▎药明康德内容团队编辑Denali Therapeutics公司今日宣布，美国FDA已授予在研疗法tividenofusp alfa（DNL310）突破性疗法认定，用于治疗亨特综合症（MPS ...

虽然tividenofusp alfa尚未获得任何卫生当局的批准，但其安全性和有效性特征仍在进行中的研究中继续评估。Denali Therapeutics是一家专注于开发神经退行性疾病和溶酶体贮积症治疗方法的生物制药公司，特别强调能够穿过血脑屏障的疗法。

Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, ...

Regenxbio's rolling BLA submission completion of RGX-121 for the treatment of patients with MPS II expected in Q1 of 2025.

REGENXBIO's partnership with Nippon Shinyaku provides $110M upfront, ensuring a cash runway for gene therapy programs in MPS ...

Regenxbio will receive $110m upfront from Nippon Shinyaku and is eligible to get up to $700m in development, regulatory, and ...

Rockville, Maryland Wednesday, January 15, 2025, 12:00 Hrs [IST] ...

Denali Therapeutics Inc. (NASDAQ: DNLI), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tividenofusp alfa (DNL310) for the treatment ...

REGENXBIO Inc. (Nasdaq: RGNX) and Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) today announced a strategic partnership for the development and commercialization of RGX-121 for the treatment of ...

Shares of US biotech Regenxbio surged 18.4% in pre-open trading Tuesday after announced a strategic partnership with Japan’s ...