Core Insights - Tenaya Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing potentially curative therapies for heart disease [1][3] - The company will participate in the Canaccord Genuity 45 Annual Growth Conference on August 12, 2025 [1] Company Overview - Tenaya's mission is to discover, develop, and deliver therapies that target the underlying causes of heart disease [3] - The company's pipeline includes clinical-stage candidates TN-201 for MYBPC3-associated hypertrophic cardiomyopathy and TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy [3] - Tenaya employs integrated capabilities such as target validation, capsid engineering, and manufacturing to create novel medicines based on genetic insights [3] - Additional products in development include TN-301, a small molecule HDAC6 inhibitor for heart failure, and various early-stage programs targeting rare genetic disorders and common heart conditions [3]

Core Viewpoint - The Rocket Pharmaceuticals class action lawsuit alleges violations of the Securities Exchange Act of 1934, focusing on misleading statements regarding the safety and clinical trial protocol of its gene therapy product RP-A501 [1][4]. Group 1: Lawsuit Details - The lawsuit is titled Ho v. Rocket Pharmaceuticals, Inc. and seeks to represent purchasers of Rocket Pharmaceuticals securities [1]. - A subsequent case, Yankov v. Rocket Pharmaceuticals, Inc., has also been filed [1]. - The lawsuit claims that Rocket Pharmaceuticals failed to disclose serious adverse events, including patient deaths, related to its clinical trials [4][5]. Group 2: Clinical Trial Allegations - Rocket Pharmaceuticals is accused of providing misleading information about its Phase 2 pivotal trial for RP-A501, particularly regarding the safety of the treatment and the introduction of a new immunomodulatory agent without proper disclosure [4]. - On May 27, 2025, the U.S. FDA placed a clinical hold on the RP-A501 study after a patient suffered a serious adverse event leading to death, which was not disclosed to investors at the time of the protocol amendment [5]. Group 3: Legal Process - Investors who purchased Rocket Pharmaceuticals securities during the class period can seek to be appointed as lead plaintiff in the lawsuit [6]. - The lead plaintiff will represent the interests of all class members and can choose a law firm to litigate the case [6]. Group 4: About Robbins Geller - Robbins Geller Rudman & Dowd LLP is a leading law firm specializing in securities fraud and shareholder litigation, having recovered over $2.5 billion for investors in 2024 alone [7]. - The firm has been ranked 1 in securing monetary relief for investors in securities class action cases for four out of the last five years [7].

Core Insights - Adverum Biotechnologies, Inc. will participate in a fireside chat at the H.C. Wainwright 5th Annual Ophthalmology Virtual Conference on August 13, 2025 [1] - The chat will feature Laurent Fischer, MD, and Szilárd Kiss, MD, discussing the company's advancements in gene therapy for ocular diseases [1] Company Overview - Adverum Biotechnologies is a clinical-stage company focused on gene therapy aimed at preserving sight in prevalent ocular diseases [3] - The company is developing a novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec), intended as a one-time intravitreal injection for patients with neovascular or wet age-related macular degeneration [3] - Adverum aims to transform the standard of care for ocular diseases by providing durable, single-administration therapies that reduce the need for frequent ocular injections [3]

NEW YORK, Aug. 05, 2025 (GLOBE NEWSWIRE) -- Bragar Eagel & Squire, P.C., a nationally recognized stockholder rights law firm, announces that a class action lawsuit has been filed against Sarepta Therapeutics, Inc. ("Sarepta" or the "Company") (NASDAQ:SRPT) in the United States District Court for the Southern District of New York on behalf of all persons and entities who purchased or otherwise acquired Sarepta securities between June 22,2023 and June 24, 2025, both dates inclusive (the "Class Period"). Inves ...

Financial Performance & Projections - The company anticipates total revenue between $640 million and $670 million in 2025, representing a 14-20% increase from 2024[93] - The company projects full-year GAAP profitability in 2027, driven by revenue growth, expense management, and potential monetization of Priority Review Vouchers (PRVs)[94, 96] - Crysvita revenue is expected to be between $460 million and $480 million in 2025, a 12-17% increase[93] - Dojolvi revenue is projected to be between $90 million and $100 million in 2025, a 2-13% increase[93] - As of June 30, 2025, the company had $538 million in cash, cash equivalents, and marketable debt securities[96] Clinical Program Updates - Phase 3 data readout for UX143 in Osteogenesis Imperfecta (OI) is expected around the end of 2025[21, 32, 97] Phase 2 data showed a 67% reduction in annualized fracture rate (AFR) with UX143[33] - Enrollment for the Phase 3 Aspire study of GTX-102 for Angelman Syndrome (AS) was completed in July 2025, and the Phase 2/3 Aurora study is expected to begin in the second half of 2025[24, 49, 60, 97] - For DTX401 in Glycogen Storage Disease Type Ia (GSDIa), BLA submission is expected in Q4 2025, with a potential launch in 2026[75, 77, 97] Phase 3 data demonstrated a 41% statistically significant reduction in daily cornstarch intake at Week 48 (p < 00001)[80] - For UX111 in Sanfilippo syndrome (MPS IIIA), the company is actively working to resolve FDA observations in the Complete Response Letter (CRL)[66] - Stage 1, Cohort 4 enrollment completion for UX701 in Wilson Disease (WD) is expected in the second half of 2025[85, 87, 97] In Stage 1, 6 out of 15 patients were completely off chelators and/or zinc therapy[88, 90]

