News

The US Food and Drug Administration has approved the first allogeneic tissue product for the treatment of children born without a thymus, a condition known as congenital athymia. The product, Rethymic, is a donor derived product that is processed and cultured and implanted into patients to help reconstitute immunity in the absence of a thymus gland. The thymus is located behind the sternum and between the lungs, and is active until puberty. The gland secretes thymosin, a hormone necessary for T cell development and production.

A new biotech company with plans to target ABC transporters, one of the oldest families of proteins, has been launched with $100 million in Series A financing by US-based Atlas Venture and three other groups. Rectify Pharmaceuticals Inc will develop small molecule drugs aimed at restoring dysfunctional ABC transporters to a normal state. These transporters play an essential role in ferrying organic and inorganic molecules across cellular membranes.

A new collaboration between Intellia Therapeutics Inc and the French ophthalmology company SparingVision SAS will seek to apply genome editing technology to the treatment of diseases of the eye. Announced on 13 October, the partnership will use Intellia’s in vivo Crispr/Cas9-based platform to investigate up to three ocular targets.

ReNeuron Group Plc has reported that exosomes were able to deliver a therapeutic protein to the brain of an animal and function better than the supply of the protein alone. Exosomes are intracellular vesicles that carry a variety of proteins and genetic material between cells. ReNeuron is exploring their use as possible delivery vehicles for  therapies targeting neurological diseases.

An antibody combination therapy has shown a statistically significant reduction in severe Covid-19 or death among patients with mild to moderate disease, the developer AstraZeneca Plc reported on 11 October. This follows the successful trial of a small molecule antiviral agent produced by Merck & Co Inc and Ridgeback Biotherapeutics LP for a similar patient population which was announced on 1 October. Using different agents, the two trials both achieved efficacy rates of 50% compared with placebo.

CureVac NV has withdrawn its application for a marketing authorisation for its first Covid-19 vaccine from the European Medicines Agency, citing plans to focus instead on a second generation product that has shown an improved immune response in an animal study. The second generation vaccine, CV2CoV, is being developed with GlaxoSmithKline Plc. It encodes for the same protein as the company’s earlier vaccine but differs in the design of non-coding regions of the mRNA backbone.

Evotec SE has made a regulatory filing in the US in preparation for a listing of its shares on the US Nasdaq market. The size of the issue of American Depositary Shares (ADSs) had not been determined as of 11 October. But it comes at a time of strong revenue growth for the Germany-based company.

The scientists Benjamin List and David MacMillan have been awarded the Nobel Prize in Chemistry for their discovery that small organic molecules are suitable as mediators of chemical reactions. Working independently of one another, Drs List and MacMillan showed that organic molecules constitute a third type of catalyst that can accelerate chemical reactions and lead them to a desired goal. Previously, scientists assumed that only enzymes and metals could perform this task.

An anti-viral medicine developed jointly by Merck & Co Inc and Ridgeback Biotherapeutics LP, has reduced the risk of hospitalisation or death in patients with Covid-19 by 50% compared with placebo, the two companies announced on 1 October. A Phase 3 trial of the drug, molnupiravir was stopped early and the developers plan to seek an emergency use authorisation from the US Food and Drug Administration in order to get the medicine onto the market quickly. Molnupiravir is a small molecule ribonucleoside analogue that inhibits the replication of the SARS-CoV-2 virus.

A Phase 3 study of Keytruda (pembrolizumab) in Asian patients with advanced hepatocellular carcinoma met is primary endpoint of overall survival, the developer Merck & Co Inc announced on 27 September. Patients in the trial had previously been treated with sorafenib, an approved treatment for kidney and liver cancers. Hepatocellular carcinoma is the most common type of primary liver cancer.