2022年10月24日

维塔疗法提高3100万美元,增加了新的肌肉萎缩症资产维塔疗法提高了B系列融资以帮助其移动导致细胞疗法资产,vta - 100,到诊所作为一个潜在的治疗2肢带肌萎缩症(LGMD2A),疾病的亚型。LGMD2A是一种局部的疾病,通常影响的肩膀,上臂,大腿和骨盆。公司使用诱导多能干细胞(iPSC)技术工程师设计特定的细胞类型替换那些有缺陷的患者。首席执行官道格拉斯·福尔克告诉BioBuzz vta - 100预计在大约18个月内进入第一次在人类研究。雷竞技 raybetvta - 100是一种自体基因治疗相结合的校正和iPSC技术来帮助修复和替换为LGMD2A患者肌肉细胞。该公司目前正在开展研究,以支持其计划试验性新药应用程序中,福尔克说。“现在,我们最大的目标是让这个诊所我们可以带来新的帮助病人,”福克说。超出vta - 100 B系列使维塔疗法获得新的资产facioscapulohumeral患者的治疗肌肉萎缩症(FSHD)。vta - 120被设计成一个自体基因治疗相结合的校正和iPSC技术来帮助修复和替换FSHD肌肉细胞治疗,就像LGMD2A是一种局部的疾病。第二个程序使用相同的技术,vta - 100,福尔克说。 Falk noted that FSHD has a similar phenotype perspective to LGMD2A. Typically, FSHD first presents with facial muscle weakness and in the scapular region. Additionally, there is proximal weakness of the pectoral and abductor muscles, which limits upper extremity function at the shoulder. Both VTA-100 and VTA-200 are designed to not only correct the genetic defect for muscular dystrophy but to also regenerate muscle in patients who have seen their muscles waste away due to the disease. The company also has a third developmental program, VTA-300. This asset is an investigational immunotherapy designed to combine proprietary chimeric antigen receptor technology and gene editing. Vita intends to develop this asset for an undisclosed indication. Since the company’s Series A in 2021, Falk said Vita Therapeutics has made significant progress. It has scaled from four employees to about 30 in the last 14 months and Falk said there are plans to bring on an additional 10 or so as it gets closer to the time VTA-100 will enter Phase 1. Beyond the expansion of its headcount, Vita has also grown physically. In May, the company secured about 11,000 square feet of office space and lab space in the University of Maryland BioPark. The new space allowed the company to significantly focus on the development of its pipeline, Falk said. It also placed the Vita team in close proximity to the University of Maryland research facilities. However, as the company continues to scale, Falk said Vita Therapeutics will begin to look for additional space. He said efforts are underway to find another facility to operate. While no lease has been signed, Falk said the company is committed to remaining in Maryland. With the combined Series A and B raises and additional funding, Vita Therapeutics has raised a total of $66 million to support its programs. The Series B was led by Cambrian BioPharma and new investor Solve FSHD. New investors included Riptide Ventures and Cedars Sinai, which participated alongside TEDCO and other existing investors. Falk said the capital organizations and companies that have continued to show support for Vita validates Vita’s cell therapy platform that targets the root cause of disease. “People have seen the progress we’ve made as well as some of the data we’ve generated internally and they continued to be impressed with what we’re doing,” Falk said. With the new financing in hand, Falk said the company will knuckle in and focus on the immediate tasks at hand so they can potentially bring new treatment options to muscular dystrophy patients.