What Sepul Bio does

Sepul Bio is a clinical-stage biopharmaceutical business unit within Laboratoires Théa S.A.S., exclusively focused on developing antisense oligonucleotide (AON) RNA therapies for inherited retinal diseases (IRDs). The company was created in May 2024 when Théa acquired two late-stage ophthalmic assets — Sepofarsen and Ultevursen — from ProQR Therapeutics, along with key program personnel. Sepul Bio's portfolio consists of exactly two investigational programs: Sepofarsen, an AON targeting the c.2991+1655A>G mutation in the CEP290 gene for Leber congenital amaurosis type 10 (LCA10), currently in a 32-participant Phase 3 trial (HYPERION, first participant dosed October 2025); and Ultevursen, an AON targeting exon 13 mutations in the USH2A gene for Usher syndrome type 2a and non-syndromic retinitis pigmentosa, in an 81-participant Phase 2b trial (LUNA, first participant dosed December 2024). Both therapies are administered via intravitreal injection and are designed to modulate mRNA splicing to restore production of functional proteins in retinal cells, addressing the underlying genetic cause rather than symptoms. Both target ultra-rare indications with no approved disease-modifying therapies.

Sepul Bio does not yet generate commercial revenue; its only near-term economics consist of clinical trial execution and milestone progress. The company is incorporated and operates from Clermont-Ferrand, France, as a wholly-owned business unit of family-owned Théa, the leading independent European ophthalmology pharmaceutical company (2,000+ employees, 35 affiliates, 75 countries), which provides corporate infrastructure and a future commercial distribution channel. The business model is a future single-time specialty pharmaceutical product sale model — one-time treatment per patient, premium orphan-disease pricing upon regulatory approval, distributed through specialized ophthalmology centers and academic medical institutions via Théa's network. Customer segments are narrowly defined patient populations with confirmed CEP290 c.2991+1655A>G or USH2A exon 13 mutations. The management team is staffed largely by former ProQR leaders and veterans from Novartis, GSK, Bayer, Astellas/Iveric Bio, Biogen, and Bausch & Lomb, with clinical site partnerships including the University of Pennsylvania Scheie Eye Institute and Ghent University Hospital.

Name

Sepul Bio

Legal name

Laboratoires Théa S.A.S.

Website

https://sepulbio.com

Company type

Private

Founded year

2024

Operating status

Operating

Headcount range

1–10 employees

Short description

Sepul Bio is a clinical-stage business unit of Laboratoires Théa developing two antisense oligonucleotide RNA therapies — Sepofarsen (Phase 3 for CEP290-associated LCA10) and Ultevursen (Phase 2b for USH2A-associated Usher syndrome 2a) — for ultra-rare inherited retinal diseases with no approved treatments.

Ownership category

akta.pro rank

Product category

Ophthalmic RNA Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH)

akta.pro secondary industry

Rare Ophthalmology Disorder Therapies (HLAIAIAK)

Keywords

• Antisense oligonucleotide therapy
• Inherited retinal diseases
• RNA therapeutics
• Ophthalmic biotechnology
• Rare disease ophthalmology

Headquarters

HQ city

Clermont-ferrand

HQ country

France

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pharmaceutical Product Revenue: Sepul Bio is a clinical-stage biopharmaceutical company developing two RNA-based therapies (Sepofarsen and Ultevursen) for inherited retinal diseases. The company does not yet generate revenue as both products are in clinical development. Future revenue will come from product sales upon regulatory approval.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Sepul Bio is developing two antisense oligonucleotide (AON) RNA-based investigational therapies, Sepofarsen and Ultevursen, for the treatment of inherited retinal diseases (Leber congenital amaurosis 10 and Usher syndrome type 2a / non-syndromic retinitis pigmentosa). Both therapies are administered via intravitreal injection and are designed to correct disease-causing genetic mutations at the RNA level, addressing the underlying cause of disease rather than just symptoms.

Product overview

Sepul Bio is a business unit of Théa exclusively focused on developing transformative RNA therapies for inherited retinal diseases. The company's portfolio consists of two flagship antisense oligonucleotide (AON) RNA therapies: Sepofarsen (targeting LCA10, Phase 3) and Ultevursen (targeting Usher syndrome type 2a, Phase 2b). Both therapies operate by addressing genetic mutations at the RNA level to enable cells to produce functional proteins essential for retinal cell function.

Differentiator

Problem solved

Functional benefit

Products and services

• Sepofarsen Investigational antisense oligonucleotide (AON) RNA therapy designed to improve visual function in patients with Leber congenital amaurosis 10 (LCA10); targets the c.2991+1655A>G mutation in the CEP290 gene to enable production of a functional protein needed for retinal cell function. Administered via intravitreal injection. Currently in Phase 3 (HYPERION).
• Ultevursen Investigational antisense oligonucleotide (AON) RNA therapy designed to stabilize visual function in patients with Usher syndrome type 2a and non-syndromic retinitis pigmentosa; targets exon 13 mutations of the USH2A gene to restore usherin protein production in retinal cells. Administered via intravitreal injection. Currently in Phase 2b (LUNA).

