Manufacturing, Gene Therapies

FDA's approval makes Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) the first gene therapy approved to treat pediatric metachromatic leukodystrophy in the US.

Cellares CEO Fabian Gerlinghaus discusses the nuts and bolts of counterflow centrifugal elutriation and electroporation innovations and optimization, while hinting at big news on future release testing of products, as a product itself.

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

The emergence of mRNA vaccines and cell and gene therapies has pushed innovation in analytical methods.

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.

Collaboration is a key component to achieving long-term success with genetic medicines.

Exagamglogene autotemcel seen as synechdoche.

Emergence of advanced manufacturing technology to ensure quality of biopharmaceutical drugs combined with efforts to identify a regulatory pathway indicate that a distributed manufacturing model is within reach.

Monoclonal antibody and viral vector manufacture share similarities, but vector scale up faces unique challenges.

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

The California Institute for Regenerative Medicine has partnered with Forge Biologics, which will manufacture AAVs to help accelerate gene therapy programs in California.

Chris Spivey, editorial director, hosts a wide ranging discussion on the COVID-19 pandemic response, future government communications, and healthcare funding for mRNA with experts Dr. Kate Broderick, Chief Innovation Officer, Maravai LifeSciences and Dr. Tom Madden, President & CEO at Acuitas Therapeutics. Highlights include supply chain modalities, ensuring a fully trained manufacturing workforce, combining delivery with gene editing innovations, epigenetic approaches to modulate gene expression moving toward the clinic. We conclude with the huge potential for RNA expression of monoclonal antibodies and protein replacement applications.

The 65,000-square-foot facility is designed with the capacity and capability to help scale the next generation of CGTs for human trials and beyond.

Further, the construction of Gritgen’s GMP facility strictly adheres to the global current GMP (CGMP) standards and complies with the regulatory guidelines of authoritative agencies including the National Medical Products Administration (NMPA), FDA, and the European Medicines Agency (EMA).

The program will improve access to adeno-associated virus gene therapy vectors.

The agreement will enable CTMC to strategically scale the recently completed good manufacturing practice facility to drive high-throughput manufacturing utilizing the automated, closed system capabilities of the Fresenius Kabi Lovo and Cue cell processing systems.

FUJIFILM Diosynth Biotechnologies has finished its new viral vector gene therapy manufacturing facility in Darlington, United Kingdom.

Part of the session "Transforming Medicine: Unleashing the Potential of Cell and Gene Therapies," Sadelain discusses CAR-T cells for immunotherapy and the future of cell/gene therapies.

The companies are collaborating to apply ShapeTX’s AAVid capsid discovery platform and transgene engineering technology in addition to Otsuka’s expertise in genetic payload design and ophthalmology to develop novel treatment options for eye diseases.

The agreement states that Orbit will implement its bead-based peptide display engine to discover peptide leads specific to targets related to specific tumors.

Rentschler Biopharma, CGT Catapult, and Refeyn aim to use automated and digital technologies to improve AAV manufacturing for gene therapies.

The launch of Alpha Teknova’s new manufacturing facility in Hollister, Calif., increases its capacity for custom reagents for life sciences applications.

The site was acquired by AGC Biologics in July 2020, and the Milan location is the first cell and gene therapy site approved in Europe for GMP manufacturing of clinical and commercial supplies.

The initial collaboration between the two companies began in 2017 with an extension announced in 2021.