Intellia Therapeutics, Inc.’s Post

#CRS2024 Reflecting on an impressive plenary lecture by Dr. Laura Sepp-Lorenzino, Ph.D. from Intellia Therapeutics, Inc., titled "Realizing the Promise of In Vivo CRISPR Therapeutics." Well in fact, it's more than a promise. Thanks to #Nanomedicine, we are now at the beginning of a true medical revolution. Dr. Sepp-Lorenzino delved into Intellia's pioneering work with CRISPR/Cas9 technologies delivered by systemic lipid nanoparticles (LNPs). Notably, onoing clinical trials have shown impressive improvements in patients with hereditary transthyretin amyloidosis (ATTR) and for hereditary angioedema (HAE), aiming to provide continuous reduction of symptoms with a single dose. HAE is a rare, autosomal dominant disorder characterized by recurrent episodes of severe swelling, which can be potentially fatal if not identified early and drastically impact the quality of life of patients. Current treatments are effective but expensive, and there is no cure yet. During the very active Q&A, Dr. Sepp-Lorenzino emphasized the key role of nanomedicine but also highlighted the variability in LNP quality (that we see in virtually all research papers nowadays): "I'm happy with my LNP... But...not all LNP are the same." she said. Couldn't agree more. At the European Technology Platform for Nanomedicine (ETPN Association), we are addressing these challenges, notably through two great European collaborative projects: METRINO Project: Producing SOPs and reference materials to help academia and industry better qualify and standardize nanomedicine, notably including LNPs indeed. NANOSPRESSO-NL: Revolutionizing the paradigm of Nanomedicine for rare diseases through bedside production of small batches of high-quality RNA-LNP, to bypass the many constraints of scale-up. We develop this concept for rare and orphan diseases like Methylmalonic acidemia (MMA), a metabolic genetic condition leading to the accumulation of substances in cells that cause damage to the brain, liver, kidneys, and other organs. We even integrate regulatory science research in the process to understand how this new concept could fit current and future regulations. I highly recommend all friends and colleagues of the Nanomedicine community to follow these two projects of interest, at least on LinkedIn. Get involved! :) This lecture truly underscored the transformative potential of CRISPR technology in modern medicine. What a fantastic period: finally, we can see treatments coming for those millions of patients who struggle with rare and orphan diseases. #CRS2024 #CRISPR #GeneTherapy #Nanomedicine #Innovation #Healthcare cc Raymond Schiffelers Olivia Lewis Marieke De Bruin Andreas Åslund Sven Even F. Borgos Solstice Pharmaceuticals B.V. UMC Utrecht Robin Schürmann Enrica Alasonati Leonardo Mortati Marco Monopoli Lorena Diéguez meriem lamghari Neill Liptrott Marco Monopoli Giovanni Tosi Steven Conlan Kathleen Spring et al.