October 24, 2024 — SOMERVILLE, Mass., Tessera Therapeutics presented new important preclinical works on the Gene Writing™ platform in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy.
The company also presented euphoric results of its in vivo gene editing programs targeting alpha-1 antitrypsin deficiency (AATD) and single-cell disease (SCD). Such advances represent an important milestone towards the application of AI-based technologies to improve the accuracy and safety of genome editing.
The data presented indicated that one Gene Writer™ treatment achieved 79% genomic modification in liver cells affected by AATD in non-human primates (NHPs).
“The continued development of our Gene Writing and delivery platforms have resulted in rewriting efficiencies in preclinical models exceeding what we anticipate will be curative levels for AATD and SCD,”
Said Michael Severino, M.D., CEO of Tessera Therapeutics.
“We believe these data represent an important step towards advancing potentially transformative in vivo genetic medicines for the treatment of two of the largest monogenic diseases, alpha-1 antitrypsin deficiency and sickle cell disease.”
These findings were strengthened by data that revealed 70 % editing efficiency in NHPs thus paving the way for in silico roi for AI-driven future clinical use.
Tessera further specified how new computational AI technologies assess the genomic safety profile related to their Gene Writing platform.
These technologies enhance specificity and minimize off-target effects, enabling new levels of gene therapy for rare diseases.
The presented data represents an advancement in the development of innovative approaches to gene therapy, as well as introducing the potential of AI-centered approaches in AATD and SCD treatment stem cells that are safe and effective.
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