The CasPlus Gene Editing Platform Technology can Correct Mutations Associated with Duchenne's Muscular Dystrophy and Cystic Fibrosis with High Efficiency and Exceptionally Low Rates of On-Target DNA Damages

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 25^th 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. 

Contact: Patricia Tolete, [email protected]

SOURCE Script Biosciences Inc