Tome Biosciences Presented Data on its Programmable Genomic Integration Technologies at ASGCT
2024年5月10日 - 7:00PM
Tome Biosciences, Inc., the programmable genomic integration (PGI)
company, today presented preclinical data highlighting its progress
in developing PGI as integrative gene and cell therapies at ASGCT’s
27th Annual Meeting in Baltimore, MD.
“Our presentations at ASGCT provided a snapshot
of the broad capabilities afforded by PGI technologies, from rapid,
efficient and multiplex editing of iPSCs and HSCs to targeted
integration of functional genes at therapeutically-relevant levels
in non-human primates,” said John Finn, PhD, Chief Scientific
Officer of Tome.
The Company is developing two technologies
capable of PGI: integrase-mediated PGI (I-PGI), which is best
suited for integrating large DNA sequences (>1,000 base pairs)
and ligase-mediated PGI (L-PGI), which is best suited for
integrating small DNA sequences (1-100s of base pairs). I-PGI works
by first inserting a unique sequence into the genome in a
user-defined location. This sequence provides a beacon for a
proprietary integrase to recognize and subsequently insert the
provided DNA sequence of interest. L-PGI can insert any small
template DNA sequence into the genome at a user-defined location.
Both technologies can edit the genome without depending on a double
stranded DNA break.
Progress on these technologies was presented at
ASGCT.
- Unveiled the Company’s lead
programs in integrative gene therapy as PKU and cell therapy as a
CD19/BCMA CAR-iNK for renal autoimmune diseases.
- Reported progress on liver
technical development for PKU, demonstrating efficient I-PGI gene
integration and functional gene expression across multiple loci and
transgenes in vitro in primary human hepatocytes and in vivo in
mice and non-human primates. In all cases, including the early
non-human primate data, the Company has achieved potentially
curative editing efficiencies for PKU.
- Reported progress on the Company’s
CD19/BCMA CAR-iNK program, showing efficient I-PGI integration of
12,000 base pairs into iPSCs, and demonstrating functional
expression in the differentiated iNK of genes designed to enhance
the efficacy and convenience of an autoimmune cell therapy, with
the ability to re-dose and reverse the treatment. The data
presented included both in vivo evidence of CD19 killing in mice
and in vitro B cell killing in blood samples from patients with
lupus.
- Reported on previously undescribed
challenges with reverse transcriptase-mediated writing of long
(~40bp) sequences in rodent non-dividing cells and potential
solutions.
- Showcased the ability of I-PGI to
efficiently edit iPSCs and HSCs, including integrating >30,000
base pairs and multiplexing four different genes at four different
loci, which enables the rapid creation of complex cell
circuitry.
- Reported the development of
proprietary assays designed to detect potential off-target editing
of integrases, including double stranded breaks (DSBs), indels,
cryptic integrations and chromosomal rearrangements.
- Demonstrated L-PGI efficiently
edited primary human hepatocytes in vitro across multiple
therapeutically-relevant genomic loci, and these edits showed
higher fidelity versus DNA edits using reverse transcriptase.
Copies of the posters and presentations will be
available on Tome’s website, www.tome.bio, following the
conference.
About TomeTome Biosciences,
Inc., is the programmable genomic integration (PGI) company. Our
technologies allow us to insert any genetic sequence of any size at
any location in the genome with site-specific precision. We are
writing the final chapter in genomic medicines, delivering cures to
patients through cell and integrative gene therapies. Follow us on
X @Tome_Bio and on LinkedIn. www.tome.bio.
PGI™, I-PGI™ and L-PGI™ are brand names and
technologies of Tome Biosciences, Inc.
Contacts:
For media:CG LifeCGL.TomeBio@cglife.com
For investors:Michelle Avery, PhD, SVP,
Corporate Affairsinvestors@tome.bio