Tessera Therapeutics
RNA Gene Writing™ level demonstrates clinically applicable levels of in vivo rewriting successful the genome of liver cells of non-human primates pursuing a azygous administration
RNA Gene Writing™ level generates tumor-clearing CAR T cells by all-RNA delivery
RNA Gene Writers™ show programmable penning to facilitate targeted DNA integration and quality to marque tiny and ample changes astatine the aforesaid clip successful quality cells
DNA Gene Writing™ level promotes sustained and precocious levels of transgene look successful non-human primates, perchance extending the scope of accepted AAV-based cistron therapy
Proprietary LNP level demonstrates the quality to present to hematopoietic stem cells and T cells in vivo in some mice and non-human primates
Company to contiguous astatine the J.P. Morgan 41st Annual Healthcare Conference connected January 9th, 2023, astatine 1:30 p.m. PST
SOMERVILLE, Mass., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Tessera Therapeutics, the biotechnology institution pioneering a caller attack successful familial medicine known arsenic Gene Writing™, contiguous announced advancements crossed its Gene Writing™ and non-viral transportation platforms including information demonstrating the imaginable of Gene Writers™ to constitute therapeutic messages successful the genome of non-human primates (NHPs), to make tumor-clearing chimeric antigen receptor (CAR)-T cells by all-RNA delivery, and the quality to people non-viral transportation systems to hematopoietic stem cells (HSCs) and T cells. The Company volition item these information and different caller advancement successful a presumption astatine 1:30 p.m. PST contiguous astatine the 41st Annual J.P. Morgan Healthcare Conference taking spot successful San Francisco, California.
“These important caller information sets successful non-human primates correspond a pivotal milestone for our Company and talk to the immense imaginable of Gene Writing to marque astir immoderate benignant of familial alteration indispensable to code quality disease,” said Michael Severino, M.D., Chief Executive Officer of Tessera and CEO-partner of Flagship Pioneering. “Our preclinical studies show that we tin present our Gene Writers to the close location, successful the liver and beyond, and marque therapeutic alterations to the genome. In familial medicine, occurrence successful NHPs is highly predictive of occurrence successful quality trials, and with these data, we are 1 measurement person to bringing our caller familial medicines into the clinic.”
Michael Holmes, Ph.D., Chief Scientific Officer of Tessera, added, “While the fields of genome engineering and non-viral transportation person made important strides, they person not yet yielded the tools needed to safely present and broadly technologist the genome to thrust perchance curative changes successful people cells with precocious efficiency, specificity, and fidelity. Building connected nature’s mode of penning DNA, we tin technologist our Gene Writers to broadly constitute and rewrite DNA sequences successful the genome crossed a wide scope of compartment types in vivo, including liver, hematopoietic stem cells, and T cells with all-RNA compositions that licence the usage of non-viral transportation platforms. This represents a existent feat for the familial medicine field.”
Tessera is processing a wide acceptable of Gene Writer™ systems to marque astir immoderate benignant of familial alteration needed to dainty oregon cure disease. RNA Gene Writers™ are based connected retrotransposon biochemistry and usage people primed reverse transcription to technologist the genome successful divers ways, without making double-strand DNA breaks, utilizing all-RNA compositions of matter. The Company’s proprietary non-viral lipid nanoparticles (LNPs) are designed to present Gene Writers™ to targeted tissues, perchance enabling the exertion of Gene Writing™ therapies in vivo. DNA Gene Writers™ are based connected recombinase oregon transposase constituent biochemistry and person been engineered to integrate a therapeutically applicable payload of choice.
RNA Gene Writers™ Make Precise Alterations, Insert Exon- and Gene-Length Sequences Using All-RNA Compositions
Tessera’s RNA Gene Writers™ show the quality to marque a precise, azygous basal brace alteration in vivo astatine the causative locus for phenylketonuria (PKU) successful NHPs utilizing each RNA-LNP compositions of matter, achieving clinically applicable rewriting efficiencies, with up to 35% of alleles successful the liver edited 7 days aft a azygous intravenous administration. Additionally, information from studies successful different preclinical models show that RNA Gene Writers™ tin close to chaotic benignant the mutation that causes sickle compartment illness astatine a precocious efficiency, resulting successful the astir implicit conversion of macromolecule accumulation from mutated sickle-causing hemoglobin to mean hemoglobin.
