Orna Therapeutics Establishes Strategic Collaboration with Vertex Pharmaceuticals to Develop Next Generation Approaches for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)
Orna Therapeutics has announced a three-year strategic research collaboration with Vertex Pharmaceuticals (VRTX) to develop next-generation gene editing therapies for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT). The partnership will utilize Orna's proprietary lipid nanoparticle (LNP) delivery solutions to enhance Vertex's therapeutic development efforts.
Under the agreement, Orna will receive an upfront payment and is eligible for potential pre-clinical, clinical, and commercialization milestone payments, along with royalties. The collaboration aims to leverage Orna's extra-hepatic LNP delivery chemistries to achieve unprecedented delivery to hematopoietic stem cells (HSCs). This marks Orna's second major partnership, highlighting the company's leadership in next-generation RNA medicines.
Orna Therapeutics ha annunciato una collaborazione strategica di ricerca di tre anni con Vertex Pharmaceuticals (VRTX) per sviluppare terapie di editing genetico di nuova generazione per la malattia falciforme (SCD) e la beta-talassemia dipendente da trasfusione (TDT). La partnership utilizzerà le soluzioni proprietarie di nanoparticelle lipidiche (LNP) di Orna per potenziare gli sforzi di sviluppo terapeutico di Vertex.
In base all'accordo, Orna riceverà un pagamento iniziale e sarà idonea a potenziali pagamenti legati a traguardi pre-clinici, clinici e di commercializzazione, oltre a royalty. La collaborazione mira a sfruttare le chemistrie di consegna extraepatica LNP di Orna per ottenere una consegna senza precedenti alle cellule staminali ematopoietiche (HSC). Questo segna la seconda grande partnership di Orna, evidenziando la leadership dell'azienda nelle medicine RNA di nuova generazione.
Orna Therapeutics ha anunciado una colaboración estratégica de investigación de tres años con Vertex Pharmaceuticals (VRTX) para desarrollar terapias de edición genética de nueva generación para la enfermedad de células falciformes (SCD) y la beta talasemia dependiente de transfusiones (TDT). La asociación utilizará las soluciones de entrega de nanopartículas lipídicas (LNP) de Orna para mejorar los esfuerzos de desarrollo terapéutico de Vertex.
En virtud del acuerdo, Orna recibirá un pago inicial y será elegible para pagos potenciales por hitos preclínicos, clínicos y de comercialización, además de regalías. La colaboración tiene como objetivo aprovechar las quimioterapias de entrega LNP extrahepáticas de Orna para lograr una entrega sin precedentes a las células madre hematopoyéticas (HSC). Esta es la segunda gran asociación de Orna, destacando el liderazgo de la empresa en medicamentos de ARN de nueva generación.
Orna Therapeutics는 Vertex Pharmaceuticals (VRTX)와 함께 겸형 적혈구 병(SCD) 및 수혈 의존형 베타 지중해빈혈(TDT)를 위한 차세대 유전자 편집 치료제를 개발하기 위한 3년 전략 연구 협력을 발표했습니다. 이 파트너십은 Vertex의 치료 개발 노력을 강화하기 위해 Orna의 고유한 지질 나노입자(LNP) 전달 솔루션을 사용할 것입니다.
계약에 따라 Orna는 선불금을 수령하며, 잠재적인 전임상, 임상 및 상용화 이정표 지급금과 로열티를 받을 수 있습니다. 협력은 Orna의 간외 LNP 전달 화학을 활용하여 조혈 줄기 세포(HSC)로의 전례 없는 전달을 달성하는 것을 목표로 하고 있습니다. 이는 Orna의 두 번째 주요 파트너십을 의미하며, 차세대 RNA 의약품 분야에서 회사의 선도적 역할을 강조합니다.
Orna Therapeutics a annoncé une collaboration de recherche stratégique de trois ans avec Vertex Pharmaceuticals (VRTX) pour développer des thérapies d'édition génique de nouvelle génération pour la drépanocytose (SCD) et la thalassémie bêta dépendante des transfusions (TDT). Le partenariat utilisera les solutions de livraison de nanoparticules lipidiques (LNP) propriétaires d'Orna pour améliorer les efforts de développement thérapeutique de Vertex.
Dans le cadre de l'accord, Orna recevra un paiement initial et pourra bénéficier de paiements potentiels liés à des étapes précliniques, cliniques et de commercialisation, ainsi que de redevances. La collaboration vise à tirer parti des chimies de livraison LNP extrahépatique d'Orna pour atteindre une livraison sans précédent aux cellules souches hématopoïétiques (HSC). Cela marque le deuxième partenariat majeur d'Orna, mettant en lumière le leadership de l'entreprise dans le domaine des médicaments à ARN de nouvelle génération.
