Finally got around to reading through the Feng Zhang laboratory’s amazing SEND (Selective Endogenous ENcapsidation for cellular Delivery) paper!

[Link: https://www.science.org/doi/10.1126/science.abg6155] The authors describe a new gene therapy delivery vehicle which leverages virus-like particles (VLPs) originally produced within human cells. These VLPs arise from ancient retroviral genomic fragments that were integrated into the human genome long ago and eventually were utilized to benefit our own physiology. Because they are recognized as ‘self’ by the immune system, the VLPs have potential as a novel gene therapy delivery modality. In this paper, Segel et al.

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Aera’s strategy is to harness these proteins, and structures, to move the cargo of genetic medicines: RNAi, antisense RNA, mRNA, or a genetic editing payload, for example. To date, proteins and nucleic acids have been packaged. The company’s first goal is to move smaller nucleic acids like ASOs and siRNA from cell to cell.

What is known about PNPs is “quite limited,” said Akinc. Their role in the human body is particularly opaque. The literature goes back only to 2018. They are called virus-like particles (VLPs) in the literature, but Aera thinks that PNP is a more technically accurate name.

Aera Therapeutics is announcing its launch today, they said, with “a vision to harness its proprietary delivery platform to unlock the potential of genetic medicines.” Akinc doesn’t specify any disease targets but emphasizes the unmet need for diseases that affect the central nervous system.

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Researchers have recently made a groundbreaking discovery in the field of kidney disease. They have found a new pathway that could potentially prevent kidney failure in thousands of people. Dr. Carl May and his team at Bristol Medical School, with funding from Kidney Research UK, have discovered a new treatment pathway for non-genetic nephrotic syndrome.

This targets the unknown factor that leads to kidney failure. Nephrotic syndrome is a rare kidney condition that causes protein to leak into the urine, affecting around 10,000 people annually in the UK. The discovery offers hope for patients, especially children, who may develop kidney failure.

Researchers from Bristol Renal have identified a receptor called PAR-1 that works in conjunction with an unknown factor to cause kidney failure in patients with idiopathic nephrotic syndrome (INS). They found that anti-PAR-1 treatments could block the effect of the factor and prevent kidneys from failing, potentially making transplantation a more viable option for more patients.