CELL AND GENE THERAPY
Specific Biologics Inc. | December 08, 2021
Specific Biologics Inc. an early-stage biotechnology company developing novel gene editing technologies, announced that it will receive more than US$527,000 to support the preclinical development of its Dualase™ gene editing platform to target a set of cystic fibrosis -causing nonsense mutations where patients currently have fewer therapeutic options available.
"Incredible advancements in the treatment of cystic fibrosis have been made since the identification of genetic mutations in the CFTR gene as the cause of CF Recent developments in gene editing technologies are creating the potential to correct these causative mutations at their source. We are truly grateful for the support of this Cystic Fibrosis Foundation award which will allow us to apply our Dualase™ gene editing technology to several frequent CFTR nonsense mutations."
Brent Stead, CEO of Specific Biologics Inc
Dualase™ cuts DNA differently than existing gene editors. This feature enables the high frequency insertion of new sequences into precise locations in the human genome. One can think of gene editing like a word processor that can find a specific spelling mistake in billions of letters and replace it with a correction. Similarly, inside a cell, Dualase™ can find a specific spelling mistake in the genome and replace it with the corrected sequence. Specific will deliver its gene editing technology using an inhalable lipid nanoparticle carrier, which will aid in delivering the gene editing machinery inside the cells of the lung. It is believed that if the CFTR nonsense mutations are corrected in enough cells, a gene editing therapeutic could provide a long-lasting benefit for CF patients.
The award will help fund preclinical testing in disease relevant models.
About Specific Biologics Inc.
Specific Biologics Inc. is a venture-backed early-stage biotechnology company on a mission to develop novel gene editing technologies to treat diseases through precision gene editing. Our two-site Dualase™ platform gene editors cut DNA in a way that optimally exploits the cell's naturally occurring DNA repair pathways. This enables two gene editing outcomes, precise DNA deletions to disrupt genes or increased repair to correct genes. Specific also develops lipid nanoparticles to deliver the gene editor to target cells and is developing a pipeline of Dualase™-based therapeutics in areas of high unmet medical need.
CELL AND GENE THERAPY
WuXi AppTec | March 08, 2022
WuXi Advanced Therapies (WuXi ATU), a wholly owned subsidiary of WuXi AppTec, today announced the launch of Tetracycline-Enabled Self-Silencing Adenovirus (TESSA™). This technology is a state-of-the-art novel process for transfection-free, scalable manufacture of adeno-associated virus (AAV) at Good Manufacturing Practice (GMP) grade. Developed by OXGENE in the United Kingdom, a WuXi Advanced Therapies company, TESSA™ will expedite AAV manufacturing and significantly reduce the cost for manufacturing cell and gene therapies, enabling global customers to deliver more accessible ground-breaking therapeutics to patients as quickly as possible.
AAV vector is a popular tool for the delivery of gene therapies in diseases such as haemophilia and Alzheimer's. Efficient processes for manufacturing AAV on a large scale are critical to meeting increasing industry demands. TESSA™ vectors can meet the scalability challenges of AAV production, which has significant implications for expanding patient access to these novel therapeutics.
The new data published in Nature Communications supports the benefits of this technology. It highlights that in the same manufacturing volume, TESSA™ vectors produced 10 times more AAV than plasmid-based manufacture, producing enough material to treat ten times the number of patients.
The TESSA™ system is a game changer for the manufacture of AAV and the advancement of cell and gene therapies to benefit patients. By improving scalability, reducing process complexity and lowering the costs of gene therapy manufacture, TESSA™ will better enable our global partners to develop and deliver life-saving gene therapies faster to more patients in need."
Dr. Ryan Cawood, Chief Scientific Officer of WuXi ATU.
As a Contract Testing Development and Manufacturing Organization (CTDMO) with operations globally, WuXi ATU's unique business model integrates powerful testing capabilities with its advanced therapies' process development and manufacturing platforms, such as TESSA™ technology for AAV manufacturing and Lenti stable solutions for lentiviral manufacturing. This allows all assay development, biosafety, viral clearance and product release testing to be completed in-house, shortening customers' timelines for the approval of advanced therapies.
About WuXi Advanced Therapies (WuXi ATU)
As the advanced therapies business unit of WuXi AppTec, WuXi Advanced Therapies is a Contract Testing, Development and Manufacturing Organization (CTDMO) that offers integrated platforms to transform the discovery, development, testing, manufacturing, and commercialization of cell and gene therapies. Our services and solutions accelerate time to market and support customer programs around the world.
Provectus Algae | August 28, 2021
Provectus Algae, an Australian biotechnology platform company specialising in the biomanufacturing of high-value compounds for a wide array of industries using microalgae, announced they will be expanding into an additional large-scale manufacturing facility.
“Since closing our seed round last year, we have been in full swing growing the team, developing products and building capacity with our proprietary production system. We are now in a strong position as we push into this next phase of the company's growth”
Located in Noosaville, Australia, the new site is expected to be operational by the end of 2021 and will complement the company’s existing facility to deliver new algae-made products to the market. The new facility will support the production of a high-performance food colouring which is currently in late stage of development. Once completed, the facility will have a 200,000 litre production capacity and expanded R&D operations to support early-stage product development and partnerships for the Food & Beverage sector. Provectus’s inaugural pilot facility will subsequently be converted to support the commercial production of a separate high-value product that is currently undergoing product testing. The company expects further expansion in the near term with plans for a supplementary 1 million litre facility already underway to support the commercialisation of multiple products already in the company development pipeline.
Provectus has engaged global food & beverage companies to craft a tailored development pipeline of products most relevant to the strengths of the company’s biomanufacturing platform. The flexibility of the company’s unique platform approach is evidenced by the speed at which lead products have moved through the development pipeline to commercial readiness.
“This is just the tip of the iceberg for Provectus Algae. The stage of development we are now at with our production platform gives us a clear line of sight to deliver some really exciting products to the food and beverage marketplace,” said Nusqe Spanton, Founder and CEO of Provectus Algae. “What we are able to achieve for our commercial partners is simply not comparable to any other offering in the marketplace. Thanks to algae, we are able to benefit from a 3.5-billion-year head start in producing some of the world’s most coveted ingredients.”
The Provectus platform addresses supply chain challenges by providing end-to-end services from product development to large-scale biomanufacturing. Importantly, the manufacturing platform is able to go from bench to industrial-scale seamlessly with a continuity of their novel production technology.
“Since closing our seed round last year, we have been in full swing growing the team, developing products and building capacity with our proprietary production system. We are now in a strong position as we push into this next phase of the company's growth,” Spanton added.
ABOUT PROVECTUS ALGAE
Provectus Algae is an Australian biotech company that programs algae to produce the world's most valuable specialty ingredients. By accelerating nature, their team is able to optimise unique microalgae to produce a whole new range of natural products, in a process described as “precision photosynthesis”. Using next-generation techniques the company has also developed a synthetic biology stack that complements and competes with existing production platforms to deliver high-performance and sustainable products.