Company Updates covers news from Brilliant Blue Biosciences. Research & Industry News highlights relevant developments published by external sources, summarised and attributed to them.
A UCLA-led team has described how they advanced an experimental CRISPR approach for sickle cell disease into a blood stem cell therapy that can be manufactured at the scale and quality required for a clinical trial.
Source: News-Medical
Read more →A clinical trial in three people tested a combination of gene-laden viruses and a drug.
Source: Nature
Read more →The FDA has accepted a BLA under its accelerated approval pathway from uniQure for ifezuntirgene inilparvovec to treat Huntington’s disease.
Source: Pharmaceutical Technology
Read more →We look at some of the key biotech developments, companies, and clinical data to watch at AHA Scientific Sessions 2026.
Source: Labiotech.eu
Read more →A single gene-editing treatment could someday protect against heart disease, high cholesterol, and other common conditions for decades, potentially replacing lifelong medications. But scientists warn that permanently altering genes introduces risks that could take years to emerge, making long-term safety a critical challenge.
Source: ScienceDaily
Read more →Kamau Therapeutics, which has stayed relatively quiet since its 2023 emergence, is sharing clinical data from its revamped sickle cell gene editing program for the first time. The startup is giving a ...
Source: Endpoints News
Read more →Rice University's Han Xiao has used his AI-supported Sequence Display technology to expand the targeting abilities of CRISPR/Cas9. In a paper recently published in Nature Communications, Xiao evolved several variants of a compact Cas9 that could efficiently target stretches of DNA previously inaccessible for editing.
Source: Medical Xpress
Read more →A study led by researchers from the Germans Trias i Pujol Research Institute (IGTP) and the Blood and Tissue Bank (BST) has developed and validated a manufacturing platform for extracellular vesicles that meets pharmaceutical quality requirements, marking a key step toward their clinical application.
Source: Phys.org
Read more →In a landmark achievement in the field of genetic medicine, researchers have reported the very first published case in a peer-reviewed journal of a successful clinical treatment of a patient with alpha-1 antitrypsin deficiency (PiZZ) using in vivo gene therapy.
Source: News-Medical
Read more →Lipid nanoparticles modified with hydroxyzine, an antihistamine compound active in the central nervous system, enable brain delivery of mRNA and gene-editing components in multiple animal models.
Source: Nature Nanotechnology
Read more →After a whirlwind third quarter that saw two FDA approvals but a major setback in Angelman syndrome, Ultragenyx is laying off staff for the second time this year. The California biopharma
Source: Endpoints News
Read more →Some genetic diseases leave cells without a crucial protein. One possible treatment is to persuade genes already inside those cells to work harder, but the necessary machinery can be difficult to deliver, especially when researchers want to target several genes at once.
Source: Phys.org
Read more →We co-develop nanocarrier and biosensing programmes with pharma, biotech and academic groups — from target selection through scale-up.
