Manufacturing, Gene Therapies

NIH reported that an investigational gene therapy developed by researchers from the University of California, Los Angeles and Great Ormond Street Hospital in London can restore the immune systems of infants and children who have severe combined immunodeficiency due to adenosine deaminase deficiency.

ViroCell will supply viral vectors and gene modified cells, to academic and corporate clients, for translational cell and gene therapies going into clinical trials.

Under the terms of the agreement, Catalent will handle the process development and CGMP manufacturing of AavantiBio’s adeno-associated viral vector-based therapeutic candidate for use in clinical trials in the US and Europe.

Avantor plans to increase its single-use manufacturing footprint by 30% and double its cleanroom space in the US and Europe.

Vertex and CRISPR have amended their collaboration agreement to include the development, manufacture, and commercialization of CTX001, an investigational CRISPR/Cas9-based gene editing therapy for sickle cell disease and transfusion-dependent beta-thalassemia.

Catalent plans to add cryogenic capabilities to its facility in Philadelphia, which will strengthen its ability to cryogenically preserve cell therapies and other biological materials.

The ATMPS and Ori Biotech collaboration aims to improve visibility and reporting of cell and gene therapy manufacturing through a pre-integrated data solution.

The UK's National Horizons Centre has been confirmed as a National Training Centre that will deliver on-site advanced therapies and vaccine manufacturing specific training.

Growth in cell and gene therapies puts pressure on the raw materials supply chain.

A growing pipeline of gene therapy candidates aimed at treating neurodegenerative diseases has prompted new guidance.

This agreement enhances GSK’s investment in cell and gene therapy manufacturing in the UK for clinical trials.

Under the terms of the acquisition, OXGENE will become a fully owned subsidiary of WuXi Advanced Therapies, WuXi AppTec’s cell and gene therapy contract testing, development, and manufacturing organization business unit.

The site will house development and GMP production capacities for cell and gene therapies, next-generation vaccines, and biopharmaceuticals, to support clients from early clinical trials through commercialization.

The expansion, to be completed in April 2021 aims to increase the CDMO’s viral-vector production capacity in Spain.

The foundation will provide funding support for the discovery and development of a single-administration, in-vivo gene therapy to cure sickle cell disease, a hereditary blood disease.

The acquisition will allow Charles River to expand its cell and gene therapy development, testing, and manufacturing capabilities, while adding to its existing portfolio of non-clinical capabilities.

Catalent will offer materials from its gene therapy facilities in Maryland to support IND-enabling studies and a Phase I/II clinical trial of the product.

Biotechnology company, Polyplus-transfection, has started construction of a new facility in France to meet growth in the cell and gene therapy market.

Evozyne’s evolution-based protein design technology will be used to research and develop proteins that could be used in the next generation of gene therapies.

Through the acquisition, Lilly will have access to Prevail’s portfolio of clinical-stage and preclinical neuroscience assets.

Leveraging real-time MRI guidance for intracranial gene therapy administration potentially improves efficacy and outcomes.

The acquisition significantly expands Precision Medicine Group’s cell and gene therapy expertise.

The collaboration will utilize ViGeneron’s novel engineered AAV capsids, vgAAV, to transduce retinal cells with intravitreal injections for an undisclosed target.

Advances in development, data management, and automation, and closer collaboration with contract development and manufacturing partners, are pushing more therapies closer to commercialization.

Through the acquisition, Eli Lilly will have access to Prevail’s portfolio of clinical-stage and preclinical neuroscience assets.

Evonik offers lipid nanoparticles for gene-based drug development and manufacturing.

The collaboration includes the development, commercialization, and supply of Orgenesis’ pipeline in South Korea and Japan and the development and commercialization of Cure Therapeutics’ pipeline on a global basis.

The gene therapy increases the ability of retina cells to make a soluble form of CD59, a protein that shields the retina from damage, to prevent additional damage and to preserve vision.

Viral clearance processes and guidance must evolve along with newer biotherapeutic modalities.

Behzad Mahdavi, PhD, will join a team of experts in the company’s Science and Technology Group to fast-track the adoption of new development and drug delivery technologies and manufacturing processes and techniques.