Yoltech Therapeutics Co., Ltd.

Single dose of YOLT-202 led to rapid, robust and dose-dependent increases in AAT levels to normal range Single dose of YOLT-202 was well tolerated with a favorable safety profile SHANGHAI--(BUSINESS WIRE)-- YolTech Therapeutics , a late clinical-stage biotechnology company developing in vivo gene editing therapies, today announced positive interim data from an investigator-initiated trial (IIT) of YOLT-202, the Company’s investigational in vivo base editing therapy, for the treatment of Alpha-1 Antitrypsin Deficiency (AATD) that demonstrated positive safety and tolerability as well as meaningful increases in AAT levels in evaluated patients treated with the 35 mg and 45 mg dose levels. “These interim findings mark an exciting and important milestone for YolTech and for patients living with severe AATD. The rapid, robust, and dose‑dependent increases in functional AAT levels observed in this study—particularly among individuals with the PiZZ genotype—underscore the transformative potential of in vivo base editing as a one‑time treatment approach,” stated Yuxuan Wu, M.D., Founder and CEO of YolTech Therapeutics. “Equally encouraging is the favorable safety pro have seen to date, which reinforces the precision and thoughtful engineering behind YOLT‑202. With these results in hand, we are more confident than ever in YOLT‑202’s potential to redefine the treatment paradigm for AATD, and we look forward to advancing this program toward an Investigational New Drug (IND) filing with the U.S. Food and Drug Administration (FDA) as we continue our mission to bring durable, life‑changing therapies to patients.” YOLT-202 is being evaluated in a first-in-human, open-label, single dose escalation study in adult AATD patients, aiming to evaluate safety and tolerability. As of February 6 th , two participants genetically confirmed as PiZZ genotype, were enrolled and dosed with YOLT-202 in both the 35 mg and 45 mg dose groups. Following administration of YOLT-202, both patients showed rapid, robust and dose-dependent increases in AAT level as early as in Week 1. AAT levels in both patients reached above the protective threshold of 11 μM. Additionally, AAT levels increased to normal range (>20 μM) in the 45 mg dose group. These newly produced AAT proteins were both structurally corrected (M-AAT) and functional, with the proportion of corrected M-AAT increasing to >95% in the 45 mg dose group. YOLT-202 demonstrated favorable safety and tolerability with manageable adverse events (AEs). No severe AEs or AEs leading to discontinuation of YOLT-202 were reported, and all AEs were classified as Grade 1. The most common AE was infusion-related reaction (IRR). Elevation of alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were asymptomatic, mild and soon recovered without medication. The ongoing IIT study (NCT07193615) is evaluating single doses administered via intravenous infusion of YOLT-202 at 35 mg, 45 mg and 55 mg dose levels. YolTech is actively preparing to IND with the FDA to support the global clinical development of YOLT-202 in AATD. About YOLT-202 YOLT-202 is an in vivo gene-editing therapy that corrects PiZ mutation to PiM for the treatment of AATD. Utilizing YolTech’s proprietary adeneine base editor, YOLT-202 is engineered to achieve on-target editing with minimal bystander activity. YOLT-202 is currently being investigated in a first-in-human IIT study designed to evaluate safety and tolerability as well as an optimal biological dose of YOLT-202 in subjects with AATD. YOLT-202 has previously been granted Orphan Drug Designation by the U.S. FDA and the Company is currently preparing to IND with the U.S. FDA to support clinical development of YOLT-202. About Alpha-1 Antitrypsin Deficiency (AATD) AATD is an inherited, genetic, autosomal co-dominant disorder caused by mutations in the SERPINA1 gene, with the most frequent deficient variants coming from the Z (Glu342Lys) and S alleles (Glu264Val). The presence of Z alleles results in misfolding and polymerization of the AAT, leading to over 95% of severe AATD patients being PIZZ. About YolTech Built on HEPDONE™ Novel Editor Platform and non-viral LNP technologies, YolTech Therapeutics is pioneering in vivo gene-editing medicines with the potential for a one-time treatment that provides lifelong benefit. The company’s expanding clinical pipeline targets genetic, metabolic, cardiovascular, and autoimmune diseases, with initial results supporting the potential for durable and transformative therapeutic benefit. Stay informed with the latest from YolTech Therapeutics: LinkedIn: linkedin.com/company/yoltech-therapeutics Website: Contacts YolTech Therapeutics Ally Yu xiaolingyu@yoltx.com Precision AQ Andrew Dymon andrew.dymon@precisionaq.com

