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{{short description|American pharmaceutical company}}
{{short description|American pharmaceutical company}}
{{Infobox company
{{Infobox company
| name = Spark Therapeutics
| name = Spark Therapeutics, Inc.
| logo = Spark Therapeutics Label.png
| logo = Spark Therapeutics Label.png
| type = Subsidiary
| type = [[Subsidiary]] of [[Hoffmann-La Roche]]
| industry = [[Biotechnology]], [[Pharmaceutical]]
| industry = [[Biotechnology]]<BR>[[Pharmaceutical]]
| founded = {{start date and age|2013}}
| fate =
| founders = [[Katherine A. High]]<BR>Jeffrey Marrazzo<BR>Jean Bennett<BR>J Fraser Wright<BR>Beverly Davidson<BR>Jennifer Wellman
| predecessor = <!-- or: | predecessors = -->
| location = [[Philadelphia]], [[Pennsylvania]]
| successor = <!-- or: | successors = -->
| revenue = {{increase}} $64 million (2018)
| founded = {{start date and age|2013}} in [[Philadelphia]], [[Pennsylvania]], [[United States of America]]
| net_income = {{increase}} -$78 million (2018)
| founders = Jeffrey Marrazzo, Katherine High
| assets = {{increase}} $814 million (2018)
| defunct = <!-- {{End date|YYYY|MM|DD}} -->
| equity = {{decrease}} $496 million (2018)
| hq_location_city = [[Philadelphia]]
| num_employees = 368 (2019)
| hq_location_country = [[United States of America]]
| parent = [[Hoffmann-La Roche]]
| area_served = <!-- or: | areas_served = -->
| website = {{URL|https://sparktx.com/}}
| key_people =
| footnotes = <ref name=10K>{{cite web | url=https://www.sec.gov/ix?doc=/Archives/edgar/data/1609351/000160935119000042/spark-201810xk.htm | title=Spark Therapeutics, Inc. 2018 Form 10-K Annual Report | publisher=[[U.S. Securities and Exchange Commission]]}}</ref>
| products =
| owner = <!-- or: | owners = -->
| num_employees =
| num_employees_year = <!-- Year of num_employees data (if known) -->
| parent = [[Hoffmann-La Roche]]
| website = {{URL|sparktx.com}}
}}
}}


'''Spark Therapeutics, Inc.''' is a developer of [[gene therapy]] treatments, which treat debilitating genetic diseases.<ref name=10K/> It was founded in 2013 and has been a subsidiary of [[Hoffmann-La Roche]] since 2020.
'''Spark Therapeutics''' is a startup pharmaceutical company that develops [[Gene therapy|gene therapies]].<ref>{{cite news |date=7 November 2017|title=Spark Therapeutics reports 3Q loss|url=https://uk.news.yahoo.com/spark-therapeutics-reports-3q-loss-124330729.html|work=[[Associated Press]]|access-date=16 November 2017}}</ref><ref name="PhilBiz1">{{cite news |first=John|last=George|date=7 November 2017|title=Spark, Pfizer amend agreement for experimental hemophilia gene therapy|url=https://www.bizjournals.com/philadelphia/news/2017/11/07/spark-pfizer-hemophilia-gene-therapy.html|work=[[Philadelphia Business Journal]]|access-date=16 November 2017}}</ref><ref>{{cite news |first=Marilynn|last=Marchione|date=10 October 2017|title=A gene therapy that could cure blindness is on the brink of getting approved|url=http://www.businessinsider.com/ap-seeing-hope-fda-panel-considers-gene-therapy-for-blindness-2017-10|work=[[Business Insider]]|access-date=16 November 2017}}</ref> It was founded in 2013 by [[Katherine A. High|Katherine High]] and Jeffrey Marrazzo in an effort to commercially develop treatments against haemophilia that High was working on at [[Children's Hospital of Philadelphia]].<ref>{{cite news |first=David|last=Crow|date=19 October 2017|title=Gene therapy helped these children see. Can it transform medicine?|url=https://www.ft.com/content/9c4fab50-b38f-11e7-a398-73d59db9e399|work=[[Financial Times]]|access-date=16 November 2017}}</ref>


