Core Viewpoint - A securities class-action lawsuit has been initiated against Sarepta Therapeutics due to safety concerns and regulatory setbacks related to its gene therapy, ELEVIDYS, alleging that the company misled investors about the treatment's safety and efficacy [1]. Company Developments - The lawsuit claims that Sarepta misrepresented ELEVIDYS as a safe and effective treatment with a clear path to regulatory approval, which has led to significant investor losses [1]. - The European Medicines Agency (EMA) rejected ELEVIDYS on July 24, 2025, after a trial involving 125 children showed no significant improvement in movement compared to a placebo, severely impacting Sarepta's European market expansion plans [3]. - The timeline of disclosures indicates that Sarepta's stock price has faced multiple declines due to safety and regulatory news, including a patient death in an ELEVIDYS trial reported on March 18, 2025, and a subsequent investigation by EU authorities [6]. Legal and Investigative Actions - Hagens Berman, a national shareholder rights firm, is investigating claims on behalf of investors who suffered substantial losses and is encouraging individuals with relevant information to come forward [4][5]. - The firm has set a deadline of August 25, 2025, for investors to be considered as Lead Plaintiff in the lawsuit, which covers the class period from June 22, 2023, to June 24, 2025 [2].

SAN FRANCISCO, Aug. 22, 2025 (GLOBE NEWSWIRE) -- A securities class-action lawsuit is underway against Sarepta Therapeutics (NASDAQ: SRPT), triggered by a series of safety concerns and regulatory setbacks for its gene therapy, ELEVIDYS. The lawsuit alleges that Sarepta misled investors by presenting ELEVIDYS as a safe and effective treatment with a clear path to broader regulatory approval. The deadline for investors with losses to be considered by the court to serve as Lead Plaintiff is August 25, 2025. Ha ...

SAN FRANCISCO, Aug. 20, 2025 (GLOBE NEWSWIRE) -- A securities class-action lawsuit is underway against Sarepta Therapeutics (NASDAQ: SRPT), triggered by a series of safety concerns and regulatory setbacks for its gene therapy, ELEVIDYS. The lawsuit alleges that Sarepta misled investors by presenting ELEVIDYS as a safe and effective treatment with a clear path to broader regulatory approval. The deadline for investors with losses to be considered by the court to serve as Lead Plaintiff is August 25, 2025. Ha ...

SAN FRANCISCO, Aug. 18, 2025 (GLOBE NEWSWIRE) -- The pending securities class action was triggered after news of safety problems with Sarepta’s (NASDAQ: SRPT) ELEVIDYS, a medication intended for the treatment of Duchenne muscular dystrophy. The deadline for Sarepta investors with losses who wish to be considered by the court to serve as Lead Plaintiff is August 25, 2025. Accordingly, national shareholders rights firm Hagens Berman urges Sarepta investors who suffered substantial losses to submit your losses ...

Oculis Holding (OCS) Fireside Chat August 13, 2025 11:00 AM ET Speaker0Good morning, and thank you for joining the HC Wainwright fifth Annual Ophthalmology Virtual Conference. For this session, we have a panel discussion, and I'd like to welcome the following panelists to share with us their view on multiple drug candidates currently in pivotal stage of clinical development for retinal disorders. Doctor Hendrik Shaw, chief medical officer of Eliot Bio. Doctor Snehao Shah, president of r and d at Oculus Hold ...

