GOX

Oral presentation at ASN on lead program, ABO-101 for primary hyperoxaluria type 1 (PH1), supporting in vivo proof of pharmacology. On-track for year-end IND/CTA filing.Oral presentation at ALS One and a poster presentation at ESGCT on lead ALS program ABO-202, highlighting in vivo reversal of ALS-associated STMN2 aberrant splicing and phenotypic outcomes in human motor neurons.Poster presentation at ESGCT demonstrating targeted gene knock-in and broader applicability of type V nuclease platform for monogenic diseases. CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announced oral and poster presentations on pipeline and early discovery programs at upcoming medical and scientific meetings: American Society of Nephrology (ASN) 2024 Kidney Week Meeting, October 23-27 in San Diego, California31st Annual European Society of Gene and Cell Therapy (ESGCT), October 22-25 in Rome, ItalyALS One 7th Annual ALS Research Symposium, November 13-15, Virtual At ASN Kidney Week, Arbor will present in vivo non-human primate (NHP) proof-of-pharmacology data supporting continued development of ABO-101, its most advanced gene editing therapeutic candidate designed to address primary hyperoxaluria type 1 (PH1) through inactivation of the HAO1 gene in the liver. Arbor is on track to file the IND/CTA for ABO-101 in PH1 by year-end. At the ALS One 7th Annual ALS Research Symposium and at ESGCT, Arbor will detail data on its lead ALS program ABO-202, supporting genetic disruption of STMN2, preventing errors in the processing of its mRNA, and elucidating a path towards proof of concept for a therapeutic CRISPR gene editing approach for the treatment of amyotrophic lateral sclerosis (ALS). The company will also showcase novel data at ESGCT demonstrating targeted gene knock-in at an endogenous promoter achieving equivalent or improved knock-in efficiency when compared to type II nucleases. This data supports the efficiency and breadth of applicability of Arbor’s type V nucleases to monogenic diseases. Details for the presentations are as follows: ASN 2024 Kidney Week:Oral Presentation Title: Development of ABO-101, A Novel Gene Editing Therapy for Primary Hyperoxaluria Type 1 Session Date and Time: Thursday, October 24, 2024, 4:30-6:00pm PDT Location: Room 23, Convention Center Presenter: Winston Yan, MD, PhD Abstract Number: TH-OR88 ESGCT 2024:Poster Title: Gene Editing with a Type V CRISPR-Cas Enzyme Disrupting the Aberrant Splicing of STMN2 as a Potential Treatment for ALS Session Date and Time: Poster Session 1, October 22, 7:30-9:00pm CEST Location: Forum, La Nuvola Presenter: Chee Yeun Chung, PhD Abstract Number: P0327 Poster Title: Targeted Knock-In at the Human TTR Locus Using Type V Nucleases Session Date and Time: Poster Session 3, October 24, 2:00-3:30pm CEST Location: Concourse Level 1 and Mezzanine Concourse, La Nuvola Presenter: Whitney J. French, PhD Abstract Number: P0603 ALS One 7th Annual ALS Research Symposium:Oral Presentation Title: Gene Editing with a Type V CRISPR-Cas Enzyme Disrupting the Aberrant Splicing of STMN2 as a Potential Treatment for ALS Session Date and Time: November 14, 3:40-4:00pm EDT Presenter: Priscilla D. Negraes, PhD About Arbor Biotechnologies, Inc.Arbor BiotechnologiesTM, a next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions - from the ultra-rare to the most common genetic diseases. Our unique suite of optimized gene editors goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need. We are rapidly advancing our lead program in primary hyperoxaluria type 1 to the clinic and focusing our research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures. For more information, please visit: arbor.bio Media Contact:Peg RusconiDeerfield Groupprusconi@deerfieldgroup.com

Arbor Biotechnologies is trimming its early-stage discovery work, leading to layoffs in the group. The move was confirmed by an Arbor spokesperson, with CEO Devyn Smith saying in a statement to Endpoints News on Friday that the company is prioritizing clinical-stage investments. “We are keenly focused on delivering new medicines and will strategically shift resources and efforts accordingly to support the transition to a clinical-stage company,” Smith said. The company did not disclose how many employees were laid off. The gene editing company was born out of the lab of CRISPR scientist Feng Zhang and launched in 2018 with a $15.6 million Series A. As the potential of gene editing grew, the company attracted more investors, closing a $215 million Series B round in 2021. The financing was co-led by Temasek, Ally Bridge Group, and TCG Crossover. The company said then that the capital would be used not only to advance programs into the clinic, but also to continue investing in a “novel discovery engine to develop the next generation of gene editing technology.” Earlier this year, Arbor bought another Zhang-founded biotech , Serendipity Bioscience, though financial details about the acquisition were undisclosed. The purchase allowed Arbor access to additional gene editing technology based around enzymes that are smaller than Cas9. The hope is that these could be used in different delivery methods that can’t accommodate the Cas9 protein. Arbor’s lead program, ABO-101, is currently in IND-enabling studies as a treatment for primary hyperoxaluria type 1 (PH1), a condition marked by abnormally high levels of oxalate in urine. This can increase the risk of certain kinds of kidney stones and in severe cases can cause kidney failure. Arbor presented preclinical data of ABO-101 in May, finding that it had “highly specific targeting of the HAO1 gene” in non-human primates that produced a “therapeutically relevant reduction in urinary oxalate.” Another liver-aimed program to treat transthyretin amyloidosis (ATTR) remains in the discovery stage. Three other CNS programs are also listed in the program, two of which are in the lead optimization phase. All three are being developed as potential treatments for amyotrophic lateral sclerosis.

