NEW
YORK, May 17, 2022 /PRNewswire/ -- SCRIPT
BIOSCIENCES INC, a biotechnology company developing curative
therapies using a novel gene editing platform
(CasPlus), and NYU Grossman School of Medicine,
presented a poster titled "Enhancement of Predictable and
Template-free Gene Editing by the Association of CAS with DNA
Polymerase" at the 25th annual American Society
of Gene & Cell Therapy meeting in Washington DC (May
16-19, 2022). Highlights of the data include:
- Development of CasPlus, a novel CRISPR/Cas based technology
that engages a DNA polymerase for high efficiency exogenous
template-free DNA repair of Cas9-induced double strand breaks, and
prevents large on-target DNA deletions and rearrangements.
Given that large DNA deletions are a major safety concern for first
generation CRISPR/Cas9 editing strategies, CasPlus provides a safer
and more efficient next-generation gene editing approach.
- Duchenne's Muscular Dystrophy (DMD): CasPlus successfully
rescued dystrophin expression in cells by restoring the reading
frame of dystrophin genes harboring frameshift mutations in exons
51 and 53. Editing efficiencies at the desired site were ~3-fold
greater with CasPlus than commonly used CRISPR/Cas9 system; no
Cas-9 induced on-target large deletions was observed.
- Uniquely CasPlus can correct the F508del mutation in the CFTR
gene that causes >80% of Cystic Fibrosis (CF) cases. Precise
insertion of three base pairs was achieved with an efficiency of
~25%, restoring the phenylalanine-encoding codon at position 508
without an exogenous DNA template.
Dr. Chengzu Long, Principal Investigator and Assistant
Professor, Division of Cardiology and The Helen and Martin Kimmel
Center for Stem Cell Biology, NYU Grossman School of Medicine,
said, "CRISPR/Cas9-mediated on-target DNA damage is an
underappreciated risk factor for safe application of genome editing
tools. While numerous approaches have already been developed to
reduce well-known off-target effects of CRISPR-mediated editing,
the CasPlus system prevents CRISPR-mediated collateral
on-target large deletions and thus offers the promise of
safer therapeutic gene editing in humans."
Anil Namboodiripad, Ph.D., CEO of Script Biosciences said, "We
are encouraged by this data and it is a step forward in our mission
to develop lasting cures for patients suffering from serious
diseases. In vivo translational studies in humanized mouse models
are ongoing. We plan to advance our lead program in DMD
towards IND-enabling studies, while also expanding our pipeline to
other gene targets".
About Script Biosciences
Script Biosciences (www.scriptbiosciences.com) is an early stage
biotechnology company committed to developing safe, efficient, and
durable cures for genetic diseases with few or no treatment options
using our proprietary CasPlus genome editing platform
(CasPlus). CasPlus corrects mutations by predictable and
efficient insertion of base pairs. The superior precision of
CasPlus paves the way towards safer genome editing in humans. The
technology was developed at New York
University with a worldwide license granted to
Script.
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SOURCE Script Biosciences Inc