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 ...

The Stock Exchange of Hong Kong Limited and the Securities and Futures Commission take no responsibility for the contents of this Application Proof, make no representation as to its accuracy or completeness and expressly disclaim any liability whatsoever for any loss howsoever arising from or in reliance upon the whole or any part of the contents of this Application Proof. Application Proof of Frontera Therapeutics IMPORTANT IMPORTANT: If you are in any doubt about any of the contents of this document, you ...

With strong 1-year returns and upside potential, Taysha Gene Therapies, Inc. (NASDAQ:TSHA) secures a spot on our list of the 12 best multibagger stocks to buy heading into 2026. Taysha Gene (TSHA) Hovers Around Its 52-Week High As It Continues to Advance Rett Syndrome Program As of December 17, 2025, Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is maintaining levels near its 52-week high, following peaks at $5.95 on December 12 and 13. The investor sentiment remains heightened as the company continues to ad ...

Biotech stocks are not for the faint of heart, as companies in the clinical stage push risk to the extreme. With key clinical data still to come, huge financial burn, and valuation almost entirely dependent on trial outcomes, these companies only offer massive upside if everything goes as planned. Here are two of the riskiest biotech stocks investors are betting on. Biotech Stock #1: Opus Genetics Opus Genetics (IRD) has emerged as one of the boldest and riskiest bets investors are making right now. Val ...

If you are interested in keeping up to date with stocks making moves within the biotech, pharma and healthcare industries, and understanding the key trends and catalysts driving valuations ahead of the market, why not subscribe to my weekly newsletter via my Investing Group, Haggerston BioHealth ?Edmund Ingham is a biotech consultant. He has been covering biotech, healthcare, and pharma for over 5 years, and has put together detailed reports of over 1,000 companies. He leads the investing group Haggerston B ...

Core Insights - Sangamo Therapeutics has initiated a rolling submission of a Biological License Application (BLA) to the FDA for isaralgagene civaparvovec, a gene therapy for Fabry disease, with expectations to complete the submission by Q2 2026 [1][4] Group 1: Study Results and Regulatory Pathway - The STAAR study demonstrated a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients, which the FDA has agreed will serve as an endpoint for accelerated approval [1][3] - Isaralgagene civaparvovec has shown a favorable safety and tolerability profile, indicating its potential as a one-time, durable treatment for Fabry disease [1][3] - The rolling submission process allows for completed modules of the BLA to be reviewed by the FDA on an ongoing basis, rather than waiting for the entire application to be submitted [2] Group 2: Designations and Company Background - Isaralgagene civaparvovec has received multiple designations from regulatory bodies, including Orphan Drug, Fast Track, and RMAT from the FDA, as well as Orphan Medicinal Product designation from the European Medicines Agency [4] - Sangamo Therapeutics is focused on genomic medicine, aiming to develop treatments for serious neurological diseases and has a pipeline that includes partnered programs and opportunities for investment [7]

Core Viewpoint - REGENXBIO Inc. will present at the 44th Annual J.P. Morgan Healthcare Conference, highlighting its advancements in gene therapy and its late-stage pipeline of treatments for rare and retinal diseases [1][2]. Company Overview - REGENXBIO is a biotechnology company founded in 2009, focused on gene therapy with a pioneering role in AAV gene therapy [3]. - The company is advancing a late-stage pipeline that includes treatments for Duchenne (RGX-202), MPS II (clemidsogene lanparvovec, RGX-121), and MPS I (RGX-111), in partnership with Nippon Shinyaku [3]. - Additionally, REGENXBIO is collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3]. - Thousands of patients have been treated using REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3]. - The investigational gene therapies from REGENXBIO have the potential to significantly impact healthcare delivery for millions [3].

Core Insights - Viatris Inc. has announced four significant regulatory milestones in its global pipeline, highlighting advancements in research and development throughout 2025 [1][6][14] Group 1: Regulatory Approvals - The U.S. FDA has approved the company's octreotide acetate for injectable suspension, a generic version of Sandostatin LAR Depot, for treating acromegaly and certain cancer-related symptoms [6][14] - This approval marks the fourth injectable FDA approval for Viatris in 2025, alongside iron sucrose, paclitaxel, and liposomal amphotericin B, showcasing the company's ability to navigate complex regulatory pathways [3][14] - The FDA has accepted the New Drug Application (NDA) for a low dose estrogen weekly patch for contraception, with a target action date of July 30, 2026 [6][14] Group 2: Pipeline Developments - The investigational low dose estrogen weekly patch is designed for women with a BMI below 30 kg/m², delivering approximately 150 mcg of norelgestromin and 17.5 mcg of ethinyl estradiol per day [4][7] - The planned 505(b)(2) NDA for the patch is supported by positive results from the Phase 3 Luminous Study, demonstrating a favorable efficacy and safety profile [5][14] - The U.S. FDA has cleared the Investigational New Drug (IND) application for MR-146, a gene therapy candidate for neurotrophic keratopathy, with plans to initiate a Phase 1/2 clinical trial in the first half of 2026 [6][9][14] Group 3: International Developments - The Japan Pharmaceuticals and Medical Devices Agency has accepted the Japanese New Drug Application (J-NDA) for pitolisant in obstructive sleep apnea syndrome, supported by positive Phase 3 data [6][11][14] - The Phase 3 trial for pitolisant showed statistically significant improvements in excessive daytime sleepiness compared to placebo, indicating its potential effectiveness [11][14]