Company Updates covers news from Brilliant Blue Biosciences. Research & Industry News highlights relevant developments published by external sources, summarised and attributed to them.
FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, for pediatric patients with Sanfilippo syndrome type A, based on cognitive data from an open-label trial in children 2 to 5 years old.
Source: CGTLive
Read more →The partners will assess automated manufacture of GenomeFrontier's virus-free CAR T process; the collaboration is Cellares' first with an Asian cell-therapy developer. The announcement concerns a product, collaboration or production plan; it does not by itself establish clinical benefit or verified scale-up performance.
Source: GEN — Genetic Engineering & Biotechnology News
Read more →Moonwalk Biosciences says new financing of $70 million will support an siRNA pipeline targeting adipose tissue and its discovery platform. The announcement concerns a product, collaboration or production plan; it does not by itself establish clinical benefit or verified scale-up performance.
Source: GEN — Genetic Engineering & Biotechnology News
Read more →SynGenSys introduced an expanded synthetic promoter library designed for biomanufacturing applications in CHO cells. The announcement concerns a product, collaboration or production plan; it does not by itself establish clinical benefit or verified scale-up performance.
Source: GEN — Genetic Engineering & Biotechnology News
Read more →PTC Therapeutics wins Sangamo's BLA-stage Fabry disease gene therapy ST-920 for $111 million in a bankruptcy auction, with STAAR data supporting accelerated approval.
Source: CGTLive
Read more →Rznomics says its participation in Lilly Gateway Labs is intended to accelerate discovery and development of RNA therapeutic candidates. The announcement concerns a product, collaboration or production plan; it does not by itself establish clinical benefit or verified scale-up performance.
Source: GEN — Genetic Engineering & Biotechnology News
Read more →FDA granted accelerated approval to pariglasgene brecaparvovec-opnr, a one-time liver-directed AAV8 gene therapy, based on a 31% reduction in cornstarch intake versus placebo.
Source: CGTLive
Read more →Oak Hill Bio announced dosing of the first participant in BEACON, a pivotal phase 3 trial evaluating rugonersen, an antisense oligonucleotide originally developed by Roche, for Angelman syndrome.
Source: CGTLive
Read more →The BaseMap ABE launch offers genome-wide measurements in relevant cells to support off-target characterisation during therapeutic development. The reported capability is a product or workflow announcement, not independent proof of clinical diagnostic performance.
Source: GEN — Genetic Engineering & Biotechnology News
Read more →DNA Script reports an ARPA-H award for a DNA manufacturing effort that incorporates its enzymatic synthesis technology. The announcement concerns a product, collaboration or production plan; it does not by itself establish clinical benefit or verified scale-up performance.
Source: GEN — Genetic Engineering & Biotechnology News
Read more →The NeX solution builds on Enzene's continuous manufacturing platform and is presented as a transportable biologics production option. The announcement concerns a product, collaboration or production plan; it does not by itself establish clinical benefit or verified scale-up performance.
Source: GEN — Genetic Engineering & Biotechnology News
Read more →A new test bed linked to the University of Maryland and NIST will support programmable, automated manufacturing workflows. The announcement concerns a product, collaboration or production plan; it does not by itself establish clinical benefit or verified scale-up performance.
Source: GEN — Genetic Engineering & Biotechnology News
Read more →We co-develop nanocarrier and biosensing programmes with pharma, biotech and academic groups — from target selection through scale-up.
