RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Rare Pediatric Disease (United States)

Structure/Sequence

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Sequence Code 32378966

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Clinical Trials associated with Sepofarsen

NCT06891443

/ RecruitingPhase 3

A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene

The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290.

Start Date04 Jun 2025

Sponsor / Collaborator

Laboratoires Théa SAS

NCT04855045

/ Unknown statusPhase 2/3

An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age With Leber Congenital Amaurosis Type 10 (LCA10) Due to the c.2991 +1655A>G (p.Cys998X) Mutation.

PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (<8 years of age) with LCA10 due to the c.2991+1655A>G mutation over 24 months of treatment.

Start Date23 Mar 2021

Sponsor / Collaborator

ProQR Therapeutics NV

NCT03913130

/ TerminatedPhase 1/2

An Open-Label, Extension Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.

Start Date13 May 2019

Sponsor / Collaborator

Laboratoires Théa SAS

100 Clinical Results associated with Sepofarsen

100 Translational Medicine associated with Sepofarsen

100 Patents (Medical) associated with Sepofarsen

Literatures (Medical) associated with Sepofarsen

01 Nov 2026·AMERICAN JOURNAL OF OPHTHALMOLOGY

Efficacy, Safety, and Durability of Gene Therapy for Inherited Retinal Diseases in the Pediatric Population: A Scoping Review

Article

Author: Stephenson, Kirk Aj ; Amato, Alessia ; Iarossi, Giancarlo

PURPOSE:

To map the published interventional gene-based therapies relevant to children with inherited retinal diseases (IRDs), with a focus on efficacy, safety, and durability, including pediatric-vs-adult patterns where available.

METHODS:

This scoping review followed a prospectively registered protocol specifying the search strategy, eligibility criteria, screening process, data-charting framework, and synthesis plan. PubMed and Embase were searched from January 1, 2008, to April 13, 2026. Retrieved records were independently screened by 2 reviewers to identify prospective interventional studies of gene-targeted therapies for molecularly confirmed IRDs enrolling participants younger than 18 years; unresolved disagreements were adjudicated by a third reviewer. Overlapping reports were grouped into trial clusters with a primary anchor record. Records with at least 3 pediatric participants and separately extractable pediatric data were classified as Tier 1 and included in the main synthesis; other eligible records were retained as Tier 2 contextual evidence. Where sufficient individual-level or subgroup-level data were available, review authors extracted descriptive pediatric-vs-adult patterns.

RESULTS:

Twenty-four records, grouped into 17 trial clusters across 8 genotypes, were included; 14 met Tier 1 criteria. RPE65-associated disease provided the largest pediatric evidence base and the most consistent efficacy and durability signals, although responses varied between trial clusters. AIPL1 gene supplementation showed substantial treated-eye functional gains with relative structural preservation at up to 4 years in 4 very young children, and high-dose GUCY2D gene supplementation improved retinal sensitivity in 3 children within a mixed-age cohort. CEP290-targeted splice modulation and gene editing showed early functional signals, but interpretation was limited by dose-related cataract with sepofarsen and few pediatric participants with EDIT-101. Evidence for RPGR, CNGB3, RS1, and MERTK did not support robust pediatric-specific efficacy conclusions. Serious ocular events were predominantly procedure-related, with no reproducible pediatric-specific toxicity signal. Formal age-stratified analyses were uncommon, and several pediatric-vs-adult patterns required review-author extraction.

CONCLUSIONS:

Pediatric benefit from IRD gene therapy depends on genotype, residual retinal structure, therapeutic platform, dose, surgical strategy, and outcome measure. The major evidence gaps are long-term pediatric durability and inconsistent age-stratified reporting. Future pediatric-inclusive trials should prespecify subgroup analyses, embed prospective long-term follow-up, use developmentally appropriate endpoints, and report individual-level data sufficient to support pediatric inference.

01 Dec 2023·American journal of ophthalmology case reports

Durable vision improvement after a single intravitreal treatment with antisense oligonucleotide in CEP290-LCA: Replication in two eyes

Author: Girach, Aniz ; Wu, Vivian ; Ho, Allen C ; Viarbitskaya, Iryna ; Swider, Malgorzata ; Sumaroka, Alexander ; Garafalo, Alexandra V ; Cideciyan, Artur V ; Jacobson, Samuel G ; Russell, Robert C ; Schwartz, Michael R ; Roman, Alejandro J

Purpose:

An intravitreally injected antisense oligonucleotide, sepofarsen, was designed to modulate splicing within retinas of patients with severe vision loss due to deep intronic c.2991 + 1655A > G variant in the CEP290 gene. A previous report showed vision improvements following a single injection in one eye with unexpected durability lasting at least 15 months. The current study evaluated durability of efficacy beyond 15 months in the previously treated left eye. In addition, peak efficacy and durability were evaluated in the treatment-naive right eye, and re-injection of the left eye 4 years after the first injection.

