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Star raises another $125M for its blood disease drug

Star Therapeutics has raised $125 million in new venture funding that will help propel its experimental blood disease drug through late-stage testing. Initially formed as a “hub-and-spoke” biotech intending to build single-drug offshoots, Star is now primarily

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Star Therapeutics has raised $125 million in new venture funding that will help propel its experimental blood disease drug through late-stage testing. Initially formed as a “hub-and-spoke” biotech intending to build single-drug offshoots, Star is now primarily focused on a singular program for the blood disorder Von Willebrand disease. The company spun out one subsidiary, Electra Therapeutics, and has taken its top program, VGA039, from preclinical development to a Phase 3 trial, according to CEO and founder Adam Rosenthal. Von Willebrand disease is the most common bleeding disorder, affecting up to 1% of the U.S. population , according to the Centers for Disease Control and Prevention. Like hemophilia, the disease prevents the blood from clotting properly, leading to frequent bleeding episodes that can “be really severe and disruptive to daily life,” Rosenthal said. The treatment landscape for hemophilia has changed dramatically in recent years with the introduction of more convenient and longer-lasting preventive therapies. But innovation has moved more slowly in Von Willebrand, which is still largely treated with replacement clotting factors that require frequent infusions or injections. The pace appears to be picking up, however. The Food and Drug Administration expanded the use of a Takeda drug for Von Willebrand in September. Star’s VGA039 is in advanced testing, as is Roche’s hemophilia drug Hemlibra . Privately held Hemab Therapeutics has a therapy in early development, too.

Adam Rosenthal is the founder and CEO of Star Therapeutics. Permission granted by Star Therapeutics

“There hasn't been any innovation because there hasn't been that recognition of the disease burden and the market potential, and I think now that is starting to change,” Rosenthal said. Star is hoping VGA309 can become for Von Willebrand what Hemlibra — one of Roche’s best-selling drugs — is for hemophilia. Like Hemlibra, VGA309 is a preventive antibody drug that’s meant to prevent bleeds for longer than available therapies and be administered through a subcutaneous injection. The drug is designed to target a molecule known as protein S, which, in turn, boosts the generation of a blood-clotting enzyme called thrombin. Unlike Hemlibra and Hemab’s drug prospect, though, VGA309 might also be useful regardless of the type of Von Willebrand a patient has. “We can treat all types of patients, and all types of bleeds,” Rosenthal said. “We're still in generation 1.0 and we're hoping to leapfrog all that incremental innovation and go straight to a subcutaneous therapy.” Star presented interim results from an early-stage study at the American Society of Hematology meeting last year. The findings “got the attention of a lot of investors,” Rosenthal said, and the company began pursuing a new funding round shortly afterwards. A Phase 3 trial began in September. Star is also exploring VGA039’s potential in other blood disorders, though it hasn’t yet disclosed specifics. Eighteen investors participated in Star’s Series D round, which was co-led by Sanofi Ventures and Viking Global Investors. In a statement announcing the financing, Sanofi Ventures managing director Jason Hafler called VGA039 “a compelling opportunity.” Rosenthal said the company is open to commercializing VGA039 on its own or teaming with a pharmaceutical partner. Star hasn’t ruled out an initial public offering either, he added. “It’s one of those rare circumstances where the market potential is so big, but the spend to get to market is actually quite small,” Rosenthal said. “It's something that an independent biotech can very easily do on their own.” Star has raised more than $300 million since its inception in 2018, including a $90 million Series C round in 2023.

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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 ↗
02What 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 ↗
03China: 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 ↗
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 ↗
05Why 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 ↗
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Peptide Therapy Guide Editorial Team

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