Orna Therapeutics hat eine dreijährige strategische Forschungskooperation mit Vertex Pharmaceuticals (VRTX) bekannt gegeben, um Therapien zur Genbearbeitung der nächsten Generation für Sichelzellanämie (SCD) und transfusionabhängige Beta-Thalassämie (TDT) zu entwickeln. Die Partnerschaft wird Orna's proprietäre Lösungen zur Lieferung von liposomalen Nanopartikeln (LNP) nutzen, um die therapeutischen Entwicklungsanstrengungen von Vertex zu verbessern.
Im Rahmen der Vereinbarung erhält Orna eine Vorauszahlung und ist berechtigt, mögliche Zahlungen für präklinische, klinische und kommerzielle Meilensteine sowie Lizenzgebühren zu erhalten. Ziel der Zusammenarbeit ist es, Orna's extrahapatische LNP-Transportchemien zu nutzen, um eine beispiellose Lieferung an hämatopoetische Stammzellen (HSC) zu erreichen. Dies markiert Orna's zweite bedeutende Partnerschaft und unterstreicht die Führungsrolle des Unternehmens im Bereich der RNA-Medikamente der nächsten Generation.
- Strategic partnership with industry leader Vertex Pharmaceuticals
- Multiple revenue streams: upfront payment, milestone payments, and royalties
- Validation of Orna's LNP delivery technology through second major partnership
- None.
Insights
The deal's structure - combining upfront payments with milestone-based compensation - aligns with industry standards for early-stage biotech collaborations. Vertex's recent success with CASGEVY (their CRISPR-based therapy for SCD/TDT) makes this partnership particularly noteworthy, as it suggests they're already planning next-generation improvements to their treatment approach.
The focus on in vivo delivery to hematopoietic stem cells (HSCs) is important - current gene editing treatments require ex vivo cell manipulation, which is complex and expensive. Success here could dramatically reduce treatment costs and expand accessibility.
For Vertex shareholders, this represents a strategic move to maintain leadership in the hemoglobinopathy space while potentially developing more scalable treatment options. The three-year timeline suggests relatively near-term deliverables.
This move is particularly strategic given the competitive landscape in genetic medicines. While Vertex currently leads in SCD/TDT treatment with CASGEVY, maintaining this position requires continuous innovation.
The collaboration with Orna could provide several competitive advantages:
- Potentially lower manufacturing costs through simplified delivery methods
- Improved treatment accessibility via non-viral delivery systems
- Possible expansion into other genetic disorders using the same delivery platform
For investors, this represents Vertex's proactive approach to maintaining market leadership while addressing key challenges in gene therapy commercialization, particularly around delivery and cost structures.
-- Collaboration to leverage Orna's differentiated lipid nanoparticle (LNP) delivery solutions for patients with SCD and TDT --
-- Orna to receive an upfront payment and is eligible to receive potential pre-clinical, clinical, and commercialization milestone payments and royalties --
"Vertex is a leader in delivering next-generation approaches to treating hemoglobinopathies, and we are excited to collaborate with them to develop in vivo therapies that leverage our proprietary technologies to achieve unprecedented delivery to HSCs," commented Amit Munshi, Chief Executive Officer of Orna. "Today's collaboration further validates our industry leading extra-hepatic LNP delivery chemistries and highlights the importance of delivery to enable the next wave of RNA medicines."
Ansbert Gadicke, M.D., Chairman of Orna and Managing Partner of MPM BioImpact added, "Today's collaboration leveraging Orna's industry-leading non-viral HSC delivery represents our second major partnership and is testament to the breadth and potential of the Company's platform. We look forward to partnering with Vertex to develop transformative therapies for SCD and TDT, while simultaneously advancing our lead panCAR™ pipeline programs in autoimmune and oncology. This deal further solidifies Orna's leadership in next generation RNA medicines and has the potential to deliver large-scale impact to patients."
About the Collaboration
Under the terms of the agreement, Orna will receive upfront payments of
About Orna Therapeutics
Orna Therapeutics is dedicated to designing and delivering a new class of fully engineered circular RNA (oRNA®) therapeutics to unlock the potential of RNA medicine to treat diseases anywhere in the body. Orna's circular RNA transcripts have advantages over traditional mRNA approaches, including simplified production, improved formulation into lipid nanoparticles, and superior protein expression. Its industry-leading LNP-based delivery systems and comprehensive editing programs position Orna to advance novel RNA medicines with vast potential to transform patient care. To learn more, visit www.ornatx.com and follow Orna Therapeutics on X and LinkedIn.
Orna Therapeutics Investor Contact:
Alex Lobo
Precision AQ
alex.lobo@precisionaq.com
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