Manufacturing, Gene Therapies

Sven Moller-Tank, PhD, director of Viral Delivery Technologies, Regeneron Genetic Medicines, Regeneron spoke about the use of bispecific antibodies to retarget AAV specificity.

Experiments conducted by the downstream technology team at Spark Therapeutics involving metal ion-containing additives showed improved capsid clearance in AAV production.

Under a partnership, ProPharm and PBL have launched the Cell Factory Box, an enclosed and fully automated cell factory manufacturing device.

A new draft guidance issued by FDA covers human- and animal-derived materials used in the manufacture of advanced therapy medicinal products.

Beqvez (fidanacogene elaparvovec-dzkt), a one-time gene therapy, helps adults with hemophilia B produce factor IX themselves instead of receiving regular intravenous infusions.

The collaboration aims to scale-up TreeFrog’s process to produce and amplify cells for Vertex’s type 1 diabetes cell therapies.

Contract manufacturer SK pharmteco will manufacture as well as conduct testing and release of Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firadenovec-vncg), a gene therapy for bladder cancer.

In back-to-back deals, Charles River will offer plasmid DNA manufacturing services for a gene therapy under development by Axovia Therapeutics and for a lead candidate being developed by Ship of Theseus.

Memel Biotech aims to offer a range of services from discovery through to formulation for preclinical through to late-stage clinical and commercial production of advanced therapies.

Univercells and Altamira Therapeutics are collaborating on nanoparticle-delivered mRNA vaccines, while Andelyn Biosciences and Grace Science are partnering on novel therapies for NGLY1 deficiency.

Cellares CEO Fabian Gerlinghaus explores advancements in counterflow centrifugal elutriation and electroporation, teasing exciting developments in product testing.

Orchard Therapeutics’ Lenmeldy (atidarsagene autotemcel) marks the first gene therapy approved in the US for treating children with metachromatic leukodystrophy.

Understanding how to apply phase-appropriate GMPs is crucial for achieving successful regulatory approval.

CSL will share access to Cytegrity, its proprietary stable production system for lentiviral vector production, with Genezen.

The milestone approval of a gene-edited therapeutic paves the way for gene-editing technologies.

The final guidance provides recommendations for developing gene therapy products incorporating genome editing of human somatic cells.

Charles River’s off-the-shelf rep/cap plasmids are intended to simplify gene therapy supply chains.

Revolutionary therapies restructure pharmaceutical manufacturing.

There are positive indications for future growth.

Tome Bioscience is facilitating the transition in biology from the editing phase to the cut-and-paste phase.

Andelyn Biosciences has been selected by Ultragenyx to manufacture UX111, that company’s gene therapy for treating Sanfilippo Syndrome.

RoslinCT will manufacture Vertex Pharmaceuticals’ CRISPR-based gene therapy, Casgevy, recently approved for treating sickle cell disease and β thalassemia.

Exagamglogene autotemcel seen as synechdoche.

A look at using best practice tech transfer methods for CGTs to increase process and analytics robustness while being scalable.

Spark Therapeutics and SpliceBio have formed a collaboration to develop a gene therapy that can treat an inherited retinal disease.