Publications & Presentations

Where science translates to action

NACFC 2020: Delivering CFTR mRNA: An Inhaled, Mutation-agnostic Approach to Treat CF
Oral Presentation, Richard Wooster, Ph.D., Translate Bio Chief Scientific Officer

NACFC 2020: Design of Next-Generation CFTR mRNA Therapeutics (MRT) for the Treatment of Patients with Cystic Fibrosis (Poster Presentation)

Immunogenicity of Novel MRNA COVID-19 Vaccine MRT5500 in Mice and Non-Human Primates.
Kalnin K., DeRosa F., et al. BioRxiv, 14 Oct. 2020, doi:10.1101/2020.10.14.337535.

NACFC 2019: Safety and Tolerability of a Single Dose of MRT5005, a Nebulized CFTR mRNA Therapeutic, in Adult CF Patients
Oral Presentation, Jonathan B Zuckerman, M.D., Maine Medical Center

NACFC 2019: Safety and Tolerability of a Single Dose of MRT5005, a Nebulized CFTR mRNA Therapeutic, in Adult CF Patients (Poster Presentation)

mRNA Delivery for Therapeutic Anti-HER2 Antibody Expression In Vivo.
Rybakova Y. Kowalski P.S,. Huang Y., Gonzalez J.T., Heartlein M.W., DeRosa F., Delcassian D., Anderson D.G., Mol. Therapy, 2019, 27(8), 1415-1423. doi: 10.1016/j.ymthe.2019.05.012. Epub 2019 May 18.

Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy.
DeRosa F, Smith L, Shen Y, et al. Molecular Therapy. doi: 10.1016/j.ymthe.2019.03.001. Epub: 2019 March 6.

Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium.
Patel AK, Kaczmarek JC, Bose S, et al. Adv Mater. 2018 Jan; e1805116. doi: 10.1002/adma.201805116. Epub 2019 Jan 4.

Optimization of a Degradable Polymer−Lipid Nanoparticle for Potent Systemic Delivery of mRNA to the Lung Endothelium and Immune Cells.
Kaczmarek JC, Kauffman KJ, Fenton OS, et al. Nano Lett. 2018 Oct 10;18(10):6449-6454. doi: 10.1021/acs.nanolett.8b02917. Epub 2018 Sep 20.

Customizable Lipid Nanoparticle Materials for the Delivery of siRNAs and mRNAs.
Fenton OS, Kauffman KJ, McClellan RL, et al. Angew Chem Int Ed Engl. 2018 Oct 8;57(41):13582-13586. doi: 10.1002/anie.201809056. Epub 2018 Sep 14.

Synthesis and Biological Evaluation of Ionizable Lipid Materials for the In Vivo Delivery of Messenger RNA to B Lymphocytes.
Fenton OS, Kauffman KJ, Kaczmarek JC, et al. Adv Mater. 2017 Sep;29(33).doi: 10.1002/adma.201606944. Epub 2017 Jul 6.

Polymer-lipid nanoparticles for systemic delivery of mRNA to the lungs.
Kaczmarek JC, Patel AK, Kauffman KJ, et al. Angew Chem Int Ed Engl. 2016 Oct 24;55(44):13808-13812. doi: 10.1002/anie.201608450. Epub 2016 Sep 30.

Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo.
Kauffman KJ, Mir FF, Jhunjhunwala S, et al. Biomaterials. 2016 Dec;109:78-87. doi: 10.1016/j.biomaterials.2016.09.006. Epub 2016 Sep 25.

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system.
DeRosa F, Guild B, Karve S, et al. Gene Ther. 2016 Oct;23(10):699-707. doi: 10.1038/gt.2016.46. Epub 2016 Jun 30.

Bioinspired alkenyl amino alcohol ionizable lipid materials for highly potent in vivo mRNA delivery.
Fenton OS, Kauffman KJ, McClellan RL, et al. Adv Mater. 2016 Apr 20;28(15):2939-2943. doi: 10.1002/adma.201505822. Epub 2016 Feb 18.

Poly(glycoamidoamine) brushes formulated nanomaterials for systemic siRNA and mRNA delivery in vivo.
Dong Y, Dorkin JR, Wang W, et al. Nano Lett. 2016 Feb 10;16(2):842-848. doi: 10.1021/acs.nanolett.5b02428. Epub 2016 Jan 13.

Optimization of lipid nanoparticle formulations for mRNA delivery in vivo with fractional factorial and definitive screening designs.
Kauffman KJ, Dorkin JR, Yang JH, et al. Nano Lett. 2015 Nov 11;15(11):7300-7306. doi: 10.1021/acs.nanolett.5b02497. Epub 2015 Oct 20.

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