Sentinel participant showed OPGx-BEST1 was well tolerated with no ocular inflammation, treatment-related adverse events, or dose-limiting toxicities at three months Early signals of functional and structural improvement observed at one month and three months12-letter BCVA gain and 23% CST reduction observed in the treated eye at three monthsFull cohort data expected in mid-year 2026 RESEARCH TRIANGLE PARK, N.C., Feb. 27, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (“Opus Genetics” or the “Com ...

Summary of enGene Conference Call Company Overview - **Company Name**: enGene - **Ticker**: ENGN - **Focus**: Non-viral genetic medicines, specifically developing a product called detalimogene voraplasmid for non-muscle invasive bladder cancer (NMIBC) [2][3] Core Points and Arguments - **Market Potential**: The NMIBC market is expected to grow significantly, with the introduction of new agents improving patient outcomes and physician practices [3] - **Product Profile**: Detalimogene is characterized as a novel class of gene therapy with a large cargo capacity, allowing for redosing and competitive cost of goods due to its non-viral nature [4][5] - **Patient Demographics**: Over 740,000 bladder cancer patients in the U.S., with 27,500 classified as high-risk NMIBC. The majority of patients are treated in community settings rather than academic centers [5][9] - **Current Treatment Landscape**: Existing treatments like BCG are in short supply and can be toxic at higher doses, leading to high rates of bladder removal surgeries with significant morbidity and mortality [6][7] - **Clinical Development**: enGene plans to provide data updates in the second half of the year, file a Biologics License Application (BLA) by year-end, and expects a potential product launch in 2027 [4][23] Clinical Data and Efficacy - **Pivotal Study**: The pivotal study has enrolled 125 patients, exceeding the target of 100. The primary endpoint is the Complete Response (CR) rate, with a reported 62% CR rate at six months [11][15] - **Tolerability**: Most adverse events are Grade 1 and 2, primarily related to catheterization, with low rates of dose interruptions and discontinuations compared to other agents [17] - **Convenience**: Detalimogene is designed for ease of use, requiring minimal special handling and storage, which is advantageous for community practices [18] Competitive Landscape - **Emerging Competitive Profile**: Detalimogene is positioned as having a competitive efficacy and tolerability profile compared to existing therapies, with a focus on community urologists who prefer products that integrate easily into their practices [19][20] - **Combination Therapy Potential**: The non-viral nature of detalimogene allows for potential combination therapies with other agents, which could enhance treatment efficacy [25] Market Dynamics - **Market Growth**: The NMIBC market is projected to grow from approximately $1 billion to over $20 billion, driven by the adoption of new therapies and changing treatment paradigms [22] - **Economic Impact**: The introduction of detalimogene is expected to benefit community practices economically, improving patient satisfaction and practice revenue through a buy-and-bill model [20][21] Additional Insights - **Pricing Strategy**: enGene's low cost of goods provides a competitive advantage in pricing, allowing flexibility in response to market dynamics [28] - **Future Data**: Anticipated updates on 12-month response data from the LEGEND study are expected in the second half of the year [27] This summary encapsulates the key points discussed during the enGene conference call, highlighting the company's strategic focus, product development, market potential, and competitive positioning in the NMIBC treatment landscape.

Core Viewpoint - A class action lawsuit has been filed against uniQure N.V. for allegedly misleading investors regarding the effectiveness of its gene therapy candidate AMT-130 and the likelihood of FDA approval [1][3]. Company Overview - uniQure N.V. is based in Amsterdam and focuses on developing and commercializing gene therapies for severe diseases, particularly Huntington's disease and other neurological disorders [2]. Legal Proceedings - The lawsuit pertains to investors who acquired uniQure securities between September 24, 2025, and October 31, 2025, with a deadline of April 13, 2026, for potential lead plaintiff appointments [2]. - The complaint claims that uniQure misrepresented the status of FDA agreement on study endpoints, which led to a significant stock price increase of nearly 250%, from $13.66 to $47.50 per share, following positive statements [3]. - On November 3, 2025, it was revealed that the FDA agreement on study design was not finalized, causing a stock price drop of over 49%, from $67.69 to $34.29 per share [4].

Core Viewpoint - Sarepta Therapeutics CEO Douglas Ingram will retire by the end of 2026 or upon the appointment of a successor, following a challenging year for the company marked by significant setbacks related to its gene therapy product, Elevidys [1]. Company Developments - The company has initiated a search for a new CEO after Ingram's announcement of his retirement [1]. - Sarepta faced a tumultuous 2025, with Elevidys linked to the deaths of two patients, leading to a request from the U.S. Food and Drug Administration (FDA) to voluntarily halt shipments of the therapy [1]. - The FDA is currently investigating the deaths associated with Elevidys, which has resulted in the therapy carrying the most serious safety warning and stringent monitoring requirements post-treatment [1]. Financial Impact - Sarepta announced 500 job cuts and halted the development of several gene therapies for limb-girdle muscular dystrophy in response to the challenges faced [1]. - The company's shares fell by 82% last year, reflecting the negative market reaction to the issues surrounding Elevidys [1]. - Following the announcement of Ingram's retirement, the stock was down approximately 4% in after-market trading [1].

