating regulatory pathways and obtaining funding, and highlight case studies of academic startups that have

commercialization aspects early in research and development. * Understanding the steps toward establishing intellectual property (IP) within academic institutions is key to conducting and

disseminating IP-yielding research. * Regenerative Medicine Advanced Therapy (RMAT) designations provide a pathway that can expedite the approval process of regenerative-medicine products. *

Grants and non-dilutive mechanisms can support preclinical studies, and dilutive mechanisms of funding and initial public offerings are the major sources of financial capital for clinical

development phases. * Academic institutions and funding agencies should implement policy changes to motivate and drive academic researchers toward the commercialization of

regenerative-medicine technologies. Access through your institution Buy or subscribe This is a preview of subscription content, access via your institution ACCESS OPTIONS Access through your

institution Subscribe to this journal Receive 12 digital issues and online access to articles $119.00 per year only $9.92 per issue Learn more Buy this article * Purchase on SpringerLink *

Instant access to full article PDF Buy now Prices may be subject to local taxes which are calculated during checkout ADDITIONAL ACCESS OPTIONS: * Log in * Learn about institutional

subscriptions * Read our FAQs * Contact customer support SIMILAR CONTENT BEING VIEWED BY OTHERS REGENERATIVE MEDICINE MEETS MATHEMATICAL MODELLING: DEVELOPING SYMBIOTIC RELATIONSHIPS Article

Open access 12 April 2021 ENDURING QUESTIONS IN REGENERATIVE BIOLOGY AND THE SEARCH FOR ANSWERS Article Open access 09 November 2023 ENGINEERING THE NEXT GENERATION OF CELL-BASED

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pre-arthroplasty patient. _Oper. Tech. Sports Med._ 30, 150956 (2022). Article  Google Scholar  Download references ACKNOWLEDGEMENTS The authors thank S. Heimann for assistance with database

searches of regenerative-medicine companies. The authors acknowledge funding support from the NIH through grants R01DE015038, R01AR071457 and R01AR078389. AUTHOR INFORMATION AUTHORS AND

AFFILIATIONS * Department of Biomedical Engineering, University of California Irvine, Irvine, CA, USA Takumi Takahashi, Ryan P. Donahue, Rachel C. Nordberg, Jerry C. Hu & Kyriacos A.

Athanasiou * School of Information Systems and Management, Muma College of Business, University of South Florida, Tampa, FL, USA Steven C. Currall * John A. Paulson School of Engineering and

Applied Sciences, Harvard University, Cambridge, MA, USA Steven C. Currall Authors * Takumi Takahashi View author publications You can also search for this author inPubMed Google Scholar *

Ryan P. Donahue View author publications You can also search for this author inPubMed Google Scholar * Rachel C. Nordberg View author publications You can also search for this author

inPubMed Google Scholar * Jerry C. Hu View author publications You can also search for this author inPubMed Google Scholar * Steven C. Currall View author publications You can also search

for this author inPubMed Google Scholar * Kyriacos A. Athanasiou View author publications You can also search for this author inPubMed Google Scholar CONTRIBUTIONS T.T. conceived the idea

for this Review and contributed to researching the data and literature, discussion, writing and editing. R.P.D. and R.C.N. contributed to researching the data and literature, discussion,

writing and editing of this manuscript. J.C.H. contributed to the discussion, reviewing and editing of the manuscript. S.C.C. and K.A.A. conceived the idea for this Review and contributed to

the discussion, reviewing and editing of the manuscript. CORRESPONDING AUTHOR Correspondence to Kyriacos A. Athanasiou. ETHICS DECLARATIONS COMPETING INTERESTS R.P.D., J.C.H. and K.A.A. are

cofounders of and hold equity in Cartilage Inc. The remaining authors declare no competing interests. PEER REVIEW PEER REVIEW INFORMATION _Nature Reviews Bioengineering_ thanks István

Hornyák, Helen Yu and John Blaho for their contribution to the peer review of this work. ADDITIONAL INFORMATION PUBLISHER’S NOTE Springer Nature remains neutral with regard to jurisdictional

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10/14/2020 (ORCA-T): https://www.globenewswire.com/news-release/2020/10/14/2108307/0/en/Orca-Bio-Receives-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-for-Orca-T.html 11/11/2020

(AB205): https://angiocrinebioscience.com/news-events/news/ 12/17/2020 (AMDC-USR):

https://www.biospace.com/article/fda-grants-rmat-designation-for-cook-myosite-s-investigational-autologous-muscle-derived-cells-for-urinary-sphincter-repair/ 1/11/2021 (RENU):

https://organogenesis.com/news-events/press-release-01112021.html 3/8/2021 (VALOCTOCOGENE ROXAPARVOVEC):

https://investors.biomarin.com/2021-03-08-BioMarin-Announces-FDA-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-Granted-to-Valoctocogene-Roxaparvovec-Investigational-Gene-Therapy-for-Hemophilia-A

3/9/2021 (RP-L201): https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-receives-fda-regenerative-medicine-0/ 4/21/2021 (ALLO-715):

https://ir.allogene.com/news-releases/news-release-details/allogene-therapeutics-announces-fda-regenerative-medicine/ OCT 2021 (REACT):

https://investors.prokidney.com/news-releases/news-release-details/prokidney-corroborates-mechanism-action-reacttm-cell-marker 11/22/2021 (CTX110):

http://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-announces-fda-regenerative-medicine-advanced/ 12/13/2021 (FT516):

https://ir.fatetherapeutics.com/news-releases/news-release-details/fate-therapeutics-highlights-positive-durability-response-data 2022 SPAC MERGER (UD):

https://medcitynews.com/2022/01/regenerative-med-biotech-prokidney-inks-825m-merger-to-back-ckd-cell-therapy/ 1/5/2022 (POSOLEUCEL):

https://ir.allovir.com/news-releases/news-release-details/fda-grants-regenerative-medicine-advanced-therapy-rmat/ 1/10/2022 (CT041):

https://www.prnewswire.com/news-releases/carsgen-announces-ct041-car-t-cell-product-candidate-granted-rmat-designation-by-the-fda-301456859.html 1/12/2022 (C-CAR039):

https://www.cellbiomedgroup.com/newsroom/fda-rmat?lang=en 2/14/2022 (APROART): https://www.ucsf.edu/news/2022/02/422276/ucsf-gene-therapy-deadly-mutation-fast-tracked-fda-review 4/12/2022

