Orna Therapeutics Establishes Strategic Collaboration with Vertex Pharmaceuticals to Develop Next Generation Approaches for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)

Rhea-AI Summary

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.

Positive

• 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

Negative

• None.

-- 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 --

WATERTOWN, Mass., Jan. 7, 2025 /PRNewswire/ -- Orna Therapeutics (through its wholly owned subsidiary ReNAgade Therapeutics Inc.) announced a three-year strategic research collaboration with Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) to utilize Orna's novel and proprietary LNP delivery solutions to enhance Vertex's efforts in developing next generation gene editing therapies for patients with SCD and TDT.

"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 $65 million, including an investment in the form of a convertible note, and is eligible to receive up to $635 million based upon the achievement of specified pre-clinical, research, development, regulatory and commercial milestones related to SCD/TDT products. Additionally, Orna is further eligible to receive up to $365 million in additional option fees and milestones per product for up to ten additional products if Vertex options rights in additional indications. Orna will be eligible to receive tiered royalties on future net sales of any products that may result from this collaboration. Vertex is funding the three-year research collaboration and holds an option to extend the research collaboration term.

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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SOURCE Orna Therapeutics

FAQ

What is the purpose of the Vertex (VRTX) and Orna Therapeutics collaboration announced in January 2024?

The collaboration aims to develop next-generation gene editing therapies for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) using Orna's proprietary LNP delivery solutions.

What financial terms are included in the VRTX-Orna Therapeutics partnership?

Orna will receive an upfront payment and is eligible for potential pre-clinical, clinical, and commercialization milestone payments, plus royalties, though specific amounts were not disclosed.

How long will the Vertex (VRTX) and Orna Therapeutics collaboration last?

The strategic research collaboration is set for a three-year period.

What technology is Orna contributing to the VRTX collaboration?

Orna is contributing its proprietary lipid nanoparticle (LNP) delivery solutions and extra-hepatic LNP delivery chemistries for delivery to hematopoietic stem cells (HSCs).