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Precigen wins immunotherapy approval; Pfizer sickle cell drug fails trial

Today, a brief rundown of news involving Precigen and Pfizer, as well as updates from the Institute for Clinical and Economic Review, Superluminal Medicines and Generation Bio that you may have missed. The Food and Drug Administration granted full approval to

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Today, a brief rundown of news involving Precigen and Pfizer, as well as updates from the Institute for Clinical and Economic Review, Superluminal Medicines and Generation Bio that you may have missed. The Food and Drug Administration granted full approval to a first-of-its-kind treatment for recurrent respiratory papillomatosis, a rare and potentially life-threatening condition caused by persistent HPV infections. Thursday’s clearance of Precigen’s Papzimeos , an immunotherapy that helps clear HPV-infected cells, was based on study results showing a little more than half of drug recipients didn't need surgery within a year of therapy. Center for Biologics Evaluation and Research director Vinay Prasad , who rejoined the FDA this week , described the approval as proof “randomized trials are not always needed to approve medical products.” That statement should be “reassuring” to biotech investors concerned about stricter regulatory standards under Prasad, wrote Cantor Fitzgerald analyst Jennifer Kim. Precigen shares rose higher Friday on the news. — Ben Fidler An experimental Pfizer drug for sickle cell disease failed to meet its goal in a Phase 3 study, the company said Friday . Testing showed that treatment with inclacumab , a drug Pfizer acquired via its 2022 buyout of Global Blood Therapeutics , failed to significantly reduce versus placebo the pain crises people with sickle cell often experience. Pfizer said it would share analyses of the data with the scientific and patient community in “due course.” Last year, the company pulled from market another sickle cell drug, Oxbryta , that it gained from Global Blood, citing safety concerns. The company plans to provide updates on Oxbryta and a third Global Blood drug, the experimental osivelotor , when they become available. — Ned Pagliarulo Eli Lilly will collaborate with biotechnology startup Superluminal Medicines to develop new drugs for cardiometabolic diseases and obesity . Through the alliance, the two intend to discover and advance small molecule medicines aimed at undisclosed G protein-coupled receptor , or GPCR, targets “relevant” to those conditions. Lilly will receive exclusive rights to the compounds emerging from the deal, while Superluminal could get up to $1.3 billion in total payouts, including an unspecified upfront payment as well as an equity investment, the companies said Thursday. — Ben Fidler Autolus Therapeutics is delaying launching its leukemia cell therapy Aucatzyl in Europe following approval there as the company “evaluates potential pricing and feasibility of market entry opportunities” in some countries. Launch in Germany is on hold and Autolus “does not anticipate any EU sales of Aucatzyl in 2025 and 2026,” the company said in its second quarter earnings report . In the U.K., where Aucatzyl has also been approved, a government cost-effectiveness monitor has initially decided not to pay for it. Autolus said it “will continue to work towards a pathway for patient access to therapy in the U.K.” Approved by the Food and Drug Administration in November 2024, Aucatzyl earned just shy of $30 million in sales in the first six months of 2025, all from the U.S. — Jonathan Gardner Generation Bio revealed preclinical results suggesting a delivery technology it’s developing can effectively send nucleic acid payloads into T cells. But the company also said this week that it may not be able to raise the funds to prove that approach works in humans and, as a result, began a strategic review that could end in a sale or merger . Generation will lay off roughly 90% of its workforce, including all of its research and development staff, by the end of October. Company shares climbed 60%, though they’ve lost much of their value since the company’s initial public offering in 2020 . — Ben Fidler

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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 ↗
02Lifestyle 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 ↗
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 ↗
04What 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 ↗
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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