凱特療法

2023年7月11日

生物技術的病毒載體平台的許可協議遵循最近阿斯特拉協議Taysha基因療法和凱特療法。Morten Sogaard 7月10日,2023年出版,阿斯特拉製藥公司的基因治療單位頒發的許可詹妮弗Arcure生物科技公司4 d分子療法引起另一個製藥合作夥伴,周一宣布與日本阿斯特拉製藥許可協議。這筆交易給阿斯特拉使用權由4 d技術在發展中治療至少一個罕見的單基因眼疾。阿斯特拉已經選擇,額外的費用,兩個基因的目標添加到協作。近10年前開始,4 d專業研究人員和製藥公司使用基因工程病毒療法進入身體。雖然許多類型的這些病毒可以在自然界中發現,4 d和其他公司一直在努力發明專門構建版本可以,例如,更好的進入特定的細胞。一個由4 d,被稱為R100中,阿斯特拉的注意力和焦點周一的交易。由一個腺相關病毒,它的設計更有效的基因植入視網膜。阿斯特拉將支付4 d 2000萬美元的前期使用R100中矢量,它將對自己的遺傳負荷。製藥公司將進行後續研究和開發,以及負責生產或任何商業化。 4DMT uses R100 in three of its clinical-stage therapies and, after initial data readouts , received interest from a number of pharma companies, including Astellas. “After going through different discussions we felt that the best place for us to start a relationship is in ophthalmology with our R100 intravitreal vector and to branch out from there,” said David Kirn, co-founder and CEO of 4D. For Astellas, the deal is its latest investment in gene therapy, following the acquisition of Audentes Therapeutics and, more recently, deals with Taysha Gene Therapies and Kate Therapeutics . The company has run into setbacks, however. Four trial volunteers died in a study of an Audentes gene therapy for X-linked myotubular myopathy, and testing remains on hold. Another trial of a separate therapy was also put on pause, but was cleared to resume earlier this year. The volunteers’ deaths were linked to signs of liver damage — an area of concern more broadly for gene therapies, many of which end up passing through the liver in some fashion. “I think there is, of course, exciting progress on gene therapy, but the challenge is really how are you going to deliver outside the liver,” said Morten Sogaard, president and division head of research and technical operations at Astellas’ gene therapy unit. Specialized vectors like those 4D has created could show a way forward. The R100 vector at the center of Monday’s deal is specific to the eye, which is immune protected and doesn’t involve the same liver risks as targeting other organs. R100 is delivered via intravitreal injection, a less invasive procedure than the subretinal injections used for other eye gene therapies, like the approved blindness treatment Luxturna. Subretinal injections require surgery under anesthesia and can risk tears or other damage to the retina. By contrast, intravitreal injections are a much shorter, outpatient procedure. The R100 vector allows this kind of delivery as it’s designed to cross the eye’s internal limiting membrane barrier. “At the end of the day, we're here to do good for patients, and some wonderful new gene therapies that will help a lot of patients is what we’re hoping to get out of it,” said Sogaard. Per deal terms, 4D is eligible to receive milestone and option fees of up to $942.5 million, as well as royalties on any approved products that result from the collaboration. The biotech said it’s continuing to develop its gene therapy tools and is open to other potential partnerships in the future. Recommended Reading