Company News

Launch of siRNA delivery company

Country
United Kingdom

A new platform company focused on the delivery of small interfering RNA (siRNA) to cells was launched on 13 October with the goal of addressing one of the main limitations of genetic medicines – reaching tissues beyond the liver. Undecimal Bio Ltd is a spin-out of Eleven Therapeutics (UK) Ltd, a synthetic medicines company. It has raised $18 million in seed financing from a syndicate co-led by 4BIO Capital and IQ Capital of the UK. Its initial therapeutic focus is renovascular disease.

Scarlet cells engage DARPA

Country
United Kingdom

In the US and elsewhere, programmes financed by governments for national defense can take many forms – including to support basic science. The Defense Advanced Research Projects Agency (DARPA) of the US, originally founded in the late 1950s to develop satellite technology, has in recent years branched out into computers and vaccines. On 23 September, DARPA stepped deeper into biology with the award of a research grant of up to $14 million to a consortium led by Scarlet Therapeutics Ltd of the UK to develop engineered, laboratory-produced red blood cells for defense.

Seed funding for RNA medicine

Country
Italy

A syndicate of venture capitalists, with support from the European Regional Development Fund, has raised seed financing of €40 million for Aptadir Therapeutics Srl of Italy to support development of a new RNA therapeutic for difficult genetic disorders. The preclinical compound is being investigated for Fragile X syndrome, an inherited generic, neurological disorder.

New oral drug for genetic disease

Country
United States

The US Food and Drug Administration has approved a new small molecule drug for a rare genetic disease that can impair the supply of thyroid hormone to the brain whilst also leaving excessive levels of the hormone in the bloodstream. The drug, Emcitate (tiratricol), was developed by Egetis Therapeutics US Inc, and received multiple designations in the run-up to  its approval on 28 September, including a Priority Review designation in March.

Sofinnova launches new fund

Country
France

Sofinnova Partners SAS has created a new fund to build and support companies developing  medical technologies with plans to launch six to eight new companies over the next five years. This is being enabled by an over-subscribed €82 million financing round announced on 28 September. While the target technologies haven’t been identified, the broad approach is to create companies from the ground up. Explaining this strategy, Antoine Papiernik, managing partner, said the investment group builds and supports “every company from day one.” These assets are then scaled up.

Gene therapy for rare disease

Country
United States

The US Food and Drug Administration has approved a new gene therapy for a rare neurological disorder that manifests at an early age – Sanfilippo syndrome type A. Until the approval, treatment was limited to managing symptoms. The therapy, Fayuvi (rebisufligene etisparvovec), is an adeno-associated serotype 9 virus therapy (AAV9) that delivers a working copy of a gene enabling the body to produce sulfamidase, an enzyme which is missing in patients with the disease. Children with deficient supplies of the enzyme cannot beak down a sugar chain known as heparan sulfate.

Forbion leads financing for Sling

Country
Netherlands

A $123 million financing has been raised for US-based Sling Therapeutics Inc, equipping it to carry out a Phase 3 trial of a candidate therapy for thyroid eye disease – an autoimmune disorder that can affect vision. Announced on 16 September, the Series C round was led by Forbion Capital Partners of the Netherlands with participation from new investor Sectoral Asset Management of Canada and existing investor TPG Life Sciences Innovations of the US.

GSK builds oncology portfolio

Country
United Kingdom

GSK Plc is to pay up to $750 million to acquire a new asset for multiple myeloma – its latest move to expand its portfolio for blood cancers. The asset is a trispecific T cell engager, an engineered antibody linking the body’s immune cells directly to antigens on cancer cells. There are currently no approved trispecific T cell engagers, but authorisations have been granted to bispecific T cell engagers, a simpler version of the same therapy. Announced on 15 September, the deal includes an undisclosed upfront fee and milestone payments.

Nicox extends cash runway

Country
France

France-based ophthalmology company Nicox SA announced an extension of its cash runway on 2 September ahead of regulatory reviews in both the US and China for its lead product NCX 470 (bimatoprost grenod) for two ocular conditions. As of 31 August, the company’s estimated cash and cash equivalents was €8.4 million compared with €4.1 million on 31 December 2025. The company said the cash position will carry it through the regulatory decision period which, if successful, would generate new revenue.