Week's news analysis

Messenger RNA technology:
who is in the race?

by the editor (with Reuters) - 25 March 2021

Moderna Inc, Pfizer and BioNtech are not the only companies to use messenger RNA (mRNA) in their vaccines projects. Several drug manufacturers are also working hard on this technology to develop treatments and vaccines for various diseases.


Arcturus Therapeutics Inc is developing vaccines for flu and Covid-19, which is in early human testing. It is also testing therapies for cystic fibrosis and heart disease. In December 2020, it has selected ARCT-032, an aerosolized LUNAR® mRNA-based therapeutic candidate, as a development candidate for Cystic Fibrosis (CF), a progressive hereditary disease. According to the company, "ARCT-032 has the potential to address the root cause of CF lung disease, which is caused by the defective, or missing, CFTR protein". Last year, CureVac returned the rights to one drug it was co-developing with the company, but can work with Arcturus on future therapies, under a collaboration deal.

Germany's BioNTech is developing mRNA-based therapies for several types of cancers and vaccines for flu. In September 2019, it penned a collaboration with the Bill & Melinda Gates Foundation to develop HIV and tuberculosis programs. The partnership included a preclinical vaccine and immunotherapy candidates to prevent HIV and tuberculosis infection. Total funding under the collaboration could reach $100 million through potential future grant funding from the Gates Foundation in order to underwrite the evaluation of these candidates in the clinic, and support the initiation of new infectious disease projects.

On February 3rd, 2021, GlaxoSmithKline and CureVac announced a new €150m collaboration to develop next generation mRNA vaccines for Covid-19, with the potential for a multi-valent approach to address multiple emerging variants in one vaccine. In details, GSK and CureVac will contribute resources and expertise to research, develop, and manufacture a number of novel mRNA vaccine candidates, including multi-valent and monovalent approaches. The aim is to offer broader protection against a variety of different SARS-CoV2 variants, and to enable a quick response to new variants potentially emerging in the future. The development program targets to introduce the vaccine in 2022, subject to regulatory approval.

German startup Ethris is developing therapies for severe pulmonary diseases. In 2017, the company negotiated a five-year strategic research collaboration with AstraZeneca on developing stabilized, nonimmunogenic modified RNA therapies for respiratory diseases. The partnership exploits Ethris' proprietary Stabilized Non-Immunogenic mRNA (SNIM®RNA) technology, which engineers chemical modifications in RNA molecules to overcome their innate instability and immunogenicity. The partners work exclusively to develop multiple targets for evaluation against asthma, chronic obstructive pulmonary disease, and idiopathic pulmonary fibrosis. Under the agreement, Ethris received $29.5 million upfront payment plus research funding. The firm was also eligible to earn potential R&D milestones and sales-related royalties.

In April 2020, Ethris GmbH and Neurimmune AG announced an exclusive partnership to develop an mRNA-based anti-SARS-CoV-2 antibody therapeutic as an inhalable Covid-19 treatment. The collaboration brought together Neurimmune's leadership in developing antibodies based on genetic information sourced directly from patients' immune cells with Ethris' mRNA platform that is able to deliver a therapeutic mRNA to the respiratory tract via inhalation. Under the deal, the companies planned to conduct research and development activities while sharing costs and revenues resulting from the collaboration.

On February 1st, 2021, Gritstone Oncology Inc has partnered with Gilead Sciences Inc to develop a mRNA based vaccine for the treatment of HIV. Gilead will use Gritstone's vaccine-like technology, which combines an adenovirus vector with self-amplifying messenger RNA, to target HIV-specific "antigens," or target proteins, that it's developing. The idea is to train the immune system to destroy HIV-infected cells. Gilead will pay Gritstone $30 million upfront and invest another $30 million in the smaller company's stock. Gritstone could receive as much as $725 million more, should Gilead exercise an option to take the treatment forward following early clinical tests, and if other, unspecified milestones are met. Gritstone has used its technology to develop cancer immunotherapies, two of which are now in clinical testing. The company's technology can also address infectious diseases. Meanwhile, it is the third partnership in HIV that Gilead has struck in the past three years, giving the California-based company at least five curative HIV therapies in development.

Kernel Biologics
Privately-held start-up Kernel is developing mRNA-based immunotherapies for cancer. It has partnered with Boeing and the International Space Station U.S. National Laboratory to launch a study of leukemia therapies to the ISS this year.

In addition to its Covid-19 vaccine, Moderna has signed partnership deals with AstraZeneca Plc, Vertex Pharmaceuticals Inc and Merck & Co to develop mRNA therapeutics.

As an exemple, in July 2016, Vertex Pharmaceuticals and Moderna entered into an exclusive research collaboration and licensing agreement aimed at the discovery and development of RNA messenger therapeutics for the treatment of cystic fibrosis (CF). The three-year collaboration will focus on the use of mRNA therapies to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with CF.

A second agreement was announced in September 2020 between the companies, aimed at the discovery and development of lipid nanoparticles (LNPs) and mRNAs for the delivery of gene-editing therapies for the treatment of cystic fibrosis (CF). The three-year research collaboration will focus on the discovery and optimization of novel LNPs and mRNAs that can deliver gene-editing therapies to cells in the lungs, enabling functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to be produced. Moderna will receive $ 75 million upfront and will be eligible to receive up to $380 million in development, regulatory, and commercial milestones, plus tiered royalties from any products that result from the collaboration.

Translate Bio-Sanofi
Translate Bio Inc's lead drug is a cystic fibrosis treatment being tested in early-to-mid stage clinical trials. It is also developing mRNA vaccines for infectious diseases, including Covid-19 and flu, along with Sanofi SA. On March 12, 2021, the companies announced the start of the Phase 1/2 clinical trial for MRT5500, an mRNA vaccine candidate against SARS-CoV-2. They expect interim results from this trial in the third quarter of 2021.

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