Quantifiable outcome

• Visual acuity improvements of approximately 8 letters gain on standard eye chart after 12 months of sepofarsen treatment in paired-eye analysis (15 individuals)
• +1 more outcomes

Segments2 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Five partnerships are on record, tiered core, supporting and clinical.

• ProQR TherapeuticscoreStrategic or Co-development Partner · 2 May 2024Sepul Bio acquired two cutting-edge ophthalmic products (Sepofarsen and Ultevursen) from ProQR Therapeutics in 2024. These assets were being developed for inherited retinal diseases and Sepul Bio, as a Théa business unit, took over late-stage development. Key personnel from ProQR joined Sepul Bio, bringing expertise in inherited retinal disease therapeutics development.
• Laboratoires Théa S.A.S.coreOthersSepul Bio is a dedicated business unit within Théa, the leading independent European pharmaceutical company specializing in eye care. Théa provides corporate infrastructure, distribution network (35 affiliates, 75 countries), and strategic oversight. Sepul Bio focuses exclusively on developing treatments for inherited retinal diseases using the acquired RNA therapy assets.
• Hope in FocussupportingStrategic or Co-development PartnerHope in Focus is a patient advocacy organization for the LCA community. They serve as a community partner working alongside Sepul Bio to advance RNA therapies for CEP290-associated LCA10, providing patient perspectives and supporting community engagement.
• University of Pennsylvania (Scheie Eye Institute)clinicalStrategic or Co-development PartnerDr. Artur Cideciyan (Research Professor of Ophthalmology and Co-Director, Center for Hereditary Retinal Degenerations) and Dr. Tomas Aleman are participating as clinical investigators in the HYPERION Phase 3 clinical trial for sepofarsen, bringing two decades of experience studying CEP290-associated LCA10.
• Ghent University HospitalclinicalStrategic or Co-development PartnerDr. Bart Leroy (Head of the Ophthalmology Department at Ghent University Hospital and Professor of Ophthalmology and Ophthalmic Genetics) is a clinical site for the HYPERION Phase 3 trial, among the first wave of clinical sites to dose participants.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Biogen: Biogen is a major neuroscience and rare disease biopharmaceutical company with historical ophthalmology interests (e.g., earlier retina collaborations) and rare disease drug development infrastructure that overlaps with Sepul Bio's IRD orphan-disease focus.
• Genentech (Roche): Genentech markets Lucentis and Vabysmo for retinal diseases and runs inherited retinal disease programs through Spark Therapeutics, representing a broad incumbent with overlapping therapeutic area and inherited retinal disease portfolio investments.
• Iveric Bio (an Astellas company): Iveric Bio (now part of Astellas) commercializes Izervay for geographic atrophy and previously developed RNA-based ophthalmology assets, making it a broad incumbent in retinal therapeutics with overlapping modality and disease-area focus.

Others

• Laboratoires Théa: Sepul Bio's parent company, Théa, is the leading independent European ophthalmology pharmaceutical company, providing both the corporate infrastructure for Sepul Bio and a directly comparable commercial-stage eye-care peer in the same therapeutic area.

Emerging players

• 4D Molecular Therapeutics: 4DMT is developing intravitreally delivered AAV gene therapies for inherited retinal diseases, including programs targeting choroideremia and RPGR-associated retinitis pigmentosa, positioning it as a competing modality (gene therapy vs RNA/AON) in the IRD space.
• Beacon Therapeutics: Beacon Therapeutics is developing gene therapies for X-linked retinitis pigmentosa (RPGR) and other IRDs, making it a peer competing for the same patient populations and specialty ophthalmology centers targeted by Sepul Bio.
• Nanoscope Therapeutics: Nanoscope is developing optogenetic and gene therapy approaches for retinitis pigmentosa and other IRDs, representing a competitive emerging modality in the same patient population targeted by Sepul Bio's ultevursen.
• Stoke Therapeutics: Stoke Therapeutics develops antisense oligonucleotide (ASO) therapies for genetic diseases including autosomal dominant optic atrophy (OPA1), making it a direct comparable in the AON modality space targeting ophthalmology genetic diseases.

Direct peers

• Editas Medicine: Editas Medicine is developing EDIT-101, a CRISPR-based in vivo gene therapy for CEP290-associated LCA10 — directly competing with Sepul Bio's sepofarsen in the same indication using an alternative (gene editing vs AON) modality.
• ProQR Therapeutics: ProQR Therapeutics is the originator of the sepofarsen and ultevursen AON programs and remains the closest direct comparable, as Sepul Bio's team, assets, and clinical development approach are inherited almost directly from ProQR's RNA therapy franchise for inherited retinal diseases.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/sepulbio

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds

Investors

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

M&A

Investments

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

Frequently asked questions about Sepul Bio