Lilly To Try ProQR’s RNA-Editing Technology In Liver, Neurology Indications
ProQR’s Axiomer platform can enable single nucleotide RNA editing, an approach that could address thousands of disease-causing genetic mutations, the company says.
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The big pharma has a gene therapy R&D site in New York from its Prevail Therapeutics buy; the RNA-focused Boston site also will conduct drug delivery research benefitting programs at both locations.
Totals for aggregate deals and in the M&A, alliance and divestment categories were similar among the 10 busiest deal makers from 2020 to 2021, but COVID-19 may have driven a more hectic deal-making pace overall in 2020.
The company is pinning hopes on its Usher syndrome and retinitis pigmentosa programs, but the disconnect between Phase I/II success and Phase II/III failure dented analysts’ enthusiasm.