Spark Therapeutics

What Spark Therapeutics does

Spark Therapeutics is a clinical-stage and now commercial gene therapy company founded in 2013 as a spinout from Children's Hospital of Philadelphia (CHOP), headquartered in Philadelphia, Pennsylvania. The company develops treatments for inherited genetic diseases using adeno-associated virus (AAV) gene delivery platforms. Its sole commercial product, Luxturna (voretigene neparvovec-rzyl), received FDA approval in 2017 as the first gene therapy for an inherited blindness condition (Leber Congenital Amaurosis caused by RPE65 gene mutations). Luxturna delivers a healthy copy of the RPE65 gene directly into the eye via AAV vector, restoring functional vision in eligible patients, with 72% of clinical trial participants regaining night vision. The product has treated hundreds of patients worldwide and created a regulatory and scientific road map that enabled over 140 subsequent gene therapy clinical trials for retinal conditions.

Spark completed its IPO on NASDAQ (ONCE/ONEM) in 2015 and was acquired by Roche for $4.8 billion in 2019. It now operates as a fully integrated subsidiary of the Roche Group under Genentech, with Luxturna commercialized through the parent's specialty ophthalmology distribution network and targeted at specialized treatment centers and genetic disease specialists. The therapy is priced as a one-time durable treatment, with approximately 99% of surveyed payers providing coverage for one-time high-cost durable gene therapies. Spark previously extended its AAV platform into hemophilia B (Beqvez, licensed to Pfizer) and choroideremia (SPK-7001), but Pfizer terminated the Beqvez license in August 2025 and the Choroideremia Research Foundation acquired SPK-7001 assets in January 2026. Luxturna generated approximately $50 million in 2025 sales, described as commercially underperforming, leaving Spark with a mature single-product profile within the Roche portfolio.

Name

Spark Therapeutics

Legal name

Spark Therapeutics, Inc.

Website

https://sparktx.com

Company type

Private

Founded year

2013

Operating status

Acquired

Headcount range

251–500 employees

Short description

Spark Therapeutics, a fully integrated Roche/Genentech subsidiary, develops AAV-based gene therapies for inherited genetic diseases. Its sole commercial product, Luxturna, is the first FDA-approved gene therapy for an inherited blindness condition (RPE65-mutated Leber Congenital Amaurosis), administered as a one-time treatment at specialized ophthalmology centers.

Ownership category

akta.pro rank

Product category

Gene Therapy Pharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

akta.pro secondary industries

Ophthalmology Gene Therapies (HLAAACAG), AAV Vector Gene Therapy Developers (HLAAACAA), Rare Ophthalmology Disorder Therapies (HLAIAIAK)

Keywords

• Gene therapy
• AAV vector therapy
• Rare disease treatments
• Ophthalmic biopharmaceuticals
• Inherited retinal disease

Headquarters

HQ city

Philadelphia

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Supply Chain, Marketing or Sales, Operations

Revenue model

1. Luxturna Sales: Revenue from commercial sales of Luxturna gene therapy for inherited retinal diseases. The therapy generated approximately $50 million in 2025 sales following Roche's acquisition of Spark Therapeutics in 2019.

Pricing tiers

Go-to-market motion1 record

Distribution channels1 record

Marketing channels3 records

Core offering

Spark Therapeutics develops and commercializes adeno-associated virus (AAV)-based gene therapies for inherited genetic diseases. Its lead commercial product, Luxturna (voretigene neparvovec-rzyl), is the first FDA-approved gene therapy for an inherited blindness condition (Leber Congenital Amaurosis), delivering a functional copy of the RPE65 gene directly into the retina to restore vision. The company also operates gene therapy development programs in hemophilia and other rare genetic disorders under the Roche/Genentech umbrella.

Product overview

Spark Therapeutics was a gene therapy company that has been fully integrated into the Roche Group (Genentech). The company's core product is Luxturna (voretigene neparvovec-rzyl), an FDA-approved gene therapy for inherited retinal diseases caused by RPE65 gene mutations. Spark Therapeutics developed gene therapies using adeno-associated virus (AAV) vectors for rare genetic diseases, with Luxturna being its sole FDA-approved product as of 2017.