Observations:

Visual function was evaluated with best corrected standard and low-luminance visual acuities, microperimetry, dark-adapted chromatic perimetry, and full-field sensitivity testing. Retinal structure was evaluated with OCT imaging. At the fovea, all visual function measures and IS/OS intensity of the OCT showed transient improvements peaking at 3-6 months, remaining better than baseline at ∼2 years, and returning to baseline by 3-4 years after each single injection.

Conclusions and Importance:

These results suggest that sepofarsen reinjection intervals may need to be longer than 2 years.

01 Jun 2022·Ophthalmology science

Restoration of Cone Sensitivity to Individuals with Congenital Photoreceptor Blindness within the Phase 1/2 Sepofarsen Trial

Article

Author: Drack, Arlene V ; Schwartz, Michael R ; Garafalo, Alexandra V ; Leroy, Bart P ; Russell, Stephen R ; Ho, Allen C ; Cideciyan, Artur V ; Swider, Malgorzata ; Jacobson, Samuel G ; Krishnan, Arun K ; Roman, Alejandro J ; Girach, Aniz ; Van Cauwenbergh, Caroline ; Wu, Vivian ; Sumaroka, Alexander

Purpose:

To understand consequences of reconstituting cone photoreceptor function in congenital binocular blindness resulting from mutations in the centrosomal protein 290 (CEP290) gene.

Design:

Phase 1b/2 open-label, multicenter, multiple-dose, dose-escalation trial.

Participants:

A homogeneous subgroup of 5 participants with light perception (LP) vision at the time of enrollment (age range, 15-41 years) selected for detailed analyses. Medical histories of 4 participants were consistent with congenital binocular blindness, whereas 1 participant showed evidence of spatial vision in early life that was later lost.

Intervention:

Participants received a single intravitreal injection of sepofarsen (160 or 320 μg) into the study eye.

Main Outcome Measures:

Full-field stimulus testing (FST), visual acuity (VA), and transient pupillary light reflex (TPLR) were measured at baseline and for 3 months after the injection.

Results:

All 5 participants with LP vision demonstrated severely abnormal FST and TPLR findings. At baseline, FST threshold estimates were 0.81 and 1.0 log cd/m² for control and study eyes, respectively. At 3 months, study eyes showed a large mean improvement of -1.75 log versus baseline (P < 0.001), whereas untreated control eyes were comparable with baseline. Blue minus red FST values were not different than 0 (P = 0.59), compatible with cone mediation of remnant vision. At baseline, TPLR response amplitude and latency estimates were 0.39 mm and 0.72 seconds, respectively, for control eyes, and 0.28 mm and 0.78 seconds, respectively, for study eyes. At 3 months, study eyes showed a mean improvement of 0.44 mm in amplitude and a mean acceleration of 0.29 seconds in latency versus baseline (P < 0.001), whereas control eyes showed no significant change versus baseline. Specialized tests performed in 1 participant confirmed and extended the standardized results from all 5 participants.

Conclusions:

By subjective and objective evidence, intravitreal sepofarsen provides improvement of light sensitivity for individuals with LP vision. However, translation of increased light sensitivity to improved spatial vision may occur preferentially in those with a history of visual experience during early neurodevelopment. Interventions for congenital lack of spatial vision in CEP290-associated Leber congenital amaurosis may lead to better results if performed before visual cortex maturity.

