Laverock Therapeutics makes significant advances in its unique programmable gene silencing platform and announces pipeline focus on advanced therapies in oncology and genetic diseases

  • – Programmable, tunable, stable and specific gene silencing has been demonstrated across multiple target genes and multiple cell types.
  • – PoC achieved with concomitant silencing of more than one gene
  • Industrialisation of platform processes delivering significant increase in throughput
  • Strategic in-house focus on development of pipeline of programmable advanced therapies in oncology and genetic disease.
  • – Potential additional application to multiple high impact therapeutic areas through partnering including, hypoimmunogenicity/type 1 diabetes, inflammatory diseases and fibrosis.

Laverock Therapeutics, a gene-silencing company with a uniquely powerful technology for the development of programmable advanced therapies to tackle major diseases, today announces significant progress in the industrialisation and validation of its technology platform enabling broadening of its potential therapeutic application. Laverock also confirms in house focus 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 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 has demonstrated 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 will be presenting these data at a number of conferences in the coming months.

In the light of the growing body of evidence supporting the Laverock platform’s unique attributes, and the platform’s capacity to rapidly advance programmes, Laverock has solidified its in house therapeutic focus on high value areas, adding genetic diseases to oncology.

To maximise the value of its platform Laverock 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.”

Laverock has commenced a financing round to fund its pipeline development and further advance the platform.