What is happening at Brilliant Blue — and around it
Company Updates covers news from Brilliant Blue Biosciences. Research & Industry News highlights relevant developments published by external sources, summarised and attributed to them.
uniQure has aligned with the FDA on what the company calls key elements of the confirmatory study design: The study is being designed as a randomized, standard-of-care trial enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Functional Capacity (TFC) at 36 months serving as the primary endpoint.
Source: GEN — Genetic Engineering & Biotechnology News
Liu ensured that his return visit to ESGCT (having spoken at the congress in Barcelona in 2019) would be one of the highlights of the conference. The “meta lesson” of the past few years in therapeutic genome editing is that different scientific approaches and applications can synergize with regulatory, manufacturing, and economic advances “to achieve the mission we’re on.”
Source: GEN — Genetic Engineering & Biotechnology News
This eBook brings together perspectives from across the AAV development and manufacturing continuum. It explores how early commercial planning can help avoid costly delays, how standardized platform approaches and off-the-shelf plasmids can streamline development, and how next-generation innovations are helping overcome long-standing manufacturing bottlenecks.
Source: GEN — Genetic Engineering & Biotechnology News
A new use of CRISPR gene editing has halted and even reversed the progression of a model of Duchenne muscular dystrophy, according to work led by scientists at the Perelman School of Medicine. The technique boosted a protein, utrophin, as a stand-in to take on the job of a different protein, dystrophin, which helps maintain healthy muscles and is in short supply in those with muscular dystrophy.
GEN 's Kevin Davies, PhD, reports from ESGCT where patient safety, expanding access to gene therapy, and progress towards an HIV vaccine were prominent themes among the early talks at ESGCT 2026.
Source: GEN — Genetic Engineering & Biotechnology News
Scientists can now edit genes in precise locations deep in the brain and body without the use of invasive surgery or implants, thanks to new work from researchers at the Wu Tsai Neurosciences Institute.
By analyzing protein coronas across 42 nanomaterials, researchers identified how surface charge, PEGylation and shape influence cellular uptake and immune evasion, enabling more predictable nanomedicine design.
A new study has revealed molecular rules governing how nanoparticles interact with biological systems, showing how their physicochemical properties shape protein corona (PC) formation and influence biological outcomes. The findings provide a framework for designing nanomedicines with more predictable biological identities.
CDMOs already play a critical role in the cell and gene therapy development and manufacturing space, says Rose Biosolutions. Emerging bioprocessing technologies, production methods, and strategies are creating even more opportunities for contractors.
Source: GEN — Genetic Engineering & Biotechnology News