Core Insights - Pacira BioSciences reported solid execution in its corporate, clinical, and commercial initiatives, advancing its 5x30 growth strategy aimed at accelerating topline growth and transitioning into an innovative biopharmaceutical organization [4][5] Financial Highlights - Total revenues for Q2 2025 were $181.1 million, an increase from $178.0 million in Q2 2024 [11] - Net product sales included $142.9 million for EXPAREL, $31.3 million for ZILRETTA, and $5.6 million for iovera° [6] - The company reported a net loss of $4.8 million, or $0.11 per share, compared to a net income of $18.9 million, or $0.41 per share in Q2 2024 [11] - Adjusted EBITDA for Q2 2025 was $54.3 million, down from $62.1 million in Q2 2024 [11] Business Developments - The company surpassed 50% enrollment in its Phase 2 ASCEND study of PCRX-201 for knee osteoarthritis, with completion expected by the end of 2025 [7] - A new $300 million revolving credit facility was established to enhance liquidity and financial flexibility [7] - A co-promotion agreement with Johnson & Johnson MedTech aims to expand ZILRETTA's market reach [7] - Manufacturing efficiencies from large-scale EXPAREL production are expected to improve gross margins and inventory management [7] Legal and Patent Developments - A favorable court ruling resulted in a $28.3 million payment from the Research and Development Foundation [8] - The company secured additional patents for EXPAREL, extending exclusivity through 2041 and 2044 [8] Financial Guidance - The company updated its full-year 2025 revenue guidance to a range of $730 million to $750 million, while increasing non-GAAP gross margin guidance to 78-80% [19]

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small molecule therapies for other ophthalmic disorders [1][3] - The company will have its CEO, George Magrath, participate in a fireside chat at the H.C. Wainwright 5th Annual Ophthalmology Virtual Conference on August 13, 2025 [1][2] Company Overview - Opus Genetics is developing AAV-based gene therapies targeting inherited retinal diseases such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - The lead candidate, OPGx-LCA5, is currently in a Phase 1/2 trial for LCA5-related mutations and has shown promising early results [3] - Additional programs include OPGx-BEST1 for BEST1-related retinal degeneration and a Phase 3-ready small molecule therapy for diabetic retinopathy, developed under a Special Protocol Assessment with the FDA [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and is being studied in two Phase 3 programs for presbyopia and dim light vision disturbances [3] - Opus Genetics is headquartered in Research Triangle Park, NC [3]

Last week, Sarepta voluntarily and temporarily paused all U.S. shipments of Elevidys. Elevidys had received traditional approval in June 2024 for ambulatory Duchenne muscular dystrophy (DMD) patients aged four years and older with a confirmed DMD gene mutation. Previously, in June 2023, it secured accelerated, conditional approval for non-ambulatory DMD patients. As gene therapies continue to evolve as a promising frontier in medical treatment, regulatory scrutiny intensifies following safety concerns. Rece ...

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) Roche will continue its dialogue with the EMA to explore a potential path forward to make Elevidys available to individuals living with DMD in the EURoche believes the benefit-risk remains positive in the ambulatory Duchenne populationElevidys is the first and only disease-modifying gene therapy for DMD Basel, 25 July 2025 - Roche (SIX: RO, ...

Core Viewpoint - Insmed Incorporated will release its second-quarter 2025 financial results on August 7, 2025, and will host a conference call for investors to discuss these results and provide a business update [1][2]. Company Overview - Insmed is a global biopharmaceutical company focused on delivering first- and best-in-class therapies for patients with serious diseases, with a diverse portfolio of approved and investigational medicines [4]. - The company is advancing programs primarily in pulmonary and inflammatory conditions, including a therapy approved in the U.S., Europe, and Japan for a chronic lung disease [4]. - Insmed's early-stage programs involve various technologies such as gene therapy, AI-driven protein engineering, and synthetic rescue [4]. Conference Call Details - The conference call for investors will begin at 8:00 a.m. ET on August 7, 2025, and can be accessed via phone or webcast [2]. - A replay of the conference call will be available approximately one hour after its completion until August 14, 2025 [3].