Cell and Gene Therapy Industry News We have previously discussed the cell and gene therapy boom in the United Kingdom. The industry is thriving not only in the UK, but around the world, and here we take a quick look at some of the major successes and deals that have taken place in the last few months. Gene therapy deals As a quick reminder, gene therapy involves the transfer of genetic material, usually in a carrier or vector, into the appropriate cells for uptake of the genetic material. The vector can either be delivered outside the body (ex vivo treatment) where the targeted cells are removed from the patient and administered to cells before they are returned to the patient, or the vectors can be injected into the body (in vivo treatment). In March this year, Novartis entered into an agreement for Voyager Therapeutics' TRACER™ adenoassociated virus (AAV) capsids to use in potential gene therapies for three neurological diseases. Under the agreement, Novartis will have the right to evaluate novel capsids from the RNA-driven TRACER™ platform and to exercise options to license capsids for exclusive use in its development of AAV gene therapies directed to three targets. Novartis also has the right to license capsids for two additional disease targets under the same terms. Voyager Therapeutics’ TRACER™ screening platform is identifying improved vectors selected to target desired cells and tissues with greater specificity, at lower doses, and with reduced risk of off-target effects compared to conventional AAVs. Initial data demonstrates TRACER™-derived capsids have enhanced tropism for the central nervous system. In February 2022, Takeda signed a deal with Code Biotherapeutics, potentially worth up to US$2 billion, to develop gene therapies for rare diseases. The partnership will make use of Code Biotherapeutics’
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3DNA non-viral, synthetic, genetic medicine delivery platform for a liver-directed programme, and also involves conducting additional studies for programmes directed to the central nervous system. 3DNA leverages targeting molecules (such as peptides, antibodies, and small molecules) which bind to cell surface proteins expressed on target cells. After binding to the target cell, the 3DNA is internalised into the cell via a receptor-mediated process. When utilised to deliver gene therapy, the gene construct then localises to the cell nucleus where a tissue specific promoter drives the expression of the desired protein.
...gene therapy “involves the transfer of genetic material, usually in a carrier or vector, into the appropriate cells for uptake of the genetic material.
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PerkinElmer, Inc. announced that its SIRION Biotech business, experienced in gene and cell therapy and vaccine development, and the Centre for Genomic Regulation, an international biomedical research centre located in Barcelona, entered into an agreement to jointly develop new generation AAV vectors for type 1 and type 2 diabetes gene therapy in the pancreas. The end goal is to develop AAV vectors that target specific pancreatic cell types and contain payloads that express therapeutic genes under control of cell-specific regulatory elements.
This new approach aims to increase the precision, safety, and efficacy of future AAV-based gene therapies for diabetes. Novartis has entered into a definitive agreement to acquire all of the outstanding share capital of Gyroscope Therapeutics for US$800 million upfront, gaining the experimental AAV2-based gene therapy GT005. This one-time treatment, which is delivered under the retina, is currently in mid-stage development for patients with geographic atrophy (GA). GA is a chronic, progressive degeneration of the macula and leads to permanent loss of vision. BioMarin Pharmaceutical Inc. and Skyline Therapeutics announced a multi-year global strategic collaboration for the discovery, development, and commercialisation of AAV gene therapies to treat genetic cardiovascular diseases. The partnership will leverage Skyline Therapeutics' AAV gene therapy platform to develop innovative gene therapies, with a focus on genetic dilated cardiomyopathies, a group of progressively advancing, devastating diseases where the ventricles of the heart dilate and cannot efficiently pump blood. There are currently no targeted treatment options. Astellas Pharma Inc is teaming up with Dyno Therapeutics to develop next-generation AAV vectors for gene therapy directed to skeletal and cardiac muscle. The partnership will rely on Dyno Therapeutics' AI-based CapsidMap™ platform to create AAV capsids with optimised tissue targeting and immuneevading properties, while also
