Arbor Biotechnologies Presents Data Supporting Clinical Development of ABO-101 and Robust Potential of Platform to Enable Therapeutic Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting
09 Mayo 2024 - 5:00AM
Arbor Biotechnologies®, a biotechnology company discovering and
developing the next generation of genetic medicines, today presents
preclinical data demonstrating the potential of ABO-101, its novel
gene editing therapeutic designed to address primary hyperoxaluria
type 1 (PH1), as well as its broader discovery capabilities and
their application to enable novel gene editing therapeutics in a
suite of presentations at the 2024 American Society of Gene and
Cell Therapy (ASGCT) 27th Annual Meeting in Baltimore, Maryland.
“At Arbor, we are building upon the potential of CRISPR-Cas
approaches by leveraging our proprietary nuclease discovery and
development platform to enable efficient identification and
optimization of novel gene editing tools for a focus on translation
of these tools into therapeutics,” said Devyn Smith, Ph.D., Chief
Executive Officer of Arbor. “This approach has fueled our robust
pipeline of genetic medicines which address a range of liver and
CNS indications, including PH1 and ALS. The data presented at ASGCT
reinforce the vast potential of our approach and demonstrate the
tremendous progress we have made advancing our platform and
programs toward the clinic.”
In an oral presentation, Arbor shares in vivo data that
highlights the therapeutic potential of its lead program, ABO-101,
to address PH1. The data show highly specific targeting of the HAO1
gene in the liver and preservation of genomic integrity upon
editing. The results further demonstrate efficient in vivo editing
of HAO1 in a preclinical model of PH1, which resulted in a
corresponding, therapeutically relevant reduction in urinary
oxalate. ABO-101 was well tolerated in nonhuman primates (NHPs) at
multiple doses. NHP data confirmed the efficacy and pharmacology of
ABO-101, with efficient editing of HAO1 yielding reduced glycolate
oxidase activity and increased serum glycolate. Together, this
strong preclinical data package and the ongoing IND-enabling
studies support continued advancement of ABO-101 toward clinical
evaluation.
Preclinical data shared in a poster presentation confirm that
novel type V nucleases, discovered and optimized using Arbor’s
discovery platform, can prevent aberrant splicing of STMN2,
providing preclinical evidence of editing in CNS cells. In vitro
studies in cellular models and human motor neurons and in vivo
studies in STMN2 transgenic mice showed that STMN2 motif disruption
increased full-length STMN2 mRNA and decreased mRNA levels for the
aberrantly spliced novel exon of STMN2 (Exon 2A). These results
show that leveraging a novel nuclease capable of achieving targeted
deletions can reverse aberrant splicing of STMN2 and demonstrate
the therapeutic potential for an effective gene editing approach
for sporadic ALS in patients with TDP43 proteinopathy.
The company also highlights the potential of its end-to-end
nuclease discovery and optimization platform in a third
presentation outlining the identification of a unique, compact type
V nuclease. Arbor used structure-guided design to enhance activity
and specificity of a compact CRISPR-Cas type V-L system identified
via metagenomic search. The resulting nuclease, ABR-004,
demonstrated the ability to achieve potent, therapeutically
relevant silencing of PCSK9 in mice and non-human primates,
signaling opportunities for future development for broader
therapeutic applications. The data demonstrate the feasibility of
applying Arbor’s discovery and targeted nuclease optimization
capabilities to develop new gene editing tools with the potential
to power the next generation of genomic medicines.
David Cheng, Chief Technology Officer at Arbor, also
participated in the scientific symposium, delivering a presentation
on the significance and future of AI in the discovery and
development of new genomic medicines as part of a session titled
The Impact of Artificial Intelligence (AI) on CGT.
Details for the presentations are as follows:Scientific
Symposium: The Impact of Generative Artificial
Intelligence (AI) on CGT
Presentation Title:
Generative AI Progress and Applications for Cell and Gene
TherapiesSession Date and Time: Wednesday, May 8,
2024, 8:00-8:25 am ETLocation: Room
318-323Presenter: David R. Cheng
Oral Presentation Title: Development Of
ABO-101, A Novel Gene Editing Therapy For Primary Hyperoxaluria
Type 1
Abstract Number:
165Session Date and Time: Thursday, May 9, 2024,
2:00-2:15 pm ETLocation: Room
307-308Presenter: Tia DiTommaso, PhD
Oral Presentation Title: Identification
and Engineering of ABR-004, a Compact, High-fidelity Nuclease for
Therapeutic Gene Editing
Abstract Number:
150Session Date and Time: Thursday, May 9, 2024,
1:30-1:47 pm ETLocation: Ballroom
3Presenter: Jeffrey Haswell, PhD
Poster Title: Disruption of Aberrant
Splicing of STMN2 by Gene Editing with a Type V CRISPR-Cas Enzyme
as a Potential Treatment for ALS
Abstract Number:
1598Session Date and Time: Friday, May 10, 2024,
12:00-7:00 pm ETLocation: Exhibit
HallPresenter: Jace Jones-Tabah, PhD
About Arbor Biotechnologies®Arbor
Biotechnologies is a next-generation gene editing company based in
Cambridge, MA. Combining the promise of CRISPR with advanced
computational AI-driven discovery, high throughput screening, and
robust protein engineering approaches, our co-founders Feng Zhang
and David Walt laid the groundwork for our proprietary discovery
engine, which has yielded an extensive toolbox of gene editors, far
exceeding the number of editors published in the literature to
date. We envision a future of gene editing that extends beyond
simple knockdowns to include precision writing, precise excisions
and large insertions. This affords us the potential to treat a
broad spectrum of patients, from ultra-rare to the most common
genetic diseases. Guided by a deep understanding of the molecular
basis of disease and our access to a unique suite of optimized
editors, we are rapidly advancing our discovery-stage programs with
an initial focus on genomic diseases of the liver and CNS for which
there are no existing functional cures. As we advance toward the
clinic with our lead program in primary hyperoxaluria type I, we
look to expand our strategic partnerships around in vivo gene
editing across multiple therapeutic areas and ex vivo cell therapy
programs to broaden the reach of our novel nuclease technology. For
more information, please visit: arbor.bio
Media Contact:Peg RusconiVerge Scientific
Communicationsprusconi@vergescientific.com