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===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 |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>


===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 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 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-7001===
===SPK-7001===

Revision as of 15:14, 17 December 2019

Spark Therapeutics
Company typePublic
Nasdaq: ONCE
Russell 2000 Component
IndustryBiotechnology, Pharmaceutical
Founded2013; 11 years ago (2013) in Philadelphia, Pennsylvania, United States of America
FoundersJeffrey Marrazzo, Katherine High
Headquarters
Philadelphia
,
United States of America
Websitesparktx.com

Spark Therapeutics is a startup pharmaceutical company that develops gene therapies.[1][2][3] It was founded in 2013 by 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.[4]

In February 2019, Roche announced it would acquire the company for $4.3 billion ($114.50 per share).[5][6]

Products and pipeline

Voretigene neparvovec

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.[7]

Fidanacogene elaparvovec

Fidanacogene elaparvovec, previously known by its study ID number SPK-9001,[8] is an experimental drug under investigation for treatment of hemophilia B. Spark partnered with pharmaceutical giant Pfizer to develop fidanacogene elaparvovec.[2] 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.[9] In July 2018, Pfizer said fidanacogene elaparvovec was entering late stage clinical trials.[10]

SPK-8011

SPK-8011 is an experimental drug under investigation for treatment of hemophilia 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.[11][12]

SPK-7001

SPK-7001 is an experimental drug under investigation for treatment of choroideremia, a genetic disorder that causes blindness.[13]

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.[13]

SPK-1001

SPK-1001 is an experimental drug under investigation for treatment of Batten disease, a fatal genetic, nervous system disorder.[13]

References

  1. ^ "Spark Therapeutics reports 3Q loss". Associated Press. 7 November 2017. Retrieved 16 November 2017.
  2. ^ a b George, John (7 November 2017). "Spark, Pfizer amend agreement for experimental hemophilia gene therapy". Philadelphia Business Journal. Retrieved 16 November 2017.
  3. ^ Marchione, Marilynn (10 October 2017). "A gene therapy that could cure blindness is on the brink of getting approved". Business Insider. Retrieved 16 November 2017.
  4. ^ Crow, David (19 October 2017). "Gene therapy helped these children see. Can it transform medicine?". Financial Times. Retrieved 16 November 2017.
  5. ^ "Roche 'steps up' for gene therapy with $4.3 billion Spark bet". 25 February 2019 – via uk.reuters.com.
  6. ^ Grover, Natalie (25 October 2019). "UPDATED: FTC staff signs off on Roche $4.3B deal for Spark — report". EndpointNews. {{cite web}}: no-break space character in |title= at position 61 (help)
  7. ^ "FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss". FDA. 19 Dec 2017. Retrieved 20 Dec 2017.
  8. ^ "Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy" (Press release). Pfizer. 16 July 2018. Retrieved 2018-07-18.
  9. ^ 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. Retrieved 16 November 2017.
  10. ^ George, John (16 July 2018). "Pfizer begins late-stage testing of Spark's hemophilia B gene therapy". Philadelphia Business Journal. Retrieved 18 July 2018.
  11. ^ Garde, Damian (7 August 2018). "Spark's gene therapy data answer some burning questions — and raise a few more". Stat. Retrieved 7 August 2018.
  12. ^ Tirrell, Meg (7 August 2018). "Spark Therapeutics shares lose a third of their value as hemophilia gene therapy trial disappoints investors". CNBC. Retrieved 7 August 2018.
  13. ^ a b c Morrison, Chris (5 March 2019). "Spark's meteoric rise from hospital-funded spinout to $4.8 billion deal". Nature Biotechnology. Retrieved 8 February 2019.
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