Drugs Therapies Trials 
Drug Discovery World
SEPTEMBER 11, 2024
A gene therapy developed by University of Florida scientists restored useful vision to most patients with rare condition Leber congenital amaurosis type I (LCA1) in a small trial. The trial also tested the safety profile of the treatment. The gene therapy itself caused mild inflammation that was treated with steroids.
Drug Discovery World
SEPTEMBER 3, 2024
Grit Biotechnology has received investigational new drug (IND) approval from the US Food and Drug Administration (FDA) for its genetically engineered tumour-infiltrating lymphocyte (TIL) product GT201. GT201 is designed to boost T cell survival and function by expressing a vital membrane-bound cytokine complex.
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Drug Discovery World
DECEMBER 19, 2023
The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.
Drug Discovery World
AUGUST 19, 2024
CV6 Therapeutics has initiated patient dosing in Phase Ia clinical trial evaluating its investigational first-in-class, specific dUTPase inhibitor CV6-168 + infusional 5-fluorouracil (5-FU). In preclinical studies, the drug has demonstrated robust anti-tumour activity in cell line and animal models with no added toxicity.
Drug Discovery World
AUGUST 6, 2024
New data has revealed a robust microdystrophin expression from a pivotal dose of gene therapy RGX-202 for Duchenne muscular dystrophy (Duchenne). It is the only gene therapy approved or in development for Duchenne that encodes for the C-Terminal (CT) domain to produce a microdystrophin that is closer to naturally occurring dystrophin.
Drug Discovery World
AUGUST 14, 2024
The US Food and Drug Administration (FDA) has approved a Phase II clinical trial for Biosyngen’s BRG01, an EBV-specific CAR-T cell therapy. This marks the first cell therapy to enter Phase lI trials in both the US and China for the treatment of relapsed/metastatic EBV-positive nasopharyngeal carcinoma.
Drug Discovery World
JULY 5, 2024
AviadoBio has launched its Phase I/II ASPIRE-FTD clinical trial at the Ohio State University in the US. ASPIRE-FTD is evaluating investigational gene therapy AVB-101 in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN).
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