Orna Therapeutics, Inc.

WATERTOWN, Mass., Jan. 9, 2025 /PRNewswire/ -- Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of circular RNA medicines and unprecedented lipid nanoparticle (LNP) delivery solutions for oncology and autoimmune diseases, today announced the appointment of Vikas Sinha to its Board of Directors. "We are thrilled to welcome Vikas to our Board of Directors. He brings a wealth of leadership experience spanning finance, corporate strategy and capital raising in the cell and gene therapy space," commented Amit Munshi, Chief Executive Officer of Orna. "We look forward to leveraging his expertise as we enter our next stage of growth and advance our panCAR™ programs for patients with cancer and autoimmune diseases." Mr. Sinha brings more than 25 years of experience working in executive finance roles in the life sciences industry. He currently serves as Chief Financial Officer and co-founder of ElevateBio, a holding company focused on building cell and gene therapy companies. Mr. Sinha also serves as President, Chief Financial Officer, and Director of AlloVir, an ElevateBio portfolio company. Concurrently, he serves as a member of the board of directors of Verona Pharma. Prior to joining ElevateBio and AlloVir, he was Chief Financial Officer of Alexion Pharmaceuticals. Before that, Mr. Sinha held various positions with Bayer across the world including Chief Financial Officer, Bayer North America and Chief Financial Officer, Bayer Yakuhin, Japan. Mr. Sinha holds an MBA from the Asian Institute of Management. He is a qualified Chartered Accountant from the Institute of Chartered Accountants of India and a Certified Public Accountant in the U.S. "I am excited to join Orna's Board of Directors and have been impressed by the breadth and depth of the Company's platforms and pipeline, which hold the potential to overcome existing limitations of cell therapies to deliver outsized impact to patients living with cancer and autoimmune diseases," said Mr. Sinha. "I look forward to working with the Orna Board and leadership to advance its lead panCAR programs towards the clinic and position the organization for future growth." 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: and follow Orna Therapeutics on X and LinkedIn. Investor Contact: Precision AQ Alex Lobo [email protected] SOURCE Orna Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

— 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, MA, USA I January 7, 2025 I 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 . SOURCE: Orna Therapeutics

The companies will focus on next-generation gene therapies for sickle cell disease and transfusion-dependent beta thalassemia. Credit: da-kuk via Getty Images. Vertex Pharmaceuticals and Orna Therapeutics have announced a three-year collaboration to develop next-generation gene therapies for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The partnership will use Orna’s lipid nanoparticle delivery technology to improve the efficiency of gene editing, potentially addressing limitations in existing therapies. Vertex will pay Orna $65m upfront, partly through a convertible note, and Orna is eligible for up to $635m in milestone payments for SCD and TDT products. Additionally, Orna could receive up to $365m per product for up to ten additional indications, alongside royalties on future sales. Vertex already holds a significant position in the gene therapy landscape. In late 2023, it secured US Food and Drug Administration (FDA) approval for its CRISPR-based therapy Casgevy (exa-cel) for both SCD and TDT, marking a milestone as the first approved gene therapy for these indications. Shortly after, bluebird bio received approval for its competing therapy, Lyfenia (lovo-cel). Casgevy involves extracting a patient’s stem cells, editing them in a laboratory using CRISPR technology to correct genetic mutations, and re-infusing the modified cells back into the patient. The groundbreaking yet resource-intensive process requires months to complete and necessitates chemotherapy preconditioning, which can lead to severe side effects. Vertex is actively exploring ways to improve on Casgevy. In July 2024, it invested $15m in South Korea’s Orum to acquire technology aimed at making preconditioning more tolerable. However, the goal is to transition to “in vivo” therapies, where genetic editing occurs directly within the body. Achieving this requires effective delivery systems to target stem cells, a challenge that Orna’s lipid nanoparticle technology could address. “Vertex is a leader in delivering next-generation approaches to treating haemoglobinopathies, and we are excited to collaborate with them to develop in vivo therapies that leverage our proprietary technologies to achieve unprecedented delivery to haematopoietic stem cells,” said Orna’s CEO Amit Munshi in the 7 January announcement. Acquiring ReNAgade Therapeutics last year, Orna Therapeutics has been advancing its platform through significant partnerships and funding. In 2022, it raised $221m in a Series B round and partnered with MSD on its circular RNA (oRNA) platform, a technology aimed at improving the performance of mRNA-based therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy