The AAV vectors of the present invention are particularly suitable for gene therapy by systemic application, since (a) transduction of the liver is eliminated or at least drastically reduced and (b) the transduction efficiency of non-hepatic tissues is increased.Jürgon Kleinschmidt...
AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes Brahim Chaqour ,Thu T. Duong &Ahmara G. Ross Article|11 August 2023 Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair ...
Outsourcing trusted-weight dry powder buffers to support expansion of a major pharmaceutical manufacturer’s gene therapy division. Learn more › AAV vector process development: achieving high purity and high yield Expert roundtable: experiences from the frontline. Learn more › Upstream and downstrea...
Today, a typical manufacturing run of an AAV-vector therapy using high-yield cell lines and large-capacity bioreactors might only produce approximately ten doses of a systemic gene therapy from a single batch at a cost of nearly $100,000 per dose (assuming approximately 1 × 10^...
1:40 pm Challenges and Process Development for Purification of Gene Therapy Vector AAV Xue Mi, PhD, Senior Scientist I, Purification Process Development, Abbvie Bioresearch Center Adeno-associated virus (AAV) is highly inefficient at packaging its genome, with up to 90% of the formed AAV caps...
Ad as a vector in gene therapy Ad vectors have the following advantages: (1) high transduction efficiency, both in quiescent and dividing cells; (2) epichromosomal persistence in the host cell; (3) broad tropism for different tissue targets; and (4) and the availability of scalable production...
Robust cGMP manufacturing platforms for viral vectors, plasmids, mRNA, and sgRNA. Accelerating gene therapy development with quality and speed.
and Zolgensma (onasemnogene abeparvovec-xioi), which harnesses an AAV (serotype 9) vector to deliver a functional copy of the SMN1gene to motor neurons in spinal muscular atrophy patients. The pipeline for AAV vector-based therapy is also increasing, with at least 250 cli...
Corning provides a broad range of cell culture systems, equipment, and solutions for advanced cell and gene therapy applications, and regulatory compliance.
Gene Synthesis services enable you to custom-clone your codon-optimized genes into your desired vector for optimal protein expression, regardless of length or complexity.