News (Medical) associated with Sepofarsen

25 Jun 2026

·allsci.com

Eli Lilly doubles down on ProQR with USD 59.2m funding round

Netherlands-based ProQR Therapeutics N.V. (Nasdaq: PRQR) announced the pricing of a combined underwritten registered direct offering and concurrent private placement totaling approximately USD 59.2 million in gross proceeds, with capital directed toward advancing its Axiomer RNA editing platform pipeline and funding general operations. The transaction is structured to retain Eli Lilly and Company as a proportional shareholder, underscoring the strategic importance of the partnership to ProQR’s development program. The registered direct offering comprises 27,624,310 ordinary shares priced at USD 1.81 per share, generating approximately USD 50 million in gross proceeds before underwriting discounts and expenses. Concurrently, Eli Lilly — an existing shareholder and strategic partner — entered into a separate share purchase agreement to acquire 5,100,780 ordinary shares at the same USD 1.81 offering price, for approximately USD 9.2 million in additional gross proceeds. Lilly’s participation is structured to allow it to maintain its pro rata beneficial ownership following the dilution created by the public offering. The two tranches are legally independent: the registered offering’s closing is not contingent on the concurrent private placement. BofA Securities, Evercore ISI, and Cantor acted as joint lead bookrunning managers, with Oppenheimer & Co. as an additional bookrunner. Proceeds will be used primarily to fund research and clinical development of current and additional pipeline candidates, with the remainder allocated to working capital and general corporate purposes. Company overview and pipeline ProQR is a Netherlands-headquartered, clinical-stage RNA therapy company with a US presence in Cambridge, Massachusetts. The company has strategically repositioned around its proprietary Axiomer platform, which harnesses the cell’s endogenous ADAR (adenosine deaminase acting on RNA) editing machinery to make targeted single-nucleotide edits in RNA. This approach is designed to correct disease-causing mutations or modulate protein expression without permanently altering the genome, and is intended to generate a new class of medicines applicable across both rare and prevalent diseases. The Axiomer platform is the foundation of ProQR’s expanded collaboration with Eli Lilly, which was originally established in September 2021 and significantly broadened in December 2022. Under the combined terms of those agreements, ProQR received USD 125 million in upfront payments and equity investments, with eligibility for up to approximately USD 3.75 billion in development, regulatory, and commercial milestone payments, plus tiered royalties. Lilly holds options to expand the collaboration further, and the December 2022 expansion included an additional USD 50 million option for Lilly to access new therapeutic targets. Lilly’s decision to participate in the concurrent private placement at the same price as public market investors reflects its continued commitment to maintaining proportional ownership in ProQR as the company executes on the Axiomer program. ProQR’s pivot to an RNA editing focus followed its 2023 divestiture of two late-stage ophthalmic RNA therapy assets — sepofarsen and ultevursen — to France-based Laboratoires Théa for EUR 8 million upfront and up to EUR 165 million in earn-out milestones. That transaction freed resources to concentrate exclusively on Axiomer-based programs. Beyond its Lilly partnership, ProQR has expanded the platform’s reach through a collaboration with the Rett Syndrome Research Trust (RSRT), which has provided a total of USD 9.1 million in funding — an initial USD 1 million grant in January 2024 followed by an USD 8.1 million expansion in December 2024 — to advance AX-2402, a candidate targeting MECP2 mutations in Rett syndrome, toward clinical development. This program extends Axiomer’s application into central nervous system diseases, adding to the metabolic and liver disease targets addressed under the Lilly partnership. The USD 59.2 million raise, combined with existing cash, is intended to extend the company’s development runway across its Axiomer pipeline. The next material catalyst for investors is likely to be updates on the Lilly collaboration programs and the progression of AX-2402 toward a planned Phase I trial in Rett syndrome, which the RSRT-funded program is designed to support. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Copyright © 2026. AllSci Corp. All rights reserved.

siRNA

03 Dec 2025

·www.theaopeninnovation.com

Théa and ProQR Therapeutics sign a new partnership

We are pleased to announce the signature today of a partnership with ProQR Therapeutics, a pioneer in next-generation RNA technology, for the further development of 2 projects: sepofarsen and ultevursen. This acquisition represents a new step for Théa in the research and treatment of rare genetic eye diseases. A fully dedicated team of experts specialized in hereditary retinal diseases (IRD) will be set up to manage these two innovative projects. Details of the next steps will be announced in the coming weeks. This strategic collaboration between Théa and ProQR Therapeutics opens up exciting new prospects in the field of eye care. To read the press release, click here.

Acquisition

01 Oct 2025

·www.theaopeninnovation.com

Sepul Bio, an innovative business unit of Théa, Doses First Participant in Phase 3 HYPERIONclinical trial of sepofarsen for CEP290-associated Leber Congenital Amaurosis Type 10

