Laverock Therapeutics advances in its programmable gene silencing platform

Laverock Therapeutics, a UK-based gene-silencing company with a technology for the development of programmable advanced therapies to tackle major diseases, has made significant progress in the industrialisation and validation of its technology platform enabling broadening of its potential therapeutic application.

Laverock also said it is focusing on the development of a pipeline of programmable advanced therapies in oncology and genetic disease including Charcot-Marie-Tooth disease.

In the course of the last year, Laverock said it has generated a complete set of data in human cells demonstrating the unique features of its technology – namely the ability to silence genes in a programmable, tunable, stable and highly specific manner across a number of target genes and cell types. Laverock’s initial data showcasing the unique properties of the technology was generated against a single target (B2M, a component of the MHC-I complex), and the Company has now designed and validated silencing RNAs against seven additional targets, demonstrating that the technology is widely applicable and not limited to specific targets or cell types.

In addition, Laverock reported that the technology can be used to concomitantly silence more than one target gene, further expanding the range of its potential therapeutic applications. Further data now also supports the use of our gene-silencing approach to control the delivery of payloads and thereby use cells to deliver biologics in a programmed manner.

To drive our in-house product development and partnering, Laverock has also materially increased the robustness and throughput of its platform with significant increases in capacity to screen and validate constructs and carry out gene editing and cell line development, the company said.

The company said it will be presenting these data at a number of conferences in the coming months of 2024.

To maximise the value of its platform Laverock said it is actively seeking partners for indications where it has already generated product concepts, for example Type 1 diabetes/conditional hypoimmunogencity, and for new programmes in other indications including regenerative medicine, fibrosis and inflammation.

David Venables, CEO of Laverock, said: “Over the course of this year, following our seed financing last year, we have made enormous progress in validating our platform, proving its versatility and wide applicability. We have also ensured that we have a robust, industrialised process capable of rapidly advancing product concepts. We believe we are uniquely positioned to generate powerful and entirely novel advanced therapies across a range of disease areas through advancing our own oncology programmes to the clinic as rapidly as possible, generating pipeline depth with further programmes in genetic diseases, and through strategic partnering.”