Research 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
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.
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Drug Discovery World
SEPTEMBER 3, 2024
The therapy is now approved by both the FDA and China Center for Drug Evaluation (CDE) to enter clinical trials in both countries for advanced solid tumour patients. According to the company, GT201 surpasses traditional TIL therapies in proliferation, tumour-killing efficacy and long-term survival with reduced dependence on IL-2.
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).
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).
Drug Discovery World
JULY 19, 2024
Dr Gonzalez-Aseguinolaza said: “This is a tremendous honour, and I am very pleased that the RFS Society recognises my dedication and work in the field of gene therapy. She then moved into rare diseases and has been working on rare hepatic diseases for the last two decades, with a focus on the development of advanced therapies.
Drug Discovery World
FEBRUARY 6, 2024
The first UK patients have received an mRNA cancer therapy – mRNA-4359 – as part of a Phase I/II clinical trial investigating its potential for treating melanoma, lung cancer and other solid tumour cancers. The study will assess the therapy administered alone or in combination with immune checkpoint inhibitor pembrolizumab.
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