Gene therapy digest – 2021 Gene therapy digest – 2021 2 year hold on Zolgensma’s intrathecal OAV-101 lifted (NOVARTIS) OAV-101 (SMN1 replacement gene therapy; Phase 3) – Novartis OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may […]
Gene therapy digest – 2021 Gene therapy digest – 2021 NMPA granted first IND approval for Belief Biomed’s gene therapy BBM-H901 (AAV gene therapy; IND approved) – Belief Biomed IND approval is for BBM-H901, an IV infusion gene therapy for […]
Gene therapy digest – 2021 Gene therapy digest – 2021 Eyevensys’ gene therapies get a financial boost EYS809 (Anti-angiogenic & anti-fibrotic; Preclinical), EYS611 (Antioxidant; IND-enabling) Series B Plus funding – $12M and Korea Investment Partners is the key investor Eyevensys’ […]
Gene therapy digest – 2021 Gene therapy digest – 2021 PRIME to boost Mustang Bio’s MB-107 before pivotal trial in XSCID MB-107 (Lentiviral gene therapy; Phase 2) – Mustang Bio/ Fortress Biotech EMA granted Priority Medicines designation to MB-107 for […]
Gene therapy digest – 2021 Gene therapy digest – 2021 XyloCor advances its lead gene therapy candidate for refractory angina to Phase 2 XC001 (AAV gene therapy; Phase 2) – XyloCor Therapeutics Phase 1 dose-escalation part of Phase 1/2 clinical […]
Gene therapy digest – 2021 Gene therapy digest – 2021 ASC Therapeutics to codevelop first in-vivo gene therapy program for maple syrup urine disease AAV gene therapy (Preclinical) – ASC Therapeutics ASC Therapeutics with University of Massachusetts Medical School (UMMS) […]
