Our RanTran Team and Research
Dr. Laura Ranum
Dr. Laura Ranum, Director of the Center for NeuroGenetics at the University of Florida, had just received her Ph.D. when her mother-in-law died of ALS in 1989. In April 2016, when her husband, Bert Ranum, was diagnosed with ALS, she had over 25 years of experience studying neurodegenerative diseases.
During that time, Laura made a paradigm-shifting discovery: the textbook rules of protein translation did not apply for repeat expansion mutations, or mutations in which small segments of the genetic code are repeated too many times. At the time, she was working on a rare disease called spinocerebellar ataxia type 8 or SCA8. She first discovered that instead of making only one RNA the repeat expansion mutation made two RNAs. Next she discovered that the protein production machinery ran amok, and instead of one RNA making one protein together these RNAs were translated into six proteins without the canonical start signal previously thought to be required to produce proteins. She called this unexpected type of protein production repeat associated non-ATG (RAN) translation.
Her scientific career and her personal life converged when the gene that causes ALS in Bert’s family, C9orf72, was also found to be a repeat expansion mutation that undergoes RAN translation. Additional research in Laura’s lab and other labs has demonstrated that RAN proteins are present in at least nine other repeat expansion diseases including frontotemporal dementia (FTD), myotonic dystrophy, and Huntington’s disease.
Although there are no effective therapies for these disorders on the market, the Ranum lab is working to understand the biological mechanisms that make repeat expansion mutations toxic and this research has opened the door to the development of therapies for C9orf72 ALS/FTD and other repeat expansion disorders. The Ranum lab has shown promising results in testing these therapies in C9orf72 BAC transgenic mice.
Bert Ranum is a corporate and securities lawyer who has advised life science companies from start-up to large public companies for 35 years. He knows the power of capital markets to provide the resources necessary to accelerate development and commercialization of preclinical drugs. So, Bert and Laura founded RanTran to take the therapeutic strategies developed in Laura’s lab out of the University and into the commercial world to fight these devastating neurodegenerative diseases. They are determined to make a difference for Bert and others who suffer from repeat expansion diseases.
RanTran is developing drugs to block RAN translation and also strategies to target RAN proteins. The Ranum Lab has shown that targeting RAN proteins using an immunotherapy approach improves behavior and survival and decreases motor neuron loss in C9orf72 BAC mice. These preclinical data provide strong evidence that RAN proteins drive disease in C9orf72 ALS/FTD.