Core Viewpoint - 4D Molecular Therapeutics announced positive results from the SPECTRA clinical trial for 4D-150 in diabetic macular edema (DME), indicating a potential shift in treatment paradigms and alignment with the European Medicines Agency (EMA) for a registrational pathway [1][6]. Clinical Trial Results - The SPECTRA trial demonstrated that 4D-150 is well tolerated with no intraocular inflammation observed at any timepoint or dose level [4] - The Phase 3 dose of 4D-150 achieved a clinically meaningful 78% reduction in treatment burden compared to the standard-of-care aflibercept 2mg every eight weeks [4][10] - Efficacy results showed a sustained gain in best corrected visual acuity (BCVA) of +9.7 letters and a reduction in central subfield thickness (CST) of -174 µm [5] Regulatory Alignment - Both the FDA and EMA have aligned on a single Phase 3 clinical trial as acceptable for regulatory submission for 4D-150 in DME, streamlining the path for marketing authorization [6][9] Product Overview - 4D-150 is designed to provide multi-year sustained delivery of anti-VEGF from a single intravitreal injection, aiming to reduce the treatment burden associated with current therapies [7][9] - The investigational agent targets both wet age-related macular degeneration and DME, addressing significant unmet medical needs in these conditions [7][11] Market Context - DME is a prevalent complication of diabetic retinopathy, affecting approximately one million individuals in the U.S., characterized by inflammation and swelling in the macula leading to vision loss [8]

Longtime drug developer Suma Krishnan was 51 when she cofounded Krystal Biotech. Now the company has one gene therapy on the market and more in the works. #ForbesOver50 (Photo: Jamel Toppin for Forbes) https://t.co/hb8RGouKMY https://t.co/5138pb6yHR ...

Core Viewpoint - Sarepta Therapeutics, Inc. has faced significant stock price decline following the reported deaths of patients in clinical trials for its gene therapy drug ELEVIDYS, leading to a securities class action lawsuit from investors [1][4][10]. Group 1: Company Developments - On July 18, 2025, Sarepta's shares closed down $7.89, a decrease of 36%, after the announcement of a third patient death related to its experimental gene therapy [1]. - The lawsuit focuses on Sarepta's disclosures regarding the safety and efficacy of ELEVIDYS, which is intended for treating Duchenne muscular dystrophy [4]. - The FDA has placed Sarepta's investigational gene therapy clinical trials for limb-girdle muscular dystrophy on clinical hold due to the reported patient deaths [10]. Group 2: Legal Actions - A securities class action lawsuit has been filed, representing investors who acquired Sarepta securities between June 22, 2023, and June 24, 2025 [2]. - The lawsuit alleges that Sarepta made false and misleading statements about the safety of ELEVIDYS and failed to disclose critical information regarding patient deaths [5][6]. - Hagens Berman, the law firm investigating the case, is urging affected investors to report their losses and is looking for individuals with knowledge to assist in the investigation [3][11]. Group 3: Regulatory and Market Impact - Following the patient deaths, Sarepta halted recruitment and dosing in some ELEVIDYS clinical studies, which has attracted regulatory scrutiny [7][8]. - The severity of adverse events related to ELEVIDYS has raised concerns about the therapy's current and future approval prospects [8]. - H.C. Wainwright has reduced its price target for Sarepta to $0, reflecting the negative market sentiment following these developments [10].

Company Overview - REGENXBIO Inc. is a biotechnology company focused on gene therapy with a mission to improve lives through its curative potential [3] - The company has been a pioneer in AAV gene therapy since its founding in 2009 [3] - REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne, RGX-121 for MPS II, and RGX-111 for MPS I, in partnership with Nippon Shinyaku [3] - The company is also collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3] - Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3] Upcoming Events - REGENXBIO will host a conference call on August 7, 2025, at 8:00 a.m. ET to discuss its financial results for Q2 2025 and operational highlights [1] - Listeners can register for the webcast and analysts can participate in the Q&A session by dialing specific numbers [2] - A replay of the webcast will be available on the company's investor website approximately two hours after the call [2]

Vinay Prasad, a top regulator at the US Food and Drug Administration, has left the agency after a controversy over his handling of Sarepta's gene therapy https://t.co/QyzJGlMYd0 ...

