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This new approach targets disorders including cystic fibrosis, muscular dystrophy, and some cancers. In lab-grown tissue from a cystic fibrosis patient who did not respond to exist...
Messenger RNA (mRNA) can be delivered into tissues and cells by encapsulating it into lipid nanoparticles (LNPs), and then injecting the nanoparticles. A target cell engulfs a LNP,...
Cancer researchers are turning a deceptively simple piece of DNA into a molecular “decoy” that could disrupt the genetic commands driving tumors. The strategy, reviewed in a new ar...
A preclinical study showed how chemically enhanced suppressor tRNAs combined with a lung-targeted delivery system can restore production of a critical protein in models of cystic f...
The treatment can reprogram immune cells that have been corrupted by the cancer, strengthening the body’s ability to attack.
Many of the more sophisticated forms of medicine, including gene therapies and immunotherapies, are challenging to deliver to the brain. At the very least there are far fewer pract...
A gene therapy that delivers instructions to produce a longer-lasting version of the clotting protein missing or deficient in hemophilia B may improve treatment effectiveness while...
A small-molecule therapy, VG-3927, targeting microglial TREM2 signaling showed neuroprotective effects in preclinical studies for Alzheimer's disease and was well tolerated in a Ph...
Our guest today is Dr. Nerissa Kreher, Chief Medical Officer of Alltrna. Nerissa brings two decades of rare disease drug development experience. The post Rewriting the rules of gen...
Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease that primarily affects the central nervous system and is marked by rapid neurodegeneration beginnin...
Mitochondrial transplantation as a form of therapy to improve mitochondrial function in aged tissues has barely started as a going concern, and only a few patients have been treate...
The PROPEL program aims to control gene expression through RNA structure, building logic-gated RNA therapies that switch on only where and when intended, without altering the genom...
The concept of targeted therapeutics is often traced back to German Nobel laureate Paul Ehrlich’s “magic bullet” in 1907, which envisioned a single entity that could both recognize...
Cystic fibrosis is an incurable genetic disorder. Since 2020, a treatment known as triple therapy has addressed the underlying cause. Researchers at Charité – Universitätsmedizin B...
The Abigail Wexner Research Institute at Nationwide Children's Hospital celebrated the Food and Drug Administration's (FDA) approval of FAYUVI for Sanfilippo syndrome type A, follo...
Researchers have engineered a custom ionizable lipid nanoparticle that delivers CAR-encoding mRNA into human T cells with over 90 percent efficiency, producing potent antitumor act...
Intravenous gene therapy extended lifespan, reduced neurological symptoms, and improved disease markers in a Sandhoff disease cat model, supporting further investigation of systemi...
The collaboration brings together Synastra’s expertise in genomic engineering, AAV vector design, rare-disease gene therapy, and translational development with NewBiologix’s propri...
New financing will help Moonwalk Biosciences expand its pipeline of siRNA medicines targeting adipose tissue while building out its proprietary drug discovery platform. The post Mo...
Through its entry into Lilly Gateway Labs, Rznomics officials say they plan to accelerate candidate discovery and development across its target gene database bank comprising over 2...
A DNA carrier checks several tumor clues before releasing its treatment, helping spare healthy cells and making chemotherapy work better in resistant brain tumors.
Rather than requiring genetic modification of the viral glycoprotein, G-Link uses a modular protein adaptor to blind native VSV-G tropism and redirect delivery after vector product...
Exosomes are one category of extracellular vesicle secreted by cells, a membrane-wrapped package of molecules, one complex component of the extensive, incompletely mapped communica...
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