Biotechnology companies Circio Holding and AaviGen have announced a research collaboration to develop heart disease gene therapies. The agreement will see the companies work to develop circVec-enhanced AAV vectors specifically engineered for targeted, low dose heart gene therapy. The collaboration focus will be on addressing genetic… Read More
Ensuring the integrity of adeno-associated virus (AAV) vectors is critical for gene therapy success, yet traditional methods can miss key structural elements. Download this new Workflow from Oxford Nanopore Technologies to discover how nanopore sequencing enables comprehensive characterisation of AAV genomes — including inverted terminal repeats (ITRs), transgenes, and… Read More
Ensuring the integrity of adeno-associated virus (AAV) vectors is critical for gene therapy success, yet traditional methods can miss key structural elements. Discover how nanopore sequencing enables comprehensive characterisation of AAV genomes — including inverted terminal repeats (ITRs), transgenes, and promoters — all in a single workflow. Download this workflow… Read More
Hansa Biopharma has announced topline results from three patients with Duchenne muscular dystrophy (DMD) treated with its imlifidase prior to receiving Sarepta’s Elevidys (delandistrogene moxeparvovec-rokl) in the SRP-9001-104 trial. After one dose of imlifidase, three patients experienced a rapid reduction of IgG antibodies, to levels ≥95% less… Read More
The US Food and Drug Administration (FDA) has decided not to recommend Ultragenyx Pharmaceutical’s UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). In its complete response letter (CRL), the FDA requested that the company provide additional information and… Read More
JCR Pharmaceuticals and Alexion, AstraZeneca Rare Disease, have entered into a license agreement for JCR’s JUST-AAV capsids to develop genomic medicines. JUST-AAV encompasses a range of vector types optimised for various target tissues—including liver-sparing, muscle-targeting, and brain-targeting variants—to expand the potential of AAV-based gene therapy. … Read More
SpliceBio has closed a $135 million Series B financing co-led by new investors EQT Life Sciences and Sanofi Ventures. There was also participation from Roche Venture Fund, as well as all existing investors: New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund, and Asabys… Read More
As the field of gene therapy advances, the need for fast, accurate, and scalable characterisation of adeno-associated virus (AAV) products and the plasmids used to produce them has never been greater. Join Oxford Nanopore Technologies and Plasmidsaurus on 11 June at 5pm BST for an… Read More
As the field of gene therapy advances, the need for fast, accurate, and scalable characterisation of adeno-associated virus (AAV) products and the plasmids used to produce them has never been greater. Join Oxford Nanopore Technologies and Plasmidsaurus on 11 June at 5pm BST for an… Read More
As the field of gene therapy advances, the need for fast, accurate, and scalable characterisation of adeno-associated virus (AAV) products and the plasmids used to produce them has never been greater. Join Oxford Nanopore Technologies and Plasmidsaurus on 11 June at 5pm BST for an… Read More
As the field of gene therapy advances, the need for fast, accurate, and scalable characterisation of adeno-associated virus (AAV) products and the plasmids used to produce them has never been greater. Join Oxford Nanopore Technologies and Plasmidsaurus on 11 June at 5pm BST for an… Read More
As the field of gene therapy advances, the need for fast, accurate, and scalable characterisation of adeno-associated virus (AAV) products and the plasmids used to produce them has never been greater. Join Oxford Nanopore Technologies and Plasmidsaurus for an expert-led webinar, hosted by DDW, showcasing new and improved genomics solutions… Read More
Hovione and Firstgene Life Sciences have announced a strategic collaboration to advance Hovione’s proprietary virus-like particle drug delivery platform for the targeted treatment of Hepatocellular Carcinoma (HCC), a therapeutic indication developed by Firstgene. Under the terms of the collaboration, Hovione will grant Firstgene an exclusive license to… Read More
The US Food and Drug Administration (FDA) has cleared BlackfinBio’s Investigational New Drug (IND) application for a Phase I/II clinical trial of novel adeno-associated virus (AAV) gene therapy, BFB-101, in children with Hereditary Spastic Paraplegia Type 47 (SPG47). The trial will be conducted in the US at… Read More
SpliceBio has dosed the first patient in the Phase I/II ASTRA study of SB-007, a dual adeno-associated viral (AAV) vector, for the treatment of Stargardt disease. Stargardt disease is an inherited retinal disorder caused by mutations in the ABCA4 gene. It is the most common form of juvenile macular degeneration,… Read More
Dyno Therapeutics and Roche have announced a second research collaboration to develop adeno-associated virus (AAV) vectors for gene therapies targeting neurological diseases. Dyno and Roche previously agreed a research collaboration and license agreement for neurological diseases and liver-directed therapies in October 2020. Under the… Read More
