6月6日13时32分，北京大学人民医院黄晓军院士团队开出了国内首款干细胞药物的首张处方，一名急性 移植物抗宿主病（aGVHD）患者在当天完成药物注射。 这款在国内获批上市的首款干细胞药物——艾米迈托赛注射液（商品名：睿铂生）每剂定价1.98万元 （含6000万单位细胞）。 据了解，该注射液8次为一个完整推荐疗程计算，整个疗程的费用大约是15.84万元，对比美国同类药 物"Ryoncil"，该价格仅为1/70。 更值得关注的是，其背后的医药企业铂生生物，已经就艾米迈托赛注射液与阿里健康展开合作。 干细胞药物，已经迈入商业化了吗？ 干细胞治疗第一单来了 所谓干细胞，指的是一类具有自我更新和分化潜能的细胞，它们能够产生特定细胞，为身体提供新的细 胞，以维持生命或替换受损细胞。在大部分胚胎干细胞转化为普通细胞后，还有少量细胞保持了增殖和 形成其他类型细胞的能力，这就是成体干细胞。它们存在于骨髓、脐带、脂肪、牙髓等组织里，人体每 天有大量细胞死亡，就由它们来补充。 因此，临床上基于干细胞的再生潜能，将其移植到人体内，发挥修复病变细胞或重建其功能的作用，从 而达到治疗疾病的目的。 近年来，干细胞的临床治疗取得了多项突破性 ...

NEW YORK, June 11, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an update on continued momentum with United States Food and Drug Administration (FDA) regarding both accelerated approval pathway for Revascor® (rexlemestrocel-L) in the treatment of patients with ischemic chronic heart failure with reduced ejection fraction (HFrEF) and inflammation, and label extension for Ryoncil® (remestemcel-L-rknd) in adu ...

以下文章来源于RimeData 来觅数据 ，作者来觅研究院 RimeData 来觅数据 . 全面的一级市场数据平台 导读：4月1日，国家卫生健康委员会发布《国家重点研发计划2025年度项目申报指南》，重磅推出五大重点专项。其中"干细胞研究与器官 修复"专项引发社会广泛关注，标志着我国正式将干细胞治疗纳入国家战略层面。近年来，干细胞疗法在治疗多种疾病方面展现出巨大潜 力，从神经系统疾病到心血管疾病，再到糖尿病等领域，都取得了令人瞩目的进展。全球范围内干细胞疗法的临床试验数量持续增加。干 细胞疗法作为创新疗法热门领域之一，近年来持续获得海内外产业及资本的关注。 01 干细胞疗法 干细胞疗法是一种利用干细胞特性来治疗疾病或修复受损组织的医疗技术，它利用干细胞的自我更新和多向分化潜能，通过将干细胞移植或诱导 分化为特定细胞类型，来修复受损组织、替代细胞或调节机体功能，从而治疗多种疾病。截至2024年12月，全球范围内已经批准或完成了116项 干细胞疗法临床试验，覆盖了帕金森病、糖尿病、癫痫、心脏病以及癌症等多种重大疾病。 干细胞有很多不同的分类方法，按发育阶段划分，可分为胚胎干细胞（ESCs）、成体干细胞（ASCs） ...

Core Insights - Mesoblast has received seven years of orphan-drug exclusive approval from the FDA for Ryoncil (remestemcel-L) to treat steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older [1][5] - The FDA's approval grants Mesoblast statutory exclusivity, preventing the approval of other mesenchymal stromal or stem cell products for this indication during the exclusivity period [2] - Mesoblast holds biologic exclusivity that prevents other sponsors from referencing the Ryoncil biologic license application until December 2036, providing a barrier against biosimilar market entry [2] - The company has a strong U.S. intellectual property position on MSC composition, manufacturing, and indications, providing commercial protection against competitors through 2044 [3][7] Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing proprietary mesenchymal lineage cell therapy technology [4] - Ryoncil is the first FDA-approved MSC therapy for SR-aGvHD in pediatric patients [5] - The company is also developing additional cell therapies for other inflammatory diseases and has established commercial partnerships in Japan, Europe, and China [6] Intellectual Property and Manufacturing - Mesoblast has over 1,000 granted patents or patent applications covering various aspects of MSC technology, expected to provide commercial protection through at least 2044 [7] - The company's proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [8]