(EXOFLO): https://directbiologics.com/fda-grants-direct-biologics-regenerative-medicine-advanced-therapy-rmat-designation-for-the-use-of-exoflo-in-covid-19-related-ards/ 4/20/2022

(POSOLEUCEL): https://ir.allovir.com/news-releases/news-release-details/fda-grants-regenerative-medicine-advanced-therapy-rmat-0/ 4/25/2022 (OBE-CEL):

https://autolus.gcs-web.com/news-releases/news-release-details/fda-grants-regenerative-medicine-advanced-therapy-rmat/ 5/13/2022 (SKINTE):

https://www.prnewswire.com/news-releases/polarityte-announces-fda-regenerative-medicine-advanced-therapy-designation-granted-to-skinte-301546677.html 5/24/2022 (NEOCART):

https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-new-cell-therapy-program-following-fda/ 6/8/2022 (ALLO-501A):

https://ir.allogene.com/news-releases/news-release-details/allogene-therapeutics-announces-fda-granted-regenerative/ 9/28/2022 (CTX130):

https://crisprtx.com/about-us/press-releases-and-presentations/crispr-therapeutics-announces-fda-regenerative-medicine-advanced-therapy-rmat-designation-granted-to-ctx130-for-the-treatment-of-cutaneous-t-cell-lymphomas-ctcl

11/8/2022 (ADP-A2M4CD8): https://www.adaptimmune.com/investors-and-media/news-center/press-releases/detail/234/adaptimmune-reports-increased-response-rate-and-durability 11/29/2022

(CB-010): https://investor.cariboubio.com/news-releases/news-release-details/caribou-biosciences-announces-fda-granted-regenerative-medicine/ 1/26/2023 (REBONUPUTEMCEL):

https://www.discgenics.com/news-posts/2023/1/26/discgenics-announces-fda-regenerative-medicine-advanced-therapy-rmat-designation-granted-to-idct-for-degenerative-disc-disease 2/7/2023

(RP-A501): https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-receives-fda-regenerative-medicine-1/ 2/8/2023 (REXLEMESTROCEL-L):

https://www.globenewswire.com/en/news-release/2023/02/08/2604535/0/en/FDA-Grants-Regenerative-Medicine-Advanced-Therapy-Rmat-Designation-for-Rexlemestrocel-L-in-Chronic-Low-Back-Pain.html

2/11/2023 (CT103A): https://www.iasobio.com/phone/info.php?id=215 3/21/2023 (NTLA-2002):

https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-fda-regenerative-medicine 5/4/2023 (HAV):

https://www.globenewswire.com/news-release/2023/05/04/2661452/0/en/Humacyte-s-Human-Acellular-Vessel-HAV-Receives-FDA-s-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-for-Urgent-Arterial-Repair-Following-Vascular-Trauma.html

5/23/2023 (RP-L301): https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-receives-fda-regenerative-medicine-2/ 5/24/2023 (RGX-121):

https://www.prnewswire.com/news-releases/regenxbio-receives-fda-regenerative-medicine-advanced-therapy-rmat-designation-for-rgx-121-gene-therapy-for-hunter-syndrome-301831944.html 1997

(US$20 M): https://investors.vcel.com/node/11886/html 1999 (US$58 M): https://www.bioworld.com/articles/392839-biomarin-completes-58m-ipo-as-first-product-nears-market?v=preview 2004 (US$21

M): https://www.intelligentinvestor.com.au/shares/asx-msb/mesoblast-limited/float 2007 (US$56 M): https://www.pehub.com/tigenix-ipo-prices-in-brussels/ 2007 REVERSE MERGER (UD):

https://www.cleveland.com/business/2007/06/athersys_goes_public_in_revers.html 2008 (UD): https://roic.ai/company/IOVA 2008 REVERSE MERGER (UD):

https://www.biospace.com/article/mimedx-announces-post-split-name-change-/ 2011 REVERSE MERGER (UD):

https://www.cfodive.com/news/reverse-merger-helped-recession-hit-axogen-regain-footing/602531/ 2013 (SEK 21 M):

https://www.marketscreener.com/quote/stock/MENDUS-AB-PUBL-17946401/news/Immunicum-AB-has-completed-an-IPO-in-the-amount-of-SEK-21-40-million-39111826/ 2013 (US$116 M):

https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-closing-initial-public-offering-and 2013 (US$40 M):

https://www.fiercebiotech.com/r-d/fate-ipo-flop-raises-fresh-concerns-about-investors-appetite-for-biotech 2013 REVERSE MERGER (UD):

https://www.biospace.com/article/china-s-b-cellular-biomedicine-group-b-announces-reverse-merger-/ 2013 REVERSE MERGER (UD):

https://www.businesswire.com/news/home/20131121005377/en/Capricor-and-Nile-Therapeutics-Complete-Merger-to-Form-Capricor-Therapeutics-Inc. 2014 (US$265 M):

https://www.fiercebiotech.com/r-d/updated-juno-banks-a-265m-ipo-pushing-next-big-thing-oncology 2015 (US$124 M):

https://www.businesswire.com/news/home/20150408005269/en/SanBio-Announces-Pricing-of-Initial-Public-Offering-on-Tokyo-Stock-Exchange 2015 (US$159 M):

https://www.globenewswire.com/news-release/2015/09/22/770163/10150220/en/REGENXBIO-Announces-Closing-of-Initial-Public-Offering-and-Full-Exercise-of-Underwriters-Option-to-Purchase-Additional-Shares.html

2015 (US$191 M): https://www.nasdaq.com/articles/adaptimmune-therapeutics-prices-upsized-ipo-17-high-end-range-2015-05-05 2015 (US$36 M):

https://www.fiercebiotech.com/r-d/kiadis-banks-36m-a-euro-ipo-to-fund-its-stem-cell-r-d 2015 (US$5.5 M):

https://www.prnewswire.com/news-releases/asterias-biotherapeutics-completes-public-offering-and-private-placement-of-common-stock-300033976.html 2015 (US$81 M):

https://ir.voyagertherapeutics.com/news-releases/news-release-details/voyager-therapeutics-announces-closing-initial-public-offering/ 2016 (US$113 M):

https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-closing-initial-public-offering 2016 (US$56 M):

https://www.fiercebiotech.com/biotech/crispr-therapeutics-raises-a-56m-ipo-but-patent-battles-potential-stock-drops-loom 2016 (US$75 M):

https://pitchbook.com/newsletter/audentes-raises-75m-in-ipo 2016 ACQUIRED BY MALLINCKRODT (UD):

https://www.fiercebiotech.com/medical-devices/mallinckrodt-acquires-regenerative-medicine-company-stratatech 2017 (US$46 M):

https://www.globenewswire.com/en/news-release/2017/09/22/1131592/0/en/Krystal-Biotech-Announces-Closing-of-Initial-Public-Offering-and-Full-Exercise-of-Underwriters-Option-to-Purchase-Additional-Shares.html

