To accelerate #mRNA vaccine and therapy research, we're excited to share our end-to-end protocol for mRNA design, synthesis and encapsulation in @natprot.nature.com. Protocol development led by @laura-leighton.bsky.social, Natasha Chaudhary and Tim Mercer.@basemrna.bsky.social rdcu.be/eqiF1

The design, manufacture and LNP formulation of mRNA for research use

Nature Protocols - This protocol describes simple, small-scale laboratory production of mRNA for research and preclinical use. This includes sequence design, DNA template production, mRNA synthesis...

rdcu.be

Today, a huge milestone for personalised #mRNA therapeutics for inherited disease! A baby with an ultra-rare mitochondrial metabolic disease was treated at UPenn with an mRNA-encoded gene-corrector. The mRNA was made to treat a single individual in just 7 months! www.nejm.org/doi/full/10....

Systemic mRNA-LNP delivery beyond the liver? Check out our recent paper demonstrating cell-specific mRNA-LNP delivery using PEG- and target cell-specific antibodies. This approach can customised to any target cell-type with off-the-shelf mRNA-LNPs. 1/ sciencedirect.com/science/arti...

Targeted mRNA delivery with bispecific antibodies that tether LNPs to cell surface markers

Efficient delivery of mRNA-lipid nanoparticles (LNPs) to specific cell types remains a major challenge for mRNA therapeutics. Conventional targeting a…

sciencedirect.com

Exited to share our latest preprint! mRNA-LNPs are efficiently delivered to healthy livers, but what about liver tumours? Our postdoc @laura-leighton.bsky.social found liver tumours efficiently take up mRNA, enabling next generation mRNA-LNP cancer therapeutics. www.biorxiv.org/content/10.1...

Developmental biology research leads to fundamental discoveries for biomedical research. RNA Vaccines. Lane et al., 1971 PMID: 5169490. Images by Brandy Walker & Amaya Moss @rkmille . @socdevbio.bsky.social 3/12

Calling on #ONT #Nanopore #sequencing experts: when sequencing through a stretch of 120nt dA using a DNA plasmid kit (outsourced in our case), what should one expect as average length in the output? The loss we are seeing is a bit extreme #RNAsky

#mRNA meets Tardigrades! Delivery of an mRNA encoding a protein from the famously tough animal can protect healthy tissue from damaging radiation. Very creative! #RNAsky www.nature.com/articles/s41...

Radioprotection of healthy tissue via nanoparticle-delivered mRNA encoding for a damage-suppressor protein found in tardigrades - Nature Biomedical Engineering

Radiation-therapy-induced DNA damage in oral and rectal epithelial cells can be reduced via the nanoparticle-mediated local delivery of messenger RNA encoding for a damage-suppressor protein found in ...

nature.com

Not the best weather forecast for Brisbane 😬

Preach!

Interested in undertaking a PhD in the transformative field #mRNA cancer vaccines? I have an earmarked #PhD scholarship available for an outstanding domestic student to join our team at UQ and @basemrna.bsky.social. If this sounds like you, get in touch. study.uq.edu.au/study-option...

New #mRNA cap structures get you more bang (protein) for your buck! Nice new research from @jmcoller.bsky.social in collaboration with Trilink shows the CleanCap M6 analogue inhibits decapping and substantially increases protein expression in vivo. www.cell.com/molecular-th...

CleanCap M6 inhibits decapping of exogenously delivered IVT mRNA

In this manuscript, Mandell and colleagues present CleanCap M6, a next generation 5’ cap analog that outperforms existing industry standards in live mice. Mandell and colleagues found that this cap ex...

cell.com

Wow this is super cool! Single-cell resolution imaging of #mRNA-LNP biodistribution in cleared mouse bodies. www.nature.com/articles/s41...

Nanocarrier imaging at single-cell resolution across entire mouse bodies with deep learning - Nature Biotechnology

An integrated experimental and deep learning pipeline reveals cell-level targeting of nanocarriers in whole bodies.

nature.com

Exciting news! @basemrna.bsky.social and Vaxxas have been awarded the $2M USD Biomedical Advanced Research and Development Authority (BARDA) Patch Forward Concept Stage Prize to develop thermostable needle-free mRNA vaccines. www.uq.edu.au/news/article...

UQ and Vaxxas win prize to advance patch-based mRNA COVID-19 vaccine

UQ’s BASE facility and biotechnology company Vaxxas have been announced as stage winners of a prestigious global prize to accelerate a patch-based mRNA vaccine for COVID-19.

uq.edu.au

#mRNA meets cell therapies! It was great to collaborate with Fernando Guimaraes's team to develop mRNA CARs and delivered them to NK cells. NKs transfected with EphA2 CAR mRNA elicited a strong anti-tumour response in animal sarcoma models. onlinelibrary.wiley.com/doi/10.1002/...

