Pipeline and Clinical Trials - Cartesian Therapeutics is pioneering mRNA cell therapies for autoimmunity, with multiple anticipated near-term catalysts[5] - Phase 3 AURORA study of Descartes-08 for Myasthenia Gravis (MG) is expected to commence in 1H25[8] - An open-label Phase 2 trial of Descartes-08 in Systemic Lupus Erythematosus (SLE) is ongoing, with data readout expected in 2H25[8] - A Phase 2 pediatric basket trial, including juvenile SLE, juvenile MG, and other conditions, is expected to initiate in 2H25[8, 12] - Dosing is underway in a first-in-human Phase 1 dose escalation trial for Descartes-15, a next-generation mRNA CAR-T candidate[8] Descartes-08 Efficacy and Safety - In a Phase 2b trial, participants treated with Descartes-08 maintained deep and durable responses over 12 months[16] - At Month 4, participants in the primary efficacy dataset experienced an average MG-ADL reduction of 5.5 points[23] - 33% of participants achieved minimum symptom expression at Month 6[23] - 80% of participants reaching Month 12 maintained a clinically meaningful response[23] - In participants with no prior exposure to biologics, the average MG-ADL reduction was 6.6 points at Month 4[26] - 57% of participants with no prior exposure to biologics achieved minimum symptom expression at Month 6[26] - 100% of participants with no prior exposure to biologics reaching Month 12 maintained a clinically meaningful response[26] - The safety profile of Descartes-08 supports outpatient administration, with no new types of adverse events reported[16, 28] Financial Position - Cartesian Therapeutics has a strong balance sheet with approximately $220.9 million as of September 30, 2024[9] - This is expected to support planned operations, including completion of the planned Phase 3 trial of Descartes-08 for MG, into mid-2027[9]

NFL super-agent Leigh Steinberg opens up about the brain trauma crisis in football and his mission to protect athletes through advocacy, research, and a groundbreaking collaboration on stem cell therapy. Learn more: https://european-wellness.eu/ The man who inspired the multiple award-winning movie “Jerry Maguire” meets the pioneering stem cell scientist who is transforming regenerative medicine. Together, they may just hold the key to healing the silent epidemic of sports-related brain injuries. A ...

Summary of Lyell Immunopharma (LYEL) Conference Call - June 25, 2025 Company Overview - **Company**: Lyell Immunopharma - **Focus**: Development of next-generation cell therapies for cancer, specifically targeting hematologic malignancies and solid tumors with CAR T cell therapies [4][5] Core Points and Arguments Product Development - **Lead Program**: Autologous CAR T cell therapy targeting CD19 and CD20, aimed at achieving more complete responses and longer remission periods [4][5] - **Clinical Trials**: Currently in pivotal clinical trials for relapsed/refractory large B cell lymphoma, with a focus on outpatient administration due to a favorable safety profile [6][19] Clinical Data - **Third-Line Setting**: - Presented data from 51 patients showing an 88% overall response rate and a 72% complete response rate, significantly higher than the approximately 50% seen with existing CD19 CAR therapies [15][16] - 71% of patients who achieved complete response remained in remission at six months, compared to about 40% for CD19 CARs [16][43] - **Second-Line Setting**: - First data presented showed a 91% overall response rate and a 70% complete response rate in patients with primary refractory disease [18][19] - The patient population included older patients with no upper age limit, broadening the inclusion criteria compared to competitors [18][65] Competitive Landscape - **Comparison with Competitors**: - Lyell's dual-targeting approach (CD19 and CD20) is positioned as a significant advancement over existing CD19-only therapies, which have limitations in patient response [7][26] - Notable competitors include Yescarta (Axi-cel) and Breyanzi (Liso-cel), both of which have shown similar response rates but with higher rates of neurotoxicity [21][23][31] - Lyell's safety profile shows lower rates of cytokine release syndrome (CRS) and neurotoxicity compared to competitors [31][32] Regulatory and Market Strategy - **Regulatory Designation**: Received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, indicating strong early data and facilitating more frequent interactions with the agency [50][56] - **Pivotal Trial Design**: A seamless phase one/two design for the pivotal trial, with plans to accelerate patient recruitment by adding more centers [51][52] - **Market Opportunity**: Targeting a significant patient population in both the second and third-line settings, with an estimated 30,000 new DLBCL cases annually in the U.S. [60] Additional Important Insights - **Patient Demographics**: The focus on older patients and those with primary refractory disease highlights the unmet need in the market and the potential for Lyell's therapies to address this gap [18][62] - **Intellectual Property**: Lyell holds a strong intellectual property position, with patents originating from UCLA, which may provide a competitive edge in the market [36][37] - **Future Outlook**: The company is optimistic about the potential for national priority review vouchers, which could expedite the approval process for their therapies [56][57] This summary encapsulates the key points discussed during the conference call, highlighting Lyell Immunopharma's strategic positioning, clinical advancements, and competitive landscape in the CAR T cell therapy market.

