OMass Therapeutics Ltd, a 2016 spin-out from the University of Oxford, has raised $310 million in a Series C financing to support development of its lead programme for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome. The funding was announced on 15 October.
AstraZeneca Plc is to explore the use of artificial intelligence in drug development and expand its oncology portfolio under two new collaborations with US companies. The first, announced on 28 September, is an equity investment and clinical collaboration with Summit Therapeutics Inc under which the companies will investigate combination therapies of a bispecific antibody from Summit with antibody-drug conjugates (ADCs) from AstraZeneca.
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.
Senior officials from the European Commission have called the union’s deficit in the trade of goods with China unsustainable and called for immediate measures to rebalance the relationship. At a briefing for journalists on 12 October, the officials said that relations with China had to go beyond dialogue and include measures to address the union’s large trade shortfall with Beijing. This followed a meeting between top trade officials from China and the EU on 8 to 9 October where the two sides issued a list of 16 issues of mutual concern. One is the trade in hybrid vehicles.
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.
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.
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 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.
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.
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.