Regenerative Medicine

Hal Barron, the chief scientific officer at GlaxoSmithKline Plc, is to become chief executive of a new regenerative medicine company that will seek to understand the process of cellular rejuvenation with the goal of slowing or even reversing the effects of disease. The company, Altos Labs Inc, will combine basic science with translational medicine across two institutes in the US and one in the UK. It is being launched with $3 billion of start-up capital, according to a statement issued on 19 January.

ReNeuron Group Plc has taken a decision to out-license a clinical-stage cell therapy programme for retinitis pigmentosa (RP), an eye disease in which the retina is damaged, and focus instead on developing its exosome technology. The decision was announced to the London Stock Exchange on 18 January.

Belgium-based UCB SA is to acquire a US developer of drugs for rare diseases in a move that will both expand its existing epilepsy franchise and give it access to gene therapy technology. The proposed acquisition of Zogenix Inc of Emeryville, California is valued at approximately $1.9 billion (€1.7 billion) and has been approved by the boards of directors of both companies.

Novartis is to pay $800 million upfront to acquire the UK gene therapy company Gyroscope Therapeutics Ltd whose lead product GT005 is in clinical development for geographic atrophy, an advanced form of dry age-related macular degeneration (AMD). The acquisition will strengthen Novartis’ position in gene therapies for the eye nearly two years after its licensee Spark Therapeutics was taken over by Roche. Spark had an approved gene therapy for retinal disease at the time it joined the Roche group.

Celyad Oncology SA, a Belgian cell therapy developer, has made a private share placement with an affiliate of the Fortress Investment Group, raising $32.5 million to help support its operations into the first half of 2023. The placement involved 6.5 million ordinary shares and was arranged by SVB Leerink.

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.