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Crystalys nets $130M more to push gout drug through late-stage tests

Less than a year after landing a big funding round, Crystalys Therapeutics has banked a second one, announcing Wednesday a $130 million Series B to support global late-stage tests, and launch preparations, for a gout drug it’s been working on. Launched last Se

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Less than a year after landing a big funding round, Crystalys Therapeutics has banked a second one, announcing Wednesday a $130 million Series B to support global late-stage tests, and launch preparations, for a gout drug it’s been working on. Launched last September with a $205 million Series A , Crystalys received support in its newest financing from more than 20 firms, many of which are the kind of “crossover” investors that back private as well as publicly traded companies. Frazier Life Sciences led the round, and firms like Wellington Management, Cormorant Asset Management, SR One and Novo Holdings pitched in. Crystalys views its drug, “dotinurad,” as a potential “best-in-class” treatment for gout, a type of arthritis caused by a buildup of uric acid in the body. While a therapy called allopurinol has long been the treatment standard for gout — which causes sudden flares of joint pain — it doesn’t work for many people and the options behind it are flawed. Amgen’s Krystexxa comes with a high cost and requires twice-weekly infusions. Another treatment, known as febuxostat, is linked with potential heart problems . Dotinurad represents a different potential solution. Originally discovered by Japanese drugmaker Fuji Yakuhin, it’s is designed to inhibit a transporter protein called URAT1 that regulates uric acid levels. Dotinurad was approved in Japan in 2020 and, since then, has been cleared in China — where Eisai owns rights — as well as the Philippines and Thailand. Crystalys acquired U.S. and European rights to the treatment from a subsidiary of Fortress Bio in 2024 . It’s since ushered the therapy into two global trials in people with severe gout cases or excess uric acid . Both are evaluating dotinurad against allipurinol over the course of 24 weeks, and should deliver results next year. The company has also started a Phase 2 trial in people who either can’t tolerate allopurinol or similar drugs, or haven’t responded to therapies like Krystexxa. “This financing strengthens our ability to advance our [trials] and positions Crystalys to achieve multiple important clinical and regulatory milestones and commercial readiness as we work to bring a potentially best-in-class treatment option to patients living with gout,” said Crystalys CEO James Mackay, in a statement. Crystalys is betting that dotinurad is a clear improvement over an old AstraZeneca drug called Zurampic, which also inhibited URAT1. Zurampic gained approval in 2015 but was associated with kidney damage and later withdrawn because of low sales . In an interview with BioPharma Dive last year, Mackay noted that dotinurad binds to its target more precisely and could better protect the kidneys. Because dotinurad is an established drug, Crystalys was able to persuade the Food and Drug Administration to allow it to go straight into late-stage development without amassing the usual early safety and efficacy data.

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01What the Artificial Neuron Cannot Do

Hersam’s next goal is a small circuit — perhaps 10 artificial neurons — where each one fires differently, and together they accomplish what would require thousands of conventional transistors. “ Silicon achieves complexity by having billions of identical devices,” Hersam said. “The brain is the opposite. It’s heterogeneous. The complexity is at the device level.” But Gaudet sees a gap no circuit design can yet fill: Biological neurons grow new connections and prune old ones, strengthening pathways that are used and weakening those that aren’t. Hersam’s lab’s printed neurons — or any other neuromorphic technology that mimics neuronal dynamics — can’t achieve that level of complexity yet. Brown is careful about the distance remaining between these printed neurons and the real thing. “Neurons are just so flexible,” he said. “They can totally change what they’re doing based on whether they’ve learned something and based on your emotional state. There’s a lot of hidden mysteries.” Sangwan suspects the device has more to reveal. “It’s a nonlinear dynamical system,” he said. “We don’t fully know how many different variables you need to explain it. It’s just the beginning.” Hersam, Sangwan, Brown, Holla, and Gaudet reported having no relevant financial disclosures. Disclosure information for study authors is available in the original study publication.

Source: www.medscape.com ↗
02China: Threat or opportunity?

One of the biggest biotech news stories of recent years is China’s continued rise as a biotech and life sciences powerhouse. China conducts a quarter of all clinical trials and drug development and has almost 1,500 new drugs in development.¹ Many China-based biotechs have benefitted from government funds, out-licencing deals with large pharmas and venture capital funding. However, policymakers in the US and EU have concerns about the possible threat to their region’s biosecurity and competitiveness as centres for health and life science research. Given China’s increased importance, ICON Biotech conducted the same biotech sector survey with 100 China-based biotech leaders. The results show that Chinese biotechs face many of the same challenges as biotechs located elsewhere. They share the same funding challenges and burdens associated with increasingly complex clinical trials and regulations.

Source: www.biopharmadive.com ↗
03Why Muscle Cells Might Do Some Heavy Lifting

Brown was studying gene therapy in the 1990s when he designed a technology to turn mRNA expression on or off in different cells. For the new mouse study, published in Nature Biotechnology , he adapted the technology to turn off mRNA expression in dendritic cells, muscle cells, or liver cells. The researchers then vaccinated the mice with each version, delivering the vaccines both intravenously and intramuscularly. “The results were pretty stunning,” Brown said. When mRNA expression was turned off in muscle cells, T-cell response went down, suggesting muscle cells play a role in immunity. When expression was turned off in liver cells, T-cell expression tripled — indicating liver cells dampen immunity. Turning off expression in dendritic cells had no effect on T-cell activation, though it did reduce the number of killer T cells by as much as half. (Interestingly, no such reduction occurred when the antigen was SARS-CoV-2 spike. Brown is now investigating why different antigens had varying effects.) Knowing all this is crucial for designing effective mRNA vaccines and therapies. That’s because different mRNA therapies require different strategies. Cancer vaccines must boost tumor-fighting killer (CD8+) T cells. For genetic disease treatments, scientists want to avoid triggering the immune system to prevent killing the very cells the mRNA is meant to modify. “Understanding the immunology is extremely important for this class of drug,” Brown said. The finding doesn’t mean dendritic cells aren’t important for mRNA vaccines to work. “It just means that the mRNA doesn’t have to get into those cells to induce an immune response,” Brown said. Instead, the antigen can be transferred to those dendritic cells.

Source: www.medscape.com ↗
04Lifestyle Matters: How do environmental and lifestyle factors influence Alzheimer’s disease?

Dr. Harrison and Finnish neuroscientist Dr. Miia Kivipelto explore the complex interplay between genetics and lifestyle in Alzheimer's development. Learn how the groundbreaking FINGER study demonstrates potential prevention strategies, and discover the latest evidence on how environmental factors, diet, and chronic conditions influence Alzheimer's risk.

Source: www.biopharmadive.com ↗
05What Comes Next

With data expected in the fourth quarter of 2026, we are prioritizing histology alongside patient-reported outcomes using the Celiac Disease Symptom Diary, one of only two instruments developed in line with U.S. Food and Drug Administration (FDA) guidance, to capture changes in symptoms such as abdominal pain and nausea. Ultimately, the broader aim is to give gastroenterologists and patients a therapeutic option for a disease that has long been managed without one. The future of drug development will not be defined by statistical significance alone, but by whether new therapies also improve the daily burden of living with celiac disease. “The first therapy to cross the line could change the field,” Geller concluded. “It would help establish celiac as a serious medical condition with options beyond a restrictive diet and open the door for what comes next.” Dr. Paul Lizzul is chief medical officer at First Tracks Biotherapeutics, a clinical ‑ stage biotechnology company advancing antibody therapeutics that modulate immune pathways implicated in autoimmune and inflammatory diseases. Marilyn Geller serves as an advisor to First Tracks Bio. Footnotes Abadie V, Jabri B. IL-15: a central regulator of celiac disease immunopathology. Immunol Rev . 2014;260(1):221-234. https://doi.org/10.1111/imr.12191. Yokoyama S, Watanabe N, Sato N, et al. Antibody-mediated blockade of IL-15 reverses the autoimmune intestinal damage in transgenic mice that overexpress IL-15 in enterocytes. Proc Natl Acad Sci U S A . 2009;106(37):15849-15854. https://doi/full/10.1073/pnas.0908834106. Anthony S, Schluns KS. Emerging roles for IL-15 in the activation and function of T-cells during immune stimulation. Research and Reports in Biology . 2015;6:25-37. https://doi.org/10.2147/RRB.S57685.

Source: www.biopharmadive.com ↗
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