With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.
ProQR announced yesterday that it had landed a global licensing and research collaboration with Eli Lilly and Company “focused on the discovery, development and commercialization of potential new medicines for genetic disorders in the liver and nervous system.”
The $1.5 billion deal will see the companies use ProQR’s proprietary Axiomer RNA editing platform technology to advance editing oligonucleotide-based therapeutics toward clinical development and commercialization. The companies plan to develop up to five targets using the Axiomer platform.
Under the terms of the deal, ProQR will receive $50 million with an upfront payment of $20 million, and will also make an equity investment of $30 million in the company through ordinary shares. ProQR could also receive an additional $1.25 billion if certain development, regulatory and commercialization targets are achieved, as well as tiered royalties of under ten percent on product sales.
“RNA editing is an exciting emerging technology, which allows transient, reversible editing, which in some indications may be an extremely attractive therapeutic approach,” said Andrew C. Adams, PhD, vice president for New Therapeutic Modalities at Lilly, in the press release from ProQR. “Through this collaboration with ProQR, we hope to utilize this technology to unlock novel treatments to improve the lives of patients across a spectrum of diseases.”
ProQR’s Axiomer RNA editing platform involves precision editing of single nucleotides in RNA in a highly targeted and specific manner. The technology is based on editing oligonucleotides, or EONs, which act to recruit endogenous ADAR enzymes (adenosine deaminases acting on RNA) to a target adenosine in a disease-associated RNA. ADAR mediates conversion of the target adenosine (A) into inosine (I).
In RNA, inosine is regarded as guanosine (G), so the change is in essence an A to G change. As such, the technology has the possibility of reversing the more than 20,000 G to A disease-causing mutations in humans.
Lilly’s latest RNA research accord follows a deal it struck in May with MiNA Therapeutics to leverage its small activating RNA (saRNA) technology platform for up to five targets. The targets, which will be chosen by Lilly, remain undisclosed.
ProQR currently has two programs in pivotal trials for Leber congenital amaurosis (LCA), the most common genetic cause of childhood blindness, and Usher syndrome, the leading cause of combined deafness and blindness. In March, the company released results from a Phase I/II trial in Usher syndrome and retinitis pigmentosa that showed some benefit on multiple measures of vision.
RNA is surely the current “it” molecule in genetic research, especially following the success of the COVID-19 mRNA vaccines. And in the last several years, dozens of CRISPR-based biotech startups have flooded the space, including one co-founded by Nobel Laureate and CRISPR inventor Dr. Jennifer Doudna called Mammoth Biosciences. The company announced this week that it had secured $195 million in financing, bringing its valuation to more than $1 billion to land it “unicorn” status.