An academic team in the US has made progress in designing an experimental gene therapy for multiple sulfatase deficiency (MSD), a lysosomal storage disorder that affects the brain, lungs, skin and skeleton and for which there are no approved treatments. The results of the preclinical study, conducted at the Children’s Hospital of Philadelphia, were published in the journal Molecular Therapy on 4 September 2024.
Asgard Therapeutics AB, a privately-held preclinical biotech based in Sweden announced on 5 September the publication in Science of preclinical data for its lead gene therapy programme AT-108. The programme is an autologous therapy that reprogrammes tumour cells into a subset of dendritic cells thereby mounting a cytotoxic T cell response in cancer. The study, co-led by Asgard and the scientists at the Pereira Lab at Lund University in Sweden, showed that it was possible to reprogramme dendritic cells in mice that were resistant to treatment with checkpoint inhibitors.
The European Medicines Agency has given a conditional marketing authorisation to a gene therapy for haemophilia B which is a one-time treatment for the rare inherited blood disorder. Announced on 31 May, the decision is for fidanacogene elaparvovec, an adeno-associated virus based therapy that is designed to deliver a functional copy of the Factor 9 gene into the body enabling patients to produce Factor 9 protein themselves. The developer is Pfizer Inc which received US regulatory approval for the same therapy in late April.
The US Food and Drug Administration has expanded the permitted medical use of a gene therapy for Duchenne muscular dystrophy (DMD), making it available to patients who are both ambulatory and non-ambulatory. DMD is a genetic disorder characterised by a progressive deterioration of muscle due to abnormalities in, or the absence of, dystrophin protein which helps keep muscle cells intact.
A gene therapy being investigated in a Phase 3 trial of boys with Duchenne muscular dystrophy (DMD) has failed to reach its primary endpoint of improvement in motor function, the developer Pfizer Inc announced on 12 June. The therapy, fordadistrogene movaparvovec, missed the primary endpoint at one year as well as key secondary endpoints compared with a placebo. The secondary endpoints included an improvement in velocity in a 10 metre run or walk. Participants were boys between the ages of four and seven who were on a daily regimen of glucocorticoids.
France-based SparingVision SA, which is developing gene therapies for retinal diseases, has presented data from an ongoing natural history study of rod-cone dystrophy showing the structural features of the disease and identifying a subset of patients with a higher rate of disease progression. This is to inform the company’s development of an ocular gene therapy for the disease which is in a separate Phase 1/2 trial in patients for severe rod-cone dystrophy.
A gene therapy being developed by Regeneron Pharmaceuticals Inc has enabled a young child, suffering from a congenital hearing loss, to overcome deafness. The child, dosed at 11 months of age, is one of two to have responded to the experimental therapy which uses gene replacement to address mutations in the OTOF gene – the cause of the disease. A second child, who received the therapy at the age of four, showed initial improvements after dosing.
A new gene therapy company with plans to start clinical development of its first product has raised $54 million in a Series A financing round. Latus Bio Inc is a spin-out of the Children’s Hospital of Philadelphia, US, with a new technology for producing adeno-associated viral vectors, one of the most common vectors in use for gene therapies. Announced on 2 May, the financing round was led by 8VC of Texas and DCVC Bio of California.
Genentech Inc and its Swiss parent, Roche, have ended a cell therapy partnership with Adaptimmune Therapeutics Plc which was focused on developing allogeneic T cell therapies for cancer. The reason for the termination, announced on 10 April, wasn’t given. However it comes at a time when other large companies are making staff redundant or divesting early-stage biotech projects. On 11 April, Syncona, the investment group, announced plans to sell a cell therapy portfolio company to Century Therapeutics Inc. This was to prioritise capital allocation on later-stage assets.
An experimental gene therapy for the rare neurological disorder, Canavan disease, has been given a regenerative medicine advanced therapy (RMAT) designation by the US Food and Drug Administration. RMAT designation is a regulatory incentive awarded by the agency to cell or gene therapy products which show promise for treating or reversing the course of a serious disease.