==History==
In February 2019, [[Roche Holding|Roche]] announced it would acquire the company for $4.3 billion ($114.50 per share).<ref>{{cite web|url=https://uk.reuters.com/article/us-spark-m-a-roche-hldg-idUKKCN1QE0L6|title=Roche 'steps up' for gene therapy with $4.3 billion Spark bet|date=25 February 2019|publisher=|via=uk.reuters.com}}</ref><ref>{{cite web |url=https://endpts.com/ftc-staff-sign-off-on-roche-4-3b-deal-for-spark-report/ |title=UPDATED: FTC staff signs off on Roche $4.3B deal for Spark — report |first=Natalie |last=Grover |date=25 October 2019 |website=EndpointNews }}</ref> In December 2019, the transaction was completed.<ref>{{cite web|url=https://www.bizjournals.com/philadelphia/news/2019/12/17/roche-completes-4-3b-purchase-of-philadelphia.html|title=Roche completes $4.3B purchase of Philadelphia gene therapy pioneer Spark Therapeutics|date=17 December 2019}}</ref>
The company was founded in 2013 by [[Katherine A. High]], Jeffrey Marrazzo, and Steven Altschuler<ref>Higgins, Robert F., and Tina Liu. "[https://store.hbr.org/product/spark-therapeutics-pioneering-gene-therapy/818059?sku=818059-PDF-ENG Spark Therapeutics: Pioneering Gene Therapy.]" Harvard Business School Case 818-059, January 2018.</ref> in an effort to commercially develop treatments against haemophilia that High was working on at [[Children's Hospital of Philadelphia]].<ref>{{cite news |first=David | last=Crow | title=Gene therapy helped these children see. Can it transform medicine? | url=https://www.ft.com/content/9c4fab50-b38f-11e7-a398-73d59db9e399 | work=[[Financial Times]] | date=19 October 2017 | url-access=subscription}}</ref>

In January 2015, the company became a [[public company]], trading under the ticker $ONCE via a $161 million [[initial public offering]]<ref>{{cite web | url=https://www.fiercebiotech.com/r-d/spark-nails-a-161m-ipo-to-fund-its-breakthrough-gene-therapy | title=Spark nails a $161M IPO to fund its 'breakthrough' gene therapy | date=30 January 2015 }}</ref> led by Chief Legal Officer Joseph La Barge.<ref>{{cite news | url=https://www.bizjournals.com/bizjournals/news/2015/01/30/spark-therapeutics-nasdaq-once-ipo.html | title=Shake Shack wasn't the day's only gonzo IPO. And this one, for Spark Therapeutics, raised more cash. | first=John | last=George | work=[[American City Business Journals]] | date=January 30, 2015}}</ref>

In December 2017, the [[U.S. Food and Drug Administration]] approved Luxturna ([[voretigene neparvovec]]-rzyl) for the treatment of patients with viable retinal cells and confirmed biallelic RPE65 mutation-associated retinal dystrophy, a genetic blinding condition caused by mutations in the RPE65 gene.<ref name=10K/> The company is currently developing several gene therapies to target a suite of diseases, including [[Haemophilia]] A and B, and several [[central nervous system]] diseases.

In December 2019, the company was acquired by [[Hoffmann-La Roche]] for $4.3 billion.<ref>{{cite news | url=https://www.bizjournals.com/philadelphia/news/2019/12/17/roche-completes-4-3b-purchase-of-philadelphia.html | title= Roche completes $4.3B purchase of Philadelphia gene therapy pioneer Spark Therapeutics | work=[[American City Business Journals]] | date=17 December 2019}}</ref><ref>{{cite press release | url=https://www.roche.com/media/releases/med-cor-2019-12-17b.htm | title=Roche concludes acquisition of Spark Therapeutics, Inc. to strengthen presence in gene therapy | publisher=[[Hoffmann-La Roche]] | date=17 December 2019}}</ref> It now continues to operate as an independent subsidiary.
<ref>{{Cite web|url=https://www.roche.com/media/releases/med-cor-2019-12-17b.htm|title=Roche concludes acquisition of Spark Therapeutics, Inc. To strengthen presence in gene therapy}}</ref> Since the acquisition by Swiss pharma [[Roche]], several key founding executives have departed, including scientist and co-founder Katherine High in February 2020,<ref>{{Cite web|url=https://www.biospace.com/article/spark-therapeutics-co-founder-katherine-high-departs-company-ahead-of-roche-merger/|title=Spark Co-Founder Katherine High Departs Company Ahead of Merger with Roche|website=BioSpace|date=21 February 2020 |language=en-US|access-date=2020-03-03}}</ref> Chief Business/Legal Officer Joseph La Barge in December 2021, and co-founder and Chief Executive Officer Jeffrey Marrazzo in April 2022 <ref name="sparktx.com">{{cite web | url=https://sparktx.com/press_releases/spark-therapeutics-announces-departure-of-ceo-and-founder-jeff-marrazzo-coo-ron-philip-named-as-successor/ | title=Spark Therapeutics Announces Departure of CEO and Founder Jeff Marrazzo; COO Ron Philip Named as Successor – Spark Therapeutics }}</ref>

