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Allogene CEO to step down; Agios drug fails key test

Today, a brief rundown of news involving Allogene and Agios Pharmaceuticals, as well as updates from AbbVie, AstraZeneca and BridgeBio Pharma that you may have missed. Allogene CEO David Chang will step aside following an eight-year run, the company announced

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Today, a brief rundown of news involving Allogene and Agios Pharmaceuticals, as well as updates from AbbVie, AstraZeneca and BridgeBio Pharma that you may have missed. Allogene CEO David Chang will step aside following an eight-year run, the company announced Thursday. Chang co-founded Allogene after serving as an executive at Kite Pharma , a cell therapy maker Gilead Sciences acquired in 2017 . The firm then raised hundreds of millions of dollars in private and public funding and, after a series of ups and downs , brought a donor-derived cell therapy for lymphoma into late-stage testing . Current research chief and Chief Medical Officer Zachary Roberts will take over as Allogene’s leader on July 1. Chang will continue to serve on Allogene’s board. An experimental drug Agios Pharmaceuticals has positioned as a “next-generation” version of its marketed medicine Pyrukynd, has failed a mid-stage trial. According to Agios, the drug, tebapivat , fell short in a study in lower-risk myelodysplastic syndromes and won’t be developed further in that indication. A Phase 2 study is still underway in sickle cell disease, however, with results expected later this year. Tebapivat activates an enzyme that’s critical to the function of red blood cells, as Pyrukynd does. But the drug is structured differently and designed to provide “optimized clinical benefits,” Agios said. AbbVie has won a U.S. clearance for a medicine it acquired in a $10 billion buyout of antibody-drug conjugate specialist ImmunoGen three years ago. Known as Decnupaz , the therapy has been cleared for use in people with blastic plasmacytoid dendritic cell neoplasma , an ultra-rare and fast-moving blood cancer with few treatment options. The clearance makes Decnupaz the third marketed ADC for AbbVie and its first for a blood malignancy. In testing, about 70% of treatment recipients had no detectable signs of cancer afterwards. The FDA also on Thursday greenlit AstraZeneca’s immunotherapy Imfinzi as a treatment for a type of bladder tumor. The clearance enables use of Imfinzi alongside another immune-boosting treatment, Bacillus Calmette-Guérin , in people with the “non-muscle invasive” form of bladder cancer . The approval was based on study results published in The Lancet last year which showed that people highly likely to relapse or see their disease progress had a 32% reduction in the risk of those outcomes, as well as death, after a year. Additionally, the agency has begun reviewing an experimental treatment BridgeBio Pharma has developed for a form of limb-girdle muscular dystrophy. The drug, BBP-418 , is being evaluated as a treatment for the “type 2I/R9” form of limb-girdle , for which there are no marketed treatments. It’s also one of a few BridgeBio rare disease therapies now nearing an approval following a string of clinical successes . The FDA is expected to make a decision on BBP-418 by Nov. 27.

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01What 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 ↗
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 ↗
03What 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 ↗
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

Editorial team for Peptide Therapy Guide.

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