[Music] [Music] [Applause] Hello everyone. My name is Jason. I'm 8 years old.I love space and I love chocolates. Today I will talk about space but maybe eat a chocolate later. Our galaxy is called the Milky Way and guess what.It has waves. These are called gravitational waves. But who makes them.Well, scientists say that when two black holes crash into each other, they make these waves. Wait a minute. What is a black hole.It's like a star, but it's not shining anymore. It's super strong and it pulls everyth ...

Company Presentation June 2025 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking sta ...

Core Viewpoint - PepGen Inc. is facing a class action lawsuit due to allegations of misleading statements regarding the safety and efficacy of its lead product candidate, PGN-EDO51, and the adequacy of its clinical trials [3][4][5]. Company Overview - PepGen Inc. is a clinical-stage biotechnology company focused on developing oligonucleotide therapeutics for severe neuromuscular and neurologic diseases, with PGN-EDO51 targeting Duchenne muscular dystrophy (DMD) [3]. Allegations and Events - The class action lawsuit claims that PepGen made false statements about PGN-EDO51's safety and effectiveness, inadequately conducted the CONNECT2 clinical trial, and overstated the product's clinical and regulatory prospects [3]. - On July 30, 2024, PepGen reported "positive clinical data" from the CONNECT1 study, but the results were deemed disappointing by analysts, leading to a nearly 33% decline in stock price [4]. - On December 16, 2024, PepGen received a clinical hold notice from the FDA regarding the CONNECT2 study, causing further stock decline [5]. - On January 29, 2025, dosing in the CONNECT1 study was paused due to safety concerns, resulting in an approximate 22% drop in stock price [6]. - On March 4, 2025, PepGen announced a voluntary temporary pause of the CONNECT2 study, leading to another nearly 19% decline in stock price [7].

Core Insights - Biogen Inc. announced topline results from the Phase 1 study of salanersen, an antisense oligonucleotide for spinal muscular atrophy (SMA), showing potential for high efficacy and once yearly dosing [1][5] - The Phase 1 study demonstrated substantial slowing of neurodegeneration and clinically meaningful improvements in motor function in children previously treated with gene therapy [2][5] - Biogen is engaging with global health authorities to advance salanersen into registrational studies based on encouraging Phase 1 data [4][5] Study Details - The Phase 1 study included two parts: a randomized placebo-controlled segment in healthy adults and an open-label segment in pediatric SMA participants who had previously received ZOLGENSMA [2][4] - Interim results from the open-label segment (n=24) indicated that both 40 mg and 80 mg doses of salanersen were well-tolerated, with a mean reduction in neurofilament light chain (NfL) of 70% at 6 months [2][4] - Exploratory data showed that half of the participants (4 out of 8) achieved new WHO motor milestones after receiving salanersen [3][4] Safety Profile - The safety profile of salanersen was generally well-tolerated, with most adverse events being mild to moderate, including pyrexia and upper respiratory tract infections [4][5] - The study's findings suggest that salanersen could address critical unmet needs in SMA treatment, building on Biogen's extensive experience in the field [3][5] Industry Context - SMA is a rare genetic neuromuscular disease affecting approximately 1 in 10,000 live births, characterized by progressive muscle atrophy and weakness [7][8] - SPINRAZA, another treatment for SMA, has been approved in over 71 countries and has treated more than 14,000 individuals worldwide, establishing a foundation of care in SMA [9][10]

-- FDA alignment on accelerated and full approval pathways for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD) ---- Jeffrey M. Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE trial investigator, will present topline del-brax data from dose escalation cohorts in oral presentation ---- Stephen Tapscott, M.D., Ph.D., Professor of Human Biology and Clinical Research, Fred Hutchinson Cancer Center, will highlight results on the charac ...

Core Insights - Avidity Biosciences announced positive topline data from the Phase 1/2 FORTITUDE™ program for delpacibart braxlosiran (del-brax) in treating Facioscapulohumeral Muscular Dystrophy (FSHD), showing improvements in function, strength, and patient-reported outcomes compared to placebo [1][3][6] - The company plans to submit a Biologics License Application (BLA) for accelerated approval in the second half of 2026 [1][3] - Del-brax is the first investigational therapy targeting the underlying cause of FSHD by directly addressing the DUX4 gene, with no approved therapies currently available for this condition [2][13] Group 1: Clinical Data and Results - The FORTITUDE™ trial included a randomized, placebo-controlled, double-blind study evaluating 39 participants on doses of 2 mg/kg or 4 mg/kg of del-brax over 12 months [5][10] - Results indicated consistent improvements in functional mobility and muscle strength, as measured by the 10-Meter Walk-Run test, Timed Up and Go, and quantitative muscle testing [6][9] - Significant reductions in biomarkers such as KHDC1L and creatine kinase were observed, indicating muscle damage [6][7] Group 2: Safety and Regulatory Pathway - Del-brax demonstrated favorable long-term safety and tolerability, with most adverse events being mild or moderate, and no serious adverse events reported [7][9] - The U.S. FDA has opened the accelerated approval regulatory pathway for del-brax, and a global Phase 3 FORWARD™ study has been initiated [3][4] Group 3: Future Developments - The ongoing biomarker cohort of the FORTITUDE trial aims to assess the impact of del-brax on KHDC1L levels, with topline data expected in Q2 2026 [7][11] - Avidity is advancing its clinical development pipeline, focusing on RNA therapeutics through its proprietary Antibody Oligonucleotide Conjugates (AOCs™) platform [16]

