Yoltech Therapeutics Co., Ltd.

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

YolTech Therapeutics, a prolific Chinese biotech that’s already begun testing four CRISPR gene editing therapies in small clinical studies, raised a $44.5 million Series B to test several more genetic medicines in humans and potentially begin its first Phase 3 trial later this year, it told Endpoints News in an exclusive interview. The new round was led by the AstraZeneca-CICC Healthcare Investment Fund, a $1 billion equity fund established by the British pharma company and the China International Capital Corporation in 2019. YolTech’s funding comes amidst soaring attention to Chinese biotech companies, and as many US gene editing companies have cut staff and trimmed once-sweeping pipelines. And while some US gene editing startups are just beginning — or still struggling — to test their first therapies in people, YolTech has wasted no time putting one experimental CRISPR therapy after another into clinical tests. “If we wanted to survive, we had to move things quickly into the clinic,” YolTech co-founder and CEO Yuxuan Wu told Endpoints. “We showed the investors that gene editing can finally be translated to the clinical stage.” YolTech’s Chief Technology Officer and co-founder Zi Jun Emma Wang said the new funding — which brings its total raised to $83 million — should support the 90-person company for the two or three years it will take to get its lead program through a Phase 3 trial. That therapy, YOLT-201, uses CRISPR to edit a gene responsible for an inherited condition called transthyretin amyloidosis, or ATTR. The US biotechs Intellia Therapeutics and Regeneron Pharmaceuticals began their own Phase 3 trial for the disease last year. The US study could wrap up in the second half of 2027. Wang told Endpoints that “it’s possible” YolTech will beat Intellia to pivotal data disclosure and approval, but downplayed that as a goal. “We’re not trying to compete with Intellia,” she said. “We are focused on generating good safety and efficacy data in the clinic to provide patients, Chinese patients, access to a drug that could be still years away without us.” Wu learned the ropes of CRISPR as a postdoc at Boston Children’s Hospital before returning to China to start his own lab at East China Normal University in Shanghai in 2018. He also co-founded a startup, BRL Medicine, to develop a CRISPR therapy for beta-thalassemia. BRL’s therapy required removing a patient’s cells and editing them in the lab. But Wu became convinced that such ex vivo editing would be too expensive for most Chinese patients. And in 2021, he was inspired by data from Intellia showing that a simple infusion could edit genes directly inside people’s bodies. A month later, he founded YolTech and teamed up with Wang, who had worked on mRNA delivery at Boston gene editing companies Tessera Therapeutics and Beam Therapeutics. Soon after Wang moved back to China, Shanghai’s renewed Covid lockdown in the spring of 2022 threatened to stall progress. But the startup’s budding team lived at the company’s headquarters for several months to continue their lab work. YolTech says it has developed its own lipid nanoparticles, scoured nature to find two million CRISPR proteins and used AI and protein engineering to assess billions of possible variants of those proteins to create its own proprietary tools for gene cutting, base editing and prime editing. While US companies have raised hundreds of millions for similar tasks, YolTech did it with a comparatively modest $17 million Series A in 2022, and a $15 million extension in 2023. “China-based investors tend to be more pragmatic, more willing to give you money only after they’ve seen some large animal data, at least, or preferably a clinically-validated program,” Wang said. “It’s very hard to raise money with only in vitro data and a good story.” K2 Venture Partners was a returning investor in the Series B, along with Grand Flight and Yunion Healthcare Ventures, both based in China, and Decheng Capital, which has offices in Shanghai, California and New York. New investors include Green Pine Capital Partners and Tianjin Venture Capital, both based in China. Since treating its first patient with ATTR in December 2023, YolTech has expanded its pipeline to a dozen programs and brought three more into the clinic. “The efficiency of this team is truly, truly impressive,” Yao Wu, a partner at K2 Venture Partners, one of YolTech’s early investors, told Endpoints. One reason is the salaries of PhD graduates are one-third to one-half the level paid in Boston or San Diego, she said. “Also, the hours are just longer.” Another factor is that YolTech’s clinical programs all started as investigator-initiated studies, or IITs. These small trials are approved and overseen by Chinese hospitals, rather than the nation’s drug regulators, and give startups a quick way to glimpse the safety and potential efficacy of experimental cell and gene therapies. They’ve also allowed the company to potentially leapfrog US competitors. In January, YolTech began an IIT for an in vivo editing therapy designed to treat beta thalassemia and sickle cell disease through an infusion of lipid nanoparticles that target the bone marrow. While numerous US drugmakers are racing to develop their own in vivo therapies for these two diseases, none have said when they will begin clinical trials. And in February, YolTech announced the first results from an IIT of a CRISPR therapy for primary hyperoxaluria 1— five months before Cambridge-based Arbor Biotechnologies dosed its first patient . YolTech is now asking the FDA to let it jump straight into a pivotal study based on its IIT data, which would catapult it even further. “We got good positive feedback, and because this is an ultra-rare disease, the trial size is manageable,” Wang said. YolTech has also begun an IIT for the genetic condition alpha-1 antitrypsin deficiency (AATD) and is preparing one for an in vivo CAR-T cell therapy . YolTech’s most advanced program uses CRISPR to break a gene, thereby reducing levels of the protein transthyretin, which causes problems for people with ATTR. It has already tested the treatment in about 25 patients in an IIT and Phase 1/2 studies with either the cardiac or nerve forms of the disease, but the company will focus on the rarer nerve form for a Phase 3. “A large-scale cardiomyopathy trial was going to require a huge amount of resources,” Wang said. “We would rather work on other indications to show early proof-of-concept in the clinic.” In a press release in March, YolTech said a single high dose reduced TTR protein by 90%, similar to data reported by Intellia. Wang said that more clinical data from the program and two others are under review at medical journals and could be published this year. Wang said about half of the patients have been followed for at least one year, but she hopes this will be enough data to convince Chinese regulators to okay a Phase 3 — likely the country’s first for a CRISPR therapy. She sees a big opportunity to make a one-time therapy that’s far more affordable than chronic treatments for ATTR, including Pfizer’s pill tafamidis, which launched with an annual cost of $225,000, and Alnylam’s siRNA drug Amvuttra , which costs $476,000 a year. “That’s out of reach for most Chinese patients,” Wang said. “We did the math, and we are able to at least match tafamidis.”

