Opus Genetics Announces FDA Clearance of IND Application for Gene Therapy Candidate OPGx-BEST1
Opus Genetics (Nasdaq: IRD) has received FDA clearance for its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy targeting BEST1-related inherited retinal disease (IRD). The company plans to initiate a Phase 1/2 clinical trial in the second half of 2025.
The trial will evaluate a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related IRD, focusing on safety, tolerability, and preliminary efficacy. This represents Opus's third ongoing clinical program, utilizing their proprietary AAV-based gene therapy platform to deliver functional BEST1 genes to retinal pigment epithelium cells.
Opus Genetics (Nasdaq: IRD) ha ottenuto l'autorizzazione della FDA per la sua domanda di Investigational New Drug (IND) relativa a OPGx-BEST1, una terapia genica mirata alle malattie retiniche ereditarie correlate al gene BEST1. L'azienda prevede di avviare una sperimentazione clinica di Fase 1/2 nella seconda metà del 2025.
Lo studio valuterà un'unica iniezione sottoretinica di OPGx-BEST1 in pazienti con diagnosi genetica confermata di IRD correlata a BEST1, concentrandosi su sicurezza, tollerabilità ed efficacia preliminare. Questo costituisce il terzo programma clinico in corso di Opus, che utilizza la propria piattaforma AAV per somministrare copie funzionali del gene BEST1 alle cellule dell'epitelio pigmentato retinico.
Opus Genetics (Nasdaq: IRD) ha recibido la aprobación de la FDA para su solicitud de Investigational New Drug (IND) para OPGx-BEST1, una terapia génica dirigida a las enfermedades retinianas hereditarias relacionadas con BEST1. La compañía planea iniciar un ensayo clínico de Fase 1/2 en la segunda mitad de 2025.
El ensayo evaluará una inyección subretinal única de OPGx-BEST1 en pacientes con IRD relacionada con BEST1 confirmada genéticamente, centrándose en la seguridad, la tolerabilidad y la eficacia preliminar. Esto representa el tercer programa clínico en curso de Opus, que emplea su plataforma basada en AAV para entregar genes BEST1 funcionales a las células del epitelio pigmentario de la retina.
Opus Genetics (Nasdaq: IRD)가 OPGx-BEST1에 대한 임상시험용 신약(IND) 신청에 대해 FDA 승인을 받았습니다. OPGx-BEST1은 BEST1 관련 유전성 망막질환(IRD)을 표적하는 유전자 치료제입니다. 회사는 2025년 하반기 중 제1/2상 임상시험을 시작할 계획입니다.
이번 시험은 유전자적으로 BEST1 관련 IRD가 확인된 환자에게 OPGx-BEST1을 단회 망막하 주입하여 안전성, 내약성 및 예비 유효성을 평가합니다. 이는 기능성 BEST1 유전자를 망막색소상피 세포에 전달하기 위해 회사의 독자적인 AAV 기반 유전자 치료 플랫폼을 활용하는 Opus의 세 번째 진행 중인 임상 프로그램입니다.
Opus Genetics (Nasdaq: IRD) a obtenu l'autorisation de la FDA pour sa demande d'Investigational New Drug (IND) concernant OPGx-BEST1, une thérapie génique ciblant les maladies rétiniennes héréditaires liées à BEST1. La société prévoit de lancer un essai clinique de Phase 1/2 au second semestre 2025.
L'essai évaluera une injection sous-rétinienne unique d'OPGx-BEST1 chez des patients présentant une IRD liée à BEST1 confirmée génétiquement, en se concentrant sur la sécurité, la tolérabilité et l'efficacité préliminaire. Il s'agit du troisième programme clinique en cours d'Opus, utilisant sa plateforme propriétaire de thérapie génique basée sur l'AAV pour apporter des gènes BEST1 fonctionnels aux cellules de l'épithélium pigmentaire rétinien.
