Eli Lilly follows up on MiNA pact with second RNA collaboration worth over $ 1 billion, this time with ProQR

Eli Lilly is doubling down on RNA research by making another major biobucks pact with small but promising biotechnology in the gene silencing space.

In May, Lilly partnered with MiNA Therapeutics to operate their small RNA activator (saRNA) technology platform for up to five targets, which were kept under wraps and would be chosen by Lilly.

Big Pharma only paid MiNA $ 25 million up front, but adds development and sales-based biobucks up to $ 245 million per target, bringing the total to $ 1.25 billion dollars if everything goes as planned.

He uses the same playbook today for ProQR, as Lilly signed an initial $ 50 million pact with $ 1.25 billion in biobucks for five targets using his RNA platform, dubbed Axiomer. While remaining silent on details, biotech CEO Daniel de Boer told Fierce Biotech that the deal focused on “genetic disorders of the liver and nervous system.”

RNA is finding its way into a lot of R&D work these days, whether it’s the messenger RNA that the whole world is now familiar with from Moderna and BioNTech’s COVID vaccines or MiNA’s saRNA.

RELATED: Lilly invests in MiNA to fund protein upregulation RNA assays

But even in normal areas of RNA technology, there are differences. Compared to other approaches focused on upregulating certain proteins (like MiNA does) by activating messenger RNA transcription, ProQR’s Axiomer technology is based on RNA base editing, typing on editing oligonucleotides (EON) to attract the ADAR editing machinery already present in human cells, which allows the body to potentially repair mutations in its own RNA and treat genetic diseases.

“This approach has the potential to reverse over 20,000 G to A mutations that are known to cause human disease,” de Boer explained. “A patient’s DNA remains unchanged and Axiomer technology avoids the use of complex delivery vectors, has a reversible mode of action and only works where the target RNA is expressed. This is the technology Lilly pays to get involved with.

ProQR has been somewhat under the radar at the news level for about a year, but is working in-house on its RNA therapies for genetic eye diseases, including two pivotal programs for Leber’s congenital amaurosis (LCA ), the most common genetic disease. cause of childhood blindness and Usher syndrome, the leading cause of deafness and blindness combined.

In March, Netherlands-based biotechnology published the results of a Phase 1/2 trial in Usher syndrome and retinitis pigmentosa that showed some benefit over several measures of vision.

Despite the pandemic, de Boer said his company was still able to enroll the pivotal phase 2/3 trial for the main sepofarsen stroke program in early 2021 “and is on track for data at during the first semester of 2022 “.

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