At the beginning of June, I was invited to tag along with Jen Farmer and a couple other representatives from FARA and the FA community as they toured two research facilities in North Carolina. Both of these labs were looking at how they could benefit FA patients, but in different ways. Heads up, I dropped a major in college because it required a science class! So I’ll dumb it down and let Jen fill in my gaps!
Tuesday, June 7: Bamboo Therapeutics in Chapel Hill, NC
Mary: This place is 5 minutes from me and I didn’t know it! Bamboo is working on gene therapy for FA, Duchenne, and a couple other conditions. They are creating viruses to deliver a correct copy of the FA gene to the cells most important in FA - nervous system and heart.They are expanding their lab so they can do this in a higher capacity. The people there were awesome! They asked us questions about our priorities for treatment so that they can work to address the real concerns in the FA community. They also asked how we feel about risk, and I said no one with FA is scared of big needles and a little blood! I’m going to keep up with this place, so I’ll report back with any cool developments
Jen: Dr. Jude Samulski is a founder of Bamboo Therapeutics and a leader in the gene therapy field because of his expertise in discovering and optimizing viruses that can be used for gene therapy. Dr. Samulski explained to us how they are optimizing the AAV virus to enhance the specificity of AAV to target organs and tissues, as well as improve the efficiency of transduction of potential therapeutic genes (frataxin in FA) into host cells. We were also given an amazing tour of their new manufacturing facility and got an upclose look at how they are planning to scale up their production with new bioreactors. As Mary mentioned we also talked with the team about symptoms of FA that are important to treat (balance, coordination, speech, vision, hearing, fatigue and cardiac disease) and what types of procedures people might be able to tolerate related to delivery of a therapy. As this was a first meeting, I see this as the very beginning of more conversations as these are important issues that we want to discuss with the wider FA community.