Capricor Therapeutics Inc CAPR on Tuesday provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the company’s lead cell therapy candidate for the treatment of Duchenne Muscular Dystrophy (DMD)—associated cardiomyopathy.DMD is an X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles.It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects ap ...

撰文丨王聪 编辑丨王多鱼 排版丨水成文 2022 年初，宾夕法尼亚大学的研究人员在 Science 期刊发表了一篇重磅研究论文 【1】 ，开发了一种 在体内原位生成 CAR-T 细胞的新技术 （ in vivo CAR- T） ，通过 注射脂质纳米颗粒 （LNP） 递送 mRNA，在体内重编程 T 细胞，使其识别心脏纤维化细胞，从而治疗心脏纤维化疾病。该方法类似于 mRNA 疫 苗，仅需简单注射，就能在体内生成 CAR-T 细胞疗法，有望解决当前 CAR-T 细胞疗法工艺复杂、周期长、价格高昂等关键难题。 不久后，该团队创立了一家名为 Capstan Therapeutics 的公司， 致力于将 in vivo CAR-T 技术应用于癌症、自身免疫疾病及纤维化疾病的治疗，该公司目前以 获得来自多家制药巨头及风投机构的 3.4 亿美元融资。该公司的 创始团队包括 Carl June （CAR-T 细胞治疗先驱） ， Drew Weissman （诺奖得主、mRNA 技 术奠基人） ， Jonathan Epstein （ in vivo CAR-T 技术发明人） 。 2025 年 6 月 11 日， Capst ...

BioRestorative Therapies (BRTX) Update / Briefing June 18, 2025 10:50 AM ET Speaker0 Thank you so much. Hey, Zakers, and welcome back to All Access. I got that wonderful guest for you today, another one, and that's Lance Allstott, the Chairman and CEO of Bio Restorative Therapies. The NASDAQ ticker is BRTX. He's joining us for another exclusive talk today. All Access starts right now. And it is my pleasure to welcome back in the Chairman and CEO of BioRestorative Therapies, Lance Allstad. It's fantastic to ...

Core Insights - Lyell Immunopharma, Inc. announced positive clinical data for LYL314, a CAR T-cell therapy for relapsed/refractory large B-cell lymphoma (LBCL), showing an 88% overall response rate and a 72% complete response rate in patients treated in the third- or later-line setting [1][3][4] Clinical Data Summary - In a Phase 1/2 trial, 25 patients in the 3L+ setting demonstrated an 88% overall response rate and a 72% complete response rate, with 71% of those achieving complete response remaining so at ≥ 6 months [1][3][4] - The efficacy evaluable population included 36 patients, with a median follow-up of 9 months for 3L+ patients and 5 months for 2L patients [2][3] - Among 51 CAR T-naive patients, 70% of those with primary refractory disease achieved a complete response [4] Safety Profile - The safety profile of LYL314 was manageable for outpatient administration, with no Grade ≥ 3 cytokine release syndrome reported and low rates of Grade 1 (22%) and Grade 2 (35%) adverse events [3][5] - Immune effector cell-associated neurotoxicity syndrome (ICANS) was reported in 6% (Grade 1), 2% (Grade 2), and 14% (Grade ≥ 3) of patients, with a median resolution time of 5 days [5] Ongoing Trials - The pivotal single-arm PiNACLE trial is currently underway, targeting approximately 120 patients with relapsed/refractory LBCL in the third- or later-line setting [6][12] - The trial aims to evaluate the overall response rate as the primary endpoint [12] Regulatory Designations - LYL314 has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA for the treatment of relapsed/refractory aggressive B-cell lymphoma [11] Manufacturing and Technology - LYL314 is designed to target B cells expressing either CD19 or CD20, utilizing a proprietary manufacturing process to enhance T-cell activity [10][9] - The Lyell LyFE Manufacturing Center™ has the capacity to produce over 1,200 CAR T-cell doses at full capacity [13]