Additional information show the quality to constitute longer sequences and full genes with precocious ratio and specificity. RNA Gene Writers™ were engineered to insert DNA sequences astatine defined people sites successful the genome, including the insertion of a 150-base brace series with single-nucleotide precision astatine efficiencies of up to 40% of alleles successful quality compartment lines. This suggests the imaginable for RNA Gene Writers™ to insert full exons oregon yet genes astatine their autochthonal tract to close familial loci wherever aggregate mutations whitethorn hap successful a diligent population. RNA Gene Writers™ person shown integration of functional CAR templates into superior quality T cells with all-RNA transportation and demonstrated potent tumor-killing enactment in vitro and in vivo in rodent tumor models. Furthermore, multiplexing – combined penning and rewriting to marque tiny and ample changes simultaneously – tin beryllium achieved and whitethorn amended alteration next-generation cell-based therapies.
DNA Gene Writers™ Drive Durable Transgene Expression
Tessera’s DNA Gene Writers™, erstwhile co-administered with an adeno-associated microorganism (AAV) containing the DNA template, promoted durable look of a transgene successful the liver of NHPs, resulting successful sustained look astatine levels 5x higher than achieved with an episomal AAV control, adjacent aft partial hepatectomy and contempt being delivered astatine one-fifth the dosage.
Novel LNP Delivery Highlights Potential for In Vivo Gene Writing successful Extrahepatic Tissues
In NHP studies, Tessera’s proprietary LNPs demonstrated the quality to present to HSCs and T cells in vivo with >30%, and >45% efficiency, respectively, demonstrating the imaginable for the instauration of additional successful vivo Gene Writing™ therapies.
The Company plans to contiguous these information astatine aboriginal technological conferences.
About Tessera’s Gene Writing™ Technology
Gene Writing™ tin marque imperishable therapeutic alterations to the quality genome, offering the imaginable for a caller class of familial medicines with wide therapeutic applications both in vivo and ex vivo. Tessera’s Gene Writer™ systems, which tin beryllium delivered arsenic RNA oregon DNA, alteration the correction of azygous nucleotides, the deletion oregon insertion of abbreviated DNA sequences, and the penning of exons oregon full genes into the genome. Gene Writers™ are based connected nature’s genome architects, mobile familial elements (MGEs)—the astir abundant people of genes crossed the histrion of life, representing astir fractional of the quality genome. Tessera’s probe motor further engineers the discovered Gene Writer candidates to optimize for efficiency, specificity, and fidelity—enabling the afloat spectrum of genome editing outcomes and bringing the anticipation of curing superior diseases with a familial ground person to reality.
About Tessera Therapeutics
Tessera Therapeutics is pioneering Gene Writing™, which consists of aggregate exertion platforms designed to connection scientists and clinicians the quality to constitute therapeutic messages into the quality genome, thereby perchance curing diseases astatine their source. Coupled with pioneering innovations successful non-viral transportation to present therapeutic messages wherever they are needed, Gene Writing™ enables the correction of azygous nucleotides, the deletion oregon insertion of abbreviated DNA sequences, and the penning of full genes into the genome, offering the imaginable for a caller class of therapeutics to recognize the committedness of familial medicine. Tessera Therapeutics was founded successful 2018 by Flagship Pioneering, a beingness sciences innovation endeavor that conceives, creates, resources, and develops first-in-category bioplatform companies to alteration quality wellness and sustainability.
For much accusation astir Tessera, delight visit www.tesseratherapeutics.com.
Contact
Anne Shelton, M.D., Ph.D.
LifeSci Communications, LLC
+1-734-276-2316
English (US)