Arbor Biotechnologies to Present Preclinical Data for ABO-101 in PH1 at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
– Data from oral presentation supported ABO-101 IND and CTA approval for clinical assessment in primary hyperoxaluria type 1 (PH1) in the Phase 1/2 redePHine study in US and UK (NCT06839235)
– Preclinical data demonstrate highly specific and durable editing of HAO1 with long-lasting reduction of urinary oxalate levels out to one-year post-single dose administration of ABO-101
– Non-human primate studies confirm preclinical efficacy and tolerability of ABO-101 with efficient editing of HAO1, reduced GO enzyme activity, and no clinical signs or adverse events
– Progeny studies demonstrate the lack of germline transmission of the intended ABO-101 edit to Hao1 in over 500 pups
CAMBRIDGE, Mass, May 13, 2025 (GLOBE NEWSWIRE) -- Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today presents IND- and CTA-enabling data for its lead clinical program, ABO-101 in primary hyperoxaluria type 1 (PH1) at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting in New Orleans, Louisiana.
'We are excited to showcase this robust preclinical data package which supported the IND clearance and CTA approval in the UK for our first clinical program' said Dan Ory, M.D., Chief Medical Officer at Arbor. 'This data increases our confidence in the potential for ABO-101 to provide a long-term clinically-meaningful treatment for PH1 patients, as we begin enrollment of our Phase 1/2 redePHine clinical study evaluating ABO-101 for the treatment of PH1, a rare genetic disorder with high unmet need'.
In an oral presentation, Arbor showcased data supporting the therapeutic potential of ABO-101 for PH1 and its clinical evaluation in the redePHine clinical study (NCT06839235). The presented data demonstrated durable and efficient dose-dependent in vivo editing of Hao1 in the Agxt KO mouse model of PH1, resulting in a corresponding reduction of approximately 40% in urinary oxalate levels that was sustained out to 1-year post-administration of a single dose of ABO-101. Administration of ABO-101 to juvenile mice resulted in editing and oxalate reduction that persisted into adulthood. An additional study evaluating the potential of treated female mice to transmit the edit to their offspring demonstrated no transmission through the germline in over 500 pups.
Data from non-human primate (NHP) studies confirmed the editing efficiency and tolerability of ABO-101 with no clinical signs or adverse events. Moreover, ABO-101 editing was shown to be highly specific to HAO1 in both primary human hepatocytes (PHH) and human splenic endothelial cells (HSEC) with no off-target editing detected above 0.07% at >1,500 potential sites, in an assay with a 10-fold greater sensitivity than reported industry standards for CRISPR nucleases.
Further details on the off-target assessment of ABO-101 and the editing profile of Arbor's Type V nucleases will be included in a poster presentation during the Thursday poster session.
Details for the oral presentation are as follows:Oral Presentation Title:
Abstract Number: 44Session: Gene and Cell Therapy for Metabolic DiseasesSession Date and Time: Tuesday, May 13, 2024, 1:30-3:15 PM CDTLocation: Room 278-282Presenter: Tia DiTommaso, PhD
Details for the poster presentation are as follows:Poster Title:
Abstract Number: 1637Session: Thursday Poster ReceptionSession Date and Time: Thursday, May 15, 2024, 5:30-7:00 PM CDTLocation: Poster Hall, Hall I2Presenter: Ivan Kristanto
About ABO-101ABO-101 is a novel, investigational gene editing medicine designed to be a one-time liver-directed gene editing treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce PH1-associated oxalate production. ABO-101 is currently being evaluated for PH1 in the redePHine Phase 1/2 clinical study (NCT06839235). 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. 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.
