Manufacturing, Gene Therapies

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.

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.

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.

The steady rise in popularity of gene therapies and other biologics is the underlying reason for the growing importance of aseptic manufacturing processes in the biopharmaceutical industry.

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.

Plasmid DNA (pDNA) is a critical raw material for the manufacture of cell and gene therapies. As this market continues to grow, so too does the demand for high-quality pDNA.

Scientists are leveraging ddPCR technology to get an accurate read on viral titer and vector copy number.

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.

Despite the successes that have already been achieved with emerging therapy development and manufacturing, companies are still facing numerous challenges.

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

In this episode of Drug Digest, industry experts discuss aseptic processing and manufacturing, including what the future holds in this area and the barriers involved with automated aseptic processing practices.

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.

In this episode of Drug Digest, editors discuss what happens in the manufacturing space from a CDMO and a cell/gene therapy perspective.

The bioreactors can integrate with Emerson’s DeltaV PK Controller in addition to S-Series and M-Series controllers.

Burgeoning CGT modalities bring promise and challenges.

mRNA may be a modality whose chief advances are yet to come.

FDA has approved Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy from BioMarin Pharmaceutical for treating severe hemophilia A in adults.

Manufacturers face the challenge of meeting growing demand for personalized biopharmaceuticals.

The agency approved Elevidys to treat pediatric patients four through five years of age with Duchenne muscular dystrophy.