Krystal Biotech (NasdaqGS:KRYS) Update / Briefing January 08, 2026 04:30 PM ET Company ParticipantsKrish Krishnan - CEODavid Sweet - Director of Clinical DevelopmentJorge Lascano - Professor of MedicineSuma Krishnan - President of Research and DevelopmentTrevor Parry - VP of Product DevelopmentConference Call ParticipantsGautam Chukka - Research AnalystJoe Pantginis - Director of Research and Senior Healthcare AnalystRitu Baral - Managing Director and Senior Biotechnology AnalystRoger Song - Senior AnalystA ...

- Multi-asset pipeline with significant targeted data readouts and milestones -- Two lead ophthalmic gene therapy programs in clinical trials with new data anticipated in 2026 -- Supplemental New Drug Application (sNDA) submitted for partnered presbyopia treatment - - Corporate presentation at J.P. Morgan Healthcare Conference on Thursday, January 15, at 8:15 a.m. PT - RESEARCH TRIANGLE PARK, N.C., Jan. 08, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company,” “Opus,” or “Opus Genetics” ...

Core Insights - Insmed Incorporated is set to present at the J.P. Morgan 2026 Healthcare Conference on January 12, 2026, at 3:00 p.m. PT / 6:00 p.m. ET [1] - The presentation will be available via a live webcast on the company's investor relations website, with an archive accessible for 30 days post-event [2] Company Overview - Insmed is a global biopharmaceutical company focused on delivering first- and best-in-class therapies for patients with serious diseases [3] - The company has a diverse portfolio that includes approved and mid- to late-stage investigational medicines, particularly in pulmonary and inflammatory conditions, with two approved therapies for chronic lung diseases [3] - Insmed's early-stage programs utilize various advanced technologies, including gene therapy, AI-driven protein engineering, and synthetic rescue [3] - Headquartered in Bridgewater, New Jersey, Insmed operates research locations across the U.S., Europe, and Japan, and has been recognized as a top employer in the biopharmaceutical industry [4]

Core Insights - Viatris Inc. (VTRS) has announced four significant regulatory achievements in its pipeline, highlighting its R&D progress for 2025 and its commitment to advancing its portfolio globally [2][9]. Regulatory Approvals - The FDA has approved Viatris' octreotide acetate for injectable suspension, a generic version of Sandostatin LAR Depot, which is used for treating acromegaly and certain types of diarrhea related to tumors [3][4]. - This approval marks VTRS' first injectable product utilizing microsphere technology and is the fourth injectable approval for the company in 2025, enhancing its generics portfolio with complex, high-value products [4]. - The FDA has accepted VTRS' new drug application (NDA) for a low-dose estrogen weekly contraceptive patch, aimed at women with a BMI below 30 kg/m², with a target action date set for July 30, 2026 [5][6]. - The patch delivers approximately 150 mcg of norelgestromin and 17.5 mcg of ethinyl estradiol daily, representing a new option for women seeking lower estrogen doses [6][7]. - The FDA has cleared an investigational new drug (IND) application for MR-146, a gene therapy candidate for neurotrophic keratopathy, with a phase I/II clinical trial planned for the first half of 2026 [8][9]. - Viatris has also received acceptance for its Japanese NDA for pitolisant in obstructive sleep apnea syndrome (OSAS), with plans to submit a separate NDA for narcolepsy by year-end [10][11]. Company Performance - Viatris has demonstrated strong performance over the past six months, with shares increasing by 34.6%, compared to the industry growth of 6.1% during the same period [12]. - The company's expansion in Emerging Markets and robust growth in Greater China are positively impacting sales, with new drug approvals expected to further enhance its portfolio [13].

4D Molecular Therapeutics (NasdaqGS:FDMT) Update / Briefing December 17, 2025 08:00 AM ET Company ParticipantsJulian Pei - Head of Investor RelationsDaniel Giraldo - VP of Equity ResearchFelix Ratjen - Paediatric RespirologistJonathan Miller - Managing Director of Biotech and Pharma Equity ResearchJennifer Taylor Cousar - Professor of Internal Medicine and PediatricsGena Wang - Managing Director of Biotech Equity ResearchRyan McElroy - Equity Research AssociateDavid Kirn - Founder and CEOConference Call Par ...

Newborn screening is the only practical means of diagnosing MLD prior to the onset of symptoms, which is key to achieving optimal outcomes for children and their families with this rapidly progressive disease As of today, 14 states—representing more than 50 percent of U.S. births—have RUSP-aligned legislation intended to expedite the addition of newly approved RUSP conditions to their respective NBS panels Other community-led efforts to enable newborn screening for MLD advancing globally TOKYO, LONDON and B ...

SOUTH SAN FRANCISCO, Calif., Dec. 11, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced it has received official notification from the U.S. Food and Drug Administration (FDA) that the clinical hold on the MyPEAK-1™ Phase 1b/2a clinical trial of TN-201 has been removed. All concerns raised by the FDA related ...

Core Insights - Abeona Therapeutics Inc. has activated The University of Texas Medical Branch (UTMB) as a Qualified Treatment Center (QTC) for ZEVASKYN, a gene-modified cellular therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][3] Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with ZEVASKYN being the first autologous cell-based gene therapy for RDEB [10] - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [10] Product Information - ZEVASKYN (prademagene zamikeracel) is the first FDA-approved autologous gene therapy for RDEB, designed to treat chronic wounds by incorporating the COL7A1 gene into a patient's skin cells [5][6] - The therapy has shown clinically meaningful results in wound healing and pain reduction with a single surgical application [5] Treatment Center Activation - The activation of UTMB as a QTC significantly enhances patient access to ZEVASKYN across Texas and the Gulf Coast region, reflecting UTMB's commitment to innovative care [3] - UTMB is recognized for its expertise in complex skin diseases and wound care, making it an ideal partner for delivering ZEVASKYN [2][3] Patient Support Services - Abeona offers a comprehensive patient support program called Abeona Assist, which provides personalized assistance, including help with insurance benefits, financial options, and logistical support for patients [3]

Core Viewpoint - Eli Lilly and Company has successfully completed a tender offer to acquire Adverum Biotechnologies, with a cash payment of $3.56 per share and potential additional payments through contingent value rights [1][2][3] Group 1: Acquisition Details - The tender offer for Adverum's shares expired on December 8, 2025, with 16,493,335 shares tendered, representing approximately 64% of the outstanding shares [2] - The acquisition is expected to be finalized on December 9, 2025, in accordance with the Agreement and Plan of Merger dated October 24, 2025 [3] Group 2: Strategic Implications - The acquisition aims to enhance gene therapy capabilities, particularly for age-related conditions such as vision loss, as stated by Andrew Adams, Lilly's group vice president [4] - Adverum is focused on developing gene therapies for ocular diseases, with a proprietary platform designed to provide durable treatments through single-administration therapies [6]

RESEARCH TRIANGLE PARK, N.C., Dec. 09, 2025 (GLOBE NEWSWIRE) -- Opus Genetics (Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced that the Independent Data Monitoring Committee (IDMC) issued a positive recommendation to continue as planned in the Company’s Phase 1/2 BEST1 clinical trial (BIRD-1), which is a multi-center, adaptive, open ...