About Arbor Biotechnologies, Inc.Arbor Biotechnologies™, a clinical stage, 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, which is capable of approaches ranging from gene knockout, excisions, reverse transcriptase editing, and large gene insertion, 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 its 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
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
an hour ago
- Yahoo
LEADING EDGE MATERIALS ANNOUNCES UP TO $4,000,000 NON-BROKERED PRIVATE PLACEMENT
LEADING EDGE MATERIALS ANNOUNCES UP TO C$4,000,000 NON-BROKERED PRIVATE PLACEMENT NOT FOR DISTRIBUTION TO U.S. NEWSWIRE SERVICES OR FOR DISSEMINATION IN THE UNITED STATES OR ANY OTHER JURISDICTION IN WHICH THE DISTRIBUTION OR RELEASE WOULD BE UNLAWFUL. ANY FAILURE TO COMPLY WITH THIS RESTRICTION MAY CONSTITUTE A VIOLATION OF U.S. SECURITIES LAWS. THIS PRESS RELEASE DOES NOT CONSTITUTE AN OFFER, OR A SOLICITATION OF ANY OFFER, TO BUY OR SUBSCRIBE FOR ANY SECURITIES IN LEADING EDGE MATERIALS IN ANY JURISDICTION. Vancouver, June 10, 2025 – Leading Edge Materials Corp. ('Leading Edge Materials' or the 'Company') (TSXV: LEM) (Nasdaq First North: LEMSE) (OTCQB: LEMIF) announces the intent to complete a non-brokered private placement of up to 25,000,000 units ('Units') at a price of C$0.16 per Unit for aggregate gross proceeds of up to C$4,000,000 (the 'Private Placement'). Leading Edge Materials intends to use net proceeds for the Company's projects, located in Sweden and Romania and for general working capital and corporate purposes. Each Unit will consist of one (1) common share (each, a 'Common Share') in the capital of the Company and one (1) Common Share purchase warrant (a 'Warrant'). Each Warrant will entitle the holder to purchase one Common Share (a 'Warrant Share') at a price of C$0.32 per Warrant Share until the date which is four (4) years from the closing date of the Private Placement (the 'Closing Date'). The Company expects certain insiders of the Company to participate in the Private Placement. Any participation by insiders in the Private Placement constitutes a 'related party transaction' as defined under Multilateral Instrument 61-101 – Protection of Minority Security Holders in Special Transactions ('MI 61-101'). However, the Company expects to rely on exemptions from the formal valuation and minority shareholder approval requirements of MI 61-101 based on the fact that neither the fair market value of the Units subscribed for by the insiders, nor the consideration for the Units paid by such insiders, would exceed 25% of the Company's market capitalization as at the date of this news release. The Private Placement is directed towards Canadian, Nordic and other international investors. All securities issued under the Private Placement, including securities issuable on exercise of the Warrants, will be delivered from Canada and are subject to a hold period expiring four months and one day from the Closing Date. The Private Placement is subject to certain conditions including, but not limited to, the receipt of all necessary regulatory approvals, including the approval of the TSX Venture Exchange. A finders' fees may be payable on a portion of the Private Placement. The securities have not been, and will not be, registered under the U.S. Securities Act, or any U.S. state securities laws, and may not be offered or sold in the U.S. or to, or for the account or benefit of, United States persons absent registration or an applicable exemption from the registration requirements of the U.S. Securities Act and applicable U.S. state securities laws. This press release shall not constitute an offer to sell or the solicitation of an offer to buy securities in the United States, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful. This news release is not a prospectus under Regulation (EU) 2017/1129 (the 'EU Prospectus Regulation'). The Company has not authorized any offer of securities to the public (as defined in the EU Prospectus Regulation) in any EEA member state and no such prospectus has been or will be prepared in connection with the Private Placement. On behalf of the Board of Directors, Leading Edge Materials Corp. Kurt Budge, CEO For further information, please contact the Company at:info@ Follow usTwitter: About Leading Edge Materials Leading Edge Materials is a Canadian public company focused on developing a portfolio of critical raw material projects located in the European Union. Critical raw materials are determined as such by the European Union based on their economic importance and supply risk. They are directly linked to high growth technologies such as batteries for electromobility and energy storage and permanent magnets for electric motors