Enhancement of anti‐sarcoma immunity by NK cells engineered with mRNA for expression of a EphA2‐targeted CAR

Human NK cells can be efficiently transfected with mRNA to safely express an EphA2-CAR without genomic alterations. EphA2-targeted CAR-NK cells exhibit enhanced cytotoxicity against paediatric sarco...

onlinelibrary.wiley.com

Out of office

Awesome work from Michael Mitchell's lab at UPenn in @natureportfolio.bsky.social identifies novel LNPs that can deliver #mRNA to the placenta. Using the placenta-targeting LNPs to deliver VEGF mRNA improves pre-eclampsia in animal models. www.nature.com/articles/s41...

Placenta-tropic VEGF mRNA lipid nanoparticles ameliorate murine pre-eclampsia - Nature

A platform for mRNA lipid nanoparticle delivery to the placenta to treat pre-eclampsia is shown to improve fetal and maternal health in mice and has potential clinical applications in obstetric d...

nature.com

Earlier this year I had the opportunity to present a TEDx talk on the transformative potential of personalised #mRNA vaccines for cancer. The recording is out now: www.youtube.com/watch?v=BnSO... #RNAsky #RNAtx @basemrna.bsky.social

How personalized mRNA vaccines could revolutionize cancer treatment | Seth Cheetham | TEDxUQ

YouTube video by TEDx Talks

youtube.com

How I imagine our lab animals talk to each other.

There are more ways to design an mRNA than atoms in the universe, so how do you choose the best one? Our new @basemrna.bsky.social software, mRNArchitect enables you to tune expression, stability, reactogenicity and synthesisability to design more potent #mRNA drugs. www.biorxiv.org/content/10.1...

mRNArchitect: optimized design of mRNA sequences

Summary: mRNA design is the critical first step in the development of a new vaccine or therapy. The mRNA primary sequence is assembled from multiple elements, including the coding sequence of the targ...

biorxiv.org

mRNA-encoded transcription factor inhibitors are a rapidly emerging class of drugs. In Science Translational Medicine a team at Harvard treated retinal disease in animals by inhibiting RUNX1. Stay tuned for our forthcoming work targeting cancer transcription factors. www.science.org/doi/10.1126/...

An mRNA-encoded dominant-negative inhibitor of transcription factor RUNX1 suppresses vitreoretinal disease in experimental models

An mRNA-encoded, dominant-negative inhibitor of RUNX1 exhibits preclinical efficacy in models of ocular diseases.

science.org

I made a starter pack for interesting people in the mRNA vaccines and therapeutics space. Message me if you want to be added. #RNAsky go.bsky.app/VSHfBce

ARC says they’ll release the Discovery Projects 2025 outcomes tomorrow. Recently, outcomes announcements have tended to happen at/around 11am Canberra time. My bot will pick up the announcement in RMS’s database and post immediately. Good luck!

Cool paper on ribozyme-mediated trans-ligation of mRNAs to deliver larger cargo. I love this concept, but the examples seem a little engineered. We can and do make conventional mRNAs up to 10kb and greater without any problems. Interested in what others think. #RNAsky www.science.org/doi/10.1126/...

Ribozyme-activated mRNA trans-ligation enables large gene delivery to treat muscular dystrophies

Ribozymes are small catalytic RNA sequences capable of nucleotide-specific self-cleavage found widespread in nature. Ribozyme cleavage generates distinct 2′,3′-phosphate and 5′-hydroxyl termini that r...

science.org

Noncoding RNA pioneer Howard Chang will be the new Chief Scientific Officer of Amgen. Interesting move. www.fiercebiotech.com/biotech/amge...

Amgen taps Stanford's Howard Chang to take CSO reins from Jay Bradner

In a few weeks, Howard Chang, M.D., Ph.D., will join Amgen, filling both the roles of senior vice president of research and chief scientific officer. | In a few weeks, Howard Chang, M.D., Ph.D., will ...

fiercebiotech.com

We're building Australia's first centre for the designing and manufacturing personalised cancer vaccines at the BASE mRNA facility. On the PhaseIII podcast I discuss the transformative potential of mRNA for cancer and our efforts to bring this tech to patients. podcasts.apple.com/au/podcast/v...

VAXXED: Game on, with BASE and WEHI

Podcast Episode · Phase III · 19/11/2024 · 26m

podcasts.apple.com

Can #mRNA technology rejuvenate aged skin? George Church's lab identified the AP-1 gene ATF3 as a key regulator of skin ageing. Delivering ATF3 mRNA rejuvenated skin cells, boosting collagen production. www.biorxiv.org/content/10.1...

Human skin rejuvenation via mRNA

Aging is characterized by a gradual decline in function, partly due to accumulated molecular damage. Human skin undergoes both chronological aging and environmental degradation, particularly UV-induce...

biorxiv.org

The greatest challenge for #mRNA-LNP therapies is targeting non-liver tissues. To overcome this, we developed customisable bispecific antibodies to tether mRNA-LNPs to cell-surface proteins, achieving efficient and selective mRNA delivery in vivo. Preprint 👇 t.co/0nJj8eVsqC