2017 (US$77 M):

https://www.globenewswire.com/en/news-release/2017/10/02/1139020/0/en/Nightstar-Announces-Closing-of-Initial-Public-Offering-and-Full-Exercise-of-Underwriters-Option-to-Purchase-Additional-American-Depositary-Shares.html

2017 REVERSE MERGER (UD):

https://www.globenewswire.com/news-release/2017/04/07/1052850/0/en/PolarityTE-TM-Inc-Announces-Completion-of-Merger-and-Acquires-Important-Intellectual-Property-for-Regenerative-Medicine-Programs.html

2018 (US$100 M): https://www.businesswire.com/news/home/20180625006210/en/Magenta-Therapeutics-Announces-Closing-of-Initial-Public-Offering 2018 (US$160 M):

https://autolus.gcs-web.com/news-releases/news-release-details/autolus-announces-closing-initial-public-offering-and-full/ 2018 (US$225 M):

https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-announces-closing-225-million-initial/ 2018 (US$373 M):

https://ir.allogene.com/news-releases/news-release-details/allogene-therapeutics-announces-closing-initial-public-offering/ 2018 ACQUIRED BY CELGENE (US$9,000 M):

https://www.geekwire.com/2018/juno-therapeutics-acquired-celgene-9b-dramatic-deal-rising-biotech-star/ 2018 ACQUIRED BY TAKEDA PHARMACEUTICAL COMPANY (US$608 M):

https://www.pharmaceutical-technology.com/news/takeda-tigenix-acquisition-608m/ 2018 REVERSE MERGER (UD):

https://endpts.com/gene-therapy-startup-rocket-pharma-reverse-merges-with-troubled-inotek-after-25m-raise/ 2018 REVERSE MERGER (UD):

https://organogenesis.com/news-events/press-release-12102018.html 2019 ACQUIRED BY BIOGEN (US$800 M):

https://investors.biogen.com/news-releases/news-release-details/biogen-completes-acquisition-nightstar-therapeutics 2019 ACQUIRED BY BIOTIME (UD):

https://www.spglobal.com/marketintelligence/en/news-insights/trending/dgy96s7WowE7oCnW-Mu7KA2 2019 ACQUIRED BY CASTLE CREEK PHARMACEUTICAL (US$63 M):

https://www.globenewswire.com/en/news-release/2019/09/12/1915187/33272/en/Castle-Creek-Pharmaceutical-Holdings-to-Acquire-Fibrocell.html 2019 ACQUIRED BY SUMITOMO DAINIPPON PHARMA (UD):

https://enzyvant.com/sumitomo-dainippon-pharma-completes-the-formation-of-the-strategic-alliance-with-roivant-sciences/ 2019 REVERSE MERGER (UD):

https://www.nbcphiladelphia.com/news/local/malvern-gene-therapy-company-completes-reverse-merger/219782/ 2020 (US$224 M):

https://investors.poseida.com/news-releases/news-release-details/poseida-therapeutics-announces-pricing-initial-public-offering/ 2020 (US$318 M):

https://www.globenewswire.com/en/news-release/2020/08/03/2072010/0/en/AlloVir-Announces-Closing-of-Upsized-Initial-Public-Offering-and-Full-Exercise-of-the-Underwriters-Option-to-Purchase-Additional-Shares.html

2020 ACQUIRED BY ASTELLAS PHARMA (US$3,000 M): https://www.spglobal.com/marketintelligence/en/news-insights/trending/VpqpVltOd5YKifSvTvBf2A2 2021 (US$150 M):

https://ir.talaristx.com/news-releases/news-release-details/talaris-therapeutics-announces-closing-initial-public-offering/ 2021 (US$304 M):

https://www.businesswire.com/news/home/20210727006010/en/Caribou-Biosciences-Announces-Closing-of-Upsized-Initial-Public-Offering 2021 (US$400 M):

https://www.bioworld.com/articles/508424-car-t-developer-carsgen-debuts-on-hkex-with-400m-ipo-targets-bcma-first?v=preview 2021 ACQUIRED BY CONSORTIUM (US$411 M):

https://www.prnewswire.com/news-releases/cellular-biomedicine-group-inc-announces-completion-of-merger-301231873.html 2021 ACQUIRED BY SANOFI (US$358 M):

https://www.fiercebiotech.com/biotech/sanofi-inks-358m-kiadis-takeover-to-acquire-nk-cell-platform 2021 REVERSE MERGER (UD):

https://www.marketscreener.com/quote/stock/HUMACYTE-INC-126300449/news/Humacyte-Inc-completed-the-acquisition-of-Alpha-Healthcare-Acquisition-Corp-from-a-group-of-share-36263997/ 2022

ACQUIRED BY CANARIA BIO (UD): https://www.koreabiomed.com/news/articleView.html?idxno=20085 2022 MERGER WITH CEND THERAPEUTICS (UD):

https://www.globenewswire.com/en/news-release/2022/09/15/2516989/18623/en/Caladrius-Biosciences-and-Cend-Therapeutics-Announce-Closing-of-Merger-and-the-Emergence-of-Lisata-Therapeutics.html

20 CELLULAR AND GENE THERAPY PRODUCTS: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products 21 CFR 312:

https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRsearch.cfm?CFRPart=312 525 NON-REGENERATIVE-MEDICINE DRUGS:

https://www.fda.gov/drugs/development-approval-process-drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products 7% IN BANK FEES FOR A US$100 MILLION IPO:

https://news.crunchbase.com/public/want-to-take-your-startup-public-heres-what-it-actually-costs-to-ipo/ 86 RMAT DESIGNATIONS:

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/cumulative-cber-regenerative-medicine-advanced-therapy-rmat-designation-requests-received-fiscal A LIST AND

RANKING OF INCUBATORS AND ACCELERATORS WORLDWIDE: https://www.andeglobal.org/publication/the-ubi-global-world-rankings-of-business-incubators-and-accelerators/ A US$20 MILLION AWARD FROM THE

CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE (CIRM): https://www.fiercebiotech.com/biotech/stemcells-inc-awarded-20-million-from-california-institute-for-regenerative-medicine-for ABECMA

(2021): https://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-adult-patients-multiple-myeloma ACCELERATORS:

https://www.feedough.com/accelerator-vs-incubator/ ACQUIRED BY CELGENE: https://www.geekwire.com/2018/juno-therapeutics-acquired-celgene-9b-dramatic-deal-rising-biotech-star/ ACQUIRED BY

MALLINCKRODT PHARMACEUTICALS: https://www.fiercebiotech.com/medical-devices/mallinckrodt-acquires-regenerative-medicine-company-stratatech ACQUIRED BY NOVARTIS:

https://www.novartis.com/news/media-releases/novartis-enters-agreement-acquire-avexis-inc-usd-87-bn-transform-care-sma-and-expand-position-gene-therapy-and-neuroscience-leader ACQUIRED IN A

REVERSE MERGER:

https://www.businesswire.com/news/home/20190930005241/en/Ocugen-Announces-Completion-of-its-Merger-with-Histogenics-to-Create-Nasdaq-Listed-Clinical-Stage-Company-Developing-Novel-Ocular-Gene-Therapies-and-Biotherapeutics

ACQUIRED THROUGH A REVERSE MERGER: https://www.massdevice.com/stemcells-soars-on-reverse-merger-with-israels-microbot-medical/ ADSTILADRIN (2022):

https://www.ferring.com/ferring-receives-approval-from-u-s-fda-for-adstiladrin-for-high-risk-bcg-unresponsive-non-muscle-invasive-bladder-cancer/ AMGEN:

https://sciencebusiness.net/news/74714/UCL-spin-out-sold-to-Amgen-in-%241-billion-deal AMOUNT FOR A SEED ROUND: https://www.paddle.com/resources/guide-to-seed-funding-for-startups ANALYSTS

CONSIDER: https://www.statnews.com/2016/06/01/stem-cell-company-shutters APPROVED AS BREYANZI:

https://news.bms.com/news/details/2021/U.S.-Food-and-Drug-Administration-Approves-Bristol-Myers-Squibbs-Breyanzi-lisocabtagene-maraleucel-a-New-CAR-T-Cell-Therapy-for-Adults-with-Relapsed-or-Refractory-Large-B-cell-Lymphoma/default.aspx

APPROVED AS RETHYMIC): https://endpts.com/after-three-decades-and-a-surprise-rejection-first-treatment-for-babies-born-without-a-thymus-secures-fda-approval/ APPROVED BY THE US FOOD AND

DRUG ADMINISTRATION: https://www.fda.gov/vaccines-blood-biologics/zolgensma APPROVED: https://www.fda.gov/vaccines-blood-biologics/stratagraft APROART:

https://www.cirm.ca.gov/our-progress/awards/ex-vivo-transduction-human-artemis-dclre1c-cdna-lentiviral-vector-aproart-cd34 ATIR101:

https://www.cancer.gov/publications/dictionaries/cancer-drug/def/allodepleted-t-cell-immunotherapeutic-atir101 AVERAGES FOR VC SERIES A, B, AND C:

https://www.evaluate.com/vantage/articles/insights/venture-financing-ipo/venture-financing-holds-steady-device-makers AVEXIS:

https://www.novartis.com/news/media-releases/novartis-enters-agreement-acquire-avexis-inc-usd-87-bn-transform-care-sma-and-expand-position-gene-therapy-and-neuroscience-leader BAYLOR COLLEGE

OF MEDICINE: https://elevate.bio/press-releases/elevatebio-announces-that-allovir-joins-its-portfolio-of-highly-innovative-cell-and-gene-therapy-companies/;

https://www.tessacell.com/about-tessa-therapeutics/ BIOVEX: https://mergr.com/amgen-acquires-biovex-group BLA CLINICAL REVIEW: https://www.fda.gov/vaccines-blood-biologics/stratagraft BLA

PHARMACOLOGY/TOXICOLOGY REVIEW OF STRATAGRAFT: https://www.fda.gov/vaccines-blood-biologics/stratagraft BLUEBIRD BIO (FOUNDED AS GENETIX PHARMACEUTICALS):

https://investor.bluebirdbio.com/news-releases/news-release-details/genetix-pharmaceuticals-renamed-bluebird-bio-announces BREYANZI (2021):

https://news.bms.com/news/details/2021/U.S.-Food-and-Drug-Administration-Approves-Bristol-Myers-Squibbs-Breyanzi-lisocabtagene-maraleucel-a-New-CAR-T-Cell-Therapy-for-Adults-with-Relapsed-or-Refractory-Large-B-cell-Lymphoma/default.aspx

CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE (CIRM): https://www.cirm.ca.gov/ CARTILAGE REPAIR PRODUCTS:

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/preparation-ides-and-inds-products-intended-repair-or-replace-knee-cartilage CARVYKTI (2022):

https://www.jnj.com/u-s-fda-approves-carvykti-ciltacabtagene-autoleucel-janssens-first-cell-therapy-a-bcma-directed-car-t-immunotherapy-for-the-treatment-of-patients-with-relapsed-or-refractory-multiple-myeloma

CASE WESTERN RESERVE UNIVERSITY:

https://case.edu/medicine/about/newsroom/our-latest-news/case-western-reserve-center-stem-cell-and-regenerative-medicine-and-athersys-receive-1m-third-frontier-grant-support-research-spinal-cord-injury

CELLULAR IMMUNOTHERAPY PRODUCT PROVENGE: https://stanmed.stanford.edu/pioneering-cancer-drug-provenge-fresh-look CFR TITLE 21:

https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/cfrsearch.cfm CGT PRODUCTS:

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/preclinical-assessment-investigational-cellular-and-gene-therapy-products CHILDREN’S HOSPITAL OF PHILADELPHIA

UNIVERSITY OF PENNSYLVANIA: https://www.chop.edu/news/childrens-hospital-philadelphia-celebrates-fda-approval-gene-therapy-inherited-blindness CITY OF HOPE NATIONAL MEDICAL CENTER:

https://www.cityofhope.org/news/phase-1-clinical-trial-of-mb-101 CLINICAL TRIALS:

https://www.fda.gov/patients/clinical-trials-what-patients-need-know/what-are-different-types-clinical-research COMPLETED AN INITIAL PUBLIC OFFERING (IPO):

https://www.globenewswire.com/en/news-release/2016/02/11/809635/0/en/AveXis-Announces-Pricing-of-Initial-Public-Offering.html CONSIDERED SAFETY DATA GENERATED FROM THE PHASE I/IIA AND PHASE