and wind power that underpin the clean energy transition towards climate neutrality. The portfolio of projects includes the 100% owned Woxna Graphite mine (Sweden), 100% owned Norra Karr Heavy Rare Earth Elements project (Sweden) and the 51% owned Bihor Sud Nickel Cobalt exploration alliance (Romania). Additional Information This information is information that Leading Edge Materials Corp. is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication through the agency of the contact person set out above, at June 10, 2025 at 23:30 PM Vancouver time. Leading Edge Materials is listed on the TSXV under the symbol 'LEM', OTCQB under the symbol 'LEMIF' and Nasdaq First North Stockholm under the symbol 'LEMSE'. Mangold Fondkommission AB is the Company's Certified Adviser on Nasdaq First North and may be contacted via email CA@ or by phone +46 (0) 8 5030 1550. Reader Advisory This press release does not constitute an offer, or a solicitation of any offer, to buy or subscribe for any securities in Leading Edge Materials in any jurisdiction. This news release may include forward-looking information that is subject to risks and uncertainties. All statements within, other than statements of historical fact, are to be considered forward-looking, including statements with respect to the closing of the Private Placement, the receipt of regulatory approvals, and the use of proceeds from the Private Placement. Although the Company believes the expectations expressed in such forward-looking information are based on reasonable assumptions, such information is not a guarantee of future performance and actual results or developments may differ materially from those contained in forward-looking information. Factors that could cause actual results to differ materially from those in forward-looking information include, but are not limited to, fluctuations in market prices, successes of the operations of the Company, the Company's ability to close the Private Placement, the Company's ability to obtain the required regulatory approvals, continued availability of capital and financing and general economic, market or business conditions. There can be no assurances that such information will prove accurate and, therefore, readers are advised to rely on their own evaluation of such uncertainties. The Company does not assume any obligation to update any forward-looking information except as required under the applicable securities laws. Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release. Important information for EEA Investors The release, announcement or distribution of this press release may, in certain jurisdictions, be subject to restrictions. The recipients of this press release in jurisdictions where this press release has been published or distributed shall inform themselves of and follow such restrictions. This press release does not constitute an offer, or a solicitation of any offer, to buy or subscribe for any securities in Leading Edge Materials in any jurisdiction. Any investment decision in connection with the Private Placement must be made on the basis of all publicly available information relating to the Company and the Company's shares/Units. The information contained in this announcement is for background purposes only and does not purport to be full or complete. No reliance may be placed for any purpose on the information contained in this announcement or its accuracy or completeness. This announcement does not purport to identify or suggest the risks (direct or indirect) which may be associated with an investment in the Company or the new shares/Units. This press release is not a prospectus for the purposes of the EU Prospectus Regulation. Leading Edge Materials has not authorized any offer to the public of Units, shares or rights in any member state of the EEA and no prospectus has been or will be prepared in connection with the Private Placement. In the United Kingdom, this document and any other materials in relation to the securities described herein is only being distributed to, and is only directed at, and any investment or investment activity to which this document relates is available only to, and will be engaged in only with, 'qualified investors' who are (i) persons having professional experience in matters relating to investments who fall within the definition of 'investment professionals' in Article 19(5) of the Financial Services and Markets Act 2000 (Financial Promotion) Order 2005 (the 'Order'); or (ii) high net worth entities falling within Article 49(2)(a) to (d) of the Order (all such persons together being referred to as 'relevant persons'). In the United Kingdom, any investment or investment activity to which this communication relates is available only to, and will be engaged in only with, relevant persons. Persons who are not relevant persons should not take any action on the basis of this document and should not act or rely on it. Attachment LEMNR