On February 23, 2022, Marrazzo named big-Pharma veteran Ron Philip as his successor. Mr. Philip currently leads the organization.<ref name="sparktx.com"/>


==Products and pipeline==
==Products and pipeline==
===Voretigene neparvovec===
===Voretigene neparvovec===
[[Voretigene neparvovec]], marketed under the tradename Luxturna, is a gene therapy approved by the [[Food and Drug Administration]] for treatment of [[Leber's congenital amaurosis]], a rare genetic eye disease.<ref>{{cite press release | url=https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589467.htm | title=FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss | publisher=[[Food and Drug Administration]] | date=19 December 2017}}</ref>
{{Main article|Voretigene neparvovec}}
Voretigene neparvovec, marketed under the tradename Luxturna, is a gene therapy approved by the [[Food and Drug Administration]] for treatment of [[Leber's congenital amaurosis]], a rare genetic eye disease.<ref>{{cite news|url=https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589467.htm|title=FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss|date=19 Dec 2017|work=[[FDA]]|access-date=20 Dec 2017}}</ref>


===Fidanacogene elaparvovec===
===Fidanacogene elaparvovec===
Fidanacogene elaparvovec, previously known by its study ID number SPK-9001,<ref>{{cite press release |last= |first= |date=16 July 2018|title=Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy|url=https://www.pfizer.com/news/press-release/press-release-detail/pfizer_initiates_pivotal_phase_3_program_for_investigational_hemophilia_b_gene_therapy|location=|publisher=[[Pfizer]]|agency= |access-date=2018-07-18}}</ref> is an [[experimental drug]] under investigation for treatment of [[hemophilia B]]. Spark partnered with pharmaceutical giant [[Pfizer]] to develop fidanacogene elaparvovec.<ref name="PhilBiz1"/> Fidanacogene elaparvovec is an [[Adeno-associated virus|adeno-associated viral vector]] which is designed to transfer a working copy of the [[Factor IX]] gene into the livers of patients who carry non-functioning copies.<ref>{{cite journal |last1=Lindsey|first1=George|date=1 December 2016|title=Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression|url=http://www.bloodjournal.org/content/128/22/3?sso-checked=true|journal=[[Blood (journal)|Blood]] |volume=128|issue=22|pages=3|access-date=16 November 2017|doi=10.1182/blood.V128.22.3.3}}</ref> In July 2018, Pfizer said fidanacogene elaparvovec was entering late stage clinical trials.<ref>{{cite news|last=George|first=John|date=16 July 2018|title=Pfizer begins late-stage testing of Spark's hemophilia B gene therapy|url=https://www.bizjournals.com/philadelphia/news/2018/07/16/pfizer-begins-late-stage-testing-of.html|work=[[Philadelphia Business Journal]]|location= |access-date=18 July 2018}}</ref>
[[Fidanacogene elaparvovec]], previously known by its study ID number SPK-9001,<ref>{{cite press release | title=Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy | url=https://www.pfizer.com/news/press-release/press-release-detail/pfizer_initiates_pivotal_phase_3_program_for_investigational_hemophilia_b_gene_therapy | publisher=[[Pfizer]] | date=16 July 2018}}</ref> is a gene therapy for the treatment of [[hemophilia B]]. It was developed by Spark in partnership with [[Pfizer]]. Fidanacogene elaparvovec is an [[Adeno-associated virus|adeno-associated viral vector]] which is designed to transfer a working copy of the [[Factor IX]] gene into the livers of patients who carry non-functioning copies.<ref>{{cite journal | last1=Lindsey | first1=George | date=1 December 2016 | title=Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression | url=http://www.bloodjournal.org/content/128/22/3?sso-checked=true | journal=[[Blood (journal)|Blood]] | volume=128| issue=22 | pages=3 | doi=10.1182/blood.V128.22.3.3| doi-access= | url-access=subscription }}</ref> It received FDA approval in 2024.<ref>{{cite press release | title=U.S. FDA Approves Pfizer's Beqvez (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B | publisher=Pfizer | via=Business Wire | date=26 April 2024 | url=https://www.businesswire.com/news/home/20240425269649/en/U.S.-FDA-Approves-Pfizer%E2%80%99s-BEQVEZ%E2%84%A2-fidanacogene-elaparvovec-dzkt-a-One-Time-Gene-Therapy-for-Adults-with-Hemophilia-B | access-date=29 April 2024 | archive-date=29 April 2024 | archive-url=https://web.archive.org/web/20240429055143/https://www.businesswire.com/news/home/20240425269649/en/U.S.-FDA-Approves-Pfizer%E2%80%99s-BEQVEZ%E2%84%A2-fidanacogene-elaparvovec-dzkt-a-One-Time-Gene-Therapy-for-Adults-with-Hemophilia-B | url-status=live }}</ref>