II TRIALS FOR AN ALTERNATE INDICATION: https://www.fda.gov/vaccines-blood-biologics/stratagraft CONVERTIBLE NOTE: https://www.investopedia.com/terms/s/senior-convertible-note.asp COOPERATIVE

PATENT CLASSIFICATION SYSTEM: https://www.cooperativepatentclassification.org/about CURRENT GOOD MANUFACTURING PRACTICE (CGMP) REGULATIONS:

https://www.fda.gov/drugs/pharmaceutical-quality-resources/current-good-manufacturing-practice-cgmp-regulations DILUTION OF EQUITY:

https://www.svb.com/startup-insights/startup-equity/startup-equity-dilution DUKE UNIVERSITY, MASSACHUSETTS INSTITUTE OF TECHNOLOGY:

https://wraltechwire.com/2021/02/18/a-blooming-success-the-rise-of-humacyte-from-humble-beginnings-to-1-1b-valuation/ DUKE UNIVERSITY:

https://endpts.com/after-three-decades-and-a-surprise-rejection-first-treatment-for-babies-born-without-a-thymus-secures-fda-approval/ DUKE UNIVERSITY:

https://endpts.com/after-three-decades-and-a-surprise-rejection-first-treatment-for-babies-born-without-a-thymus-secures-fda-approval/ ‘END OF PHASE’ MEETINGS:

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/otp-ind-milestone-type-b-meetings ENZYVANT THERAPEUTICS GMBH LICENCED THE TECHNOLOGY FROM DUKE UNIVERSITY:

https://www.prnewswire.com/news-releases/enzyvant-to-develop-novel-biologic-therapy-for-complete-digeorge-syndrome-300386922.html ESTABLISHED IP:

https://patents.justia.com/assignee/histogenics-corporation EXCEEDING US$20 BILLION IN 2021: https://alliancerm.org/sector-report/2021-annual-report/ EXCESS OF THESE AMOUNTS:

https://grants.nih.gov/grants/guide/notice-files/NOT-HL-22-065.html FDA APPROVAL OF JCAR017:

https://news.bms.com/news/details/2021/U.S.-Food-and-Drug-Administration-Approves-Bristol-Myers-Squibbs-Breyanzi-lisocabtagene-maraleucel-a-New-CAR-T-Cell-Therapy-for-Adults-with-Relapsed-or-Refractory-Large-B-cell-Lymphoma/default.aspx

FERRING PHARMACEUTICALS: https://ferringusa.com/?press=ferring-and-blackstone-life-sciences-invest-over-570-million-usd-in-novel-gene-therapy-for-bladder-cancer-patients FINANCIAL EXIT

STRATEGIES: https://www.investopedia.com/terms/b/business-exit-strategy.asp FOUNDED IN 2013:

https://www.prnewswire.com/news-releases/spark-therapeutics-launched-with-50-million-in-financing-to-advance-late-and-mid-stage-gene-therapy-programs-with-clinical-proof-of-concept-228752221.html

FRED HUTCHINSON CANCER CENTER, ST JUDE CHILDREN’S RESEARCH HOSPITAL: https://ir.mustangbio.com/news-events/press-releases/detail/68/mustang-bio-and-st-jude-childrens-research-hospital FRED

HUTCHINSON CANCER RESEARCH CENTER MEMORIAL SLOAN-KETTERING CANCER CENTER SEATTLE CHILDREN’S RESEARCH INSTITUTE:

https://www.forbes.com/sites/matthewherper/2014/08/05/why-this-cancer-fighting-company-has-raised-300-million-in-just-12-months/ FRED HUTCHINSON CANCER RESEARCH CENTER MEMORIAL

SLOAN-KETTERING CANCER CENTER SEATTLE CHILDREN’S RESEARCH INSTITUTE UNIVERSITY OF CALIFORNIA, SAN FRANCISCO ST JUDE CHILDREN’S RESEARCH HOSPITAL:

https://www.businesswire.com/news/home/20171101006552/en/Juno-Therapeutics-Reports-Third-Quarter-2017-Financial-Results FUNDING CAPS: https://www.sbir.gov/about GENESIS SEED FUND:

https://research.rutgers.edu/researcher-support/commercialize-your-innovation/launch-startup/new-ventures/genesis-seed-fund GILEAD SCIENCES:

https://www.gilead.com/news-and-press/company-statements/kite-pharma HARVARD UNIVERSITY, MASSACHUSETTS INSTITUTE OF TECHNOLOGY: https://news.mit.edu/2001/sicklecell HARVARD UNIVERSITY,

WASHINGTON UNIVERSITY IN ST LOUIS, STANFORD UNIVERSITY, SAN RAFFAELE TELETHON INSTITUTE FOR GENE THERAPY, UNIVERSITY OF BASEL:

https://investor.magentatx.com/news-releases/news-release-details/magenta-therapeutics-announces-485m-series-financing-transform/ HARVARD UNIVERSITY:

https://hsci.harvard.edu/company-startups HARVARD UNIVERSITY: https://hsci.harvard.edu/company-startups HEMGENIX (2022):

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b HIGHLY COMPETITIVE: https://grantengine.com/sbir-sttr-paylines-by-nih-institute/

IMLYGIC (2015): https://www.amgen.com/newsroom/press-releases/2015/10/fda-approves-imlygic-talimogene-laherparepvec-as-first-oncolytic-viral-therapy-in-the-us INITIAL TARGETED ENGAGEMENT

FOR REGULATORY ADVICE ON CBER/CDER PRODUCTS (INTERACT) MEETING: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/otp-interact-meeting INTERPRETATIONS OF SECTION

506(G)(8) BY THE US FOOD AND DRUG ADMINISTRATION (FDA): https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/resources-related-regenerative-medicine-therapies

‘INVENTIVE STEP’: https://www.wipo.int/pct/en/texts/ispe/13_01_02.html INVESTIGATIONAL NEW DRUG APPLICATION: https://www.fda.gov/vaccines-blood-biologics/stratagraft IPO:

https://pitchbook.com/blog/ipo-process-explained JLABS: https://jnjinnovation.com/home JOHNS HOPKINS UNIVERSITY: https://www.sdbj.com/technology/capricor-therapeutics-relocates-la-jolla/