SHANGHAI, Aug. 22, 2024 /PRNewswire/ -- YolTech Therapeutics, a pioneering clinical-stage gene editing company dedicated to delivering lifelong cures, today announced a major milestone: the first patient has been dosed with YOLT-203, the company's cutting-edge in vivo genome editing candidate. This marks the commencement of an Investigator-Initiated Trial (IIT) and represents a global first in the application of in vivo gene editing therapies for Primary Hyperoxaluria Type 1 (PH1). YOLT-203 is designed as a single-dose, potentially curative therapy, harnessing the power of YolTech's proprietary YolCas12™ editor. This trial sets a new standard in the field, pushing the boundaries of what gene editing can achieve in treating life-threatening genetic diseases. Primary hyperoxaluria is a genetic disorder associated with hepatic oxalate overproduction, leading to increased renal oxalate excretion. PH1 as the most commonly diagnosed subtype, is a childhood-onset autosomal recessive disease form caused by AGXT gene mutations and absence or dysfunction of alanine-glyoxylate aminotransferase. Most patients ultimately progress to renal failure and require intensive hemodialysis as a bridge to a dual liver/kidney transplant to resolve the metabolic defect in the liver and replace the terminally damaged kidneys. YOLT-203, independently developed by YolTech, is powered by YolTech's proprietary YolCas12™ editor, a novel CRISPR/Cas gene editing tool developed through YolTech's metagenomics discovery and protein engineering platform named HEPDONE® (High-Throughput Evolution Platform for Discovery and Optimization of Novel Editors) system. YolCas12™ has demonstrated significantly high gene editing activity in both prokaryotic and eukaryotic systems and has been proven to achieve high-efficiency in vivo editing via LNP-mRNA delivery in mice and non-human primates. YoLT-203 is utilizing YolCas12™ editor to target HAO1 gene, significantly preventing pathogenic protein expression in pre-clinical study, promising to provide effective treatment options for PH1 child and adult patients. The clinical study of YOLT-203, initiated by investigators, is a single-arm, open-label, dose-escalation clinical trial, aiming to evaluate the safety and tolerability of YOLT-203 in the Chinese population with PH1. 7 subjects are estimated to be enrolled. The first adult subject received the dose on 5th Aug 2024 and the first child patient were treated on 20th Aug 2024. This trial represents a pioneering effort as the first global clinical trial of an in vivo gene editing therapy targeting PH1. "PH1 is a devastating disease that can lead to life-threatening kidney damage and systemic complications," said Dr. Yuxuan Wu, Founder and CEO of YolTech. "As the first in vivo gene editing therapy for PH1, YOLT-203, using our proprietary YolCas12™ editor, has the potential to transform treatment options for patients suffering from this debilitating disease. We believe YOLT-203 is expected to bring significant clinical benefits to both children and adults affected by this condition, and we look forward to sharing and reporting more data from this program in the future." YolTech remains committed to progressing the clinical development of YOLT-203 and working closely with regulatory authorities, healthcare professionals, and patient advocacy groups to further the advancement of gene editing therapies. **About YOLT-203** YOLT-203 is an innovative in vivo gene editing therapy designed using YolTech's proprietary YolCas12™ system, a novel CRISPR/Cas tool developed through the High-Throughput Evolution Platform (HEPDONE®). This therapy is encapsulated within lipid nanoparticles (LNPs) that facilitate targeted delivery to the liver, where it addresses the underlying genetic mutations responsible for primary hyperoxaluria type 1 (PH1). Upon intravenous administration, the LNPs are recognized and internalized by liver cells. Once inside, the YolCas12™ editor protein, guided by the corresponding guide RNA (gRNA), targets and corrects mutations in the AGXT gene, aiming to reduce harmful oxalate levels in the blood, thereby offering a potential one-time, curative treatment for PH1. **About YOLT-203-101 Study** The YOLT-203-101 trial is a single-arm, open-label, single-dose, dose-escalation clinical study designed to evaluate the safety and tolerability of YOLT-203 in Chinese patients diagnosed with primary hyperoxaluria type 1 (PH1). The study will also preliminarily assess the impact of a single administration of YOLT-203 on plasma oxalate levels, a critical biomarker of therapeutic efficacy in PH1. The trial's dose-escalation phase will involve three dose groups, employing an accelerated titration approach to escalate doses from the starting level to the highest planned dose. Following the one-year main study, participants will enter a long-term follow-up period of fifteen years to monitor the sustained effects and safety of the treatment. **About YolTech** YolTech Therapeutics is a pioneering gene editing company dedicated to developing medicines delivering lifelong cures with serious diseases. YolTech has built a leading high-throughput evolution platform and innovative LNP delivery system. It possesses strong capabilities in novel Cas and base editor discovery and exceptional in-house LNP production capacity for GMP manufacturing, with independent intellectual property rights and core patent protection globally. YolTech's lead pipeline, YOLT-201, became the first LNP-mediated in vivo gene editing drug in China to enter registered clinical development in 2024. The company is also advancing gene editing therapies for familial hypercholesterolemia (FH) and primary hyperoxaluria type 1 (PH1) with promising early clinical results. For more information on the YOLT-203-101 trial, please visit: or follow YolTech on LinkedIn Contacts Colin LIN Business Development & Investor Relations [email protected] +86 180 2100 7750 SOURCE YolTech Therapeutics