AccurEdit Therapeutics, a startup based in Suzhou, China, has raised $75 million in Series A funding to advance its growing pipeline of CRISPR-based medicines — among the largest rounds on record for a Chinese gene editing company. The funding, which hasn’t previously been reported by Western media, closed in September, AccurEdit confirmed to Endpoints News . The investment brings total funding to $116 million since AccurEdit’s founding in 2021. While the full sum may be small by US standards, AccurEdit — like many other Chinese biotech startups — is already proving it can do more with less. In an exclusive interview with Endpoints, founder and CEO Yongzhong Wang said AccurEdit has already tested CRISPR therapies for two cardiovascular diseases — transthyretin amyloidosis and familial hypercholesterolemia — in about 40 patients. Data from AccurEdit presented last year suggest efficacy and safety are on par with, and perhaps slightly better than, those of similar therapies being developed by companies in the US. These results are why Wang says he’s not too concerned about perceptions that Chinese genetic medicine developers are largely imitating therapies developed by Western companies. “Perception holds a lot of power, but I don’t think — in the long term — perception is as powerful as data,” he said. AccurEdit is also working on a third program that appears to be a first in the gene editing field. It’s trying to inhibit a gene called HSD17B13, which has emerged as a promising target for metabolic dysfunction-associated steatohepatitis (MASH), a prevalent liver disease previously known as NASH. While other drugmakers, including GSK and Regeneron Pharmaceuticals, are in the early stages of designing or testing therapies that inhibit the same gene, AccurEdit’s approach would be a permanent fix instead of one that needs to be taken over time. “This is a new target — the efficacy is not proven yet, so it’s kind of risky still,” Wang said. Clinical tests could begin in the second half of the year. The new funding could support the company for about three years and help push at least two of its gene editing programs into Phase 2 studies and at least one of them into a Phase 3 study, Wang said. Like many emerging Chinese biotech leaders, Wang got his start in the US. He earned his PhD at Tufts University and then worked at nearby Genzyme for five years. When Wang moved back to China in 2011, he rose through the ranks at Kanghong Biotech, becoming its CEO and helping take the company public. Wang later hopped to leadership roles at other companies. After working on protein-based drugs for over a decade, Wang and a team of about 10 people who had stuck with him between companies were ready for a new challenge in genetic medicines. Lipid nanoparticles had just proven their worth in the first Covid-19 vaccines, and Wang thought that nanoparticles would be key to making gene editing affordable. “We will be able to reduce the price to a very low level,” Wang said. Promising data from Intellia Therapeutics helped. In June 2021, the Cambridge, MA-based company became the first to show that CRISPR could safely and effectively edit a problematic gene in people with transthyretin amyloidosis (ATTR), a heart and nerve disease. A month later, AccurEdit and another Chinese startup called YolTech Therapeutics were founded to develop CRISPR therapies, with an initial focus on ATTR. AccurEdit’s first major investors were China-based Legend Capital and Boston-based Cormorant Asset Management. They didn’t participate in the startup’s most recent funding round, which was led by two public Chinese companies: China Medical System Holdings and Tibet Pharmaceutical. AccurEdit says it was the first in China to test an in vivo gene editing therapy — one administered directly inside the body — in August 2023. That patient was treated in an investigator-initiated study , a popular approach in China, cleared by hospitals rather than national regulators. In AccurEdit’s lead program for ATTR, the highest dose tested (1 mg/kg) reduced transthyretin protein levels in the blood by more than 90% within four weeks. That reduction has kept stable for at least 72 weeks, according to data the company shared with Endpoints. While Intellia has reported similar results in Phase 1 studies, Wang emphasized that AccurEdit has seen far lower rates of liver enzyme elevations than the rival’s therapy. (One of Intellia’s Phase 3 studies is on hold after a patient died in November.) AccurEdit said a Phase 2 study of its therapy is ongoing in China, and the company has clearance for a Phase 1 study in the US. AccurEdit’s second program aims CRISPR at the well-studied gene PCSK9. Inhibiting the gene reduces low-density lipoprotein cholesterol (LDL-C) Verve Therapeutics, a Boston company that Eli Lilly acquired for $1 billion last year, reported in April 2025 that the highest dose it tested (0.6 mg/kg) of its similar therapy reduced LDL-C by 53% and PCSK9 protein by 60%. AccurEdit has seen even better results, albeit at higher doses (0.75 mg/kg and 1.0 mg/kg). Its therapy reduced LDL-C by almost 62% and PCSK9 by 88%, according to data shared with Endpoints. The startup is also jumping on the red-hot trend of in vivo CAR-T therapies , using lipid nanoparticles to reprogram immune cells to fight cancer or autoimmune disease directly in the body. The company hopes to begin a human study this year.

SHANGHAI, Sept. 11, 2025 /PRNewswire/ -- YolTech Therapeutics, a clinical-stage biotech company pioneering in vivo genome editing therapies, today announced the closing of its approximately $45 million Series B financing led by the AstraZeneca-CICC healthcare investment fund. The proceeds will support the advancement of YolTech's clinical programs and global strategic execution. Dr. Yuxuan Wu, Founder and CEO of YolTech, "This financing marks an important milestone as we continue advancing our pipeline toward transformative therapies. We are committed to turning cutting-edge gene-editing science into real-world treatments for patients worldwide." About YolTech Therapeutics Founded in 2021, YolTech Therapeutics is advancing next-generation in vivo gene-editing therapies designed for one-time treatment. The company has built a fully integrated platform encompassing proprietary CRISPR nucleases (YolCas), base editors (YolBE), and a cutting-edge lipid nanoparticle delivery system (Yol-LNPs), enabling precise, efficient, and tissue-specific gene editing across a broad range of therapeutic areas. In 2024, YolTech's lead program YOLT-201, a CRISPR-based therapy for transthyretin amyloidosis (ATTR), became the first in vivo gene-editing therapy in China to enter Phase I/IIa clinical trial. Since then, the company has advanced four clinical-stage programs addressing ATTR, familial hypercholesterolemia (HeFH), primary hyperoxaluria type 1 (PH1), and β-thalassemia/sickle cell disease (TDT/SCD). YolTech operates a cGMP-compliant manufacturing facility supporting clinical supply through Phase III and early commercial scale-up, ensuring manufacturing consistency and scalability across programs. With one of the most extensive in vivo gene-editing clinical pipeline globally, YolTech continues to lead in the field. Its base-editing therapy YOLT-101, developed for familial hypercholesterolemia (HeFH), became the first in vivo base-editing program to receive IND clearance in both China and the United States. SOURCE YolTech Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED