Tessera Therapeutics Presents Non-Human Primate (NHP) Data in Alpha-1 Antitrypsin Deficiency (AATD) Program at the Federation of American Societies for Experimental Biology Meeting on Genome Engineering
- First-time demonstration in NHPs of therapeutically relevant levels of editing at the SERPINA1 locus responsible for AATD with a single dose of RNA Gene Writer
- Dr. Cecilia Cotta-Ramusino, Tessera’s Senior Vice President of Technology Development and Head of Platform, highlighted these data in an invited presentation on June 19th at 5:25 pm CEST
/EIN News/ -- SOMERVILLE, Mass., June 20, 2024 (GLOBE NEWSWIRE) -- Tessera Therapeutics, a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, presented progress across two of its preclinical programs, including first-time demonstration in NHPs of efficient in vivo rewriting at the SERPINA1 locus responsible for AATD. Genomic DNA analysis performed on whole liver samples taken from NHPs after a single administration of RNA Gene Writer showed an estimated 56% rewriting efficiency in hepatocytes1 on average. A supporting cDNA analysis showed that an average of 64% of mRNA transcripts contained the edited sequence. These NHP data build upon the highly efficient in vivo correction observed across two AATD mouse disease models presented recently at the American Society of Gene and Cell Therapy 27th Annual Meeting. Additional data were presented on in vitro and in vivo correction of the pathogenic mutations responsible for AATD and sickle cell disease (SCD). These data were shared in an oral presentation at the Federation of American Societies for Experimental Biology (FASEB) Meeting of Genome Engineering: Research and Applications taking place in Rome, Italy, June 16 – 20, 2024.
“To our knowledge, this is the first proof-of-concept demonstration in NHPs of in vivo genome editing of the locus responsible for AATD,” said Michael Severino, M.D., CEO of Tessera Therapeutics. “We are encouraged by the progress we have made in achieving therapeutically relevant levels of rewriting in NHPs after a single intravenous injection, which resulted in levels of edited mRNA that exceeded those of normal transcripts seen in asymptomatic carriers. We are excited to continue rapidly advancing our program for AATD towards the clinic. Our goal is to offer patients with AATD a potentially curative therapy by restoring the native gene locus to the wild-type sequence to address both the lung and liver manifestations of AATD.”
Tessera’s Senior Vice President of Technology Development and Head of Platform, Cecilia Cotta-Ramusino, Ph.D., discussed these data in an oral presentation titled “Writing DNA with RNA: Genome Engineering by Target-Primed Reverse Transcription” on June 19th at 5:25 pm CEST during the Session VI: Delivery and In Vivo Genome Editing.
1 Based on the assumption that 60% of liver cells are comprised of hepatocytes
About Tessera Therapeutics
Tessera Therapeutics is pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, with the aim to unlock broad new therapeutic frontiers. Our Gene Writing platform is designed to write therapeutic messages into the genome by efficiently changing single or multiple DNA base pairs, precisely correcting insertions or deletions, or adding exon-length sequences and whole genes. Our proprietary lipid nanoparticle delivery platform is designed to enable the in vivo delivery of RNA to targeted cell types. We believe our Gene Writing and delivery platforms will enable transformative genetic medicines to not only cure diseases that arise from errors in a single gene, but also modify inherited risk factors for common diseases and create engineered cells to treat cancer and potentially autoimmune and other diseases. Tessera Therapeutics was founded in 2018 by Flagship Pioneering, a life sciences innovation enterprise that conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability.
For more information about Tessera, please visit www.tesseratherapeutics.com.
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