Idea or Concept
uniQure Announces Positive Interim Data Update Demonstrating Slowing of Disease Progression in Phase I/II Trials of AMT-130 for Huntington’s Disease
uniQure, Huntington’s disease, AMT-130, gene therapy, Phase I/II trials, disease progression, neurofilament light protein (NfL), Unified Huntington’s Disease Rating Scale (cUHDRS), Regenerative Medicine Advanced Therapy (RMAT) designation
US Speaker Promises Vote on BIOSECURE Act to Halt Federal Contracts with Chinese Biotech Companies
BIOSECURE Act, House Speaker Mike Johnson, Chinese biotech companies, federal contracts, healthcare data, national security
UniQure Wins First FDA RMAT in Huntington’s Disease After Strong Phase I/II Data
UniQure, Huntington’s disease, gene therapy, AMT-130, FDA, RMAT designation, Phase I/II clinical trials, neurodegenerative disorder, neurofilament light (NfL), disease progression, composite Unified Huntington’s Disease Rating Scale (cUHDRS).
Annovis Bio Reports Promising Phase III Study Results for Buntanetap in Parkinson’s Disease Treatment
Annovis Bio, Buntanetap, Parkinson’s Disease, Phase III Study, Neurodegenerative Disorders, Cognitive Function, Motor Functions, Unified Parkinson’s Disease Rating Scale (MDS-UPDRS)
FDA Rejects Rocket Pharmaceuticals’ Gene Therapy for Ultra-Rare Immune Disease, Requests Additional Manufacturing Information
FDA, Rocket Pharmaceuticals, Gene Therapy, Leukocyte Adhesion Deficiency-I (LAD-I), Manufacturing Information, Complete Response Letter
Lykos Faces Setback as ICER Concludes Insufficient Evidence for MDMA-Based PTSD Treatment
Lykos Therapeutics, MDMA, PTSD, ICER, Insufficient Evidence, Clinical Trials, FDA Approval
US Supreme Court Rejects Purdue’s Multibillion-Dollar Opioid Pact
US Supreme Court, Purdue Pharma, Opioid Settlement, Sackler Family, Bankruptcy, Opioid Crisis
CDC Expands RSV Vaccine Recommendations for Adults 75 and Older
RSV vaccine, CDC, adults 75 and older, vaccination recommendations, public health
Vertex Announces Positive Results for VX-880 in Type 1 Diabetes Treatment
Vertex Pharmaceuticals, VX-880, Type 1 Diabetes, Stem Cell Therapy, Insulin Independence, Severe Hypoglycemic Events
Emergent Biosolutions Sells Baltimore Facility for $30 Million
Emergent Biosolutions, Baltimore facility, sale, Bora, $30 million