Laverock Therapeutics is expanding its therapeutic pipeline into in vivo CAR-T and genetic medicine, building on its existing ex vivo cell therapy programmes.

Laverock expands into in vivo therapeutics

The biotechnology company is developing in vivo programmes in oncology and genetic medicine using its programmable gene control technology.

Laverock’s platform is designed to provide programmable and tunable control of gene expression across multiple targets and pathways. The company has previously applied the technology to ex vivo cell therapy programmes, including autologous T cells and induced pluripotent stem cell-derived macrophages targeting solid tumours.

Its new in vivo programmes will initially focus on CAR-T therapy for solid tumours and genetic medicines for nervous system disorders and metabolic disease.

In vivo CAR-T aims to generate therapeutic T cells directly within the patient, potentially removing some of the manufacturing and logistical requirements associated with conventional ex vivo approaches.

Laverock said its gene control platform is being developed to regulate multiple targets or pathways simultaneously and to provide context-specific control of gene expression.

The company’s oncology programme will focus on the application of in vivo CAR-T to solid tumours, an area where developing effective cell therapies presents additional challenges because of the tumour microenvironment.

David Venables, CEO of Laverock Therapeutics, said: “Building on our success in developing ex vivo therapeutics, we are extremely excited to announce the launch of our in vivo programmes, and realise the benefits these cutting edge technologies can bring to patients.”

Gene control technology targets multiple pathways

In genetic medicine, Laverock plans to use its technology for in vivo gene silencing through the direct delivery of gene-modifying tools.

The initial target areas are nervous system disorders and metabolic disease. These programmes are intended to address diseases where controlling several genes or biological pathways may be important to treatment.

Laverock’s platform uses recoded microRNAs to provide programmable control of gene expression. The company said the approach is designed to allow gene regulation to be adjusted according to specific biological conditions.

This differs from approaches that rely on permanently altering a single genetic target, although the clinical relevance of the technology will depend on results from development programmes.

The expansion brings together two areas of advanced therapy development: engineered cell therapies and genetic medicines. Both approaches are being investigated as potential ways of treating diseases that are difficult to address with conventional medicines.

Laverock is also continuing to develop ex vivo applications of its gene control platform alongside the new in vivo programmes.

In vivo CAR-T development moves into solid tumours

CAR-T therapies have produced clinical responses in several blood cancers, but extending the approach to solid tumours remains an area of research.

Solid tumours can present challenges for engineered T cells, including the physical tumour environment and biological signals that can suppress immune activity.

Laverock is developing its programmable gene control technology to address these factors by controlling the activity of multiple genes or therapeutic payloads.

The company has not disclosed clinical-stage data for its new in vivo programmes in this announcement, so the potential efficacy and safety of the approach remain to be established through further development.

Laverock said it has technology partnerships in these areas and is seeking further collaboration and co-development opportunities.

The company has raised more than £20M in seed funding to date and is developing its own pipeline alongside partnered programmes.

The new programmes extend Laverock’s focus from ex vivo cell therapies into in vivo approaches spanning oncology and genetic medicine, with the initial development areas covering solid tumours, nervous system disorders and metabolic disease.