–The Study Met Primary and All Secondary Efficacy Endpoints– –TPIP Was Well Tolerated in the Study, with 75% of Patients Titrating to the Highest Dose– –Insmed to Immediately Engage with FDA to Inform Phase 3 Trial Design with Studies Expected to Begin Before End of 2025 for PH-ILD and in Early 2026 for PAH– –Insmed to Host Investor Call at 8:00 AM ET on Tuesday, June 10, 2025– BRIDGEWATER, N.J., June 10, 2025 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical compan ...

Company Overview - Gyre Therapeutics is a biopharmaceutical company focused on advancing fibrosis-first therapies across various organ systems affected by chronic diseases [1][6] - The company is headquartered in San Diego, CA, and is primarily engaged in the development and commercialization of Hydronidone for liver fibrosis, including metabolic dysfunction-associated steatohepatitis (MASH) [6] Clinical Development - Gyre has successfully dosed the first volunteer in a Phase 1 clinical trial for F230, a novel endothelin A receptor antagonist aimed at treating pulmonary arterial hypertension (PAH) [1][4] - The Phase 1 trial is designed to evaluate the safety, tolerability, and pharmacokinetics of F230 in healthy volunteers [4] - F230 is a fully synthetic small molecule that selectively blocks the ETA receptor, intended to reduce pulmonary vascular remodeling and lower pulmonary pressure, which are critical factors in PAH progression [3] Market Potential - Gyre's entry into the PAH field is significant due to the condition's rarity, progressive nature, and high mortality rate, with limited treatment options available [2] - The PAH market in China was valued at $370 million in 2023 and is projected to grow to $480 million by 2031, highlighting the commercial potential for Gyre's F230 [2] Pipeline and Future Plans - F230 joins Gyre's pipeline alongside its lead candidate Hydronidone (F351), which has met the primary endpoint in a pivotal Phase 3 trial for chronic hepatitis B (CHB)-induced liver fibrosis [5] - A New Drug Application (NDA) submission for F351 to China's National Medical Products Administration (NMPA) is planned for the third quarter of 2025 [5] - Gyre is also planning a pre-IND meeting with the U.S. Food and Drug Administration for an expected Phase 2 trial in MASH fibrosis [5]

Mark J. Bachleda, PharmD, MBA appointed as independent member of the Board of DirectorsDavid Shook, MD appointed as Chief Medical Officer, Mark Meltz, JD as General Counsel and Corporate Secretary, and Jarrad Aguirre, MD, MBA as Senior Vice-President of Medical Affairs SOUTH SAN FRANCISCO, Calif., June 09, 2025 (GLOBE NEWSWIRE) -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical-stage company advancing a pipeline of next-generation CAR T-cell therapies for patients with cancer, today announced the appoi ...

Summary of Verve Therapeutics (VERV) FY Conference Call - June 09, 2025 Company Overview - **Company**: Verve Therapeutics - **Focus**: Gene editing for cardiovascular disease, specifically targeting lifelong cholesterol lowering to treat coronary heart disease Key Points and Arguments 1. **HEART-two Data Announcement**: The company recently disclosed data from the HEART-two trial, which showed promising results for their gene editing therapy aimed at lowering LDL cholesterol levels [3][4] 2. **Unmet Need**: Current cholesterol-lowering therapies have a high dropout rate, with up to 50% of patients discontinuing treatment within a year. Verve aims to provide a one-time therapy that offers lifelong efficacy [5] 3. **Efficacy Results**: In the HEART-two trial, the therapy demonstrated an LDL reduction of up to 60%, with a mean reduction of 59% in the highest dose group. All patients in this group experienced over 50% lowering [8][21] 4. **Safety Profile**: The therapy was well tolerated, with only one infusion-related reaction reported among 14 patients. No significant laboratory abnormalities were observed [8][9] 5. **Durability of Effect**: The company reported that the LDL reduction was sustained at 60% two years post-infusion, supporting the vision of a one-time therapy for atherosclerotic cardiovascular disease [10][11] 6. **Comparison of Products**: The difference between the first-generation product (VRRB101) and the second-generation product (VRRB102) lies in the delivery system, which has been improved to enhance safety and efficacy [12][14] 7. **Regulatory Environment**: Recent discussions with the FDA indicate a supportive stance towards gene editing technologies, particularly those addressing chronic diseases like atherosclerosis [45][48] 8. **Market Research Findings**: Surveys indicate a significant openness among patients and physicians towards a one-time gene editing therapy, with about one-third of patients preferring this option over traditional therapies [50][53] 9. **Cost of Manufacturing**: The estimated cost to manufacture the therapy is around $3 per dose, suggesting potential for flexible pricing strategies compared to traditional gene therapies [55][56] 10. **Partnership with Eli Lilly**: The company is preparing to present data to Eli Lilly for potential opt-in for further development, which could extend Verve's cash runway [62][69] Additional Important Insights - **Dosing Strategy**: The company is shifting towards fixed dosing rather than weight-based dosing, as the therapy primarily targets the liver [24][26] - **Long-term Safety Considerations**: The company believes that the low risk of off-target effects, combined with the significant risk of untreated cardiovascular disease, will favor the adoption of their therapy [40][41] - **Future Developments**: Verve is also working on additional products targeting ANGPTL3 and Lp(a), leveraging the same delivery system as VRRB102 [64][66] This summary encapsulates the critical insights from the conference call, highlighting the company's innovative approach to gene editing in cardiovascular disease and its potential impact on patient care and market dynamics.