Yahoo
5 hours ago
- Yahoo
CTA hosts pop-up events to solicit rider feedback
CHICAGO (WGN) — Transit riders this month have an opportunity to speak with Chicago Transit Authority personnel to offer feedback that will be used by the agency to plan for the upcoming budget and help prioritize future investments and strategies. The CTA this week announced a series of pop-up 'CTA Chats' at several rail stations and bus terminals, with the first being held Tuesday at the Union Station Transit Center. The recently passed budget for Illinois' 2026 fiscal year did not include reform or additional funding for the state's transit agencies. In Chicagoland, the Regional Transportation Authority faces a $770 million fiscal cliff and has warned of transit cuts on an 'unprecedented scale.' 'We are in a unique time for transit in our region, and it important that decisions we are making and projects and programs we are prioritizing are grounded in our riders' priorities and vision for the future,' acting CTA President Nora Leerhsen said in a statement. Union Station Transit Center 6/10/25(Tuesday) 4 p.m. – 6 p.m. Illinois Medical District (Blue Line) 6/11/25(Wednesday) 4 p.m. – 6 p.m. Garfield (Green Line) 6/12/25(Thursday) 4 p.m. – 6 p.m. Navy Pier Bus Terminal 6/14/25(Saturday) 12 p.m. – 2 p.m. Loyola (Red Line) 6/17/25(Tuesday) 4 p.m. – 6 p.m. Paulina & Howard Bus Terminal 6/18/25(Wednesday) 4 p.m. – 6 p.m. Belmont/Kimball (Blue Line Bus Terminal) 6/19/25(Thursday) 4 p.m. – 6 p.m. 95th Dan Ryan (Red Line) 6/21/25(Saturday) 12 p.m. – 2 p.m. Jefferson Park (Blue Line) Bus Terminal 6/24/25(Tuesday) 4 p.m. – 6 p.m. Roosevelt (Red/Orange Green Line) 6/25/25(Wednesday) 4 p.m. – 6 p.m. Midway Orange Line Bus Terminal 6/26/25(Thursday) 4 p.m. – 6 p.m. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Yahoo
6 hours ago
- Yahoo
CRISPR Therapeutics AG (CRSP) Rises Higher Than Market: Key Facts
The latest trading session saw CRISPR Therapeutics AG (CRSP) ending at $42.94, denoting a +2.02% adjustment from its last day's close. The stock outperformed the S&P 500, which registered a daily gain of 0.55%. At the same time, the Dow added 0.25%, and the tech-heavy Nasdaq gained 0.63%. Shares of the company have appreciated by 11.97% over the course of the past month, outperforming the Medical sector's gain of 3.49% and the S&P 500's gain of 6.29%. Investors will be eagerly watching for the performance of CRISPR Therapeutics AG in its upcoming earnings disclosure. On that day, CRISPR Therapeutics AG is projected to report earnings of -$1.54 per share, which would represent a year-over-year decline of 3.36%. At the same time, our most recent consensus estimate is projecting a revenue of $5.89 million, reflecting a 1032.88% rise from the equivalent quarter last year. Regarding the entire year, the Zacks Consensus Estimates forecast earnings of -$5.54 per share and revenue of $39.95 million, indicating changes of -27.65% and +7.06%, respectively, compared to the previous year. It's also important for investors to be aware of any recent modifications to analyst estimates for CRISPR Therapeutics AG. These revisions typically reflect the latest short-term business trends, which can change frequently. With this in mind, we can consider positive estimate revisions a sign of optimism about the company's business outlook. Our research shows that these estimate changes are directly correlated with near-term stock prices. To exploit this, we've formed the Zacks Rank, a quantitative model that includes these estimate changes and presents a viable rating system. The Zacks Rank system, which ranges from #1 (Strong Buy) to #5 (Strong Sell), has an impressive outside-audited track record of outperformance, with #1 stocks generating an average annual return of +25% since 1988. Over the past month, there's been a 1.68% fall in the Zacks Consensus EPS estimate. CRISPR Therapeutics AG is currently a Zacks Rank #3 (Hold). The Medical - Biomedical and Genetics industry is part of the Medical sector. At present, this industry carries a Zacks Industry Rank of 76, placing it within the top 31% of over 250 industries. The Zacks Industry Rank evaluates the power of our distinct industry groups by determining the average Zacks Rank of the individual stocks forming the groups. Our research shows that the top 50% rated industries outperform the bottom half by a factor of 2 to 1. Be sure to use to monitor all these stock-influencing metrics, and more, throughout the forthcoming trading sessions. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
