Nonsense mutations have been identified in over 1,800 rare and ultra-rare diseases. ELX-02 is in clinical development for systemic administration for cystic fibrosis. There are over 300 IRDs associated with nonsense mutations. Cystinosin is a ubiquitous cystine-selective transport channel in the lysosomal membrane. There have been few randomized trials of PCD treatment to date. Based on a publication from Prior, et al. ( Upcoming studies will confirm these findings. Each of these families is described briefly below. Orphan drug designation must be requested before applying for marketing approval. Promotion of indications not covered by the SmPC is specifically prohibited. 28 As of December 31, 2020, 40% of our employees were female and 60% were male. We may never achieve or maintain profitability. &# 8226; We will need substantial additional funding. 31 Risk Factors Investing in our common stock involves a high degree of risk. We have never generated any revenue from product sales and may never be profitable. We will need substantial additional funding. In addition, our product candidates, if approved, may not achieve commercial success. We could be subject to additional tax liabilities. Moreover, the length of the extension could be less than we request. Third parties may challenge our patents.
