Company News

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.

Therapy for blood disorder

Country
United States

Patients with polycythaemia vera, a rare blood disorder that causes the body to make too many red blood cells, will be eligible for treatment with a new peptide therapeutic approved by the US Food and Drug Administration on 28 August. Mimrylo (rusfertide) is a hepcidin mimetic that works by mimicking the action of hepcidin, a hormone that regulates the body’s use of iron. Too little iron can lead to iron-deficiency anaemia while too much can be toxic.

Drug for autoimmune disease

Country
United States

The US Food and Drug Administration approved a new treatment for dermatomyositis on 27 August – the first oral drug for a disease that can cause progressive damage to the muscles, skin and lungs and an impaired quality of life. Existing treatments include combinations of chronic steroids and antirheumatic drugs which have not been effective in controlling the disorder. The new treatment, Lisraya (brepocitinib), works as a Janus kinase inhibitor, blocking pathways that play a key role in the body’s immune responses.

Win for pancreatic cancer drug

Country
United States

A new small molecule drug has won approval from the US Food and Drug Administration to treat patients with metastatic pancreatic ductal adenocarcinoma – an aggressive cancer with a poor prognosis for survival. The drug, Rasonque (daraxonrasib), has been authorised for adults who have received at least one prior systemic therapy for their cancer, or who are not candidates for multiagent systemic therapy.

ReCode tightens focus

Country
United States

US-based ReCode Therapeutics Inc has strengthened its ties with the Cystic Fibrosis Foundation as it progresses an experimental messenger RNA (mRNA) therapy for the disease through the clinic. Simultaneously, it promoted Heather Clark on 1 July to the role of chief executive from vice president and head of the company’s cystic fibrosis franchise. Ms Clark joined ReCode in 2022. She has nearly 30 years of experience in rare disease drug development of which more than half were spent at Vertex Pharmaceuticals Inc, developer of five marketed cystic fibrosis products.