Ac-SSO110 has now received FDA orphan drug designation and investigational new drug application clearance for the treatment ...

The company has already kicked off an open-label Phase I trial of ABX1100 in Canada following positive results from a study of healthy volunteers.

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...

Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced that it has received U.S. Food and Drug Administration (FDA) ...

The Washington University trial comes as part of a larger platform study which hopes to understand if Alzheimer's disease can ...

Astellas Pharma Inc. (TSE: 4503) (President and CEO: Naoki Okamura, "Astellas") today announced the submission of a New Drug Application (NDA) to Japan's Ministry of Health, Labour and Welfare (MHLW) ...

Nader Yaghoubi, M.D., Ph.D., PathMaker’s Co-Founder and CEO, stated, “We are pleased to announce IRB approval for the CALM study, which allows us to commence enrollments shortly. Given the mechanisms ...

By creating gene editors not found in nature, or optimizing existing editors, AI can improve the accuracy, effectiveness, and accessibility of gene editing.

This study will evaluate HCW9302 in patients with moderate to severe alopecia areataMIRAMAR, Fla., Feb. 03, 2025 (GLOBE ...

United Therapeutics Corporation , a public benefit corporation, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug application (IND) to initiate ...