BRL Medicine Inc

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.”

SHANGHAI, June 5, 2025 /PRNewswire/ -- Qure Biotechnology (Shanghai) Co., Ltd. (QureBio) announced that it has completed a Series C1 financing round. The financing raised nearly CNY 100 million (approximately USD 14 million) and was led exclusively by Efung Capital. This infusion of capital will be used to accelerate QureBio's clinical trials and advance its pipeline of novel antibody therapeutics. Therapeutic Focus and Technology Platforms QureBio specializes in developing bispecific and multispecific antibody and protein therapeutics to address unmet medical needs in cancer, autoimmune diseases, and inflammatory disorders. The company has established a suite of proprietary technology platforms – including its I2T platform and its T-cell engager and NK-cell engager platforms – which form the foundation for its pipeline of novel drug candidates. Using these platforms, QureBio has built a robust pipeline of therapeutics aimed at previously intractable diseases. Pipeline Highlights Key pipeline developments include: Q-1802 (Claudin18.2/PD-L1 bispecific antibody): Received regulatory clearance to initiate clinical trials in both China and the United States in March 2021. Phase II patient enrollment is nearly complete, and preparations are underway for Phase III clinical trials in China. Q-1801 (SIRPα/PD-L1 bispecific antibody): Obtained clinical trial approvals in China and the US, and is poised to begin clinical studies. PD-1 Antibody-Cytokine Fusion Candidate: An innovative fusion protein with dual functions as a PD-1 checkpoint inhibitor and a cytokine modulator, demonstrating a favorable therapeutic window. This candidate targets an area where similar approaches by larger biopharmaceutical companies have faced setbacks, and QureBio is seeking partners to accelerate its global development. Strategic Partnerships and Industry Recognition QureBio's proprietary antibody engineering technology has garnered broad industry recognition. Leveraging its platform capabilities, the company has established collaborations with leading biotechnology and pharmaceutical firms – including BRL Medicine, BioMap, Hengrui Pharma, and Precision Scientific – to co-develop novel therapies. These partnerships underscore QureBio's status as an emerging leader in innovative biopharmaceuticals. About QureBio Qure Biotechnology (Shanghai) Co., Ltd. (QureBio) is a biopharmaceutical company founded in 2017 and based in Shanghai, China. The company specializes in the research and development of innovative antibody and protein therapeutics, focusing on bispecific and multispecific antibodies for oncology, autoimmune diseases, and inflammatory disorders. QureBio has built multiple proprietary technology platforms for antibody discovery and engineering, enabling a robust pipeline of drug candidates aimed at addressing critical unmet medical needs in China and around the world. Qu , Xiangdong, the founder of Qure Bio, said: We express our appreciation to Efung Capital, a leading Chinese VC/PE focusing on Biomedicine, for its trust and support in QureBio. As an innovation-driven biotech company focused on pioneering biopharmaceuticals, we will continue leveraging our proprietary bispecific and multispecific antibody technology platforms to develop novel therapies for unmet clinical needs include malignancies, autoimmune diseases, and inflammation-based disorders. We look forward to collaborating with partners to develop novel therapies to benefit patients worldwide. Fan, Rongkui, lead investor in this round of financing from Efung Capital, said: There are numerous companies engaged in the development of Claudin18.2 targets, and different products have demonstrated promising therapeutic potential across various treatment lines. As a promising model, the Claudin18.2 bispecific antibody holds the potential to achieve unique clinical value in first-line therapy for gastric cancer. Efung Capital is optimistic about the advancement of Q-1802 into Phase III clinical trials, aiming to provide gastric cancer patients with innovative products that offer differentiated efficacy and align with national circumstances. Additionally, the company's founding team has a deep understanding of tumor immunology mechanisms, with core members having previously led R&D efforts at multinational pharmaceutical companies such as Amgen, Eli Lilly, and Pfizer, combining international perspectives with the ability to localize industrialization. The core team has demonstrated exceptional efficiency in key areas such as target validation, molecular design, and process development, and has subsequently developed an early-stage differentiated R&D pipeline covering solid tumors and autoimmune diseases. About Efung Capital Efung Capital is one of the earliest professional biopharmaceutical investment institutions in China. Its investment team consists of a group of PhDs in biopharmaceuticals from top universities in China and abroad, focusing on global biopharmaceutical VC/PE investments. It has conducted in-depth screening and invested in companies such as CHIPSCREEN, FRONTIER BIOTECHNOLOGIES Inc., Ascentage Pharma, Lifotronic, HARBOUR BIOMED, Obio Technology, ASIERIS, 3D-Medicines, Genuine Biotech, Apexigen, Centrexion, and Elicio, among other high-quality domestic and international companies. With its outstanding investment performance and exceptional research and investment capabilities, Efung Capital has been honored with numerous industry accolades, including multiple awards such as the Qianke Top 30 Investment Institutions in China's Healthcare Sector, China Venture Capital Top 100, China Venture Capital Top 10 Best Exit Cases of the Year, China Venture Capital Top 10 Best Biomedical Investment Institutions of the Year, China Venture Capital Top 30 Best Venture Capital Institutions in the Guangdong-Hong Kong-Macao Greater Bay Area of the Year, China Venture Capital Top 100 Chinese Venture Capital Institutions of the Year, Fund of Funds Top 30 Best Healthcare Investment Institutions of the Year, and China's Most Dynamic Drug Innovation Investment Institutions. It has been consecutively named one of the "Top 10 Venture Capital Institutions in Shenzhen" for multiple years and has been featured in an exclusive interview by the world-renowned journal Nature. Efung Capital aims to drive the industrialization of biotechnology achievements through capital investment. SOURCE QureBio Ltd. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

