Manufacturing, Gene Therapies

FDA has granted expanded approval to Sarepta Therapeutics for Elevidys to treat DMD in non-ambulatory patients, in addition to ambulatory patients.

CGT manufacturing processes need automation as well as standardization, according to Lonza’s Joe Garrity and Jerry Jiang.

Joe Garrity and Jerry Jiang from Lonza share their insights on the latest trends and challenges for the commercialization of new CGTs.

BioPharm International chats with Dr. Monika Paulė, CEO and co-founder of Caszyme about the evolution of CRISPR.

CGT Catapult and CATTI have developed aligned training standards for the manufacture of advanced therapies.

Cellular Origins will apply its robotic platform to establish automated CGT manufacturing at CGT Catapult’s manufacturing innovation center in Stevenage, UK.

The Mirus Bio acquisition will boost Merck KGaA’s viral vector manufacturing services.

The organizations plan to combine etherna’s experience in mRNA and lipid-based nanoparticles with UHasselt’s autoimmune research to treat a variety of auto-immune diseases.

Studies conducted by a team at Oxford Biomedica demonstrated that HIC can be used to effectively separate non-deamidated capsids from deamidated capsids.

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A new draft guidance issued by FDA covers human- and animal-derived materials used in the manufacture of advanced therapy medicinal products.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