About us Our team News Laboratoires Théa Contact us About us Our team News Laboratoires Théa Contact us ©Eveon Home » News » Sepul Bio, an innovative business unit of Théa, Doses First Participant in Phase 3 HYPERIONclinical trial of sepofarsen for CEP290-associated Leber Congenital Amaurosis Type 10 Clermont-Ferrand, France, Wednesday 1 October 2025 – Sepul Bio, an innovative business unit of Théa, dedicated to the advancement of RNA therapies for inherited retinal diseases, has today announced the dosing of its first participants in the Phase 3 HYPERION clinical trial for sepofarsen, in individuals with CEP290- associated Leber Congenital Amaurosis Type 10 (LCA10). “There are currently no effective treatments for inherited retinal diseases like CEP290-associated LCA10,” says Dr. Artur Cideciyan, PhD, Research Professor of Ophthalmology and Co-Director, Center for Hereditary Retinal Degenerations at the Scheie Eye Institute of the University of Pennsylvania. “Together with Dr. Tomas Aleman, MD, we have studied this childhood blindness for two decades. We are now excited to participate in the HYPERION phase 3 clinical trial and dose one of the first participants. The HYPERION study is evaluating the potential positive impact of sepofarsen, an RNA therapy, on the vision of individuals with LCA10. We know that the c.2991+1655A>G (p.Cys998X) variant in the CEP290 gene stops retinal cells from producing an essential protein needed for vision. By addressing this variant specifically, sepofarsen aims to restore cell function in the retina, offering the LCA10 community potentially life-changing therapies.” LCA is a rare genetic eye disorder that primarily affects the retina, which is the light sensitive tissue at the back of the eye. It is a congenital condition, meaning it is present from birth, and causes severe vision impairment or blindness. It results from genetic mutations that disrupt the function of the retina’s photoreceptor cells. Common symptoms, such as loss of acuity, nystagmus (involuntary eye movements), nyctalopia and impaired peripheral vision, typically emerge in infancy. LCA encompasses various subtypes, each linked to specific genetic mutations and different levels of visual impairment. “The launch of the HYPERION study is an exciting milestone for the LCA community,” said Courtney Coates, Executive Director, Hope in Focus. “We have been eagerly waiting for the re-initiation of the sepofarsen program, and today’s announcement reflects renewed hope for the community. As a community partner, we look forward to working alongside the Sepul Bio team as they advance RNA therapies to potentially help children, adults, and families affected by CEP290- associated LCA10.” “CEP290- associated LCA10 is a devastating retinal disease resulting in blindness, for which there are no therapies,” said Dr. Bart Leroy, Head of the Ophthalmology Department at Ghent University Hospital and Professor of Ophthalmology and Ophthalmic Genetics at Ghent University, Ghent, Belgium. “We are proud to continue to bring forward sepofarsen as a medicine through the new HYPERION clinical trial. We are delighted to be part of the first wave of clinical sites to dose participants. By directly targeting this defect, sepofarsen is designed to restore retinal cell activity and bring the possibility of transformative therapies to the LCA10 community.” About the Phase 3 HYPERION study HYPERION, or SB-110-007, is a two-year double-masked, randomized, placebo-controlled, paired-eye study to evaluate the efficacy, safety and tolerability of sepofarsen in 32 participants with LCA10 due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290 gene. About Sepul Bio As a dedicated business unit of Théa, Sepul Bio is at the forefront of advancing transformative RNA therapies for inherited retinal diseases, with a particular emphasis on the further development of two cutting-edge ophthalmic products—sepofarsen and ultevursen. Sepofarsen targets LCA10, a rare genetic ailment causing severe visual impairment, while ultevursen is designed to slow vision loss in individuals with a mutation in exon 13 of the USH2A gene suffering from retinitis pigmentosa. www.sepulbio.com About Théa Théa is the leading independent European pharmaceutical company specialized in the research, development, and commercialization of eye care products. Based in Clermont Ferrand, France, this family-owned and run company comprises more than 2000 collaborators and has expanded by opening 35 affiliates and offices in Europe, North Africa, North and South America, and the Middle East. Its products are available in 75 countries. www.thea.com Feel free to contact us to submit your project or ideas. By submitting this form, I accept that the information entered will be used, to answer my request. The recipient of the data is Théa. Please read our Privacy Policy Théa Open Innovation 12 rue Louis-Blériot 63017 CLERMONT-FERRAND cedex FRANCE Tel. +33 (0)4 73 98 14 36 About us Our team News Laboratoires Théa Contact us About us Our team News Laboratoires Théa Contact us © Théa Open Innovation 2024 | Personal data protection | Terms & conditions | Legal terms | Site Map

Phase 3

100 Deals associated with Sepofarsen

External Link

KEGG	Wiki	ATC	Drug Bank
D12488	-	S01XA29	DB16327

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Retinal Dystrophies	Phase 3	Belgium	ProQR Therapeutics NV	23 Mar 2021
Retinal Dystrophies	Phase 3	Brazil	ProQR Therapeutics NV	23 Mar 2021
Retinal Dystrophies	Phase 3	Canada	ProQR Therapeutics NV	23 Mar 2021
Retinal Dystrophies	Phase 3	Germany	ProQR Therapeutics NV	23 Mar 2021
Retinal Dystrophies	Phase 3	Italy	ProQR Therapeutics NV	23 Mar 2021
Retinal Dystrophies	Phase 3	Netherlands	ProQR Therapeutics NV	23 Mar 2021
Retinal Dystrophies	Phase 3	United Kingdom	ProQR Therapeutics NV	23 Mar 2021
Blindness	Phase 3	United States	ProQR Therapeutics NV	04 Apr 2019
Blindness	Phase 3	Belgium	ProQR Therapeutics NV	04 Apr 2019
Blindness	Phase 3	Brazil	ProQR Therapeutics NV	04 Apr 2019

