First approval of TAAV synthetic DNA material for use in clinical trial

2022-09-27

基因疗法

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.

About TAAV

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.

TAAV Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding TAAV's manufacturing technology and process. These forward-looking statements involve risks and uncertainties, many of which are beyond TAAV's control. Known risks include, among others: TAAV may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory requirements, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and for the production its product, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office or European drug authorities. Any of the foregoing risks could materially and adversely affect TAAV business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. TAAV or its parent companies do not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

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SOURCE TAAV