• Strong pipeline of pre-clinical drug candidates 

• Moving into the clinical trial phase later this year

• Major collaboration with US biopharmaceutical firm Mallinckrodt agreed

What does Silence do?

The name provides a hint. It is a specialist in the field of gene silencing. Its technology uses RNA interference, a biological process in which RNA molecules inhibit gene expression or translation, by neutralising targeted molecules.

How’s it doing

Silence Therapeutics PLC (LON:SLN) has made a clinical trial application for its lead candidate, SLN124, which is being developed to treat iron overload disorders.

SLN124 already has orphan drug designation in Europe, which could shorten its timeline to market while providing the approved product with additional exclusivity.

Silence describes SLN124 as a “promising candidate medicine” for patients with iron overload disorders such as ß -Thalassemia, Myelodysplastic Syndrome and Hereditary Hemochromatosis.

A phase Ib, first-in-human trial is scheduled to start in the third quarter.

Another drug in Silence’s pipeline is SLN500, which will be used to treat rare diseases.  Silence announced in July that it has agreed a partnership with US biopharmaceutical firm Mallinckrodt PLC (NYSE: MNK) for SLN500.

Silence will be responsible for taking SLN500 through pre-clinical studies and a phase I trial. After that, Mallinckrodt will take over clinical development and responsibility for global commercialisation.

Meanwhile, an investigational new drug/clinical trials application for SLN360, a treatment for a range of cardiovascular disease, is anticipated to be filed in the second half of 2020.

What the boss says:

In the group’s 2018 results statement in March, chief executive David Horn Solomon said:  "2018 was a defining year for Silence Therapeutics, with transformational change throughout the business.

 “With the first RNAi therapeutics now approved by the FDA, effectively creating a new class of medicines, we are working hard to consolidate our position as a leading developer in this exciting new field.

“During the year we have made great progress in advancing our lead product SLN124 towards the clinic and we are due to commence in-human clinical trials later in 2019 to demonstrate safety and tolerability.

"We look forward to unlocking more of Silence Therapeutics' potential in 2019 and beyond for the benefit our shareholders and, importantly, for the patients of these devastating conditions."