Crispr Technology & Applications - Crispr 基因编辑技术能够对患者的 DNA 进行精确编辑，从而识别新的治疗靶点并纠正某些疾病，扩大可治疗疾病的范围 [2] - Crispr 技术与 AI 结合，加速识别靶点和疗法，从而加快新疗法上市的速度 [3] - 癌症，尤其是无法以其他方式治疗的儿童疾病和罕见癌症，可能是 Crispr 技术首先产生影响的领域 [5] Regulatory Landscape - FDA 对新的治疗方法和技术平台持支持态度，并倾向于快速批准 [5] - 美国卫生与公众服务部 (HHS) 部长承诺支持细胞和基因治疗领域，这对行业来说是一个巨大的推动力 [7] - 众议院拨款委员会也表达了对细胞和基因治疗领域的支持，释放了积极信号 [7] Manufacturing & Supply Chain - 该公司主要在美国本土进行生产，对进口尤其是来自中国的依赖程度不高 [9] - 公司在马萨诸塞州进行生产，并在匹兹堡进行扩张 [9] - 匹兹堡的团队致力于支持当地学校的 STEM (科学、技术、工程和数学) 项目 [9]

Vera Therapeutics (VERA) FY 2025 Conference June 11, 2025 03:15 PM ET Speaker0 Okay. Good afternoon. We'll continue with the next session. I'm Paul Choi, and I cover the SpidCap biotech sector here at Goldman Sachs. It's my pleasure to have Vera Therapeutics here on stage with us. To my immediate left is CEO, Marshall Fortis, and to my far left, Chief Medical Officer Rob Brenner. Maybe what we'll do is let Marshall kick it off with maybe some high level comments or an introduction of Avera, just kind of wha ...

Scholar Rock Holding (SRRK) FY 2025 Conference June 11, 2025 09:20 AM ET Speaker0 Joining us, I'm Salveen Richter, biotechnology analyst at Goldman Sachs, and it's a pleasure to have, with us David Halal, the CEO of ScholarRock. So, David, to to start here, you recently took over as the CEO of ScholarRock after after serving as the Chairman of the Board of Directors since 2017, and you were previously the CEO of Alexion. Can you discuss your decision to step into this role, and your vision for the company, ...

NewAmsterdam Pharma Company (NAMS) 2025 Update / Briefing June 11, 2025 09:00 AM ET Speaker0 Alright. Good morning, everybody. Welcome to New Amsterdam's R and D Day. Thank you for everyone to show up who's live here as well as everyone who's on the webcast. We have a exciting day planned for you. Speaker1 Let's see here. Speaker0 you know, we may be making forward looking statements today, so please everyone pay attention to this slide. So just to kind of give you a brief overview of what will be discussed ...

Welcome to R&D Day June 11, 2025 Disclaimer Forward Looking Statements Certain statements included in this presentation (together with oral statements made in connection herewith, this "Presentation") that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements generally are accompanied by words such as "believe," "may," "will," "estimate," "continue," "anticipate," ...

NEWARK, N.J., June 11, 2025 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL; NYSE American: RFL-WT), today reported its financial results for the third quarter and first nine months of fiscal year 2025 ended April 30, 2025. “We are pleased to have completed our merger with Cyclo Therapeutics and look forward to reporting the topline data from the 48-week interim analysis of the pivotal Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 anticipated ...

ALKIVIA data demonstrate significant improvement in muscle strength and physical function in myositis patients treated with efgartigimodRHO data show efgartigimod achieved sustained reduction in autoantibodies and improved functional outcomes in patients with Sjogren’s disease; program granted U.S. FDA Fast Track designationargenx committed to new therapeutic areas in rheumatology with ongoing Phase 3 studies in myositis (ALKIVIA) and Sjogren’s disease (UNITY) June 11, 2025, 12:01 AM CET Amsterdam, the Net ...

Financial Data and Key Metrics Changes - The company is executing a Fit for Growth program aimed at optimizing its cost structure, with a target of achieving $800 million in net cost savings by the end of the year [44] - The company has successfully executed activities necessary to yield $1 billion in growth and $800 million in net savings [44] Business Line Data and Key Metrics Changes - The company has shifted its focus from primarily neuroscience to include immunology and nephrology, with significant investments in four ongoing product launches [4][9] - The company has nine programs in phase three or phase three ready, indicating a robust pipeline for future growth [7] Market Data and Key Metrics Changes - The U.S. launch of Skyclaris is in a steady growth phase, while initial launches in Europe are progressing similarly [48] - The company has received approval in Brazil for Skyclaris, which is expected to be an important market [49] Company Strategy and Development Direction - The company is focused on redeploying capital from its MS business to support new growth opportunities in nephrology and immunology [5] - The company is actively pursuing business development opportunities, including the acquisition of Hai Bio, to enhance its pipeline [7][8] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the foundational position of the company for future capital deployment and growth opportunities [6] - The company is monitoring potential changes in drug pricing policies and tariffs, which could impact negotiations in the EU [14][15] Other Important Information - The company is excited about the potential of its pipeline assets, particularly in rare diseases and kidney diseases, as highlighted in an upcoming investor presentation [40][41] - The company is leveraging AI technology to identify patients for its therapies, indicating a focus on innovative approaches in patient engagement [49] Q&A Session Summary Question: How is the firm balanced now in terms of various disease areas? - The company is leveraging existing expertise in rare diseases while expanding into nephrology and immunology through strategic acquisitions [10] Question: What is the comfort level among PCP providers with the use of blood-based biomarker tests for Alzheimer's? - PCP providers are already using blood-based biomarkers alongside PET scans, indicating a growing acceptance of these tests [26] Question: What are the expectations for the upcoming readouts from the company's trials? - The company anticipates steady progression in growth and is optimistic about the potential for inflection points with new diagnostic tests and treatment methods [27]

Orchestra BioMed Holdings (OBIO) 2025 Conference June 05, 2025 03:45 PM ET Speaker0 Okay, Speaker1 good to go. So thanks everybody for joining for our next session. I'm Matt Taylor, the US Medical Supplies and Devices analyst here at Jefferies. I'm joined by David Hockman, the CEO of Orchestra Lab. Speaker0 Nice to be here. Speaker1 And we have about half an hour for moderated Q and A. And I'll get things rolling here with kind of a high level question. So I think let's start with your business model is rea ...

Disc Medicine (IRON) 2025 Conference June 05, 2025 10:30 AM ET Speaker0 Excellent. All right. Good morning, everyone. Welcome to twenty twenty five Jefferies Global Healthcare Conference. My name is Roger Song, one of the seniors covers MEKAP at Jefferies. It's my pleasure to have the next fireside chat with DISCMADISON and Theo Zhang. Welcome, Zhang. Speaker1 Yeah, great to be here. Speaker0 Awesome. All right. I think, you know, we had an interview with McCurry yesterday at our conference, and then pretty ...