Spark Therapeutics

{{Infobox company

| name = Spark Therapeutics, Inc.

| logo = Spark Therapeutics Label.png

| type = Subsidiary of Hoffmann-La Roche

| industry = Biotechnology
Pharmaceutical

| founded = {{start date and age|2013}}

| founders = Katherine A. High
Jeffrey Marrazzo
Jean Bennett
J Fraser Wright
Beverly Davidson
Jennifer Wellman

| location = Philadelphia, Pennsylvania

| revenue = {{increase}} $64 million (2018)

| net_income = {{increase}} -$78 million (2018)

| assets = {{increase}} $814 million (2018)

| equity = {{decrease}} $496 million (2018)

| num_employees = 368 (2019)

| parent = Hoffmann-La Roche

| website = {{URL|https://sparktx.com/}}

| footnotes = {{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}}

}}

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

History

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

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

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. 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.{{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}}{{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}} It now continues to operate as an independent subsidiary.

{{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}} Since the acquisition by Swiss pharma Roche, several key founding executives have departed, including scientist and co-founder Katherine High in February 2020,{{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}} Chief Business/Legal Officer Joseph La Barge in December 2021, and co-founder and Chief Executive Officer Jeffrey Marrazzo in April 2022 {{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 }}

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

Products and pipeline

=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.{{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}}

=Fidanacogene elaparvovec=

Fidanacogene elaparvovec, previously known by its study ID number SPK-9001,{{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}} 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.{{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 | volume=128| issue=22 | pages=3 | doi=10.1182/blood.V128.22.3.3| doi-access= | url-access=subscription }} It received FDA approval in 2024.{{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 }}

=SPK-8011=

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.{{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 | date=7 August 2018}}{{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}}

=SPK-7001=

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

=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.