Financial Data and Key Metrics Changes - Revenue for Q2 2025 was $5.3 million, a decrease from $11.1 million in Q2 2024, primarily due to a $7.1 million drop in collaboration revenue and a $2.1 million decrease in contract manufacturing revenue [18][19] - Research and development expenses increased to $35.4 million in Q2 2025 from $33.7 million in the same period in 2024, driven by a $6.3 million rise in external program spending [19] - Cash, cash equivalents, and investment securities totaled $377 million as of June 30, 2025, up from $367.5 million at the end of 2024, attributed to net proceeds of $80.5 million from a follow-on offering [20][21] Business Line Data and Key Metrics Changes - The company is advancing AMT-130 for Huntington's disease, with pivotal data expected in September 2025, and has received breakthrough therapy designation from the FDA [6][7] - AMT-191 for Fabry disease and AMT-260 for mesial temporal lobe epilepsy are also progressing, with initial data from AMT-191 expected at the ICIEM Conference in September [10][17] Market Data and Key Metrics Changes - The company has 14 clinical sites in the U.S. screening patients for AMT-260, with strong interest from the epilepsy community following early positive data [10][17] - There are approximately 35,000 diagnosed Huntington's disease patients in the U.S., with potentially three times that number undiagnosed [70] Company Strategy and Development Direction - The company is focused on preparing for a potential commercial launch of AMT-130 in 2026, with disciplined investments in commercial planning and recruitment for key roles [9][21] - The strategy includes leveraging prior knowledge from Hemgenics to validate the manufacturing process for AMT-130 [12][13] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming pivotal data and regulatory progress, indicating a transformational second half of 2025 [22][24] - The company is committed to delivering transformative therapies for patients with serious unmet needs, emphasizing the urgency of effective treatments for Huntington's disease [23][24] Other Important Information - The company has achieved alignment with the FDA on the statistical analysis plan and CMC requirements for AMT-130, which is crucial for the planned BLA submission [8][12] - The company is actively monitoring the safety and efficacy of AMT-130, including a new cohort for patients with lower striatal volumes [14][59] Q&A Session Summary Question: Does the FDA expect a minimum threshold for clinical benefit versus the enrolled HD on CUHDRS? - Management indicated that the FDA has not requested a minimum clinical effect and is optimistic about the three-year data supporting accelerated approval [26] Question: Can you walk us through the AMT-130 procedure from a patient's perspective? - The procedure is described as minimally invasive, with patients typically recovering quickly and returning to work within days [31][34] Question: What are the expectations for the fourth cohort in the AMT-130 trial? - The goal is to document the safety of administering AMT-130 to patients who would have otherwise been excluded due to lower striatal volumes [58] Question: Are there any differences expected in the regulatory path to approval in Europe versus the U.S.? - The company has not yet met with EMA for scientific advice and is currently focused on the U.S. regulatory path [61] Question: What are the next steps for AMT-260? - The company plans to increase the number of active sites for AMT-260 and is confident in patient enrollment in the second half of the year [59]

Core Insights - uniQure N.V. has achieved alignment with the FDA on the statistical analysis plan and CMC requirements for AMT-130, supporting a planned BLA submission in Q1 2026 [1][4] - The company expects to present topline three-year data for AMT-130 in September 2025 [1][4] - Early clinical data for AMT-260 shows a 92% reduction in seizures for the first patient treated, with no serious adverse events reported [1][4] - The appointment of Kylie O'Keefe as Chief Customer and Strategy Officer is aimed at strengthening the leadership team [1][4] - As of June 30, 2025, the company has approximately $377.0 million in cash and equivalents, expected to fund operations into the second half of 2027 [1][5] Recent Developments - The FDA's alignment supports an Accelerated Approval pathway for AMT-130, with a focus on the three-year change in the composite Unified Huntington's Disease Rating Scale [4] - The final statistical analysis plan was submitted to the FDA in July 2025, and a PPQ campaign has been initiated [4] - AMT-130 has received Breakthrough Therapy designation from the FDA based on Phase I/II trial evidence [4] Financial Performance - Revenue for Q2 2025 was $5.3 million, a decrease from $11.1 million in Q2 2024, attributed to changes in license and collaboration revenue [6] - Research and development expenses increased to $35.4 million in Q2 2025 from $33.7 million in Q2 2024, driven by external program spending [9] - Selling, general and administrative expenses decreased to $13.5 million in Q2 2025 from $15.8 million in Q2 2024 [10] - The net loss for Q2 2025 was $37.7 million, compared to a net loss of $56.3 million in the same period in 2024 [13][21] Clinical Programs - AMT-260 is in clinical trials for refractory mesial temporal lobe epilepsy, with initial data showing significant seizure reduction [7] - AMT-191 for Fabry disease and AMT-162 for ALS are also in development, with initial data presentations expected in September 2025 and the first half of 2026, respectively [7] Leadership Changes - Kylie O'Keefe's appointment as Chief Customer and Strategy Officer is part of the company's strategy to enhance its commercial and medical affairs [1][4]

In a major win for Sarepta, US regulators are recommending that patients who can walk be allowed to take its gene therapy Elevidys again https://t.co/lnGkzEsubE ...

Core Viewpoint - Rocket Pharmaceuticals is undergoing a strategic reorganization to extend its operational runway and focus on its adeno-associated virus (AAV) cardiovascular platform, which includes several clinical programs [1][2][3]. Group 1: Corporate Reorganization - The restructuring initiative will prioritize resources on clinical programs for Danon disease, PKP2-associated arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy, while also addressing the complete responses to the CRL for KRESLADI [2][3]. - The company plans to reduce its workforce by approximately 30%, which, along with other cost-saving measures, is expected to decrease 12-month operating expenses by nearly 25% [3]. Group 2: Financial Outlook - Rocket anticipates that its existing cash resources will fund operations into the second quarter of 2027 [3]. - Analyst Jason Zemansky from Bank of America downgraded Rocket from Buy to Neutral and reduced the price forecast from $9 to $4, citing weakened investor confidence due to recent failures in the gene therapy sector [5][6]. Group 3: Program Delays and Regulatory Challenges - The company expects delays in the Fanconi Anemia and Pyruvate Kinase Deficiency programs, with regulatory approval for RP-L102 in 2026 now deemed unlikely [4]. - A clinical hold was placed by the FDA on the Phase 2 pivotal trial of RP-A501 for Danon disease, further complicating the company's outlook [4].

A new report saying Serepta would need to conduct new studies in order to show the FDA that its musculardrophe drug Elevus is safe. And just this morning, EU regulators not recommending approval of that treatment. As a result, that stock is down another 13%.Joining us right now on that and what to expect from the other biotech names reporting next week is Saline Richtor, lead biotech analyst at Goldman Sachs. and Saline, let's go ahead and start with the news of the day since it's out with Surrepta. Um, jus ...