CRISPR base editing has delivered a landmark n-of-1 gene therapy for an infant with carbamoyl phosphate synthetase 1 (CPS1) deficiency, in a clinical milestone that may redefine how rare genetic diseases are treated. CPS1 deficiency is a mitochondrial disorder caused by a loss-of-function mutation in the CPS1 gene that impairs the urea cycle and leads to life-threatening hyperammonaemia.
The US Food and Drug Administration has approved a new allogeneic encapsulated, cell-based gene therapy for a rare neurodegenerative disease that affects the macula, a portion of the eye that processes central vision. The product, Encelto (revakinagene taroretcel) is a therapy that delivers a recombinant protein to the retina to slow down the progression of the disease. Delivery is by way of a surgical intravitreal implant by an ophthalmologist.
bluebird bio Inc is to be acquired by two private equity groups in a move to provide more capital to the gene therapy developer which was founded by two academics in 1992 and now has three approved products on the market. The transaction, which was announced on 21 February, will transform bluebird into a private enterprise from its current status as a publicly listed concern on the Nasdaq market. The buyers are Carlyle Group Inc and SK Capital Partners LP.
A new cell therapy has been approved by the US Food and Drug Administration to treat paediatric patients from two months of age who experience complications from a stem cell transplant for blood cancer or an immune system disorder. The treatment, Ryoncil (remestemcell-L rknd), is an allogeneic therapy made up from mesenchymal stromal cells and isolated from the bone marrow of healthy adult donors. It is indicated for the treatment of acute graft-versus-host disease in patients undergoing transplants who do not respond to corticosteroids.
Novartis is to tap the engineering expertise of Vyriad Inc of the US in order to discover and develop in vivo chimeric antigen receptor (CAR) T cell therapies. Announced on 20 November, the agreement will take cell-based gene therapy technology to a new level by enabling the CAR recombinant protein to be delivered to T cells in their natural environment. This will be done by using a lentiviral delivery system to target and transduce resting T cells and reprogramme them while they are still inside the body. The reprogrammed cells would become potential treatment for patients.
Biopharma executives in Europe and Asia, together with their financial supporters, have launched a new company to develop therapies that will both activate and inhibit the function of regulatory T cells (Tregs). Tregs are white blood cells that play a key role in regulating the immune system to ensure that the body responds appropriately to foreign antigens and neoantigens. Announced on 18 October, Regimmune/Kiji TX is the result of a merger between Kiji Therapeutics of France and Regimmune Ltd of Taiwan. The Japan based venture capital group DCI Partner Co Ltd helped execute the merger.
Purespring Therapeutics Ltd, a gene therapy company developing treatments for kidney diseases, has raised £80 million from private investors to bring its lead product into clinical development. This is the latest in a series of transactions among biotech companies in the kidney disease sector but the first for a preclinical gene therapy. Purespring’s product candidate, PS-002, is an adeno-associated viral vector with a gene payload for the treatment of IgA nephropathy (IgAN), a chronic kidney disease primarily affecting young adults.
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.