science

Spark Therapeutics

Spark Therapeutics is a science topic covered in the lgStudy science library. This page brings together a partial reference excerpt, illustrations, worked examples, real-world applications and a short study plan, so you can understand Spark Therapeutics rather than just read about it. In short: Spark Therapeutics, Inc. was 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.

Key takeaways

Spark Therapeutics belongs to science; place it in that map before memorising details.
Learn the definition first, then one example that makes the definition concrete.
Connect Spark Therapeutics to a quantity you can measure, compute or draw — that is where exam questions come from.
Reproduce the core statement of Spark Therapeutics from memory before moving on to harder problems.

Reference excerpt

Spark Therapeutics, Inc. was 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 Altschuler in an effort to commercially develop treatments against haemophilia that High was working on at Children's Hospital of Philadelphia. In January 2015, the company became a public company, trading under the ticker $ONCE via a $161 million initial public offering led by Chief Legal Officer Joseph La Barge. 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. It now continues to operate as an independent subsidiary. Since the acquisition by Swiss pharma Roche, several key founding executives have departed, including scientist and co-founder Katherine High in February 2020, Chief Business/Legal Officer Joseph La Barge in December 2021, and co-founder and Chief Executive Officer Jeffrey Marrazzo in April 2022 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.

Fidanacogene elaparvovec Fidanacogene elaparvovec, previously known by its study ID number SPK-9001, 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. It received FDA approval in 2024.

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.

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

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.

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

References

External links Official website

Worked examples

Example 1 — a first encounter with Spark Therapeutics

Start with the simplest possible case. Write down what Spark Therapeutics claims or describes in one sentence, then invent the smallest concrete situation in which that sentence is true. In science, the smallest case is usually a single object, a single equation or a single measurement. Check that every symbol or term in your sentence has a meaning in that case.

Example 2 — changing one variable

Take the situation from Example 1 and change exactly one quantity: double it, halve it, or set it to zero. Predict what should happen to Spark Therapeutics before you calculate. Comparing your prediction with the result is the fastest way to find out whether you understand the idea or only the words.

Example 3 — an exam-style question

Typical questions about Spark Therapeutics ask you to (a) state it precisely, (b) apply it to given data, and (c) explain a limitation. Practise writing all three answers in under five minutes; the third part is what separates a full-mark answer from an average one.

Applications of Spark Therapeutics

In research: Spark Therapeutics appears in science research whenever the underlying quantities have to be modelled precisely. Papers usually cite it as a starting assumption and then explore where it breaks down.
In technology and industry: Engineering practice reuses Spark Therapeutics in design rules, simulations and safety margins. Knowing the idea lets you read a specification sheet and understand why the numbers look the way they do.
In the classroom: Spark Therapeutics is common in secondary-school and first-year university syllabi. It links to neighbouring topics 2013 establishments in Pennsylvania, 2015 initial public offerings, 2019 mergers and acquisitions, so understanding it makes those chapters shorter.
In everyday life: Look for Spark Therapeutics outside the textbook — in sport, cooking, traffic, electronics or the sky above you. An example you found yourself is remembered far longer than one you were given.

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How to study Spark Therapeutics in 20 minutes

Read the reference excerpt below once, without taking notes.
Close the page and write down what Spark Therapeutics means in your own words.
Compare your version with the excerpt and mark what you missed.
Work through the three examples above with pen and paper.
Explain Spark Therapeutics out loud to somebody else — or to Teacher Smith in the lgStudy chat.

Frequently asked questions

What is Spark Therapeutics in simple terms?

Spark Therapeutics, Inc. was 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.

Why does Spark Therapeutics matter?

Because it connects several science ideas at once: it gives you a definition you can apply, a quantity you can calculate, and a way to check whether a result is plausible.

How should I study Spark Therapeutics?

Read the excerpt, restate it from memory, then work through the examples and applications listed on this page. The five-step study plan above takes about twenty minutes.

What does this page cover?

It gives you a compact reference excerpt plus original lgStudy explanations, examples, applications and study material on Spark Therapeutics.