Cizzle Biotechnology is focusing on the gene Ciz1 and its variant, found in small-cell lung cancer. The trigger for the start-up's formation was the finding that cells require Ciz1 to grow, and that variant forms could be used for selective targeting of cancer cells, especially small-cell lung cancer. The company's founders have shown that blocking the disrupted form of Ciz1, using a small interfering RNA (siRNA) targeted to the variant, prevented cell proliferation and growth in vitro. The specificity of the Ciz1 variant also gives it potential as a biomarker for early detection of lung cancer.

While not identifying specific situations raising potential questions, the FDA commissioner uses his appearance at agency's annual rare disease event to advise regulatory decisions may be revisited as more data becomes available. 

NGM’s Phase II data for FGF19 analog aldafermin shows ability to reduce liver fat and improve fibrosis, but also raises LDL-C. Company says it gets a broader effect this way and can manage the increased cholesterol.