Spruce Biosciences (NasdaqCM:SPRB) FY Conference February 25, 2026 03:20 PM ET Company ParticipantsJavier Szwarcberg - CEOConference Call ParticipantsLeland Gershell - Managing Director and Senior Biotechnology AnalystLeland Gershelleveryone, back to Oppenheimer's 36th Annual Healthcare and Life Sciences Conference. I'm Leland Gershell, one of the analysts here at the firm, up next, we have Spruce Bio, ticker SPRB. This is a company that we cover, we like the story very much. We have an outperform rating. O ...

The Gross Law Firm Reminds Shareholders of a Lead Plaintiff Deadline of April 14, 2026 in REGENXBIO Lawsuit - RGNX [Accessibility Statement] Skip NavigationNEW YORK, Feb. 24, 2026 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of REGENXBIO Inc. (NASDAQ: RGNX).Shareholders who purchased shares of RGNX during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any r ...

PDUFA action date set for August 23, 2026 If approved, DTX401 will be the first treatment to address the underlying cause of GDSIa NOVATO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the Biologics License Application (BLA) seeking approval of DTX401 AAV gene therapy (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia (GSDIa). The FDA gra ...

Core Viewpoint - uniQure N.V. is set to report its fourth quarter and full year 2025 financial results on March 2, 2026, highlighting its ongoing commitment to advancing gene therapies for severe medical conditions [1]. Group 1: Financial Reporting - The financial results will be announced before market open on March 2, 2026, followed by a conference call at 8:00 a.m. ET [1]. - The event will be accessible via webcast on uniQure's website, with a replay available for 90 days post-event [2]. Group 2: Company Overview - uniQure is recognized for its pioneering work in gene therapy, particularly for hemophilia B, marking a significant milestone in genomic medicine [3]. - The company is advancing a pipeline of proprietary gene therapies targeting conditions such as Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease, among others [3].

Core Insights - Insmed reported total revenues of $606.4 million for the full year 2025, with significant contributions from its products BRINSUPRI and ARIKAYCE [1][2] - The company anticipates full-year 2026 revenues for BRINSUPRI to be at least $1 billion and for ARIKAYCE to be between $450 million and $470 million [1][2] - Insmed's net loss for the fourth quarter of 2025 was $328.5 million, compared to a net loss of $235.5 million in the same quarter of 2024 [2][3] Financial Performance - Total revenues for the fourth quarter of 2025 were $263.8 million, a 153% increase compared to $104.4 million in the fourth quarter of 2024 [2] - ARIKAYCE revenues for the fourth quarter of 2025 were $119.2 million, reflecting a 14% growth year-over-year, while full-year revenues reached $433.8 million, a 19% increase from 2024 [2] - BRINSUPRI generated $144.6 million in the fourth quarter and $172.7 million for the full year 2025, marking its successful U.S. commercial launch [1][2] Research and Development Updates - Insmed is advancing its clinical programs, including the Phase 3 ENCORE trial for ARIKAYCE and additional Phase 3 studies for TPIP in patients with pulmonary conditions [1][2] - The company plans to submit a supplementary new drug application for ARIKAYCE in the second half of 2026, pending positive topline data from the ENCORE trial [1][2] - Insmed is also developing INS1148 and INS1201, targeting various respiratory and genetic diseases, with IND filings expected in 2026 [1][2] Cash Position and Future Guidance - As of December 31, 2025, Insmed had approximately $1.4 billion in cash, cash equivalents, and marketable securities, positioning the company for continued investment in its pipeline [1][2] - The company expects to maintain a focus on commercialization and expansion of its product offerings, particularly BRINSUPRI and ARIKAYCE, throughout 2026 [1][2] - Insmed anticipates that pre-clinical research programs will comprise less than 20% of overall expenditures, indicating a strategic focus on advancing clinical development [2]

Core Viewpoint - Opus Genetics, a clinical-stage biopharmaceutical company, has announced a private placement of 7,374,632 shares of Series B Non-Voting Convertible Preferred Stock at a price of $3.39 per share, aiming to raise approximately $25 million to fund its gene therapy programs and general corporate purposes [1][2]. Group 1: Private Placement Details - The private placement involves the issuance of 7,374,632 shares of Series B Non-Voting Convertible Preferred Stock, which will convert into common stock upon stockholder approval [1]. - The gross proceeds from the private placement are expected to be $25 million before deducting offering expenses [1]. - The financing is led by Adage Capital Management, with participation from Trails Edge Capital Partners and Marshall Wace [1]. Group 2: Use of Proceeds - The net proceeds from the private placement will be utilized to advance gene therapy clinical programs, as well as for working capital and general corporate purposes [2]. - The company anticipates that its pro forma cash balance of $70 million at year-end 2025 will fund operations into the first half of 2028, excluding potential proceeds from callable warrants or future milestone payments [2]. Group 3: Closing and Legal Aspects - The offering is expected to close on February 18, 2026, subject to customary closing conditions [3]. - Sidley Austin LLP is serving as counsel to Opus Genetics for this transaction, and the company did not use a placement agent [3]. - The shares issued in the private placement have not been registered under the Securities Act and may not be offered or sold in the U.S. without proper registration or exemption [4]. Group 4: Company Overview - Opus Genetics is focused on developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs) [6]. - The company is working on seven AAV-based programs targeting various genetic causes of severe retinal disorders, including OPGx-LCA5 and OPGx-BEST1 [6]. - Additionally, Opus Genetics is advancing Phentolamine Ophthalmic Solution 0.75% for pharmacologically induced mydriasis, with potential applications in presbyopia and low-light visual disturbances [6].