PEC Encap

1922年，班廷等人发现并提纯了胰岛素，将其成功应用于糖尿病的治疗，这一发现拯救了千千万万的糖尿病患者，尤其是1型糖尿病（T1D）患者。然而，对于1型糖尿病患者来说，只能通过胰岛素治疗来维持，尚无其他任何办法解决导致疾病的根本问题——胰岛细胞无法自己产生胰岛素。 近年来，随着干细胞技术的成熟和应用，基于人多能干细胞诱导分化而来的胰岛细胞的胰岛细胞替代疗法开始进行人体临床试验，有望实现1型糖尿病的功能性治愈，让患者不再依赖注射胰岛素。 2022年7月11日，Vertex Pharmaceuticals 宣布将以3.2亿美元现金收购 ViaCyte 公司，收购后，Vertex 将拥有 ViaCyte 的三款治疗糖尿病的细胞疗法在研管线，其在人了干细胞系、干细胞分化领域的知识产权、细胞疗法 GMP 设备，以及其与 CRISPR Therapeutics 合作获得的新型低免疫干细胞资产，进一步补充自身在糖尿病功能性治愈领域的领先地位。 ViaCyte 与 CRISPR Therapeutics 合作开发一款基于基因编辑的干细胞疗法（VCTX210）来治疗1型糖尿病。今年2月份，他们在1期临床试验中完成了首例患者给药，该1期临床试验预计在今年12月完成。VCTX210 使用 CRISPR-Cas9 基因编辑技术敲除了 B2M、PD-L1、HLA-E 基因，从而避免细胞转移后的免疫排斥。外界认为，VCTX210 疗法是 Vertex 公司决定收购 ViaCyte 的关键驱动力。 ViaCyte 公司是一家干细胞疗法公司，通过重编程技术，将人类干细胞诱导分化为产生胰岛素的细胞，封装到微型装置中，然后一直到糖尿病患者胰腺内，并在胰腺内再生胰岛细胞、产生胰岛素。 ViaCyte 公司发明了一种可重复的专利技术方法，能够将期专有的人多能干细胞（CyT49）分化为胰腺内胚层细胞（也称为胰腺前体细胞、PEC-01细胞）。整个过程模拟人类胰腺的自然发育，在每个步骤中，通过添加特定类型和数量的生长因子、生长培养基和补充剂，引导多能干细胞分化为PEC-01细胞，整个细胞制造过程约为30天。 2021年11月，ViaCyte 公司在 Cell Stem Cell 和 Cell Reports Medicine 期刊上发表 了正在进行中的人体1/2期临床试验（VC02-101， NCT03163511）数据，数据表明，VC-02 疗法可以在患有严重1型糖尿病的患者中产生胰岛素。在该研究中，植入 ViaCyte 的 PEC-Direct 设备的17名患者中，部分患者在植入后六个月就检测到了阳性 C 肽（胰岛素生物标志物）。数据还表明，干细胞来源的胰腺内胚层细胞可以分化为产生胰岛素的胰岛细胞。 早在2019年，Vertex 公司就斥资9.5亿美元收购了 Semma Therapeutics，从而获得了其开发的基于干细胞的胰岛细胞替代疗法——VX-880。 2021年10月18日，Vertex 公司宣布，一名严重的1型糖尿病的患者在接受 VX-880 的干细胞来源的完全分化的胰岛细胞替代疗法后，实现了胰岛细胞功能的“稳健恢复”，这些恢复的胰岛细胞可以让患者调节胰岛素甚至自己产生胰岛素，每天的胰岛素使用量减少91%，血糖控制得到显著改善。 该疗法的安全性与典型的免疫抑制剂治疗相似，且没有报告与治疗相关的严重副作用。VX-880 也是目前处于临床阶段的众多糖尿病细胞疗法中进展最为迅速的。 此外，Vertex 公司还在开发其封装胰岛细胞的疗法，这一疗法将无需对患者进行免疫抑制，并计划在今年向FDA提交临床试验申请。 参考资料：https://viacyte.com/pipeline/https://www.genengnews.com/metabolic-disorders/diabetes/vertex-to-acquire-viacyte-for-320m-growing-diabetes-cell-therapy-pipeline/ 内容来源于网络，如有侵权，请联系删除。

Vertex Pharmaceuticals is developing a cell therapy for type 1 diabetes that replaces the insulin-producing cells lost to the chronic condition. In paying $320 million to acquire ViaCyte, Vertex is getting its hands on the company that was the first to demonstrate this feat in clinical testing. Boston-based Vertex framed the acquisition agreement announced Monday as complementary to its own research efforts. Privately held ViaCyte brings additional tools, technologies and assets, Vertex CEO Reshma Kewalramani said in a prepared statement. In type 1 diabetes, the immune system destroys the islet cells in the pancreas that produce insulin. Patients require regular doses of insulin, either by injection or infused from an insulin pump, to maintain levels of the glucose-regulating hormone. Vertex’s type 1 diabetes cell therapy candidate, VX-880, is comprised of islet cells that are grown in a lab. These cells are infused into a patient along with immunosuppressive therapies to keep the immune system from rejecting them. During the annual meeting of the American Diabetes Association last month, Vertex presented data from two patients who were given half of the targeted dose of cells. Both patients showed changes in blood glucose. One patient achieved insulin independence 270 days after dosing while the other showed a 30% reduction in infused or injected insulin at day 150. Vertex added VX-880 to its pipeline in 2019, paying $950 million up front to acquire startup Semma Therapeutics. At the time of the deal, Semma was preclinical. The Phase 1/2 study underway has