The past 9 months since joining RSRT have been amongst the most exciting of my professional career. Never in the history of life sciences has the development of scientific technologies that can benefit individuals with Rett been so rapid.
In my role as RSRT’s Chief Scientific Officer, ensuring that these technologies are put to work on behalf of your loved ones is a responsibility I take to heart. I have had the privilege of representing individuals with Rett Syndrome in many ways over the past several months. With frequent trips to universities up and down the East Coast of the USA, as well as in Europe, I am in close contact with the numerous professors we fund to better understand their research, their challenges and importantly, gauge their progress.
Our Gene Therapy Consortium is developing new coatings on the outside of the vector viruses called AAV so that they distribute better throughout the brain. The investigators that we have funded to work on reactivating the healthy but silent copy of MECP2 have made a breakthrough in doing this in a selective manner. The scientists working on DNA editing have the single base editors ready to go and are now testing them on patient derived cells collected through our Outcome Measures & Biomarkers Development (OMBD) initiative.
In my dual role as RSRT’s Chief Business Officer I have also engaged with dozens of pharmaceutical and biotech companies educating them about our Roadmap to a Cure and assessing their interest in Rett. I have counseled academics on the importance of protecting their discoveries by filing intellectual property (IP) and then brokered introductions to companies in the hopes that the IP will be licensed and developed.
These interactions are already proving worthwhile. One of the largest pharmaceutical companies in the world, which has a keen interest in gene therapy, is also very interested in our MECP2 reactivation efforts. Our RNA editing efforts, already a hot area of science, is generating considerable interest amongst established companies, start ups and investors. Two companies already have option agreements with the universities involved in our Gene Therapy Consortium 2.0 to move concepts forward once progress is made in the academicians’ labs.
Taking advantage of networking opportunities is core to what my colleagues and I do on behalf of the Rett community and in that spirit I’ve also attended conferences on a variety of topics as well as a very productive BIO partnering event in Philadelphia.
Despite all this progress and activity, there are hurdles to overcome and big unknowns remain. These unknowns can only be answered through the systematic and rigorous drug development process that is required by the FDA. Questions such as the body’s immune reaction to the viral delivery vehicle and/or to the DNA and RNA editors we would need to deliver to the brain. We will proceed cautiously yet aggressively to answer these questions and overcome the challenges.
My colleagues and I have big plans for the remainder of the year. The ability to safely and effectively deliver biologic therapeutics diffusely to the brain is fundamental and relevant to all of our curative strategies. So a major objective of RSRT is to identify and support delivery technologies that extend beyond AAV9. We are in close contact with a number of professors and companies working in this area and are actively soliciting proposals.
Beyond Rett, we have also developed a roadmap for MECP2 Duplication Syndrome and have identified and received commitments from investigators to submit proposals for a number of exciting interventions. With funding from duplication families we hope to launch several pivotal projects.
As I said in my introductory blog, I was hired at RSRT to move the needle and that is exactly what I intend to do.
If you have questions or would like to have a phone call to delve more deeply into any of the above topics feel free to contact me. I am always available to schedule some time to chat.