Enabling organ-specific targeting using LNPs
On June 29, ReCode Therapeutics, a gene therapy company, announced the closing of a $120 million Series B+ financing round. The round was led by Leaps by Bayer, the investment arm of Bayer, and AyurMaya, an affiliate of Matrix Capital Management, with participation from Amgen Ventures. The company previously closed an $80 million Series B round of funding in October 2021. Proceeds from the financing will help ReCode diversify into CNS, liver, and oncology and continue to advance ReCode’s lead programs in primary ciliary dyskinesia and cystic fibrosis mRNA drugs into the clinic. The funds will also help ReCode improve its technology platform and expand its R&D into gene modification and small interfering RNA therapeutics.
Contrary to earlier LNPs, which were only capable of delivering to the liver, ReCode’s organ-specific targeted delivery platform, SORT-LNP, enables the delivery of nucleic acid drugs, including mRNA, siRNA, and DNA, to specific organs.
ReCode’s technology features the creative introduction of a “fifth element” into lipid nanoparticles (LNPs). Conventional LNPs mostly consist of four components: ionizable cationic lipid, amphiphilic phospholipid, polyethylene glycol lipid, and cholesterol. ReCode received a license from the University of Texas Southwestern Medical Center to use Dan Siegwart’s research to add a fifth lipid to the LNP, enabling it to load cargo like mRNA, tRNA, siRNA, and gene editing drugs and target organs other than the liver. The SORT LNP (selective organ targeting LNP) technology is one of the “seven technologies to watch in 2022,” according to Nature.
The development of gene drugs has been limited by the ability to target organs other than the liver. Dr. Suliman, CEO of ReCode, said, “the unique new selective organ-targeted lipid nanoparticle delivery platform allows for direct delivery of gene drugs to the organs and cells most affected by disease, improving drug efficacy.”
At the American Thoracic Society 2022 Annual Meeting in May, ReCode presented preclinical data from the PCD and CF programs that demonstrated how SORT-LNP delivery of mRNA can specifically target disease-associated cells without significant exposure in other tissues.
It is reported that ReCode will submit a clinical trial application for a primary ciliary dyskinesia (PCD) mRNA therapy (mRNA for DNAl1 delivered via SORT-LNP) to the FDA in the fourth quarter of this year and will submit a clinical trial application for a cystic fibrosis (CF) mRNA therapy (mRNA for CFTR delivered via SORT-LNP) in the middle of next year.
ReCode also said that SORT-LNP can also target the spleen, including the T and B cells therein, after tuning, opening the door to the possibility of in vivo in situ CAR-T therapies.
