Atidarsagen autotemcel is a gene therapy for metachromatic leukodystrophy (MLD).
gene therapy
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory
Skysona: Gene Therapy for Cerebral Adrenoleukodystrophy
A single dose of the elivaldogene autotemcel will cure a deadly disease.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Roctavian: Gene Therapy for Hemophilia A
A single infusion of valoctocogene roxaparvovec can completely cure severe hemophilia A.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Zynteglo: Gene Therapy for Beta-thalassemia
One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Trikafta/Kaftrio: World’s First Triple Therapy for Cystic Fibrosis
The combination drug will help almost all cystic fibrosis patients.
Posted inClinical Trials, Rare/Orphan Diseases
Vyjuvek: Effective Treatment for Dystrophic Epidermolysis Bullosa
Krystal Biotech’s beremagene geperpavec gene therapy will save from imminent death.
Posted inAutoimmune Diseases, Neuroscience, Oncology, Pharma & Biotech
Bit Bio: Mass and Problem-Free Production of Any Human Cells
Human cells are needed for scientific research, development of new drugs, and creation of cell therapies.