Core Insights - Mesoblast Limited has successfully launched Ryoncil (remestemcel-L) for treating children with acute graft versus host disease (GVHD) shortly after receiving FDA approval, marking it as the first approved mesenchymal stromal cell therapy in the US [2][11] - The company is well-positioned financially, with a cash reserve of US$182 million (A$290 million), allowing for the expansion of Ryoncil's indications to other serious pediatric inflammatory diseases and adult acute GVHD [5][12] - Mesoblast is also preparing for a Type B meeting with the FDA to discuss the accelerated approval pathway for Revascor (rexlemestrocel-L) in treating ischemic chronic heart failure with reduced ejection fraction [5][12] Financial Highlights - The net operating cash spend for the quarter was US$12.7 million [5] - Cash on hand at the end of the quarter was US$182 million (A$290 million) [5] - Mesoblast raised A$260 million (US$161 million) through a global private placement primarily to existing major shareholders [12] Operational Highlights - Ryoncil became commercially available in the US on March 28, 2025, with Federal Medicaid coverage, and 15 infusion kits have been purchased for patient treatment [5] - The company has onboarded ten priority transplant centers, with plans to onboard an additional ten in the current quarter [5] - Mesoblast has expanded Ryoncil coverage to over 104 million US lives insured by commercial and government payers [5] Product Development - Ryoncil is being developed for additional indications, including adult SR-aGvHD and biologic-resistant inflammatory bowel disease [13] - Revascor is under development for chronic heart failure and has received RMAT designation from the FDA [12] - The confirmatory Phase 3 trial for rexlemestrocel-L in chronic low back pain is actively enrolling patients, with a focus on opioid cessation [12] Corporate Developments - Mesoblast has strengthened its Board of Directors with the appointment of Dr. Gregory George and Ms. Lyn Cobley [12] - The company was added to the S&P/ASX 200 Index effective March 6, 2025 [12] Intellectual Property - Mesoblast holds a strong global intellectual property portfolio with over 1,000 granted patents or applications covering its mesenchymal stromal cell technology [14] Manufacturing Capabilities - The company utilizes proprietary manufacturing processes to produce industrial-scale, cryopreserved, off-the-shelf cellular medicines [15] Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing its proprietary mesenchymal lineage cell therapy technology [10]

Company Overview - Mesoblast is a global leader in developing allogeneic cellular medicines for severe inflammatory diseases, utilizing proprietary mesenchymal lineage cell therapy technology [4][6] - The company has received FDA approval for RYONCIL (remestemcel-L), the first mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease in pediatric patients [5][6] Recent Developments - Mesoblast appointed Lyn Cobley to its Board of Directors, who brings over 30 years of experience in the financial services industry, including senior roles at major banks [1][3] - Cobley expressed enthusiasm about Mesoblast's recent FDA approval and the company's potential for future growth, including commercialization and new indications for prevalent diseases [3] Strategic Initiatives - The company is focused on developing additional cell therapies for various indications, including adult SR-aGvHD and biologic-resistant inflammatory bowel disease, as well as rexlemestrocel-L for heart failure and chronic low back pain [6] - Mesoblast has established commercial partnerships in key markets such as Japan, Europe, and China [6] Intellectual Property and Manufacturing - Mesoblast holds a strong global intellectual property portfolio with over 1,000 granted patents or applications, providing commercial protection expected to last until at least 2041 in major markets [7] - The company utilizes proprietary manufacturing processes to produce industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability for patients worldwide [8]

Core Insights - Mesoblast has expanded coverage for its product Ryoncil® (remestemcel-L) to 104 million insured lives in the U.S. through government and commercial payers [1][2] - Ryoncil® is the first FDA-approved mesenchymal stromal cell therapy for steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older [4] Coverage Expansion - 37 out of 51 states provide fee-for-service Medicaid coverage for Ryoncil®, covering 20 million lives, which is 80% of total Medicaid fee-for-service lives [2] - Mandatory coverage for an additional 24 million lives will begin on July 1, 2025 [2] - Commercial plans cover 84 million lives, excluding medical exceptions policies, indicating that the actual commercial coverage is likely higher [2] Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing a proprietary mesenchymal lineage cell therapy technology platform [3] - The company is also developing additional therapies for other inflammatory diseases and conditions such as heart failure and chronic low back pain [5] Intellectual Property - Mesoblast holds a strong global intellectual property portfolio with over 1,000 granted patents or applications, providing commercial protection expected to last until at least 2041 in major markets [6] Manufacturing Capabilities - The company has proprietary manufacturing processes that yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [7]