JOHNSON & JOHNSON: https://johnsonandjohnson.gcs-web.com/news-releases/news-release-details/johnson-johnson-announces-completion-merger-centocor-inc/ JUNO THERAPEUTICS (FOUNDED AS FC

THERAPEUTICS): https://pitchbook.com/profiles/company/60580-27#overview JUNO THERAPEUTICS:

https://www.geekwire.com/2018/juno-therapeutics-acquired-celgene-9b-dramatic-deal-rising-biotech-star/ KATHOLIEKE UNIVERSITEIT LEUVEN GHENT UNIVERSITY:

https://www.flandersinvestmentandtrade.com/invest/en/news/tigenix-raise-20-million-euro-product-trials KYMRIAH (2017):

https://www.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm-ctl019-children-and-young-adults-b-cell-all-refractory-or-has-relapsed-least-twice

KYMRIAH: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/kymriah-tisagenlecleucel LUXTURNA (2017):

https://sparktx.com/press_releases/fda-approves-spark-therapeutics-luxturna-voretigene-neparvovec-rzyl-a-one-time-gene-therapy-for-patients-with-confirmed-biallelic-rpe65-mutation-associated-retinal-dystrophy/

LUXTURNA TO BE APPROVED BY THE US FOOD AND DRUG ADMINISTRATION:

https://sparktx.com/press_releases/fda-approves-spark-therapeutics-luxturna-voretigene-neparvovec-rzyl-a-one-time-gene-therapy-for-patients-with-confirmed-biallelic-rpe65-mutation-associated-retinal-dystrophy/

M&A: https://www.sba.gov/business-guide/grow-your-business/merge-acquire-businesses MACI (2016):

https://investors.vcel.com/news-releases/news-release-details/fda-approves-maci-treatment-symptomatic-cartilage-defects-knee MALLINCKRODT PHARMACEUTICALS:

https://mallinckrodt.gcs-web.com/news-releases/news-release-details/mallinckrodt-announces-us-fda-approval-stratagraftr-allogeneic/ MASSACHUSETTS INSTITUTE OF TECHNOLOGY:

https://organogenesis.com/about-us/ MASSACHUSETTS INSTITUTE OF TECHNOLOGY: https://organogenesis.com/about-us/ MGTA-456:

https://investor.magentatx.com/news-releases/news-release-details/magenta-therapeutics-announces-updated-phase-2-data-mgta-456/ NATIONAL CANCER INSTITUTE:

https://www.stocktitan.net/news/IOVA/iovance-biotherapeutics-announces-first-patient-randomized-in-phase-1fnhlejx69wn.html NATIONWIDE CHILDREN’S HOSPITAL OHIO STATE UNIVERSITY:

https://www.nationwidechildrens.org/newsroom/news-releases/2013/10/avexis-biolife-licenses-spinal-muscular-atrophy-sma-patent-portfolio-from-nationwide-childrens NATIONWIDE CHILDREN’S

HOSPITAL:

https://www.prnewswire.com/news-releases/abeona-therapeutics-and-nationwide-childrens-hospital-receive-prestigious-global-genes-champions-of-hope-award-for-patient-advocacy-227092241.html

NIH DATA MANAGEMENT AND SHARING POLICY: https://oir.nih.gov/sourcebook/intramural-program-oversight/intramural-data-sharing/2023-nih-data-management-sharing-policy NIH’S COMMERCIALIZATION

READINESS PILOT (CRP) PROGRAMME: https://seed.nih.gov/support-for-small-businesses/commercialization-enhancement-programs/crp NO MAJOR SAFETY CONCERNS WERE GENERATED FROM THE PRECLINICAL

STUDIES: https://www.fda.gov/vaccines-blood-biologics/stratagraft NSF-FUNDED INNOVATION-CORPS (I-CORPS): https://beta.nsf.gov/funding/initiatives/i-corps/about-i-corps OBTAINED REGENERATIVE

MEDICINE ADVANCED THERAPY DESIGNATION: https://www.prnewswire.com/news-releases/us-fda-designates-mallinckrodts-stratagraft-as-regenerative-medicine-advanced-therapy-300489200.html ONLY THE

PHASE IB AND PHASE III TRIALS WERE CONSIDERED FOR EFFICACY: https://www.fda.gov/vaccines-blood-biologics/stratagraft ORCA-T:

https://ashpublications.org/blood/article/140/Supplement1/654/487614/Precision-Engineered-Cell-Therapy-Orca-T OTL-103:

https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-announces-fda-regenerative-medicine/ OVER 1,000 NEW DEVICES:

https://www.fda.gov/about-fda/center-devices-and-radiological-health/cdrh-reports UNIVERSITY OF OXFORD, UNIVERSITY OF PENNSYLVANIA: https://www.adaptimmune.com/our-company PATENT COOPERATION

TREATY (PCT): https://www.wipo.int/pct/en/faqs/faqs.html PEAK SALES FORECAST: https://www.europeanpharmaceuticalreview.com/news/179320/cd19-car-t-agents-to-boost-blood-cancer-market/ PHASE

0 GRANTS: https://www.sbir.gov/sites/default/files/SBIR-Table_StateMatchingPhase0_Sept2020.pdf PLEDGED A US$50 MILLION COMMITMENT FROM THE CHILDREN’S HOSPITAL OF PHILADELPHIA:

https://www.prnewswire.com/news-releases/spark-therapeutics-launched-with-50-million-in-financing-to-advance-late-and-mid-stage-gene-therapy-programs-with-clinical-proof-of-concept-228752221.html

PRE-IND MEETING: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/otp-pre-ind-meetings PRIVATE EQUITY (PE): https://www.investopedia.com/terms/p/privateequity.asp

PRODUCT REVENUE OF BREYANZI: https://bioprocessintl.com/bioprocess-insider/therapeutic-class/bms-sees-car-t-sales-rocket-in-line-with-increased-capacity/ PROMOTION AND TENURE-INNOVATION AND