===SPK-8011===
===SPK-8011===
SPK-8011 is an experimental drug under investigation for treatment of [[Haemophilia A|hemophilia A]]. It is entering [[Phases_of_clinical_research#Phase_III|phase III]] clinical trials in the United States. The therapy transfers a working copy of the [[Factor VIII]] gene into patients who lack one. In Phase II clinical trials, 2 of 7 patients receiving the highest dose of the drug suffered immune responses. One patient had to be hospitalized. The reactions against the treatment were seen as a set back, though Spark suggested that the responses could be controlled with steroids, and promised to move forward with Phase III testing.<ref>{{cite news |last=Garde|first=Damian|date=7 August 2018|title=Spark's gene therapy data answer some burning questions — and raise a few more|url=https://www.statnews.com/2018/08/07/sparks-latest-gene-therapy-data-answer-some-burning-questions-and-raise-a-few-more/|work=[[Stat (website)|Stat]]|location= |access-date=7 August 2018}}</ref><ref>{{cite news |last=Tirrell|first=Meg|date=7 August 2018|title=Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors|url=https://www.cnbc.com/2018/08/07/spark-therapeutics-gene-therapy-reduces-bleeding-in-hemophilia-a-trial.html|work=[[CNBC]]|location= |access-date=7 August 2018}}</ref>
SPK-8011 ([[Dirloctogene samoparvovec]]) is an experimental drug under investigation for treatment of [[Haemophilia A]]. It is entering [[Phases of clinical research#Phase III|phase III]] clinical trials in the United States. The therapy transfers a working copy of the [[Factor VIII]] gene into patients who lack one. In Phase II clinical trials, 2 of 7 patients receiving the highest dose of the drug suffered immune responses. One patient had to be hospitalized. The reactions against the treatment were seen as a set-back, though Spark suggested that the responses could be controlled with steroids, and promised to move forward with Phase III testing.<ref>{{cite news | last=Garde | first=Damian | title=Spark's gene therapy data answer some burning questions — and raise a few more|url=https://www.statnews.com/2018/08/07/sparks-latest-gene-therapy-data-answer-some-burning-questions-and-raise-a-few-more/ | work=[[Stat (website)|Stat]] | date=7 August 2018}}</ref><ref>{{cite news | last=Tirrell | first=Meg | title=Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors | url=https://www.cnbc.com/2018/08/07/spark-therapeutics-gene-therapy-reduces-bleeding-in-hemophilia-a-trial.html | work=[[CNBC]] | date=7 August 2018}}</ref>


===SPK-7001===
===SPK-7001===
SPK-7001 is an experimental drug under investigation for treatment of [[choroideremia]], a genetic disorder that causes blindness.<ref name="NBT1">{{cite news |last=Morrison | first=Chris | title=Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal | url=https://www.nature.com/articles/d41587-019-00007-6 | work=[[Nature Biotechnology]] | date=5 March 2019}}</ref>

SPK-7001 is an experimental drug under investigation for treatment of [[choroideremia]], a genetic disorder that causes blindness.<ref name="NBT1">{{cite news |last=Morrison|first=Chris|date=5 March 2019|title=Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal|url=https://www.nature.com/articles/d41587-019-00007-6|work=[[Nature Biotechnology]]|location= |access-date=8 February 2019}}</ref>


