CHARLESTOWN, Mass., Oct. 01, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from its neuromuscular and cardiac programs at the World Muscle Society (WMS) 2025 Annual International Congress, October 7-11, 2025, in Vienna, Austria, and at the European Society of Gene & Cell Therapy (ESGCT) 2025 Annual Congress, October 7-12, 2025, in Seville, Spain. “Our presentations at WM ...

In this article, we will take a look at some of the most promising biotech stocks to buy according to hedge funds.If there’s one sector that is gaining popularity each day, it’s definitely the biotechnology market. For the past few years, this sector has appeared to be one of the most dynamic and rewarding in the stock market.From record-breaking advancements in gene therapy and precision medicine to novel drug development, biotech gems perfectly combine healthcare with innovation, and where there’s innovat ...

Key Takeaways Vyjuvek is now approved for DEB patients from birth, with self-administration at home allowed.Vyjuvek posted $184.2M in 1H25 sales, with growth expected from the U.S., EU, and Japan labels.KRYS prioritizes oncology candidate KB707, with an FDA end-of-phase II meeting set for October.Krystal Biotech, Inc.’s (KRYS) lead drug Vyjuvek is a non-invasive, topical, redosable gene therapy approved in the United States, Europe, and Japan for the treatment of dystrophic epidermolysis bullosa (DEB).Kryst ...

Healthcare innovation and biotechnology are standing out right now as areas with a lot of promise for investors. September's rate cut has brightened prospects, while R&D, boosted by AI, is driving new drug discoveries. Health and biotech ETFs and investing could make for a worthwhile consideration as equities uncertainty rises. Amid that interest, investors may want to consider how active investing can outperform passive biotech approaches to life sciences in some key ways. See more: Rising Active ETF TCAF ...

Pediatric participants demonstrated large gains in cone-mediated vision; therapy remains well tolerated with no ocular serious adverse events or dose-limiting toxicitiesLasting, durable responses observed out to 18 months in adult participantsExpected FDA Meeting in Q4 2025Management to Host Webcast and Conference Call Today at 8:30 A.M. ET RESEARCH TRIANGLE PARK, N.C., Sept. 30, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies ...

LEXINGTON, Mass. and AMSTERDAM, Sept. 29, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the closing of its previously announced underwritten public offering of 6,736,841 ordinary shares at a public offering price of $47.50 per share, which includes 947,368 ordinary shares issued pursuant to the exercise in full by the underwriters of their option to purchase additional ordinary s ...

About this content About Ian Lyall Ian Lyall, a seasoned journalist and editor, brings over three decades of experience to his role as Managing Editor at Proactive. Overseeing Proactive's editorial and broadcast operations across six offices on three continents, Ian is responsible for quality control, editorial policy, and content production. He directs the creation of 50,000 pieces of real-time news, feature articles, and filmed interviews annually. Prior to Proactive, Ian helped lead the business outpu ...

Accessibility StatementSkip Navigation ROCKVILLE, Md., Sept. 29, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced Chief Medical Officer, Steve Pakola, M.D., will present at the International Congress of the World Muscle Society taking place in Vienna, Austria, October 7-11, 2025. The podium presentation will share new analysis of previously presented 12-month functional data from the Phase I/II trial of RGX-202, including individual patient improvement on the North Star Ambulatory Assessme ...

Core Viewpoint - uniQure N.V. has announced a public offering of 5,789,473 ordinary shares priced at $47.50 per share, aiming to raise approximately $300 million in gross proceeds before expenses [1] Group 1: Offering Details - The offering includes pre-funded warrants for certain investors to purchase 526,316 ordinary shares at the public offering price minus a nominal exercise price [1] - A 30-day option has been granted to underwriters to purchase up to 947,368 additional ordinary shares at the public offering price [1] - The offering is expected to close on or about September 29, 2025, pending customary closing conditions [1] Group 2: Management and Underwriters - Leerink Partners, Stifel, Guggenheim Securities, and Van Lanschot Kempen are acting as bookrunning managers for the offering, with H.C. Wainwright & Co. as the lead manager [2] Group 3: Company Background - uniQure is focused on gene therapy, with a significant milestone achieved through the approval of its gene therapy for hemophilia B, representing over a decade of research [5] - The company is advancing a pipeline of gene therapies targeting severe diseases, including Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [5]

A new gene therapy has slowed the progression of Huntington’s disease by as much as 75%, according to the results of a newly announced trial. We explain what this could mean for patients https://t.co/rfIICk3QQ1 ...