Differentiator

Problem solved

Functional benefit

Brands

• Luxturna (voretigene neparvovec-rzyl): FDA-approved gene therapy for Leber Congenital Amaurosis (inherited blindness caused by RPE65 gene mutations)

Products and services

• Luxturna (voretigene neparvovec-rzyl) One-time AAV-based ocular gene therapy delivering a functional copy of the RPE65 gene to the retina to restore vision in patients with inherited retinal diseases (Leber Congenital Amaurosis) caused by biallelic RPE65 mutations; administered at specialized ophthalmology treatment centers and covered by approximately 99% of surveyed payers.

Quantifiable outcome

• 72% of clinical trial participants regained night vision
• +1 more outcomes

Named customers1 record

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Two partnerships are on record, tiered minor and core.

• Choroideremia Research Foundation (CRF)minorStrategic or Co-development Partner · 1 January 2026The Choroideremia Research Foundation acquired all assets related to Spark Therapeutics' discontinued SPK-7001 gene therapy for Choroideremia in January 2026, including intellectual property, orphan drug designations, and Phase I/II clinical trial data. CRF is analyzing five-year follow-up data to identify ophthalmic endpoints for potential future regulatory action.
• RochecoreOthersRoche acquired Spark Therapeutics for $4.8 billion in 2019, making it a fully integrated subsidiary operating under Genentech. Ron Philip served as CEO of Spark during this acquisition.

Scale indicators5 records

Recent moves6 records

Expansion highlights3 records

Broad incumbents

• Genentech (Roche): Genentech is the parent organization that fully integrated Spark Therapeutics after the 2019 Roche acquisition. As a broad incumbent in ophthalmology and rare disease biopharmaceuticals, Genentech encompasses Spark's operations and uses its established commercial infrastructure to distribute Luxturna.

Direct peers

• Sarepta Therapeutics: Sarepta develops gene therapies for rare genetic diseases, including Elevidys for Duchenne muscular dystrophy (AAV-based). It is a direct peer in AAV gene therapy commercialization for rare monogenic disorders and operates in the same high-cost one-time treatment reimbursement environment.
• BioMarin Pharmaceutical: BioMarin is a rare-disease biopharmaceutical company developing AAV-based gene therapies (notably Roctavian for hemophilia A), directly comparable to Spark's AAV gene therapy platform targeting rare monogenic disorders. Both companies operate in the high-cost one-time gene therapy commercial model with similar payer and patient-access dynamics.
• bluebird bio: bluebird bio is a gene therapy company developing lentiviral-based treatments for rare genetic diseases (sickle cell disease, beta-thalassemia, cerebral adrenoleukodystrophy). It is a peer in the rare-disease gene therapy commercial space, operating with similar one-time treatment pricing and payer negotiation dynamics.
• Adverum Biotechnologies: Adverum is a clinical-stage gene therapy company developing AAV-based therapies for ocular diseases including wet AMD and diabetic retinopathy. It is the most direct competitor in ocular AAV gene therapy, sharing Spark's modality, indication area, and delivery approach (intraocular injection).
• Krystal Biotech: Krystal Biotech developed Vyjuvek, the first FDA-approved topical gene therapy (for dystrophic epidermolysis bullosa). It is a peer in commercializing AAV-based gene therapies for rare monogenic diseases and shares the one-time treatment, high-cost reimbursement paradigm with Luxturna.
• uniQure: uniQure is a gene therapy company developing AAV-based treatments for rare diseases including hemophilia B (etranacogene dezaparvovec) and Huntington's disease. It shares Spark's AAV platform heritage, rare-disease focus, and one-time gene therapy commercial model.
• REGENXBIO: REGENXBIO is an AAV gene therapy platform company developing treatments for rare diseases including retinal conditions (ABBV-RGX-314). Its proprietary AAV technology and ocular gene therapy focus directly parallel Spark's AAV platform and Luxturna ocular gene therapy approach.

Emerging players

• Solid Biosciences: Solid Biosciences is a clinical-stage gene therapy company developing AAV-based treatments for Duchenne muscular dystrophy and other rare neuromuscular disorders. It shares Spark's AAV gene therapy modality and rare monogenic disease focus but is earlier-stage and less commercially diversified.
• Editas Medicine: Editas Medicine is a clinical-stage gene editing company developing CRISPR-based therapies for inherited retinal diseases (EDIT-101 for LCA10) and other genetic conditions. It overlaps with Spark in ocular genetic disease targeting, though using a different (editing) modality.

Market position

Strengths1 record

Weaknesses1 record

Competitive moat4 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xx.com/spark_tx
• Facebookfacebook.com/1028132703905824
• LinkedInlinkedin.com/company/spark-therapeutics-inc

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds5 records

Investors7 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Spark Therapeutics