A little-known Chinese biotech startup has zipped ahead of US companies in one of the gene editing field’s most competitive races. YolTech Therapeutics announced Monday that it started a clinical trial of an experimental CRISPR therapy for transfusion-dependent beta thalassemia, a genetic blood disease. But rather than removing a patient’s cells and editing them in the lab, as companies in the US have already done, the Shanghai-based biotech plans to edit the cells directly in the bone marrow with a simple transfusion. The trial is the first public disclosure globally of so-called in vivo editing therapy for a blood disease. It’s a potentially cheaper, easier and safer alternative to Casgevy, the sole commercial gene editing treatment made by CRISPR Therapeutics and Vertex in Boston. Several US drugmakers, including well-funded startups and public companies, are working on in vivo therapies for sickle cell disease. Many believe that an in vivo therapy is the only way to make treatments broadly available for beta thalassemia and sickle cell disease — which can be treated with the same approach. But designing lipid nanoparticles that can reach stem cells of the bone marrow is challenging, and YolTech’s announcement that it found a solution ready for clinical testing came as a surprise. YolTech’s beta thalassemia drug is its fourth gene editing therapy to enter clinical studies in a little over a year. Until now, its programs have mimicked ones pioneered by American companies, and the 80-person biotech hasn’t attracted much attention since it was founded in 2021. But that’s starting to change. YolTech, which raised a $32 million Series A in December 2023, has moved at lightning speed with a fraction of the funding of other gene editing companies. CEO and co-founder Yuxuan Wu told Endpoints News on Tuesday that he is “looking for some potential collaborators in the United States and Europe” and took meetings with several pharma companies at the JP Morgan Healthcare Conference in San Francisco this month. If the startup is successful, it will build upon the recent trend of biotech investors and pharma companies turning to China as a source of new drugs . So far, the biggest investments have centered around cancer and obesity drugs. But YolTech wants to prove that Chinese companies can compete in cutting-edge fields like gene editing, too. “We don’t want to just stop at being considered a Chinese company. Hopefully, we will be a global company as well,” Zi Jun Emma Wang, YolTech’s chief technology officer and co-founder, told Endpoints. “It doesn’t matter where the science originates from. Good science should be good science.” Wu cut his teeth on CRISPR a decade ago as a postdoc in Dan Bauer’s lab at Harvard and Boston Children’s Hospital, where he researched editing for blood diseases. Bauer himself was a colleague and former trainee of Boston Children’s hematologist Stuart Orkin, whose work using gene editing to boost fetal hemoglobin would become the trick employed in Casgevy. Wu continued his work on CRISPR when he opened his own lab at East China Normal University in Shanghai in 2018. He also co-founded BRL Medicine, a Shanghai startup making its own CRISPR-edited cell therapies for beta thalassemia and sickle cell. It was a tough time to be a gene editor in the country. The same year, Chinese scientist He Jiankui had announced the birth of babies whose genes were edited as embryos. The swift global condemnation put a damper on gene editing research in China and stalled BRL’s work, because hospitals didn’t want anything to do with CRISPR. Over the next few years, Wu increasingly began to view cell-based therapies as too complex and expensive for China, an idea that’s only solidified since Casgevy’s approval in the US in 2023. The treatment is an onerous process: Doctors first harvest a patient’s bone marrow cells and ship them to a lab where they are altered with CRISPR. Before the edited cells are reinfused, the patient receives chemotherapy to clear out the old diseased cells. Casgevy costs $2.2 million per patient in the US, and that doesn’t include a lengthy hospital stay. Roughly a year after its launch, CRISPR Therapeutics said that 50 patients have had their cells collected. In 2021, Wu co-founded YolTech to develop in vivo therapies as an alternative. That summer, Intellia Therapeutics reported the first glimpse of promising data from therapies that used lipid nanoparticles to deliver CRISPR into the liver to treat transthyretin amyloidosis. YolTech soon began working on its own therapy for that disease, as well as two other liver-targeted programs. All three are now in early-stage clinical studies. Wang and Wu said that the in vivo program for beta thalassemia (which is more common in China than sickle cell disease) has always been a focus for YolTech, but the company kept quiet about its progress until November, when scientists from YolTech and East China Normal University published a preprint on bioRxiv describing how an in vivo therapy could boost fetal hemoglobin in mice. Wang said that the nanoparticles described in the preprint are “from a couple years ago,” noting that essentially every aspect of the therapy has been optimized since then. The experimental drug, dubbed YOLT-204, uses lipid nanoparticles designed to target hematopoietic stem cells of the bone marrow to shuttle mRNA molecules encoding the company’s own base editors. Those CRISPR tools will make a single letter change to a gene that controls production of fetal hemoglobin, boosting levels of that oxygen-ferrying protein. Crucially, the new nanoparticles are decorated with a molecule that homes in on bone marrow cells, although the company isn’t disclosing details of the key ingredient yet. “We screened thousands of formulations,” Wang said. “I would not claim that we solved the problem, but we have a good candidate to test in the clinic.”