S1, E6: Alltrna and tRNA therapeutics for rare genetic diseases with CEO Michelle Werner and rare diseases expert Dr David Weinstein

In this episode of Raising Biotech, Surani explores the untapped world of tRNA therapeutics with Flagship Pioneering-founded company Alltrna. The company raised $109 million in a series B in August 2023. Surani talks to CEO Michelle Werner about the inception of Alltrna, the company's tRNA therapeutic thesis and her journey to the role. She also talks about the company's plans to study its technology in rare genetic liver diseases and the potential to accelerate clinical trials with the use of basket trials. Dr David Weinstein, a rare diseases pediatrician and owner of Weinstein Rare Disease and Clinical Development Consulting also joins the podcast to give his take on the potential of tRNA therapeutics to serve thousands of diseases. He gives his thoughts on the company's mission, potential clinical trial challenges ahead and how the therapeutics might fit into the treatment paradigm. Timestamps: 01:58 - Background of Alltrna and tRNA therapeutics for rare genetic diseases 06:00 - Company formation under Flagship and Michelle's personal backstory 11:35 - Focussing on stop-codon diseases of the liver for first clinical trials 12:55 - Dr Weinstein talks about tRNA therapies for rare genetic diseases 16:55 - The use of basket trials to accelerate clinical trials 18:30 - Efficacy and safety considerations & regulatory pathway 21:45 - How tRNA therapies would fit into the treatment paradigm 24:05 - Potential for tRNA in other genetic diseases beyond the liver  26:00 - Alltrna's future pipeline and business strategy  For any comments, questions, feedback or suggestions you can connect directly with Surani Fernando on LinkedIn or email: [email protected] Music composed by: Yrii Semchyshyn (Coma Media) Hosted on Acast. See acast.com/privacy for more information.