Summary of Adverum Biotechnologies (ADVM) FY Conference - August 13, 2025 Company Overview - **Company**: Adverum Biotechnologies - **Ticker**: ADVM - **Focus**: Developing potential one-time gene therapies for debilitating ocular diseases, specifically targeting wet age-related macular degeneration (AMD) [1][5] Core Points and Arguments - **Product Development**: - Adverum is in Phase 3 of developing Ixavec, an intravitreal gene therapy delivering aflibercept, which is the gold standard for wet AMD treatment [3][6] - Ixavec aims to provide sustained aflibercept production in the retina, potentially for life [6] - **Clinical Trial Results**: - In the OPTIC and LUNA trials, Adverum achieved 50% injection freedom and an 80% reduction in treatment burden for hard-to-treat patients [7] - The ARTEMIS trial is currently enrolling rapidly, indicating strong interest from investigators and patients [7] - **Market Need**: - There are over 1.5 million wet AMD patients in the US and over 20 million globally, with a significant annual diagnosis rate [9] - Despite existing anti-VEGF therapies, many patients experience vision decline due to under-treatment and missed injections, highlighting a large unmet need for more durable treatments [9][10] - **Competitive Landscape**: - The gene therapy approach is seen as transformational, offering the potential for injection-free treatment, which is highly favored by retina specialists [11][12] - Adverum's Ixavec is positioned as best-in-class with the highest injection-free rates compared to competitors [28] - **Patient Preference**: - In the LUNA trial, 93% of patients preferred Ixavec over previous treatments, and 95% would choose it if diagnosed with wet AMD in both eyes [25] - **Phase 3 Trial Details**: - The ARTEMIS trial will randomize approximately 284 patients into two cohorts: one receiving Ixavec and the other receiving aflibercept [27] - The primary endpoint is non-inferiority in best-corrected visual acuity (BCVA) at 52-56 weeks [27] Additional Important Content - **Safety Profile**: - Ixavec has shown a favorable safety profile with no serious adverse events related to the treatment, and inflammation, when it occurred, was manageable with local steroids [23][24] - **Long-term Efficacy**: - Patients in the OPTIC trial maintained vision levels and showed significant reductions in central subfield thickness over four years, indicating a disease-modifying effect [14][22] - **Market Potential**: - Wet AMD represents a large market opportunity for gene therapy, with the potential for mass market pricing due to high prevalence and annual incidence [28] - **Future Plans**: - Adverum plans to initiate a second Phase 3 trial, Aquarius, in the second half of the year and release additional long-term follow-up data from the LUNA trial in Q4 [29]

Summary of Tenaya Therapeutics (TNYA) FY Conference Call Company Overview - **Company**: Tenaya Therapeutics (TNYA) - **Founded**: 2016 - **Mission**: Focus on therapies for heart disease, including both rare genetic forms and prevalent forms [3][4] Pipeline and Clinical Development - **Clinical Stage Assets**: Three clinical stage assets currently in development [4] - **Gene Therapies**: Focus on gene therapies for genetic causes of cardiomyopathy and a small molecule for HFpEF [4] - **Clinical Sites**: Over 40 clinical sites active in seven countries for patient recruitment [4] Gene Therapy Programs TN-201 for MYBPC3 Positive Hypertrophic Cardiomyopathy - **Target Disease**: Leading genetic cause of hypertrophic cardiomyopathy, affecting approximately 120,000 patients in the US [15] - **Mechanism**: Addresses deficiency of myBPC3 protein, leading to heart thickening and potential heart failure [16][17] - **Natural History Study**: MyCLIMB study with over 220 children enrolled to characterize disease progression [20][21] - **Phase 1b Study**: Completed dosing of high dose cohort; initial results show improvement in heart function [25][27] - **Upcoming Data**: Full cohort data expected in Q4 2025, with focus on safety and efficacy [37] TN-401 for PKP2-Related Arrhythmogenic Right Ventricular Cardiomyopathy - **Target Disease**: Accounts for about 40% of arrhythmogenic right ventricular cardiomyopathy cases, approximately 70,000 patients in the US [44] - **Mechanism**: Aims to add a copy of the human gene to improve desmosome function and reduce arrhythmia burden [46] - **Biopsy Data**: Expected in Q4 2025, focusing on vector copy number, RNA, and protein levels [50][51] Safety and Efficacy - **AAV9 Vector**: Selected for its extensive safety database and effectiveness in cardiac applications [5][6] - **Safety Record**: No significant safety issues reported in ongoing trials; adverse events consistent with other gene therapies [13][14] - **Patient Outcomes**: Initial data shows symptomatic improvement in patients treated with TN-201 [25][27] Small Molecule Program TN-301 - **Status**: Completed first-in-human study with 72 patients; well tolerated with no dose-limiting toxicities [55] - **Future Plans**: Exploring opportunities to advance the program, focusing on HFpEF and potentially severe rare diseases [56] Key Takeaways - **Market Opportunity**: Both TN-201 and TN-401 target large orphan conditions with significant unmet medical needs [44][46] - **Data Releases**: Important data updates expected in Q4 2025 for both gene therapy programs, which could influence future clinical development strategies [37][50]

Vinay Prasad, who was ousted as the top vaccine and gene therapy regulator at the US Food and Drug Administration, is returning to his role https://t.co/snfLbLJi9Y ...

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].