Opus Genetics (Nasdaq: IRD) hat von der FDA die Zulassung seines Investigational New Drug (IND)-Antrags für OPGx-BEST1 erhalten, eine Gentherapie, die auf BEST1-assoziierte erbliche Netzhauterkrankungen (IRD) abzielt. Das Unternehmen plant, in der zweiten Hälfte des Jahres 2025 eine Phase-1/2-Studie zu starten.
Die Studie wird eine einmalige subretinale Injektion von OPGx-BEST1 bei genetisch bestätigten BEST1-IRD-Patienten untersuchen und dabei Sicherheit, Verträglichkeit und erste Wirksamkeitsdaten erfassen. Dies ist Opus' drittes laufendes klinisches Programm, das seine eigene AAV-basierte Gentherapieplattform nutzt, um funktionsfähige BEST1-Gene an die retinalen Pigmentepithelzellen zu liefern.
- FDA clearance received for OPGx-BEST1 IND application
- Phase 1/2 trial to begin in second half of 2025
- Preclinical work shows promising results in disease models
- Third clinical program in pipeline demonstrates portfolio depth
- No current approved treatments for BEST1-related IRDs
- Early-stage clinical development with uncertain outcomes
Insights
Opus receives FDA IND clearance for BEST1 gene therapy, advancing toward clinical trials for an untreated inherited retinal disease.
The FDA's clearance of Opus Genetics' IND application for OPGx-BEST1 represents a significant regulatory milestone in their clinical development program. This approval enables the company to initiate their planned Phase 1/2 trial in the second half of 2025, making this their third ongoing clinical program.
This regulatory advancement is particularly notable because it addresses BEST1-related inherited retinal diseases (including Best disease or vitelliform macular dystrophy), which currently have no approved treatments. The condition causes macular degeneration through BEST1 gene mutations, leading to progressive vision loss and potential blindness.
The planned multi-center, open-label study will evaluate a single subretinal injection of OPGx-BEST1, focusing on safety, tolerability, and preliminary efficacy in patients with genetically confirmed BEST1-related IRD. Importantly, the study will track functional and anatomical endpoints, including changes in visual function and retinal structure - critical measures for establishing therapeutic value.
Mechanistically, OPGx-BEST1 utilizes Opus's proprietary AAV-based gene therapy platform to deliver a functional copy of the BEST1 gene directly to retinal pigment epithelium (RPE) cells. This approach is supported by preclinical evidence demonstrating restoration of BEST1 protein expression and improved retinal function in disease models, suggesting potential therapeutic efficacy if these results translate to humans.
– Phase 1/2 trial expected to initiate in 2H 2025 in patients with BEST1 inherited retinal disease
RESEARCH TRIANGLE PARK, N.C., Aug. 18, 2025 (GLOBE NEWSWIRE) -- Opus Genetics (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related IRD.
Best disease, or vitelliform macular dystrophy, is a rare, inherited retinal condition causing macular degeneration by mutations in the BEST1 gene, leading to progressive vision loss and, in some cases, blindness.
With this IND clearance, Opus Genetics plans to initiate a Phase 1/2 clinical trial in the second half of 2025. The multi-center, open-label study will evaluate the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related IRD. The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.
“The FDA’s clearance of our BEST1 IND is a significant step forward for the IRD community and for our mission at Opus Genetics focused on restoring vision for patients,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “BEST1-related IRDs have no approved treatments today, leaving patients and families with uncertainty about the future of their vision. The OPGx-BEST1 trial will be our third ongoing clinical program, reflecting the depth of our pipeline and our commitment to advancing multiple therapies in parallel for patients with urgent, unmet needs.”
OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models.
About Opus Genetics
Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders. The Company’s pipeline features AAV-based gene therapies targeting inherited retinal diseases including Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa. Its lead gene therapy candidates are OPGx-LCA5, which is in an ongoing Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1, a gene therapy targeting BEST1-related retinal degeneration. Opus Genetics is also advancing Phentolamine Ophthalmic Solution
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, expectations regarding us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, our Quarterly Reports on Form 10-Q for the quarters ended March 31, 2025 and June 30, 2025, and our other filings with the U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.
Contacts:
Investors
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
ir@opusgtx.com
Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com
Source: Opus Genetics, Inc.
FAQ
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