Company’s Lipid Nanoparticle (LNP) formulation successfully delivered nebulized mRNA to target cells and demonstrated translation of DNAI1 protein essential to ciliary movement in preclinical models
Newly-made DNAI1 protein detected in ciliated cells, club cells, and basal (stem) cells in human bronchial epithelial (hBE) cells and NHPs
LNP-formulated DNAI1 mRNA rescued ciliary function in cell-based PCD models and cells derived from knock-out mice
Menlo Park, Calif. and Dallas, Texas – August 5, 2021 – ReCode Therapeutics (the “Company”), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today presented encouraging preclinical data from the Company’s inhaled mRNA-based molecular therapy program for the treatment of primary ciliary dyskinesia (PCD) during an oral and poster session at the 2021 PCD on the Move Virtual Scientific Conference.
“The data featured today demonstrate that delivery of our LNP-formulated mRNA leads to production of DNAI1 protein in the target cells and rescue of ciliary function. These results further demonstrate the potential of our novel mRNA approach as a disease-modifying therapy for primary ciliary dyskinesia, a rare lung disease that drastically affects length and quality of life,” said David Lockhart, Ph.D., CEO & President, ReCode Therapeutics. “These data underscore the power of our LNP platform to deliver mRNA-based therapies to treat PCD and are an important step in advancing our program toward human clinical studies.”
ReCode Therapeutics is developing an mRNA-based therapy for the treatment of PCD caused by mutations in DNAI1. The DNAI1 mRNA is formulated in a proprietary lipid nanoparticle (LNP), nebulized and delivered as an aerosol directly into the airway. In vivo data demonstrated successful delivery of the formulated mRNA and showed that it was well-distributed in the lungs of non-human primates (NHPs) after a single, low dose administration. Newly-made DNAI1 protein was detected in ciliated cells, as well as club and basal (stem) cells, which are precursors of ciliated cells, in human bronchial epithelial (hBE) cells and in NHPs.
The ex vivo data presented for the LNP-formulated DNAI1 mRNA confirmed that newly translated DNAI1 protein was incorporated throughout the ciliary axoneme of human ciliated cells. In this model, the incorporated DNAI1 protein could be detected for two weeks following treatment. Further, the mRNA rescued ciliary function in cell-based PCD models and in the presence of mucus. In cells derived from PCD knock-out mice, the delivered mRNA rescued ciliary function and the activity persisted for weeks following the final dose.
Details on ReCode’s Oral and Poster Presentations at the 2021 PCD on the Move Scientific Conference
Oral Presentation Session: PCD Scientific Reports
Presenter: Mirko Hennig, Ph.D., Principal Scientist, ReCode Therapeutics
Date & Time: Thursday, August 5, 2021 at 11:45 – 11:50 am ET
Poster Title: mRNA-based Therapies for Primary Ciliary Dyskinesia
This presentation and the virtual poster summarizing the findings are now available to registered conference attendees on the PCD Foundation’s website.
About Primary Ciliary Dyskinesia (PCD)
Primary ciliary dyskinesia (PCD) is a disease characterized by deficient mucociliary clearance (MCC), chronic respiratory tract infections and premature death. This rare disease is caused by mutations in more than 40 different genes that result in dysfunctional cilia and loss of MCC. PCD is a life-limiting disease with no disease-modifying treatments available to patients.
About ReCode Therapeutics
ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using its powerful LNP delivery technology to target organs and tissues beyond the liver. The Company’s pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia. The Company is leveraging its proprietary LNP platform and nucleic acid technologies and utilizing systemic and direct delivery for mRNA-mediated replacement and gene editing/correction in target cells, including stem cells. For more information, visit www.recodetx.com and follow us on Twitter @ReCodeTx and LinkedIn.
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