AGT

SHANGHAI, April 21, 2025 /PRNewswire/ -- Rona Therapeutics, a biotechnology company pioneering innovative RNA-targeted therapies, announced that the Investigational New Drug (IND) application for RN1871, a small interfering RNA (siRNA) drug targeting angiotensinogen (AGT), has been approved by China's National Medical Products Administration (NMPA). RN1871 is designed to silence AGT mRNA expression in the liver, precisely inhibiting the production of the key precursor protein in the Renin-Angiotensin-Aldosterone System (RAAS). The RAAS pathway plays a key role in blood pressure regulation, and its overactivation is closely linked to the progression of hypertension. Preclinical studies have demonstrated that RN1871 delivers long-lasting blood pressure control offering a promising new therapeutic option for over 1.5 billion hypertension patients worldwide. Stella Shi, CEO and Founder of Rona Therapeutics, stated: "The IND approval of RN1871 marks a significant breakthrough in our pursuit of innovative therapies for chronic diseases. By leveraging siRNA technology to treat disease fundamentally, RN1871 achieves sustained efficacy with less dosing frequency. We look forward to bringing this innovative therapy to hypertension management where more compliant and safe treatment is needed to improve cardiovascular events and other comorbidities." Dr. Alex M. DePaoli, Chief Medical Officer and Executive Vice President of Translational Medicine, added: "RN1871's preclinical data showed potent AGT suppression which reduces blood pressure significantly with a favorable safety profile. We plan to advance RN1871 into Phase I trials to evaluate its safety, pharmacokinetics, and preliminary efficacy in humans. This progress reflects our team's expertise in translating scientific insights into clinical solutions." About Rona Therapeutics Rona Therapeutics is a global leader in nucleic acid therapeutics discovery and development, specializing in the treatment of cardiometabolic and neurological diseases. Rona Therapeutics is committed to developing the best and first-in-class siRNA drugs with differentiation and innovation to address unmet needs and improve outcome in cardiovascular diseases, obesity, MASH and neurological diseases through both hepatic and extra-hepatic delivery of proprietary siRNA. For Further information,Please visit: or contact: [email protected] WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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.

When some former MyoKardia execs reunited with $300 million for a new “heart health” biotech earlier this year, they were tight-lipped about their in-licensed clinical-stage assets. The company, called Kardigan, has now quietly disclosed one of the drugs in its pipeline. The startup is putting tonlamarsen, an investigational antisense medicine for adults with uncontrolled hypertension, into a Phase 2 trial , according to the US clinical trials database. The trial will compare a monthly under-the-skin administration of tonlamarsen versus placebo in about 700 patients. It’s slated to wrap up in February 2027. The experimental medicine comes from Ionis Pharmaceuticals, which is focusing on launching new medicines on its own after decades as one of the leading RNA biotechs. A Kardigan spokesperson told Endpoints News that tonlamarsen is one of several cardiovascular therapies that Kardigan is developing. An Ionis spokesperson confirmed that Kardigan received the exclusive global development and commercialization rights to the treatment candidate. Ionis took tonlamarsen (formerly ION904) through a much smaller, 48-person Phase 2 study in 2022. At the American Heart Association’s annual confab in November 2023, the company said the mid-stage trial showed ION904 had “significantly reduced” levels of angiotensinogen (AGT) versus placebo. The drug aims to reduce blood pressure by inhibiting AGT production, Ionis has said. Tassos Gianakakos, who steered MyoKardia to a $13 billion exit to Bristol Myers Squibb in 2020, is CEO and chair of Kardigan. The heart drug at the center of that deal was subsequently approved as Camzyos. As for Kardigan, Gianakakos and team reeled in $300 million in January from Perceptive Advisors, ARCH Venture Partners and Sequoia Heritage to build out a pipeline of cardio drugs through a mix of internal R&D and “strategic in-licensing and acquisitions.”