Spyre Therapeutics Inc (SYRE) FY Conference June 09, 2025 09:20 AM ET Speaker0 Thank you for joining us. I'm Paul Choi, and I cover biotechnology here at the firm. It's our pleasure to welcome Spire, and we have Cameron here on on stage with us. Maybe to to kick it off, Cameron, can just give us a brief overview of Spire and just sort of the key programs and technologies under pending pending the company? Yeah. Speaker1 Of course. And so and thanks for having us. It's a great event as always. So the backgro ...

– New blinded safety and efficacy data from 30 patients in ongoing Phase 2 trial to be unveiled June 13, 2025 – – ISSCR 2025 is the premier global event for stem cell research, drawing nearly 4,000 leaders from academia, biotech, and pharma – – BioRestorative to release new clinical data to the public prior to market open on June 13th – MELVILLE, N.Y., June 09, 2025 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (“BioRestorative,” “BRTX” or the “Company”) (NASDAQ: BRTX), a clinical-stage regenerative me ...

2025 年 6 月 5 日，加州大学欧文分校的研究人员在 Cell 子刊 Cell Stem Cell 上发表了题为： Harnessing human iPSC-microglia for CNS-wide delivery of disease-modifying proteins 的研究论文。 该研究利用 CRISPR 基因编辑修饰的 人类诱导多能干细胞来源的小胶质细胞 （iMG） ，实现了 中枢神经系统 （SNS） 范围内的疾病修正蛋白递送，并验证了 撰文丨王聪 编辑丨王多鱼 排版丨水成文 血脑屏障 （Blood-Brain Barrier，BBB）， 是指脑毛细血管壁与神经胶质细胞形成的血浆与脑细胞之间的屏障，和由脉络丛形成的血浆和脑脊液之间的屏障，仅 允许特定类型的分子从血流进入大脑神经元和其他周围细胞。 血脑屏障的存在，对于阻止有害物质由血液进入大脑具有重要意义，但这也同时阻止了绝大部分小分子和大分子药物 （例如多肽，蛋白质和核酸） 的转移，严重 限制了中枢神经系统疾病 （例如神经退行性疾病、脑肿瘤，脑部感染和中风等） 的治疗。 尽管该领域近年来取得了一些进展，但我们仍然迫切需要能够跨越血脑屏 ...

PRINCETON, N.J., June 06, 2025 (GLOBE NEWSWIRE) -- via IBN – Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, today spotlights the efforts of Ellen Kim, M.D., Lead Principal Investigator for the Company’s Phase 3 FLASH (1 and 2) studies in early stage cutaneous T-cell lymphoma (CTCL), in advancing HyBryte™ (synthetic hypericin) as a potential new th ...

EDAP TMS (EDAP) 2025 Conference June 05, 2025 04:20 PM ET Speaker0 Okay. Alright. Good afternoon. My name is Michael Sarcone. I'm an analyst on the US Medical Supplies and Devices team, this is day two of the Jefferies twenty twenty five New York Healthcare Conference. This is a session with EDAP Focal One. And with us from the company, we've got Ryan Rhodes, CEO Ken Moback, CFO and John Francis who heads the IR function. Ryan Rhodes is going to give a presentation, some prepared commentary and then we'll o ...

I-Mab (IMAB) 2025 Conference June 05, 2025 03:45 PM ET Speaker0 Great. I think we can get started. Good afternoon, everyone. Thank you for attending Jefferies Healthcare Conference. My name is Yifan Shi from Jefferies Biotech Research Team. Today, we are very pleased to have Doctor. Sheng Fu, CEO of IMAP, and Doctor. Philip Dennis, CMO of IMAP, in for this session. Welcome. Speaker1 Thank you. Speaker0 So, to get start, so for those who may be less familiar with your story, could you please provide a high l ...