Confirmed wild-type CFTR delivery and expression in conducting airway cells of patients with class I mutations KB407 transduction confirmed in all six patients with successful bronchoscopies irrespective of modulator-status; percentage of conducting airway cells transduced in each patient ranged from 29.4% to 42.1% Registrational repeat dosing CORAL-3 study design submitted to FDA in late December; anticipating enrollment in study to start in 1H 2026 following alignment with the FDA Investor call to be he ...

Key Takeaways Krystal Biotech shares have climbed 57.2% in a year, far outperforming the biotech industry and sector.Vyjuvek uptake remains strong after FDA label expansion and approvals in the U.S., Japan and Europe.KRYS is advancing a broad pipeline across respiratory, ophthalmology, oncology, dermatology and aesthetics.Shares of Krystal Biotech (KRYS) have surged 57.2% in a year compared with the industry’s growth of 17.1%. The stock has also outperformed the industry and the sector in this time frame.Th ...

Round led by new investor Life Sciences at Goldman Sachs Alternatives with participation by new investor the Retinal Degeneration Fund, and supported by existing investorsFunds will advance laru-zova toward commercialization for treatment of X-linked retinitis pigmentosa and accelerate development of additional pipeline candidates LONDON and CAMBRIDGE, Mass., Jan. 08, 2026 (GLOBE NEWSWIRE) -- Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading clinical-stage biotechnolog ...

Could the tide be turning for Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) after a surprising shift in analyst sentiment?As investors digest the latest upgrade, what factors are driving this newfound optimism for the biotech giant?Bank of America (BofA) Securities on Wednesday upgraded Regeneron Pharmaceuticals, citing several factors, including higher sales for key products.Analyst Tazeen Ahmad upgraded Regeneron from Underperform to Buy, and raised the price forecast from $627 to $860.BofA's prior Underpe ...

Enrollment in 4D-150 Phase 3 wet AMD clinical trials exceeding expectations; 4FRONT-1 trial remains on track to complete enrollment in Q1 2026, with 381 patients randomized or approved to randomize as of January 6, 20264D-150 PRISM wet AMD 2-year Phase 2b data expected mid-20264D-150 DME global Phase 3 trial initiation expected Q3 2026; 2-year SPECTRA Phase 1/2 data expected H2 2026Appointed Glenn Sblendorio to Board of Directors, adding deep commercial and operating experience, including in retina therapeu ...

We recently published 10 Stocks on Fire Ahead of 2026. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the best performers on Tuesday. Ultragenyx rebounded by 15.52 percent on Tuesday to close at $22.78 apiece as investors resorted to bargain-hunting following the previous day’s steep 46 percent fall, dragged by disappointing clinical trial results for its brittle bone disease treatment. In a statement on Monday, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said that two of its phase 3 clinical tr ...

Core Insights - Ocugen (OCGN) is advancing its gene therapy programs for retinal diseases, with plans to file three regulatory applications in the next three years, resulting in a stock increase of 75.1% in 2025 compared to the industry's 20.9% rise [1] Group 1: OCU400 for Retinitis Pigmentosa (RP) - Enrollment is nearing completion in the phase III liMeliGhT study for OCU400, targeting retinitis pigmentosa, with top-line data expected in Q4 2026 [2][8] - Ocugen plans to file a biologics license application (BLA) for OCU400 in 2026, with a rolling submission starting in the first half of 2026 [2] - OCU400 has the potential to treat multiple gene mutations associated with RP with a single subretinal injection, unlike the only approved gene therapy that targets a specific mutation [3] Group 2: OCU410 for Stargardt Disease - The phase II/III GARDian3 study for OCU410ST, aimed at treating Stargardt disease, is ongoing, with interim data expected in mid-2026 and a BLA filing planned for the first half of 2027 [4][8] - There are currently no FDA-approved treatments for Stargardt disease, highlighting the potential market opportunity for OCU410ST [4] Group 3: OCU410 for Geographic Atrophy (GA) - OCU410 is in phase II development as a one-time gene therapy for geographic atrophy, with data expected in Q1 2026 and phase III starting in mid-2026 [5][8] - Currently, patients with GA have only one treatment option, which requires multiple injections and addresses only one aspect of the disease, while OCU410 aims to address multiple aspects [6]

Lexeo Therapeutics, Inc. (NASDAQ:LXEO) is among the Best Get Rich Fast Stocks to Buy Right Now. As of December 24, Lexeo Therapeutics, Inc. (NASDAQ:LXEO) is a consensus buy from all eleven analysts covering the stock. While the target price ranges from $12 to $30, the median price target of $19 translates to an upside potential of 79.92%. On December 18, Raymond James initiated coverage on Lexeo Therapeutics, Inc. (NASDAQ:LXEO) with a ‘Strong Buy’ rating and a price target of $25, citing solid results fr ...

Based on the Phase I/II results, the FDA indicated in October that it is open to an accelerated approval pathway, including discussions around a Biologics License Application for LX2006. Pivotal data are expected in 2027, and Lexeo plans to initiate the registrational study in the first half of 2026, pending finalization of the trial protocol.The company’s leading program is LX2006, an AAV-based gene therapy designed to treat Friedreich’s ataxia cardiomyopathy by delivering a functional frataxin gene via in ...

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