ENTREPRENEURSHIP (PTIE) COALITION: https://ptie.org/ PROVENGE (2010): https://stanmed.stanford.edu/pioneering-cancer-drug-provenge-fresh-look/ QUALITY MANAGEMENT SYSTEM:

https://www.nidcr.nih.gov/sites/default/files/2017-12/quality-management-of-clinical-research-brief-overview.doc RAISED A TOTAL OF US$310 MILLION:

https://www.crunchbase.com/organization/juno-therapeutics/company_financials RAISED AN ADDITIONAL US$72.8 MILLION IN SERIES B FUNDING:

https://www.prnewswire.com/news-releases/spark-therapeutics-raises-728-million-in-oversubscribed-financing-260806381.html RAISED US$304 MILLION THROUGH ITS INITIAL PUBLIC OFFERING:

https://www.nasdaq.com/articles/juno-therapeutics-prices-upsized-ipo-24-largest-2014-biotech-ipo-22-billion-market-cap REASON FOR NONCOMPLIANCE:

https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=312&showFR=1 REGENERATIVE MEDICINE ADVANCED THERAPY (RMAT) DESIGNATION:

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation REGENERATIVE MEDICINE MINNESOTA: https://www.regenmedmn.org/

RETHYMIC (2021):

https://enzyvant.com/enzyvant-receives-fda-approval-for-rethymic-allogeneic-processed-thymus-tissue-agdc-a-one-time-regenerative-tissue-based-therapy-for-pediatric-congenital-athymia/

REVERSE MERGER: https://www.investopedia.com/terms/r/reversetakeover.asp REXLEMESTROCEL-L: https://go.drugbank.com/drugs/DB17606 RISK EVALUATION AND MITIGATION STRATEGY PROGRAMME:

https://www.fda.gov/drugs/drug-safety-and-availability/risk-evaluation-and-mitigation-strategies-rems ROCHE:

https://technical.ly/startups/spark-therapeutics-acquired-roche-bill-marrazzo-university-city-science-center-gene-therapy/ ROUGHLY 90% OF APPLICATIONS FILED ARE UTILITY PATENTS:

https://www.uspto.gov/web/offices/ac/ido/oeip/taf/data/patdesc.htm SAN RAFFAELE-TELETHON INSTITUTE FOR GENE THERAPY:

https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-announces-fda-regenerative-medicine/ SBA LOANS: https://www.sba.gov/funding-programs/loans SBIR AND STTR

PROGRAMMES: https://www.sbir.gov/node/2120519 SECONDARY PUBLIC OFFERINGS: https://www.cbinsights.com/research-what-is-a-secondary-offering SECTION 361:

https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=1271 SECTION 506(G)(8) OF THE UNITED STATES (US) FEDERAL FOOD, DRUG, AND COSMETIC ACT:

https://www.congress.gov/114/plaws/publ255/PLAW-114publ255.htm SEOUL NATIONAL UNIVERSITY: https://www.koreabiomed.com/news/articleView.html?idxno=20085 SETTLED A LEGAL DISPUTE:

https://www.wsj.com/articles/juno-therapeutics-settles-patent-dispute-with-novartis-1428326691 SEVERAL EXPEDITED PROGRAMMES:

https://www.fda.gov/patients/learn-about-drug-and-device-approvals/fast-track-breakthrough-therapy-accelerated-approval-priority-review SKINTE: https://www.woundsource.com/product/skinte

SKYSONA (2022): https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-receives-fda-accelerated-approval-skysonar-gene SPARK THERAPEUTICS:

https://sparktx.com/press_releases/spark-therapeutics-enters-into-definitive-merger-agreement-with-roche/ STANFORD UNIVERSITY: https://orcabio.com/who-we-are/ STANFORD UNIVERSITY:

https://www.medeortx.com/about-us.php STRATAGRAFT (2021): https://mallinckrodt.gcs-web.com/news-releases/news-release-details/mallinckrodt-announces-us-fda-approval-stratagraftr-allogeneic/

STRATAGRAFT: https://www.stratagraft.com/ STRATATECH: https://www.fiercebiotech.com/medical-devices/mallinckrodt-acquires-regenerative-medicine-company-stratatech SUBSEQUENTLY APPROVED:

https://www.ns-healthcare.com/news/breyanzi-large-b-cell-lymphoma/ SUMITOMO PHARMA:

https://endpts.com/after-three-decades-and-a-surprise-rejection-first-treatment-for-babies-born-without-a-thymus-secures-fda-approval/ TECARTUS (2020):

https://www.gilead.com/news-and-press/press-room/press-releases/2020/7/us-fda-approves-kites-tecartus-the-first-and-only-car-t-treatment-for-relapsed-or-refractory-mantle-cell-lymphoma

TECHNOLOGY FOR BRENYANZI: https://www.forbes.com/sites/matthewherper/2014/08/05/why-this-cancer-fighting-company-has-raised-300-million-in-just-12-months/?sh=449f6d8b50d5 TECHNOLOGY FOR

STRATAGRAFT: https://pathology.wisc.edu/2021/06/21/stratagraft-approved-by-fda/ TECHNOLOGY OF ZOLGENSMA:

https://www.nationwidechildrens.org/newsroom/news-releases/2013/10/avexis-biolife-licenses-spinal-muscular-atrophy-sma-patent-portfolio-from-nationwide-childrens THREE FDA-APPROVED PRODUCTS:

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/cber-regenerative-medicine-advanced-therapy-rmat-approvals THREE TYPES OF PATENT:

https://www.uspto.gov/patents/basics/general-information-patents “TREAT, MODIFY, REVERSE, OR CURE A SERIOUS OR LIFE-THREATENING CONDITION”:

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation TT11: https://www.tessacell.com/pipeline/ UNIQURE (FOUNDED AS

AMSTERDAM MOLECULAR THERAPEUTICS): https://www.biopharmadive.com/news/hemophilia-gene-therapy-fda-approval-hemgenix-csl-uniqure/636999/ UNIVERSITÉ DE MONTRÉAL UNIVERSITY OF TORONTO:

https://www.signalsblog.ca/catching-up-with-excellthera-and-co-founder-guy-sauvageau/ UNIVERSITY COLLEGE LONDON: https://www.uclb.com/portfolio/our-spinouts/orchard-theapeutics-plc/

UNIVERSITY OF CALIFORNIA SAN DIEGO (UCSD): https://innovation.ucsd.edu/commercialize/licensing.html UNIVERSITY OF CALIFORNIA, BERKELEY: https://ipira.berkeley.edu/caribou-biosciences-inc