===SPK-3006===
===SPK-3006===
[[SPK-3006]] is an experimental drug under investigation for treatment of [[Pompe disease]], a genetic disorder that leads to failure to correctly metabolize [[glycogen]].<ref name="NBT1"/>

SPK-3006 is an experimental drug under investigation for treatment of [[Pompe disease]], a genetic disorder that leads to failure to correctly metabolize [[glycogen]].<ref name="NBT1"/>


===SPK-1001===
===SPK-1001===

SPK-1001 is an experimental drug under investigation for treatment of [[Batten disease]], a fatal genetic, nervous system disorder.<ref name="NBT1"/>
SPK-1001 is an experimental drug under investigation for treatment of [[Batten disease]], a fatal genetic, nervous system disorder.<ref name="NBT1"/>


==References==
==References==
{{reflist}}
{{Reflist|}}


==External links==
==External links==
*[http://sparktx.com/ Official Website]
* {{Official website|https://sparktx.com/}}


[[Category:Biotechnology companies of the United States]]
[[Category:Biotechnology companies of the United States]]
[[Category:Pharmaceutical companies of the United States]]
[[Category:Biopharmaceutical companies]]
[[Category:Haemophilia]]
[[Category:Haemophilia]]
[[Category:Pharmaceutical companies established in 2013]]
[[Category:Pharmaceutical companies established in 2013]]
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[[Category:Health care companies based in Pennsylvania]]
[[Category:Health care companies based in Pennsylvania]]
[[Category:Virotherapy]]
[[Category:Virotherapy]]
[[Category:Companies listed on NASDAQ]]
[[Category:Companies formerly listed on the Nasdaq]]
[[Category:2015 initial public offerings]]
[[Category:2015 initial public offerings]]
[[Category:Announced mergers and acquisitions]]
[[Category:2019 mergers and acquisitions]]
[[Category:Roche]]

[[Category:American subsidiaries of foreign companies]]

{{US-company-stub}}
{{med-company-stub}}

Latest revision as of 06:48, 4 December 2024

Spark Therapeutics, Inc.
Company typeSubsidiary of Hoffmann-La Roche
IndustryBiotechnology
Pharmaceutical
Founded2013; 12 years ago (2013)
FoundersKatherine A. High
Jeffrey Marrazzo
Jean Bennett
J Fraser Wright
Beverly Davidson
Jennifer Wellman
HeadquartersPhiladelphia, Pennsylvania
RevenueIncrease $64 million (2018)
Increase -$78 million (2018)
Total assetsIncrease $814 million (2018)
Total equityDecrease $496 million (2018)
Number of employees
368 (2019)
ParentHoffmann-La Roche
Websitesparktx.com
Footnotes / references
[1]

Spark Therapeutics, Inc. is a developer of gene therapy treatments, which treat debilitating genetic diseases.[1] It was founded in 2013 and has been a subsidiary of Hoffmann-La Roche since 2020.

History

[edit]

The company was founded in 2013 by Katherine A. High, Jeffrey Marrazzo, and Steven Altschuler[2] in an effort to commercially develop treatments against haemophilia that High was working on at Children's Hospital of Philadelphia.[3]

In January 2015, the company became a public company, trading under the ticker $ONCE via a $161 million initial public offering[4] led by Chief Legal Officer Joseph La Barge.[5]

In December 2017, the U.S. Food and Drug Administration approved Luxturna (voretigene neparvovec-rzyl) for the treatment of patients with viable retinal cells and confirmed biallelic RPE65 mutation-associated retinal dystrophy, a genetic blinding condition caused by mutations in the RPE65 gene.[1] The company is currently developing several gene therapies to target a suite of diseases, including Haemophilia A and B, and several central nervous system diseases.

