RNA Therapeutics Receives Feedback from FDA for the First-in-class mRNA-based Delivery of Therapeutic Protein Promising Affordable Biological Drugs

RNA Therapeutics, a clinical-stage biotechnology company focused on innovative applications of mRNA-based delivery systems for therapeutic proteins and vaccines, announced that it has received a written response to its Pre-Investigational New Drug Application (pre-IND) questions submitted to the U.S. Food and Drug Administration (FDA) regarding the development of its lead asset, RNAT-89 (BLA-761423), an mRNA LNP formulation to express darbepoetin, as the first such product presented to the FDA to seek advice on the naming of the product, nonclinical and clinical testing and comparison with Aranesp (Amgen) indicated for the treatment of anemia due to chronic kidney disease (CKD) and non-myeloid malignancies where anemia is due to the effect of concomitant myelosuppressive chemotherapy. There is no biosimilar to Aranesp in the US. The global market for erythropoietin (EPO) drugs, including darbepoetin, was valued at $9,734.1 million in 2022 and is projected to reach $16,926.21 million by 2032, with a compound annual growth rate (CAGR) of 5.7%. The current treatment cost with darbepoetin is about $1,000, which will be reduced to less than $50, making it affordable globally.

FDA Reviews mRNA Protein Delivery pre-IND

The pre-IND meeting with the FDA clarified several issues, the first being how the product would be named. There was no history of naming such a product, and the FDA agreed with RNA Therapeutics’ suggestion to call it “Darbepoetin mRNA (xxxx) Injection.” The FDA has not approved any therapeutic protein product delivered by mRNA technology, so this naming plan is significant information. It should be noted that this naming will apply only if the mRNA expresses the same protein, not its scFV or conjugated form for the mRNA therapy developers. The FDA also agreed with RNA Therapeutics composition, release specification, and nonclinical and clinical testing protocols substantially reduced compared to a new BLA, even though RNAT-89 is not considered a biosimilar.

“The completion of the pre-IND discussion with the FDA is a significant milestone for RNA Therapeutics, and we believe it validates our development strategy for RNAT-89,” said Dr. Sarfaraz K. Niazi, CEO of RNA Therapeutics. “This the first mRNA-based therapeutic protein filed with the FDA, and given its low cost of goods, we will be able to sell our product at a fraction of the cost of any product manufactured by the traditional recombinant process,” said Dr. Matthias Magoola, Chairman of RNA Therapeutics. “This is also a revolution for biological drugs that we project will be delivered through the body’s bioreactor, the ribosomes, making them safer and more effective, and at a minute fraction of the cost of biological drugs that remain unaffordable to 80% of patients worldwide; all this will go away once RNA Therapeutics demonstrates this design that we hope is followed by many,” added Dr. Magoola.

About RNA Therapeutics

RNA Therapeutics is a clinical-stage biotechnology company developing novel treatments and preventions using mRNA-based delivery of therapeutic proteins and vaccines. Its pipeline includes vaccines against Alzheimer’s disease, diabetes type I, HIV vaccine against active and latent viruses, antibody-transferrin conjugates for neurodegenerative disorders, and cancer treatment. RNA Therapeutics has over 100 US Patents on its technology. RNA Therapeutics is fully funded to take all its products to the IND approval stage when it licenses out to major pharmaceutical companies.

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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