UNIVERSITY OF CALIFORNIA, BERKELEY: https://ipira.berkeley.edu/intellia UNIVERSITY OF CALIFORNIA, IRVINE:

https://innovation.uci.edu/2016/11/20161129special-feature-stem-cell-strides-asterias-biotherapeutics/ UNIVERSITY OF CALIFORNIA, IRVINE:

https://innovation.uci.edu/2020/06/uci-startup-company-jcyte-partners-with-santen-pharmaceutical/ UNIVERSITY OF CALIFORNIA, LOS ANGELES: https://labusinessjournal.com/news/arie-belldegrun/

UNIVERSITY OF CALIFORNIA, LOS ANGELES: https://www.biopharmadive.com/news/Kite-Pharma-UCLA-tcell-allogeneic/423263/ UNIVERSITY OF CALIFORNIA, SAN FRANCISCO:

https://www.ucsf.edu/news/2022/02/422276/ucsf-gene-therapy-deadly-mutation-fast-tracked-fda-review UNIVERSITY COLLEGE LONDON: https://www.autolus.com/about-us/ UNIVERSITY OF COLORADO

UNIVERSITY OF ILLINOIS AT CHICAGO: https://www.fiercebiotech.com/biotech/eye-specialist-ocugen-looks-east-for-second-round-fundraising UNIVERSITY OF EASTERN FINLAND:

https://www.uef.fi/en/article/university-of-eastern-finland-and-ferring-ventures-to-continue-collaboration-in-gene-and-cell UNIVERSITY OF FLORIDA:

https://ir.axogeninc.com/press-releases/detail/811/axogen-inc-receives-david-j-gury-company-of-the-year UNIVERSITY OF FLORIDA:

https://news.drgator.ufl.edu/2013/09/26/uf-teams-with-company-to-advance-gene-therapy-treatments/ UNIVERSITY OF LOUISVILLE:

https://www.uoflnews.com/post/uofltoday/uofl-born-company-secures-100-million-to-advance-cell-therapy/ UNIVERSITY OF MASSACHUSETTS UNIVERSITY OF CALIFORNIA, SAN FRANCISCO STANFORD

UNIVERSITY: https://www.umassmed.edu/es/news/news-archives/2014/02/voyager-therapeutics-targets-novel-gene-therapies-to-combat-diseases/ UNIVERSITY OF MINNESOTA:

https://www.nytimes.com/2002/12/11/business/technology-politically-correct-stem-cell-is-licensed-to-biotech-concern.html UNIVERSITY OF OXFORD:

https://www.ox.ac.uk/news/2015-11-09-£23-million-boost-oxford-spinout-company UNIVERSITY OF PENNSYLVANIA:

https://pci.upenn.edu/regenxbio-receives-fda-fast-track-designation-for-its-novel-gene-therapy-candidate/ UNIVERSITY OF PENNSYLVANIA:

https://www.novartis.com/news/media-releases/novartis-kymriah-receives-fda-regenerative-medicine-advanced-therapy-designation-follicular-lymphoma UNIVERSITY OF PENNSYLVANIA:

https://www.pennmedicine.org/news/news-releases/2019/september/university-pennsylvania-announces-exclusive-alliance-novartis-development-new-focused-relationship UNIVERSITY OF WASHINGTON,

CHILDREN’S HOSPITAL BOSTON MASS GENERAL HOSPITAL FOR CHILDREN:

https://iscrm.uw.edu/fate-therapeutics-a-company-started-in-seattle-with-intellectual-property-from-iscrm-has-announced-its-first-clinical-trials/ UNIVERSITY OF WISCONSIN:

https://pathology.wisc.edu/2021/06/21/stratagraft-approved-by-fda/ UNIVERSITY OF WISCONSIN — MADISON: https://news.wisc.edu/gov-evers-state-leaders-tour-uw-biotech-spinoff-stratatech/

UPDATED THE US POLICY GUIDANCE: https://www.whitehouse.gov/ostp/news-updates/2022/08/25/ostp-issues-guidance-to-make-federally-funded-research-freely-available-without-delay/ US$10 MILLION

IN SERIES C: https://www.wsj.com/articles/DJFVW00120150109eb19ozrd9 US$13.1 MILLION: https://www.pehub.com/histogenics-adds-131-million/ US$34 MILLION:

https://www.massdevice.com/histogenics-raises-34-million-after-prochon-biotech-merger/ US$49 MILLION:

https://www.fiercebiotech.com/biotech/histogenics-closes-49-million-series-a-fundraising-to-support-commercial-development-of-0 US$5.35 MILLION:

https://www.prweb.com/releases/2008/05/prweb908944.htm/ US$65 MILLION IN SERIES D: https://www.fiercebiotech.com/biotech/avexis-secures-65-million-financing US$65 MILLION THROUGH AN INITIAL

PUBLIC OFFERING: https://www.bizjournals.com/boston/blog/bioflash/2014/12/with-65m-offering-histogenics-becomes-states-17th.html US$9.0 MILLION:

https://www.fiercebiotech.com/biotech/histogenics-secures-9-million-second-round-of-financing VYJUVEK (2023): https://www.fda.gov/vaccines-blood-biologics/vyjuvek WEIZMANN INSTITUTE:

https://www.haaretz.com/israel-news/2017-08-31/ty-article/kite-pharmas-success-is-nothing-compared-to-tevas-failure/0000017f-dbae-df9c-a17f-ffbef7f70000 WEILL CORNELL MEDICINE:

https://angiocrinebioscience.com/about-us/our-story/ WESTERN NEW YORK INCUBATOR NETWORK SBIR/STTR ASSISTANCE PROGRAMME: https://www.wnyincubators.com/sbir-sttr-assistance Y COMBINATOR

MANAGEMENT: https://www.ycombinator.com/ YESCARTA (2017):

https://www.gilead.com/news-and-press/press-room/press-releases/2020/7/us-fda-approves-kites-tecartus-the-first-and-only-car-t-treatment-for-relapsed-or-refractory-mantle-cell-lymphoma

ZOLGENSMA (2019): https://www.novartis.com/news/media-releases/avexis-receives-fda-approval-zolgensma-first-and-only-gene-therapy-pediatric-patients-spinal-muscular-atrophy-sma ZYNTEGLO

(2022): https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-fda-approval-zynteglor-first-gene-therapy RIGHTS AND PERMISSIONS Springer Nature or its

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Takahashi, T., Donahue, R.P., Nordberg, R.C. _et al._ Commercialization of regenerative-medicine therapies. _Nat Rev Bioeng_ 1, 906–929 (2023). https://doi.org/10.1038/s44222-023-00095-9

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