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03913143 (ILLUMINATE, ARVO2026)	Phase 3	Leber Congenital Amaurosis 10 CEP290-associated	15	Sepofarsen	qvzpyxarxf(tyfwgktilf) = trzulbzphz iwtnjhopjz (nccjnjzvly ) View more	Positive	03 May 2026
				Sham injection	qvzpyxarxf(tyfwgktilf) = xtrmabcxxc iwtnjhopjz (nccjnjzvly ) View more
NCT06891443 (HYPERION, ARVO2026)	Phase 3	Leber Congenital Amaurosis 10	62	Sepofarsen (Pediatric (<18 years))	dggorrmoiy(kaajbxqdxr) = Most AEs were mild and related to the injection procedure hrdnwlclfm (jrerxuexxc )	Positive	03 May 2026
				Sepofarsen (Adult (≥18 years))
NCT03913130 (INSIGHT, CTgov)	Phase 1/2	Blindness \| Leber Congenital Amaurosis 10	9	QR-110	gysubfzbno = idbkxcbqpl whkvwiamao (ekldljvepa, dnbsfuuiul - dwrfiflrfs) View more	-	04 Jan 2023
NCT03140969 (Pubmed \| Nat Med)	Phase 1/2	Leber Congenital Amaurosis 10	11	sepofarsen (Untreated eye)	sadzvuanbq(yedmkttwlw) = Eight patients developed cataracts, of which six (75.0%) were categorized as serious (2/3 with 160 µg/80 µg; 4/5 with 320 µg/160 µg), as lens replacement was required. dmdxaglcyy (mqktonydaw ) View more	Positive	04 Apr 2022
EURETINA2021	Not Applicable	Leber Congenital Amaurosis	-	Sepofarsen 160 µg	nexyqvqnja(dfjxowwtni) = wxmosmcevx diytaqustm (okdimjhsgh ) View more	-	01 Sep 2021
				Sepofarsen 80 µg	nexyqvqnja(dfjxowwtni) = qzeucqnojm diytaqustm (okdimjhsgh ) View more
NCT03913130 (ARVO2021)	Phase 1/2	Leber Congenital Amaurosis 10	9	Sepofarsen	utnaimweat(ytsoforzhv) = One patient developed cataract at Month 9 in the second treated eye ynxlrncoql (eajylmdgst )	-	01 Jun 2021
NCT03140969 (CEP290, AAO2020)	Phase 1/2	Leber Congenital Amaurosis 10 p.Cys998X mutation in the CEP290 gene	11	Sepofarsen 320/160-µg	jqzmwwanjx(bbzfobjvrh) = Reported cases of cataract were 3 in the 160/80-µg group and 5 in the 320/160-µg group gnvbpptqsf (lkhprvbyay ) View more	Positive	13 Nov 2020
NCT03140969 (ARVO2020)	Phase 1/2	Leber Congenital Amaurosis	11	sepofarsen 160/80 µg	ycyjqjshyl(tmoawppdor) = Although sepofarsen treatment is associated with cataract development, it is well tolerated and shows improvement in BCVA, FST, and mobility course. The 160/80 µg dose has a more favorable benefit:risk profile than 320/160 µg ecdkwboqfz (qnezuoizoh ) View more	-	01 Jun 2020
				sepofarsen 320/160 µg
NCT03140969 (PQ-110-001, GlobeNewswire) Manual	Phase 1/2	Leber Congenital Amaurosis 10	11	sepofarsen	xsabqbvvqj(micuwzcenv) = wbdgovngvq qhyytgfkqy (npfhunpbwu ) View more	Positive	10 Oct 2019
				Placebo	xsabqbvvqj(micuwzcenv) = wfocltqfav qhyytgfkqy (npfhunpbwu ) View more
NCT03140969 (CEP290, ARVO2019)	Phase 1/2	Leber Congenital Amaurosis	10	QR-110 160/80 µg	cmpchpffjl(fbaspqkxcb) = results also demonstrated greater stability fpvqvkgonv (riecabudyn )	Positive	01 Jul 2019
				QR-110 320/160 µg

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