Core Insights - FibroBiologics, Inc. is a clinical-stage biotechnology company focused on developing therapeutics for chronic diseases using fibroblast technology [1][4] - The company will present updates on its recent milestones and research at the BIO International Convention 2025, highlighting advancements in wound healing, multiple sclerosis, and psoriasis [1][2] Company Overview - FibroBiologics holds over 275 patents issued and pending, covering various clinical pathways including wound healing, multiple sclerosis, disc degeneration, psoriasis, orthopedics, human longevity, and cancer [4] - The company aims to transform the treatment landscape for chronic diseases by addressing underlying biological dysfunction rather than merely managing symptoms [2][4] Upcoming Events - The presentation at the BIO International Convention is scheduled for June 17, 2025, at 10:45 a.m. ET, at the Boston Convention & Exhibition Center [2] - The company is set to begin a Phase 1/2 clinical trial for diabetic foot ulcers later this year, marking a significant step in its research efforts [2]

Summary of Arcellx (ACLX) Investor Relations Event - June 13, 2025 Company Overview - **Company**: Arcellx (ACLX) - **Event**: Investor Relations event held in Milan - **Focus**: Development of a novel cell therapy, Anitosel, targeting multiple myeloma Key Industry Insights - **Market Size**: - The second line plus market for multiple myeloma is estimated at **$12 billion** - The frontline market is projected to reach **$20 billion** - The fourth line plus population is approximately **$3.5 billion** [7][8][9] - **Market Research**: - Conducted with over **300 hematologists and oncologists** to assess market share and therapy adoption [11] - Anitosel is expected to capture a **58% market share** in 2024, increasing to **83%** in 2025 [12][13] Product Development and Clinical Trials - **Anitosel**: - A novel synthetic binder developed to address limitations of biologic-based binders, focusing on safety, scale, and manufacturability [5][6] - Strong clinical data in relapsed and refractory myeloma populations [6] - **IMMAGINE-one Study**: - Phase 2 study with **129 patients** enrolled, showing a **97% overall response rate** and **68% complete response rate** [31][33] - Median progression-free survival (PFS) of **30.2 months** and no delayed neurotoxicities reported [32][39] Launch Strategy - **Launch Timeline**: Anticipated launch of Anitosel in the U.S. in **mid to late 2026** [5] - **Market Access**: - Plans to access **80% of covered lives** within **30 days** of launch and **90% within 90 days** [21][22] - **Manufacturing Capacity**: - Expected to cover the majority of the fourth line plus population at launch, with a global potential of over **24,000 doses** [17][19] Operational Excellence - **Turnaround Time**: Expected turnaround time for Anitosel is less than **17 days**, with a high in-spec rate of **96%** [18][19] - **Partnership with Kite**: - Collaboration with Kite to leverage their expertise in cell therapy, enhancing operational efficiency and reducing costs [24][21] Financial Outlook - **Profitability**: Expected gross margins of **≥70%** at launch, with profitability anticipated at less than **$1 billion** in Anitosel sales [23][24] - **Capital Management**: As of Q1, the company has **$565 million** remaining on its balance sheet, reflecting operational efficiency [23] Safety and Efficacy - **Safety Profile**: - Anitosel demonstrated a predictable and manageable safety profile with no delayed neurotoxicities observed [39][40] - **85%** of patients experienced grade one or less cytokine release syndrome (CRS) [36] Conclusion - **Market Positioning**: Anitosel is positioned to be a leading CAR T therapy in the myeloma space, with a strong focus on patient safety, operational excellence, and market access strategies [28][29]

Phase 1b study suggests a promising safety profile and highlights the potential of a novel dual-targeting CD19/CD20 CAR T in patients with relapsed or refractory disease75-80% complete response rate among evaluable patients at the recommended Phase 2 dose  MILAN, June 13, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) announced today the first clinical data from an ongoing Phase 1b study for JNJ-90014496 (JNJ-4496), an investigational dual-targeting anti-CD19/CD20 bispecific autologous chimeric antigen ...