GEN5 天
Rare Genetic Disorder Helps Explain Link Between Cancer Immunotherapy and Increased Infections
Studying patients with PD-1 or PD-L1 deficiency may help inform new approaches to fine-tuning immunotherapies to maximise ...
WebMD13 天
First Brain-Injected Gene Therapy Approved by FDA
The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called ...
Nature10 天
Deficiency of methyl-CpG binding protein-2 in CNS neurons results in a Rett-like phenotype ...
The CNS-specific phenotype of Mecp2 deficiency is unexpected considering that the gene is expressed ubiquitously and thought to act as a general repressor of transcription. It remains a major ...
Science Daily5 天
Gene Therapy News
Sep. 9, 2024 — Researchers tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs ...
GlobalData on MSN18 天
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) ...
World's youngest patient receives groundbreaking gene therapy in Cincinnati
A groundbreaking gene therapy treatment at Cincinnati Children's Hospital has given a 3-year-old boy named Sriansh a new ...
PD-1 deficiency reveals the impact of checkpoint inhibition on B cell function
A multinational collaboration co-led by the Garvan Institute of Medical Research has uncovered a potential explanation for why some cancer patients receiving a type of immunotherapy called checkpoint ...
Monthly Prescribing Reference17 天
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
Too Old to Operate4 天
FDA Approves Kebilidi for Aromatic L-Amino Acid Decarboxylase Deficiency
The U.S. Food and Drug Administration has approved Kebilidi (eladocagene exuparvovec-tneq) for the treatment of adult and ...
Becker's Hospital Review12 天
1st gene therapy delivered directly into brain approved
The FDA granted accelerated approved to the first gene therapy directly administered into the brain on Nov. 14. The therapy, Kebilidi, was approved to treat adults and children with aromatic L-amino ...
BioWorld18 天
FDA says yes to PTC for Kebilidi gene therapy in AADC deficiency
PTC Therapeutics Inc.’s accelerated go-ahead from the U.S. FDA for recombinant adeno-associated virus serotype 2-based gene therapy Kebilidi (eladocagene exuparvovec-tneq) to treat aromatic L-amino ...