ABO-101

Arbor Biotechnologies\' series C financing follows a $215 million series B in 2021.\n As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials.The series C was led by Arch Venture Partners and TCGX, with new investors QIA, Partners Investment, Revelation Partners and Kerna Ventures joining alongside other existing investors, according to a March 18 release.The raise will support ABO-101, a CRISPR-based gene editing therapy meant to treat primary hyperoxaluria type 1 (PH1). The FDA granted the therapy both orphan drug and rare pediatric disease designations in early February.A two-part phase 1 trial of ABO-101 is slated to start this month, according to ClinicalTrials.gov. The first part of the study will test single ascending doses of the gene therapy in adults with PH1, while the second portion will test a recommended dose in children with the disease.ABO-101 is Arbor’s first program to enter human trials.The new cash will also help other branches of Arbor’s pipeline, according to the release, including an editing program for an undisclosed rare liver disease and another program targeting amyotrophic lateral sclerosis. “Arbor is developing a differentiated portfolio with first-in-class potential to deliver on the promise of CRISPR-based genetic medicines,” Keith Crandell, partner and cofounder at Arch Venture Partners, said in the release. “Arbor has established a track record of pipeline focus, coupled with execution and capital efficiency, to yield strong preclinical data supporting its pipeline.”PH1 is caused by a malfunctioning copy of the AGXT gene, which makes an enzyme called alanine-glyoxylate aminotransferase (AGT). Though AGT is created in the liver, its absence causes the buildup of oxalate downstream in the kidneys. This buildup causes symptoms like frequent kidney stones, loss of bladder control and painful urination, and, in serious cases, it can lead to kidney failure.Arbor sprouted out of the shadows in 2018 with a $15.6 million series A and a newfound CRISPR-associated enzyme called Cas13d. This enzyme’s smaller size makes packaging it into gene therapy vectors easier.Arbor followed up its debut with much bigger money at the end of 2021, signing a deal with Vertex worth up to $1.2 billion and closing a $215 million series B.Arbor’s $73.9 million series C raise comes as the gene editing field undergoes a period of self-reflection. Pfizer recently discontinued its hemophilia gene therapy Beqvez, which carried a list price of $3.5 million per person and seemingly has not been given to any patients since its April 2024 approval. And bluebird bio, a gene therapy darling once valued at $10 billion, was sold to a pair of venture capital firms last month for a comparatively paltry $29 million.Massachusetts-based Arbor is forging ahead into the clinic without Chief Scientific Officer John Murphy, Ph.D., who plans to retire. The firm is currently searching for his replacement.

Dec. 19, 2024 -- Arbor Biotechnologies™, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for ABO-101, a novel gene editing therapeutic designed to address primary hyperoxaluria type 1 (PH1). The redePHine Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of ABO-101 in both adult and pediatric patients with PH1. “We are thrilled to advance ABO-101 to the clinic as we believe it has the potential to be a first-in-class treatment for PH1, a rare disease with a high unmet need, and reinforces the promise of our precisely tailored gene editing approach,” said Dan Ory, M.D., Chief Medical Officer of Arbor. “ABO-101 is supported by a strong suite of preclinical data demonstrating specific and durable in vivo editing of HAO1 and corresponding, therapeutically relevant reductions in urinary oxalate in PH1 disease models.” ABO-101 is a liver-targeted gene editing therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1). PH1 is a rare genetic disorder in which enzyme deficiencies in the liver lead to the overproduction and buildup of oxalate, resulting in kidney stones eventually leading to end stage kidney disease and systemic oxalosis. ABO-101 is designed to knock down HAO1 gene expression in the liver, thereby providing durable reduction in oxalate production. “The FDA approval of the ABO-101 IND marks a significant milestone for the hyperoxaluria community. At OHF, we are committed to partnering with industry leaders to advance care, research, and patient-focused drug development for hyperoxaluria. Arbor’s innovative CRISPR-based approach represents a groundbreaking opportunity in genomic medicine, with the potential to transform the lives of patients with PH1,” said Kim Hollander, Executive Director of The Oxalosis and Hyperoxaluria Foundation. “The initiation of this study marks an important milestone both for patients with PH1 and in the growth of our company as we begin advancing our pipeline of first-in-class programs into the clinic,” said Devyn Smith, CEO of Arbor. “There is a tremendous need for innovative approaches to treat genetic conditions – from ultra-rare to the most common genetic diseases – and we’re excited for the potential of our next-generation genetic medicines that can address a vast spectrum of devastating diseases. I applaud the hard work of our team at Arbor for getting us to this stage.” ABO-101 is a novel, investigational gene editing medicine designed to be a one-time treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce PH1-associated oxalate production. ABO-101 consists of a lipid nanoparticle (LNP), licensed from Acuitas Therapeutics, encapsulating messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA which specifically targets the human HAO1 gene. Arbor Biotechnologies™, 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. The company’s 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. With Arbor’s lead program, ABO-101 for the treatment of primary hyperoxaluria type 1, progressing into clinical trials, the company continues to focus their 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. The content above comes from the network. if any infringement, please contact us to modify.