Manufacturing, Gene Therapies

The next-generation IRO platform was found to significantly outperform the legacy Prodigy system, showing improvements in cell growth, CAR+ cell yield, and overall efficiency.

Emerging therapies, such as cell and gene therapies, come with unique challenges that must be planned for in advance.

The latest developments in emerging therapies, including new innovations and collaborative efforts in manufacturing.

Viral vectors and other complex biologic modalities require more specificity and higher sensitivity to detect and distinguish contaminants.

Rentschler Biopharma now offers a new lentiviral vector manufacturing toolbox at its site in Stevenage, UK.

Under the license agreement, Andelyin Biosciences will expand its AAV Curator Platform by including MyoAAV plasmids developed by Broad Institute of MIT and Harvard.

Optimizing AAV vector manufacturing will be necessary to meet anticipated future market demand.

AAV and lentivirus both have pros and cons in their use for specific gene therapy applications.

BioIVT has launched new cleanroom manufacturing space using Germfree’s technology that boosts its cell and gene therapies development capabilities.

Under the expanded agreement, Univercells and University of Pennsylvania will evaluate certain bioreactors for scalable gene therapy production.

The acquisition of uniQure’s Lexington, Mass., manufacturing operations will boost Genezen’s late-phase and commercial gene therapy development and manufacturing services.

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.

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.

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.

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.