MENLO PARK, Calif.--(BUSINESS WIRE)--ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced the publication of a research article in Science. The article titled "In vivo editing of lung stem cells for durable gene correction in mice" describes the ability of Selective Organ Targeting (SORT) lipid nanoparticles (LNPs) to facilitate high levels of persistent gene editing in lung stem cells when given systemically via intravenous injection.
Professor Daniel Siegwart, Ph.D., the lead author and a co-founder of ReCode Therapeutics, led his team at UT Southwestern, in collaboration with ReCode researchers, to develop SORT LNPs that could efficiently deliver gene-editing tools away from the liver and directly to cells in the lung. Key findings include:
- Delivery of Gene Editors to Lung Stem Cells. This is the first study to show that LNPs carrying gene editing tools can effectively correct a gene in lung stem cells when administered intravenously and delivered through systemic circulation. Lung stem cells are the progenitors for many functional cells in the lung that play a role in rare genetic diseases such as cystic fibrosis (CF) and primary ciliary dyskinesia (PCD).
- Lasting Disease-Modifying Therapies. This approach enabled over 70% editing efficiency in mouse lung stem cells, sustaining the effect for up to 660 days, a result which, if translated into the clinic, may offer the potential for sustained benefits for patients with genetic lung diseases.
- Potential for a New Cystic Fibrosis Treatment. Proof of concept was demonstrated for correction of the CF mutation CFTR R553X, which is untreatable with currently available small molecule therapies. The new technique restored CFTR protein function in cells derived from CF patients, potentially offering a path for a durable treatment for cystic fibrosis.
“Achieving long-lasting gene correction in lung stem cells highlights the growing potential of SORT LNPs to deliver effective treatments for conditions like cystic fibrosis and could pave the way for our technology to advance durable therapies for other genetic diseases,” said Marco Weinberg, Ph.D., Head of Research at ReCode. “This publication is a testament to Dan’s scientific vision and ongoing collaboration with the ReCode team to develop the Selective Organ Targeting (SORT) lipid nanoparticles (LNPs) platform, with the goal of achieving targeted delivery of genetic medicines to patients with lung disorders.”
These findings will support ReCode’s ongoing research efforts to translate SORT LNP-based gene editing into the clinical setting, leading to durable and potentially curative therapies for patients with genetic lung disorders.
Further studies are needed to explore the mechanisms underlying efficient delivery to airway stem cells, assess safety and tolerability in larger animals, and evaluate efficacy in human trials.
About ReCode Therapeutics
ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues, and cells implicated in disease, enabling improved efficacy and potency. ReCode’s lead programs include RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene, and RCT2100 for the treatment of the 10-13% of cystic fibrosis patients who have Class I or other mutations in the CFTR gene that do not respond to currently approved CFTR modulators. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated using the SORT LNP delivery platform. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases, including musculoskeletal, central nervous system, liver, and infectious disease indications.
ReCode has been recognized by the San Francisco Business Times and Silicon Valley Business Journal as a Best Place to Work. For more information, visit www.recodetx.com and follow us on LinkedIn and Instagram.
