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.
The Roche Group has entered into a collaboration and licensing agreement with Shape Therapeutics Inc of Seattle, US giving it access to an RNA editing technology that reportedly has promise for neurological disorders. Shape was founded in 2018, and since then has raised $147.5 million in Series A and Series B financing rounds. The deal with Roche, announced on 24 August, could generate as much as $3 billion in milestone payments for Shape should the collaboration yield marketable products.
T-knife Therapeutics Inc has raised $110 million in a Series B financing round to advance its preclinical portfolio of T cell receptor (TCR) therapies for cancer. The technology originated in Germany and will be developed and commercialised by teams based in Berlin and San Francisco, US.
A US company developing models of cancer tumours derived from the tissues of actual patients has received $70 million in Series A financing in order to advance its diagnostic technology. Xilis Inc of Durham, North Carolina has taken the concept of the organoid, a miniaturised organ, and adapted it to oncology. The financing was led by Mubadala Capital with participation by the new investors GV (formerly Google Ventures) and Life Sciences Partners (LSP) as well as existing investors.
Two European companies have decided to combine their expertise to develop a candidate gene therapy for the treatment of hereditary angioedema (HAE), a rare protein deficiency that can cause swelling in multiple parts of the body. Pharming Group NV, which already has an HAE product on the market, is to pay Orchard Therapeutics Plc for rights to a preclinical gene therapy programme for the same disease. The therapy, OTL-105, is an investigational ex vivo autologous haematopoietic stem cell therapy delivered with a lentiviral vector.
A new regenerative medicines company deploying regulatory T cells as potential therapies was launched in the US on 23 June with a Series A financing of $95 million. Abata Therapeutics plans to treat patients with severe autoimmune and inflammatory diseases, starting with non-relapsing multiple sclerosis, a disease for which there are no approved therapies. The Series A funding was provided by a syndicate led by Third Rock Ventures. Other investors included the biotech company ElevateBio LLC and the charity Juvenile Diabetes Research Foundation.