Business: The billion-dollar biotech

  title={Business: The billion-dollar biotech},
  author={Elie S. Dolgin},
Moderna Therapeutics has big ambitions and a bankroll to match. How a fledgling start-up became one of the most highly valued private drug firms ever. 
Nanotechnology-Based Weapons: A Potential Approach for COVID-19
The present review particularly emphasizes the perception of several nanoformulation-based approaches as an appropriate means to safeguard mankind against COVID-19.
Introduction to RNA Vaccines.
In this chapter, key developments in RNA vaccines are reviewed and the contents of this volume of Methods in Molecular Biology are outlined.
Alternative strategies in cardiac preclinical research and new clinical trial formats
This work examines recent gold standard randomized clinical trials and presents possible modifications to increase lead candidate throughput: adaptive designs, master protocols and drug repurposing.
Recent Progress in Nanotechnology for COVID-19 Prevention, Diagnostics and Treatment
This review article comprehensively discusses the use of nanotechnology for COVID-19 based on three main categories: prevention, diagnostics and treatment and critically review the benefits of nanomaterials along with their applications in personal protective equipment, vaccine development, diagnostic device fabrication and therapeutic approaches.
Toward Nanotechnology-Enabled Approaches against the COVID-19 Pandemic
Nanoimmunity by design can help to design materials for immune modulation, either stimulating or suppressing the immune response, which would find applications in the context of vaccine development for SARS-CoV-2 or in counteracting the cytokine storm, respectively.
mRNA-Based Genetic Reprogramming.
ER Intrabodies: Potent Molecules for Specific Knockdown of Proteins Passingthe ER
A very promising protein knockdown technique is based on recombinant antibody fragments expressed inside the ER (ER intrabodies). ER intrabodies mediate inhibition of the function of proteins passing
Single domain antibodies for the knockdown of cytosolic and nuclear proteins
  • T. Böldicke
  • Biology, Chemistry
    Protein science : a publication of the Protein Society
  • 2017
The development of new endosomal escape protein domains and cell‐penetrating peptides for efficient transfection broaden the application of inhibiting sdAbs, and the generation of relatively new cell‐specific nanoparticles such as polymersomes and polyplexes carrying cytosolic/nuclear sdAb‐DNA or –protein will pave the way to apply cytosol/ nuclear sdAbs for inhibition of viral infection and cancer in the clinic.
ER-targeted Intrabodies Mediating Specific In Vivo Knockdown of Transitory Proteins in Comparison to RNAi
Specificity and off-target effects (OTE) of these molecules as well as the therapeutic potential of ER intrabodies and RNAi have been compared.
Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia
Successful usage of RNA transcript therapy (RTT) is demonstrated as an exogenous human FXN supplementation strategy in vitro and in vivo, specifically to dorsal root ganglia (DRG), providing the first demonstration that RTT can be used for the delivery of therapeutic mRNA to DRG.


Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability.
It is found that mRNAs containing pseudouridines have a higher translational capacity than unmodified m RNAs when tested in mammalian cells and lysates or administered intravenously into mice at 0.015-0.15 mg/kg doses.
Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin
Results demonstrate that HPLC-purified, pseudouridine-containing mRNAs encoding therapeutic proteins have great potential for clinical applications and are evaluated using erythropoietin (EPO)-encoding mRNA complexed with TransIT-mRNA.
Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction
It is shown that intramyocardial injection of synthetic modified RNA (modRNA) encoding human vascular endothelial growth factor-A (VEGF-A) results in the expansion and directed differentiation of endogenous heart progenitors in a mouse myocardial infarction model.
Direct gene transfer into mouse muscle in vivo.
RNA and DNA expression vectors containing genes for chloramphenicol acetyltransferase, luciferase, and beta-galactosidase were separately injected into mouse skeletal muscle in vivo and expression was comparable to that obtained from fibroblasts transfected in vitro under optimal conditions.
Reversal of diabetes insipidus in Brattleboro rats: intrahypothalamic injection of vasopressin mRNA.
In Brattleboro rats, injection into the hypothalamus of purified mRNAs from normal rat hypothalami or of synthetic copies of the vasopressin mRNA leads to selective uptake, retrograde transport, and expression of vasoppressin exclusively in the magnocellular neurons.