Core Insights - DuPont de Nemours, Inc. (DD) has launched AmberChrom TQ1 chromatography resin aimed at purifying oligonucleotides and peptides for biopharma applications, enhancing its product portfolio and establishing a new benchmark in the industry [1][2][3] Group 1: Product Launch and Features - The AmberChrom TQ1 chromatography resin is an agarose-based product that offers higher loading capacity, increased throughput, and lower pressure requirements compared to competing products, making it suitable for high-resolution separations and large-scale purifications [2] - The resin is designed to work in conjunction with existing AmberChrom XT and CG chromatography resins, facilitating the purification of various oligonucleotide feeds and aiding in the crude purification of peptide feeds [3] Group 2: Performance Validation - DuPont has published internal application studies using real-world drug feeds to validate the performance characteristics of the AmberChrom TQ1 resin, supporting its integration into processes from drug discovery to commercialization [3] Group 3: Financial Outlook - For the second quarter, DuPont projects net sales of approximately $3.2 billion, operating EBITDA of around $815 million, and adjusted earnings per share of about $1.05, reflecting a seasonal increase in sales [6] - The company has maintained its full-year 2025 guidance, indicating confidence in its financial performance moving forward [6] Group 4: Stock Performance - DuPont's stock has experienced a decline of 14.5% over the past year, which is less severe compared to the industry's overall decline of 26.6% [5]

Core Insights - Avidity Biosciences has completed enrollment in the FORTITUDE biomarker cohort for delpacibart braxlosiran (del-brax), targeting accelerated approval for treating facioscapulohumeral muscular dystrophy (FSHD) [1][2] - The company plans to present topline data from the FORTITUDE trial in Q2 2025, with regulatory alignment anticipated for a global Phase 3 trial [1][6] - Del-brax aims to be the first approved therapy for FSHD, a rare disease affecting approximately 45,000 to 87,000 individuals in the U.S. and EU [2][10] Company Overview - Avidity Biosciences focuses on RNA therapeutics, specifically Antibody Oligonucleotide Conjugates (AOCs™), to address rare neuromuscular diseases [11] - The company is advancing clinical programs for multiple conditions, including myotonic dystrophy type 1 and Duchenne muscular dystrophy, alongside FSHD [11] Clinical Trial Details - The FORTITUDE trial is a Phase 1/2 randomized, placebo-controlled study evaluating del-brax in 90 participants with FSHD [3] - The trial assesses safety, tolerability, pharmacokinetics, and pharmacodynamics, using biomarkers like DUX4-regulated genes and MRI measures [3][4] - The ongoing biomarker cohort evaluates del-brax at a dose of 2 mg/kg every six weeks for 12 months, focusing on changes in DUX4-regulated gene expression [5] Efficacy and Safety Data - Initial data from the 2 mg/kg dose of del-brax show over 50% reductions in DUX4-regulated genes, alongside trends of functional improvement and favorable safety [2][9] - The trial's design includes three dose cohorts, with the 2 mg/kg every six weeks identified for future studies [4] Future Plans - Avidity aims to share additional data and key milestones from the del-brax program in Q2 2025, including regulatory updates and topline results [2][6]

Codexis, Inc. (NASDAQ:CDXS) Q4 2024 Earnings Conference Call February 27, 2025 4:30 PM ET Company Participants Carrie McKim - Director of Investor Relations Stephen Dilly - President and Chief Executive Officer Kevin Norrett - Chief Operating Officer Georgia Erbez - Chief Financial Officer Conference Call Participants Richard Miller - Cantor Fitzgerald, L.P. Matthew Stanton - Jefferies Group LLC Allison Bratzel - Piper Sandler Companies Dan Arias - Stifel Financial Corp. Jacob Johnson - Stephens Inc. Matthe ...