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Rocket lays off staff; Abivax capitalizes on immune drug data

Today, a brief rundown of news involving Rocket Pharmaceuticals and Atara Biotherapeutics, as well as updates from Abivax, Gate Bioscience and Matchpoint Therapeutics that you may have missed. Rocket Pharmaceuticals is laying off approximately 30% of its workf

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Today, a brief rundown of news involving Rocket Pharmaceuticals and Atara Biotherapeutics, as well as updates from Abivax, Gate Bioscience and Matchpoint Therapeutics that you may have missed. Rocket Pharmaceuticals is laying off approximately 30% of its workforce and focusing resources on gene therapies for three inherited heart conditions, the company said Thursday . Rocket expects the cuts will reduce its operating expenses by one-quarter, which, together with existing cash reserves and the potential sale of a regulatory fast pass it might receive in the future, should help keep the company afloat into the second quarter of 2027. The Food and Drug Administration last year rejected an approval application for Rocket’s gene therapy Kresladi , which the company hopes to resubmit. Rocket said it anticipates delays in advancing two other programs for Fanconi anemia and and pyruvate kinase deficiency. — Ned Pagliarulo Atara Biotherapeutics and partner Pierre Fabre Pharmaceuticals are taking a second shot at FDA approval of their T cell immunotherapy tab-cel , announcing Thursday that the agency has accepted their resubmission after rejecting an initial attempt over manufacturing in January. The FDA will decide on approval by Jan. 10, Atara said. Tab-cel was developed for previously treated adults and children who have post-transplant lymphoproliferative disease that’s positive for the Epstein-Barr virus. — Ned Pagliarulo Abivax expects to raise at least $650 million via a sale of its American Depositary shares that it priced Thursday. The secondary stock offering follows the company’s announcement this week of Phase 3 study results for an ulcerative colitis drug that impressed analysts and investors. Should the investment banks underwriting the offering exercise their options to purchase additional stock, Abivax could bring in nearly $750 million, which would be some ten times its current cash holdings. It expects the new funds will give it operating runway through 2027 and support further testing of the drug, obefazimod , in inflammatory bowel disease. — Ned Pagliarulo Gate Bioscience , a California-based biotechnology company, announced Thursday that it has entered into collaboration and licensing agreement with Eli Lilly . The deal includes an upfront payment, equity investment, and could also hold potential milestone and royalty payouts. A Gate spokesperson said the fresh funding provides more than a year of runway for the company and, all told, the deal value could reach up to $856 million. The company is backed by well-known life sciences investors like Versant Ventures , a16z , Arch Venture Partners and Alphabet's venture capital arm. It aims to create "molecular gates," a new class of medicines designed to stop cells from secreting disease-causing proteins. Through their collaboration, Lilly and Gate hope to identify therapies that can eliminate "specific difficult-to-drug proteins" and thereby provide novel ways to treat diseases with "high unmet medical need." — Jacob Bell Privately held Matchpoint Therapeutics is getting validation of its drug research from Novartis , which has agreed to pay the Watertown, Massachusetts-based biotech as much as $60 million in upfront and research funding fees. Under the deal , Matchpoint is responsible for advancing an undisclosed program through preclinical testing, at which point Novartis holds an option to exclusively license it. Matchpoint specializes in drugs that work by binding covalently to their target, and is taking aim at an unnamed transcription factor involved in inflammatory disease. — Ned Pagliarulo GSK will have to wait a little longer to learn whether its multiple myeloma drug Blenrep can make a return to the U.S. market . The FDA was set to decide on approval by July 23, but extended its review by three months to "review additional information" the company provided to support its application. Blenrep's new decision date is Oct. 23. An advisory committee voted last week that GSK's trial data did not prove a favorable benefit-risk balance for the patients the company hopes to treat. — Ned Pagliarulo

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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 ↗
03What 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 ↗
04China: 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 ↗
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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