In December 2019, the company was acquired by Hoffmann-La Roche for $4.3 billion.[6][7] It now continues to operate as an independent subsidiary. [8] Since the acquisition by Swiss pharma Roche, several key founding executives have departed, including scientist and co-founder Katherine High in February 2020,[9] Chief Business/Legal Officer Joseph La Barge in December 2021, and co-founder and Chief Executive Officer Jeffrey Marrazzo in April 2022 [10]

On February 23, 2022, Marrazzo named big-Pharma veteran Ron Philip as his successor. Mr. Philip currently leads the organization.[10]

Products and pipeline

[edit]

Voretigene neparvovec

[edit]

Voretigene neparvovec, marketed under the tradename Luxturna, is a gene therapy approved by the Food and Drug Administration for treatment of Leber's congenital amaurosis, a rare genetic eye disease.[11]

Fidanacogene elaparvovec

[edit]

Fidanacogene elaparvovec, previously known by its study ID number SPK-9001,[12] is a gene therapy for the treatment of hemophilia B. It was developed by Spark in partnership with Pfizer. Fidanacogene elaparvovec is an adeno-associated viral vector which is designed to transfer a working copy of the Factor IX gene into the livers of patients who carry non-functioning copies.[13] It received FDA approval in 2024.[14]

SPK-8011

[edit]

SPK-8011 (Dirloctogene samoparvovec) is an experimental drug under investigation for treatment of Haemophilia A. It is entering phase III clinical trials in the United States. The therapy transfers a working copy of the Factor VIII gene into patients who lack one. In Phase II clinical trials, 2 of 7 patients receiving the highest dose of the drug suffered immune responses. One patient had to be hospitalized. The reactions against the treatment were seen as a set-back, though Spark suggested that the responses could be controlled with steroids, and promised to move forward with Phase III testing.[15][16]

SPK-7001

[edit]

SPK-7001 is an experimental drug under investigation for treatment of choroideremia, a genetic disorder that causes blindness.[17]

SPK-3006

[edit]

SPK-3006 is an experimental drug under investigation for treatment of Pompe disease, a genetic disorder that leads to failure to correctly metabolize glycogen.[17]

SPK-1001

[edit]

SPK-1001 is an experimental drug under investigation for treatment of Batten disease, a fatal genetic, nervous system disorder.[17]

References

[edit]
  1. ^ a b c "Spark Therapeutics, Inc. 2018 Form 10-K Annual Report". U.S. Securities and Exchange Commission.
  2. ^ Higgins, Robert F., and Tina Liu. "Spark Therapeutics: Pioneering Gene Therapy." Harvard Business School Case 818-059, January 2018.
  3. ^ Crow, David (19 October 2017). "Gene therapy helped these children see. Can it transform medicine?". Financial Times.
  4. ^ "Spark nails a $161M IPO to fund its 'breakthrough' gene therapy". 30 January 2015.
  5. ^ George, John (January 30, 2015). "Shake Shack wasn't the day's only gonzo IPO. And this one, for Spark Therapeutics, raised more cash". American City Business Journals.
  6. ^ "Roche completes $4.3B purchase of Philadelphia gene therapy pioneer Spark Therapeutics". American City Business Journals. 17 December 2019.
  7. ^ "Roche concludes acquisition of Spark Therapeutics, Inc. to strengthen presence in gene therapy" (Press release). Hoffmann-La Roche. 17 December 2019.
  8. ^ "Roche concludes acquisition of Spark Therapeutics, Inc. To strengthen presence in gene therapy".
  9. ^ "Spark Co-Founder Katherine High Departs Company Ahead of Merger with Roche". BioSpace. 21 February 2020. Retrieved 2020-03-03.
  10. ^ a b "Spark Therapeutics Announces Departure of CEO and Founder Jeff Marrazzo; COO Ron Philip Named as Successor – Spark Therapeutics".
  11. ^ "FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss" (Press release). Food and Drug Administration. 19 December 2017.
  12. ^ "Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy" (Press release). Pfizer. 16 July 2018.
  13. ^ Lindsey, George (1 December 2016). "Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression". Blood. 128 (22): 3. doi:10.1182/blood.V128.22.3.3.
  14. ^ "U.S. FDA Approves Pfizer's Beqvez (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B" (Press release). Pfizer. 26 April 2024. Archived from the original on 29 April 2024. Retrieved 29 April 2024 – via Business Wire.
  15. ^ Garde, Damian (7 August 2018). "Spark's gene therapy data answer some burning questions — and raise a few more". Stat.
  16. ^ Tirrell, Meg (7 August 2018). "Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors". CNBC.
  17. ^ a b c Morrison, Chris (5 March 2019). "Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal". Nature Biotechnology.
[edit]