MiNA Therapeutics Demonstrates First-Ever In Vivo Delivery of RNAa Therapy to Erythroid Progenitors, Paving the Way for Functional Cure in Beta-Hemoglobinopathies

Jan 29, 2024 07:29 CST Updated 07:29

MiNA Therapeutics

Innovative Drug Developer for Cancer and Liver Disease Treatment

January 29, 2024

eMedClub News

Recently, MiNA Therapeutics, a pioneer in small activating RNA (RNAa, saRNA), announced the latest preclinical in vivo study data for its RNAa product targeting fetal hemoglobin (HbF). For the first time, it was demonstrated that RNAa therapy can be efficiently delivered to erythroid progenitor cells in the bone marrow, offering an effective treatment for β-hemoglobinopathies. The field of hematopoietic stem cells is about to witness a new branch and competition in gene therapy.

MiNA’s HbF product aims to increase the transcription of the γ-globin (HBG) gene, enabling patients with β-hemoglobinopathies to produce higher levels of HbF as a compensatory form of hemoglobin, with the potential to achieve functional cures for severe inherited blood disorders such as sickle cell disease and β-thalassemia.

In non-human primates, MiNA encapsulates the RNAa complex within liposomes called NOV340, which can be delivered to the bone marrow via intravenous injection, achieving over 60% transfection efficiency in erythroid colony-forming units (CFU-E) and pro-erythroblasts (Pro-E). An equivalent level of delivery efficiency was also observed in peripheral blood mononuclear cells.

The 4th International Symposium on Gene Editing and Gene Therapy Technologies

Discovery of Novel AAV Vectors and Capsid Engineering
Enhancing Non-Liver Tissue-Specific Targeting Strategies
Process Development and Clinical Application of Dual AAV Vectors
The Innovative Path to Large-Scale Production of AAV Vectors
Advances in Gene Therapy for Neurodegenerative Diseases
Delivery Strategies for In Vivo Gene Editing Therapy
Differentiated Innovation in Ophthalmic Gene Therapy
Gene Therapy from Genetic Diseases to Metabolic Disorders
Innovation in Gene Therapy Mediated by Novel Vectors
AAV Vectors for Cancer Immunotherapy
Efficient Targeted Insertion or Replacement of Large DNA Fragments
Underlying Innovation and Safety of Gene Editing Tools
The Application Prospects of Epigenetic Technology

999 piecesFree single-session ticket,

Limited Time Offer!

Collaboration, Speaking Engagements, and Synchronous Recruitment Ongoing

The liposome delivery technology NOV340 can effectively deliver RNAa therapy in vivo without the need for harmful pre-treatment or complex cellular engineering. Previously, the pharmacodynamic activity of RNAa therapy delivered via NOV340 has been confirmed in clinical studies, including those for the first RNAa product.

Current Status and Development Trends of Hematopoietic Stem Cell Gene Therapy

eMedClub

As the world's first CRISPR/Cas9 gene editing therapy, Casgevy, has been successively approved by the U.S. FDA for two indications, hematopoietic stem cell gene editing therapy has also attracted more attention.

As early as 2016, the world's first hematopoietic stem cell gene therapy, Strimvelis, was launched in Europe. In the following years, bluebird bio’s Zynteglo and Skysona, as well as Orchard’s Libmeldy, were successively marketed. The most approved indication is thalassemia. Currently, the standard treatment options for thalassemia in clinical practice are regular blood transfusions or hematopoietic stem cell transplantation, which, although effective, also bring significant side effects and long-term treatment requirements that greatly trouble patients. Gene therapy, on the other hand, can better avoid these drawbacks and is expected to become a primary treatment method for this disease in the future, with enormous market potential.

Facing mature technologies with products already on the market, challenges in CMC processes and production costs are gradually being overcome for commercialization. Gene editing tools continue to iterate and optimize, deepening their advantages in precision, simplicity, and efficiency. In this context, RNAa therapy, which has only recently entered the scene, seems somewhat behind. However, RNAa does not alter the genome but instead utilizes inherent gene activation mechanisms to restore or promote normal gene function on a small scale, thereby influencing protein regulation pathways within cells—a mechanism that sets it apart in the highly competitive field of gene therapy. After obtaining the foundational patent license for saRNA in 2014, MiNA Therapeutics initiated the world's first clinical study of an saRNA drug, MTL-CEBPA, in 2016. Current research results targeting advanced liver cancer have been particularly remarkable. The recent announcement of MiNA’s preclinical study results undoubtedly signals that the field of hematopoietic stem cell gene therapy is about to experience the impact of a new treatment, ushering in another round of technological optimization and acceleration.

Reference: Corporate official website

Statement and Copyright Notice

Disclaimer: This article aims to convey industry development information and explore the frontier progress of biomedicine. The content of the article represents the author's viewpoint, and does not represent the position of EBC Pharma, nor does it constitute any value judgment, investment advice, or medical guidance. If necessary, please consult a professional for investment or visit a regular hospital for medical advice.

Copyright Statement:This article is from the content team of Yima Guest. Individuals are welcome to share it on their social media platforms, but unauthorized reproduction by media or institutions in any form to other platforms is prohibited. For reprint authorization, please leave a message at the bottom of the article.

DianDian "Share”、“Like" and "In view", charge me up a bit~"