REGENXBIO Announces Closing of Strategic Partnership with Nippon Shinyaku for MPS Diseases
REGENXBIO (RGNX) has finalized its strategic partnership with Nippon Shinyaku for the development and commercialization of two gene therapy treatments: RGX-121 (clemidsogene lanparvovec) for Mucopolysaccharidosis II (Hunter syndrome) and RGX-111 for Mucopolysaccharidosis I (Hurler syndrome) in the United States and Asia.
The collaboration aims to advance these potential treatments for rare genetic disorders, with RGX-121 positioned to potentially become the first gene therapy for MPS II, targeting FDA approval by late 2025.
REGENXBIO (RGNX) ha finalizzato la sua partnership strategica con Nippon Shinyaku per lo sviluppo e la commercializzazione di due trattamenti di terapia genica: RGX-121 (clemidsogene lanparvovec) per la Mucopolisaccaridosi II (sindrome di Hunter) e RGX-111 per la Mucopolisaccaridosi I (sindrome di Hurler) negli Stati Uniti e in Asia.
La collaborazione mira a far progredire questi potenziali trattamenti per rari disturbi genetici, con RGX-121 posizionato per diventare potenzialmente la prima terapia genica per la MPS II, puntando all'approvazione della FDA entro la fine del 2025.
REGENXBIO (RGNX) ha finalizado su asociación estratégica con Nippon Shinyaku para el desarrollo y la comercialización de dos tratamientos de terapia génica: RGX-121 (clemidsogene lanparvovec) para la Mucopolisacaridosis II (síndrome de Hunter) y RGX-111 para la Mucopolisacaridosis I (síndrome de Hurler) en los Estados Unidos y Asia.
La colaboración tiene como objetivo avanzar en estos tratamientos potenciales para trastornos genéticos raros, con RGX-121 posicionado para convertirse potencialmente en la primera terapia génica para la MPS II, buscando la aprobación de la FDA para finales de 2025.
REGENXBIO (RGNX)는 Nippon Shinyaku와 두 가지 유전자 치료제의 개발 및 상용화를 위한 전략적 파트너십을 체결했습니다: RGX-121 (clemidsogene lanparvovec)은 뮤코다당증 II형 (헌터 증후군) 치료를, RGX-111은 뮤코다당증 I형 (훌러 증후군) 치료를 위해 미국과 아시아에서 진행됩니다.
이번 협력은 희귀 유전 질환을 위한 잠재적 치료제를 발전시키는 것을 목표로 하며, RGX-121은 MPS II에 대한 최초의 유전자 치료제가 될 가능성이 있으며, 2025년 말까지 FDA 승인을 목표로 하고 있습니다.
REGENXBIO (RGNX) a finalisé son partenariat stratégique avec Nippon Shinyaku pour le développement et la commercialisation de deux traitements par thérapie génique : RGX-121 (clemidsogene lanparvovec) pour la Mucopolysaccharidose II (syndrome de Hunter) et RGX-111 pour la Mucopolysaccharidose I (syndrome de Hurler) aux États-Unis et en Asie.
La collaboration vise à faire progresser ces traitements potentiels pour des troubles génétiques rares, RGX-121 étant positionné pour devenir potentiellement la première thérapie génique pour la MPS II, avec l'objectif d'obtenir l'approbation de la FDA d'ici fin 2025.
REGENXBIO (RGNX) hat seine strategische Partnerschaft mit Nippon Shinyaku für die Entwicklung und Vermarktung von zwei Gentherapien finalisiert: RGX-121 (clemidsogene lanparvovec) für Mukopolysaccharidosen II (Hunter-Syndrom) und RGX-111 für Mukopolysaccharidosen I (Hurler-Syndrom) in den USA und Asien.
Die Zusammenarbeit zielt darauf ab, diese potenziellen Behandlungen für seltene genetische Erkrankungen voranzutreiben, wobei RGX-121 möglicherweise die erste Gentherapie für MPS II werden könnte, mit dem Ziel, bis Ende 2025 die FDA-Zulassung zu erhalten.
- Strategic partnership secured with Nippon Shinyaku for US and Asia markets
- RGX-121 positioned to be first-to-market gene therapy for MPS II
- Potential FDA approval for RGX-121 expected by late 2025
- None.
Under the terms of the agreement, REGENXBIO and Nippon Shinyaku will develop and commercialize RGX-121 (clemidsogene lanparvovec) for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome in
"RGX-121 and RGX-111 represent potentially transformative new medicines for patient populations in great need of new options," said Curran M. Simpson, President and CEO of REGENXBIO. "This partnership bolsters our ability to make important progress on these programs, and we look forward to serving the MPS community with Nippon Shinyaku."
RGX-121 is on track to be the first gene therapy for MPS II, with potential FDA approval as early as late 2025.
About RGX-121
RGX-121 is a potential one-time AAV therapeutic for the treatment of boys with MPS II. RGX-121 expressed protein is structurally identical to normal I2S. Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS.
RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the U.S. Food and Drug Administration and advanced therapy medicinal products (ATMP) classification from the European Medicines Agency.
About RGX-111
RGX-111 is designed to use the AAV9 vector to deliver the α-l-iduronidase (IDUA) gene to the central nervous system (CNS). By providing rapid IDUA delivery to the brain, RGX-111 could potentially help prevent the progression of cognitive deficits that otherwise occurs in MPS I patients. Positive interim data from a Phase I/II trial of RGX-111 were reported in February 2023. RGX-111 has received Orphan Drug Product, Rare Pediatric Disease and Fast Track designations from the U.S. Food and Drug Administration.
ABOUT REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.regenxbio.com.
ABOUT NIPPON SHINYAKU
Based on Nippon Shinyaku's business philosophy, "Helping people lead healthier, happier lives," we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. Please visit our website (www.nippon-shinyaku.co.jp/english/) for products or detailed information.
FORWARD-LOOKING STATEMENTS
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's collaboration with Nippon Shinyaku and REGENXBIO's future operations, clinical trials, and regulatory plans. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the anticipated completion of REGENXBIO's proposed transaction with Nippon Shinyaku, the outcome of REGENXBIO's proposed collaboration with Nippon Shinyaku, whether the milestones contemplated by the proposed transaction will be achieved, the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2023, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the
Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
George E. MacDougall
Investor Relations
IR@regenxbio.com
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SOURCE REGENXBIO Inc.
FAQ
When is RGNX's RGX-121 gene therapy expected to receive FDA approval?
What diseases will RGNX and Nippon Shinyaku target in their partnership?
What markets are covered in RGNX's partnership with Nippon Shinyaku?
What is the significance of RGNX's RGX-121 in the MPS II treatment landscape?
