A stem cell-derived therapy has restored the function of pancreatic islet cells in a patient with Type 1 diabetes in what is being described as the first demonstration of a potentially curative treatment for the disease. The therapy is being developed by Vertex Pharmaceuticals Inc on the basis of research conducted by Douglas Melton, a professor at Harvard University and co-director of the Harvard Stem Cell Institute in Cambridge, US.
Blackstone Life Sciences, an active investor in the biopharma sector, is to provide $150 million upfront to Autolus Therapeutics Plc to support its lead T cell product, obecabtagene autoleucel, for acute lymphoblastic leukaemia (ALL), as well as earlier oncology projects.
A new collaboration between Intellia Therapeutics Inc and the French ophthalmology company SparingVision SAS will seek to apply genome editing technology to the treatment of diseases of the eye. Announced on 13 October, the partnership will use Intellia’s in vivo Crispr/Cas9-based platform to investigate up to three ocular targets.
ReNeuron Group Plc has reported that exosomes were able to deliver a therapeutic protein to the brain of an animal and function better than the supply of the protein alone. Exosomes are intracellular vesicles that carry a variety of proteins and genetic material between cells. ReNeuron is exploring their use as possible delivery vehicles for therapies targeting neurological diseases.
Anjarium Biosciences AG, a Swiss company with technology for delivering gene therapies to patients without the use of a viral vector, has raised CHF 55.5 million (€50.96 million) from a group of investors co-led by Gimv and Abingworth LLP. The money will be used to expand the company’s staff and advance several of its programmes towards the clinic.
France-based Coave Therapeutics (formerly Horama SAS) has entered into a licensing deal, giving Théa Open Innovation, an ophthalmology company, rights to co-develop and commercialise a gene therapy for retinitis pigmentosa.
Astellas Pharma Inc has reported the death of a participant in a clinical trial of a gene therapy for the treatment X-linked myotubular myopathy, a rare neuromuscular disease caused by mutations in the MTM1 gene. The participant, who developed an adverse event during the trial, passed away on 9 September. “The cause of death is still pending,” the company announced on 14 September.
The US Food and Drug Administration has placed a clinical hold on a Phase 1/2 trial of a gene therapy for adults with phenylketonuria, a genetic disorder caused by the deficiency of an enzyme needed to break down an amino acid in certain foods. The clinical hold was based on findings from a preclinical study showing that six out of seven mice receiving the highest dose of the therapy developed liver tumours. One of the tumours was cancerous. BioMarin Pharmaceutical Inc, the developer, announced the clinical hold on 6 September.
France-based Eyevensys SA has raised $12 million from a syndicate led by Korea Investment Partners to accelerate development of its preclinical programme, EYS809, for treating wet age-related macular degeneration. The Series B round extension was also supported by existing investors. The Eyevensys technology is a non-viral gene therapy delivery platform that uses an electrotransfection system to deliver DNA plasmids encoding therapeutic proteins into the eye.
Two US biotech companies have launched a new partnership in order to use gene editing to develop medicines for multiple diseases including Type 1 diabetes. Vertex Pharmaceuticals Inc, known for its work in cystic fibrosis, and Arbor Biotechnologies Inc, a genetic disease specialist, will used Crispr gene editing technology to develop ex vivo engineered cell therapies. The new partnership, announced on 24 August, builds on a collaboration established in 2018.