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'''Spark Therapeutics, Inc.''' is a developer of [[gene therapy]] treatments, which treat debilitating genetic diseases.<ref name=10K/> It is a subsidiary of [[Hoffmann-La Roche]].
'''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.


==History==
==History==
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>
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>https://www.fiercebiotech.com/r-d/spark-nails-a-161m-ipo-to-fund-its-breakthrough-gene-therapy</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 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 LUXTURNATM ([[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 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.
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|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">https://sparktx.com/press_releases/spark-therapeutics-announces-departure-of-ceo-and-founder-jeff-marrazzo-coo-ron-philip-named-as-successor/</ref>
<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"/>
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==
The company has 3 gene therapy product candidates in clinical development: (i) SPK-8011, a candidate in the SPK-FVIII program for hemophilia A; (ii) SPK-8016, a product candidate for the hemophilia A inhibitor market; and (iii) SPK-7001, targeting choroideremia, or CHM. SPK-9001, a lead product candidate in the SPK-FIX program for hemophilia B, is being developed in partnership with [[Pfizer]].<ref name=10K/>

===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>
[[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>


===Fidanacogene elaparvovec===
===Fidanacogene elaparvovec===
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 an [[experimental drug]] under investigation for treatment of [[hemophilia B]] 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=free }}</ref> In July 2018, fidanacogene elaparvovec entered late stage clinical trials.<ref>{{cite news | last=George | first=John | 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=[[American City Business Journals]] | date=16 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]]. 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-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===
Line 48: Line 46:


===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===

Latest revision as of 06:48, 4 December 2024

Spark Therapeutics, Inc.
Company typeSubsidiary of Hoffmann-La Roche
IndustryBiotechnology
Pharmaceutical
Founded2013; 11 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]
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