A newly developed therapy inspired by bacteria residing within tumors offers a different way to combat cancer by targeting how tumor cells produce energy.

Merck has announced that the U.S.

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Investing.com -- Dyne Therapeutics Inc (NASDAQ:DYN) stock rose 3.6% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its DYNE-251 treatment for Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. The designation was based on data from the company’s ongoing DELIVER clinical trial. The breakthrough status provides Dyne with enhanced FDA support, including senior-level involvement, early and frequent communication with FDA reviewers, and eligibility for rolling and priority review, potentially reducing the review timeline from 12 to 8 months. "This Breakthrough Therapy Designation for DYNE-251 is a testament to its potential as a next-generation therapy designed to bring meaningful functional improvement to individuals with DMD for whom exon 51 skipping can lead to the production of near full-length dystrophin," said Doug Kerr, chief medical officer of Dyne. The company reported that DYNE-251 has demonstrated sustained functional improvement through 18 months, as assessed by key measures such as time to rise and stride velocity 95th centile. This marks the second breakthrough therapy designation for Dyne’s lead programs, with DYNE-101 having received the same status for myotonic dystrophy type 1 earlier this year. Dyne has completed enrollment of 32 patients in the Registrational Expansion Cohort of the DELIVER trial, with data expected in late 2025. The company anticipates a potential Biologics License Application submission for U.S. Accelerated Approval in early 2026. DYNE-251 has also received Fast Track, Orphan Drug, and Rare Pediatric disease designations from the FDA and Orphan Drug designation from the European Medicines Agency for DMD treatment. This article was generated with the support of AI and reviewed by an editor. For more information see our T&C. Don't miss out on the next big opportunity! Stay ahead of the curve with ProPicks AI – 6 model portfolios fueled by AI stock picks with a stellar performance this year... In 2024 alone, ProPicks AI identified 2 stocks that surged over 150%, 4 additional stocks that leaped over 30%, and 3 more that climbed over 25%. That's an impressive track record. With portfolios tailored for Dow stocks, S&P stocks, Tech Stocks, and Mid Cap stocks, you can explore various wealth-building strategies. So if DYN is on your watchlist, it could be very wise to know whether or not it made the ProPicks AI lists.

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Investing.com -- Shares of uniQure N.V. (NASDAQ:QURE) surged 39% following the announcement that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to AMT-130, its gene therapy candidate for the treatment of Huntington’s disease. This significant regulatory milestone, which aims to expedite the development and review process of AMT-130, has been well-received by investors, signaling potential for an accelerated path to market for the treatment. The FDA’s Breakthrough Therapy designation is supported by clinical data from ongoing Phase I/II trials, which have indicated that AMT-130 could slow disease progression. The optimism surrounding uniQure’s stock is further bolstered by a comprehensive set of FDA designations for AMT-130, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations, reflecting the drug’s potential to address the urgent need for effective treatments for Huntington’s disease. uniQure’s Chief Medical Officer, Walid Abi-Saab, M.D., highlighted the significance of the designation, stating, “Receiving Breakthrough Therapy designation underscores both the urgent need for effective treatments for Huntington’s disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression.” He expressed gratitude for the FDA’s commitment to advancing gene therapies for patients with critical unmet needs and conveyed the company’s eagerness to work with the agency to deliver AMT-130 to patients as swiftly as possible. Huntington’s disease is a debilitating neurodegenerative disorder characterized by motor symptoms, behavioral abnormalities, and cognitive decline, resulting in progressive deterioration. The disease affects approximately 70,000 people in the U.S. and Europe, with no current disease-modifying therapies available. The Breakthrough Therapy designation is particularly significant for uniQure, as it indicates that AMT-130 may offer substantial improvement over existing therapies for a serious condition, based on preliminary clinical evidence. This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Immunotherapy Revolutionizes Cancer Treatment, Helping Patients Avoid Surgery: 'A Game-Changer for the Future' Kelly Spill's life changed in an instant

STELLANEWS.LIFE（ステラニュース・ライフ）は、科学技術・医療・ライフサイエンスの分野における研 | STELLANEWS.LIFE（ステラニュース・ライフ）は、科学技術・医療・ライフサイエンスの分野における研

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In a significant leap for mental health treatment, the U.S.