Amsterdam, The Netherlands – May 7, 2025
Ascend Advanced Therapies and Annogen will combine their expertise in AAV biology, vector manufacturing and genetic engineering to improve the quality of recombinant AAV particles used in gene therapy applications.
While AAV remains the gene therapy vector of choice for in-vivo gene delivery, and much effort has gone into improving AAV vector yields, the sector is now becoming more aware of the importance of vector quality, which often suffers when platforms are optimized for yields.
The collaborative effort announced today is aimed at optimizing sequences in the rep open reading frame to further improve the full/empty capsid ratio during upstream manufacturing which from a scientific point a view could provide an increase in vector genome yields but also, more potent gene therapy vectors and more effective gene therapies.
Annogen supports advanced therapy manufacturers in building and optimizing gene expression systems including promoters and expression loci. In previous work at Annogen the SuRE™ platform was used to perform saturation mutagenesis screen of cis-regulatory elements in the rep ORF, thereby measuring the functional consequence of each single nucleotide mutation. This exhaustive interrogation identified clear repressor and activator domains and mutations affecting these.
Ascend is a specialist AAV development partner working to support the next generation of life-science innovators in bringing gene therapy products to patients. Ascend’s preclinical labs are in Munich, Germany, with clinical, commercial and fill-finish capabilities located in Rockville, MD and Alachua, Florida. Their team of experts will help identify mutations that lead to more effective AAV genome encapsidation and can translate to improved gene therapy vectors containing full-length genomes.
“Building on from the initial proof-of-concept work we performed to better understand the genetic underpinnings of cap, helper and rep isoform expression during AAV production, we are grateful to now partner with experts at Ascend to apply these findings to improve vector quality, efficacy and safety”, said Dr. Joris van Arensbergen, founder and CEO of Annogen.
Ascend’s CSO Markus Hoerer added that, “although our split 2-plasmid AAV production system (EpyQ) already produces very high-quality particles at good yields, in a field that is moving quickly, we want to stay ahead of the curve. For this reason, we have been investing in development projects that improve AAV yield but never at the cost of quality”.
About Ascend
Ascend Advanced Therapies (Ascend) is a gene to GMP CDMO specializing in high-quality, cost-effective advanced therapy development and manufacturing. Established in clinical AAV innovation since 1991, Ascend and ABL, Inc. merged in late 2024 to offer expanded commercial facilities and a versatile platform for gene therapies, oncolytics, vaccines and immunotherapies. A collaborative CMC approach backed by therapeutic development roots helps the team guide clients from clinic to commercialization while balancing yield, quality and cost. Investors include; EW Healthcare Partners Monograph, Abingworth, Petrichor, DCVC Bio, 4BIO Capital, Cathay Health, Ajinomoto, Deerfield, Digitalis Ventures. Learn more at www.ascend-adv.com.
About Annogen
At Annogen we serve biopharmaceutical and agricultural companies with bespoke gene expression engineering. We use our SuRE™ technology to develop cis-regulatory DNA elements to be used for controlled gene expression and to study the impact of non-coding mutations for thousands of variants in parallel. In addition, we offer the AIM™ service to identify favorable gene insertions and their expression levels for more than 100,000 integrations in parallel. Combined, these platforms have demonstrated substantial improvements towards the development of tissue-specific and condition-specific gene expression that is required for the next generation of therapies and crops. For more information, please visit www.annogen.bio.
