Pseudohypoparathyroidisms

Jonathan Weiss/Shutterstock ProQR Therapeutics is collaborating with Eli Lilly to bolster the former's Axiomer RNA editing platform and create new medicines that could treat a range of genetic disorders. The partnership combines the companies' resources to focus on Axiomer, a technology that can edit single nucleotides present in RNA in a specific and highly targeted manner. Editing oligonucleotides, also called EONs, enables scientists to recruit endogenous adenosine deaminases acting on RNA (ADAR) enzymes to specific adenosine in RNA associated with certain diseases. ADAR can then convert the target adenosine (A) into inosine (I), which is equivalent to an A to G change, as I in RNA is interpreted as guanosine (G). By doing so, scientists might be able to reverse the over 20,000 G to A mutations in humans, many of which are known to cause different diseases, including McCune-Albright syndrome, Albright hereditary osteodystrophy, and sporadic endocrine tumors. ProQR's Axiomer RNA base editing platform can target many diseases by using the cell's editing capacity to make specific single nucleotide edits in RNA to reverse a mutation. While further study is still needed to explore this method's scope and possible outcomes fully, the platform represents a significant opportunity for researchers to potentially find cures for specific diseases that were otherwise untreatable. ProQR and Lilly's goal is to develop as many as five drug targets. "RNA editing is an exciting emerging technology, which allows transient, reversible editing, which in some indications may be an extremely attractive therapeutic approach. 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," said Andrew C. Adams, Ph.D. vice president for New Therapeutic Modalities at Lilly, in a press release. According to ProQR founder and chief executive Daniel A. de Boer, the partnership not only strengthens the company's financial standing but, more importantly, expands Axiomer's application potential beyond its core therapeutic focus of developing therapies for genetic eye disease. Now, Axiomer's technology might be the key to finding treatments for patients diagnosed with genetic nervous system and metabolic disorders. In an interview about the merits of the Axiomer platform, ProQR senior vice president for innovation Bar Klein said: "It most certainly offers new possibilities in RNA modulation, both to ProQR and other companies that believe in the potential of RNA technology. Axiomer platform can help push current borders, I dare say that it could change the entire playing field!" As part of the deal, ProQR will receive $50 million from Lilly, consisting of a $20 million payment upfront and $30 million in equity investment in the form of ordinary shares. ProQR is also set to receive $1.5 billion for development, regulatory, and commercialization milestones, plus tiered royalties.