a targeted enrollment of 17 patients. It was cleared to resume last week with the resolution of a clinical hold placed on the study in May; the FDA said at the time that there was insufficient information to support dose escalation. San Diego-based ViaCyte has three programs in development for type 1 diabetes. The most advanced is VC-02, stem cell-derived islet cells that are housed in an implantable pouch. This pouch connects with a patient’s blood vessels, enabling them to interact with the transplanted cells. In June 2021, ViaCyte reported clinical data at nine months indicating that transplanted cells were producing insulin in the first patient treated with this approach. Like the Vertex therapy, VC-02 requires immunosuppressive drugs to prevent rejection. A second ViaCyte program, VC-01, could reduce this risk with a pouch designed to prevent the patient’s immune cells from directly coming in contact with the transplanted islet cells. ViaCyte presented additional data for both programs at the American Diabetes Association’s meeting in June. The results are important for showing the potential of cell therapy in type 1 diabetes, according to William Blair analyst Raju Prasad. “Together, the data demonstrated—for the first time in humans—that implanted pancreatic progenitor cells can functionally differentiate into mature, glucose-responsive, (endogenous) insulin-producing islet cells,” Prasad wrote in a note sent to investors Monday morning. Vertex’s main interest in ViaCyte might be its third program, which could eliminate the need for immunosuppressive therapy. The cells in this experimental therapy, VCTX210, are edited with CRISPR technology to evade detection by the immune system. ViaCyte is developing VCTX210 under a partnership with CRISPR Therapeutics. The Switzerland-based gene-editing company is already a partner to Vertex. The two firms are collaborating on gene-editing treatments for two blood disorders, sickle for sickle cell disease and beta thalassemia. The collaboration has also expanded to two muscular disorders, Duchenne muscular dystrophy and myotonic dystrophy type 1. Prasad sees synergies between the type 1 diabetes approaches of Vertex and ViaCyte due to the similarities in the technology, development and manufacturing of the two cell therapies. Those similarities will benefit both CRISPR Therapeutics and Vertex in the development of a CRISPR-edited therapy with a reduced risk of prompting an immune response, he said. However, the ViaCyte acquisition will still need to pass antitrust muster. The Federal Trade Commission has raised concerns about the anti-competitive effects of some pharmaceutical mergers, discussing the topic most recently during a virtual workshop last month. There are other companies developing innovative new treatments for type 1 diabetes. Beta Bionics has also reached clinical testing with a medical device designed to take on the role of a patient’s pancreas. A drug candidate from Provention Bio is currently under FDA review. The technology that might be closest to ViaCyte’s is a cell pouch in development under Canada-based Sernova. This pouch, designed to house therapeutic cells that could offer an alternative to daily dosing with drugs, is in early clinical development in type 1 diabetes.