YOLT-101 will be a new treatment possibility for familial hypercholesterolemia. Credit: aipicte/Shutterstock. YolTech Therapeutics has received the US Food and Drug Administration (FDA) approval for its investigational new drug (IND) application for YOLT-101 to treat heterozygous familial hypercholesterolemia (HeFH). YOLT-101 is an in vivo base editing therapy designed to target proprotein convertase subtilisin/kexin type 9 (PCSK9). Its innovative approach lies in its potential to durably reduce blood low-density lipoprotein cholesterol (LDL-C) levels through a single-dose treatment. The technology behind YOLT-101 incorporates YolTech’s adenine base editor, known as hpABE5, which consists of nCas and a new deaminase evolved from Hafnia paralvei. To deliver this therapeutic agent, the company employs an advanced lipid nanoparticle (LNP) delivery system. hpABE5 can perform precise A•T to G•C base conversions without inducing DNA double-strand breaks, thus minimising risks associated with chromosomal abnormalities and off-target effects. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence YolTech Therapeutics co-founder and CEO Yuxuan Wu stated: “The FDA IND clearance marks a significant milestone for YolTech. In vivo gene editing represents a new generation of therapeutics — offering one-time, durable solutions for chronic and genetic diseases. “We are committed to advancing breakthrough gene editing solutions that offer transformative benefits for patients living with severe genetic and cardiovascular diseases.” Currently under evaluation in an ongoing investigator-initiated trial (IIT), YOLT-101 has shown promising results with a favourable safety profile and substantial LDL-C–lowering effects. Familial hypercholesterolemia is a genetic disorder stemming from mutations affecting LDL metabolism-related genes such as LDLR, Apolipoprotein B (APOB) and PCSK9. Individuals suffering from FH typically experience elevated LDL-C levels, which contribute to a heightened risk of developing early-onset atherosclerotic cardiovascular diseases. Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now