Synthetic DNA material to be used in upcoming approved Huntington's disease study in France may be key to scaling gene therapy production
SAN SEBASTIAN, Spain, Sept. 27, 2022 /PRNewswire/ -- TAAV Biomanufacturing Solutions, SL (TAAV) announced today the anticipated first use in a clinical trial of company-manufactured doggybone DNA™ (dbDNA™)1, a precisely engineered synthetic DNA material. TAAV-produced dbDNA™ is a component of an Asklepios BioPharmaceutical, Inc. (AskBio) gene therapy candidate for the treatment of early-stage Huntington's disease, which was recently authorized by France's National Agency for Safety of Medicines and Health Products (ANSM) for an upcoming Phase I/II clinical trial.
TAAV's dbDNA™ is produced using a synthetic manufacturing process that eliminates the use of fermenters and bacteria, which are required in more common plasmid DNA manufacturing. Synthetic manufacturing removes significant levels of bacterial contaminants, resulting in the production of a synthetic DNA product with an improved safety profile for the manufacture of recombinant adeno-associated virus (rAAV) gene therapy vectors2.
"The successful completion of this first clinical trial involving TAAV-manufactured dbDNA™ would mark a significant milestone for TAAV and bring us one step closer to commercial validation of this groundbreaking technology," said Alfredo Martinez, TAAV's Chief Operating Officer. "Clinical success of this first treatment would put us on the path to potentially increasing access to this gene therapy with a safer, more effective DNA material that can be produced faster, in much higher volumes and at lower costs. We look forward to that and to doing the same for others in the future."
The synthetic manufacturing process consistently produces large amounts of dbDNA™ in reduced times, which can be measured in days versus the months that are typical when producing plasmid DNA. Further, since dbDNA™ does not contain a plasmid backbone, it significantly reduces the risk of introducing bacterial DNA sequences into patients, improving the overall quality of gene therapy products.
"We knew very early on that manufacturing at scale and at the lowest cost possible would be central to realizing the full promise of gene therapies," stated Sheila Mikhail, CEO and Co-Founder of AskBio, TAAV's parent company. "Products like dbDNA™ could transform how AskBio and others efficiently and effectively increase availability of AAV therapeutics, creating a new industry standard for transfection-based AAV therapeutic manufacturing."
TAAV produces dbDNA™ as a lower cost, higher yield alternative to plasmid DNA and makes this available for use by all biotechnology companies across the world who are bringing life changing AAV gene therapies to the clinic.
TAAV Biomanufacturing Solutions, SL (TAAV), a wholly owned subsidiary of Asklepios Biopharmaceuticals (AskBio) and Bayer AG, is a cGMP manufacturer of doggybone DNA™ (dbDNA™), a Synthetic DNA material used for adeno associated virus (AAV) gene therapies. dbDNA™ is manufactured using an enzymatic process and produced under ISO classified production suites following GMP standards for research, clinical and commercial applications of synthetic DNA in AAV therapeutic vectors. Synthetic dbDNA™ is as an alternative to plasmid DNA, commonly used in AAV manufacturing, and leads to higher yields of DNA material. The use of synthetic dbDNA™ material can significantly shorten manufacturing timelines and facilitate faster production of AAV with an increased safety profile by eliminating residual bacterial sequences of plasmid DNA in the AAV product. TAAV was founded in 2020 and became 100% wholly owned by AskBio in 2022. Company headquarters, manufacturing facilities and labs are in San Sebastian, Spain. For more information, go to taav.com.
1doggybone™, doggybone DNA™ and dbDNA are trademarks of Touchlight Genetics Limited.2Technology for making dbDNA™ is licensed from Touchlight IP Ltd.
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