News

A team of scientists at the VIB research institute and KU Leuven in Belgium has discovered that an amyloid-beta precursor protein, APP, modulates neuronal signal transmission by binding to a specific receptor called GABA_BR1a. This has implications for treating Alzheimer’s disease and probably other disorders.

Alzheimer’s-affected brains are clogged with amyloid-beta plaques. These fragments are produced from a precursor protein whose normal function has remained unclear for decades. 

A wave of clinical trial applications for new cell and gene therapies has reached the US Food and Drug Administration triggering a review of clinical guidance documents for developers. In an announcement on 15 January, Scott Gottlieb, the FDA commissioner, said new guidances are being drafted for gene replacement therapies as well as for cell-based gene therapies such as the chimeric antigen receptor (CAR) T cell drugs.

Scientists at the University of Cambridge have identified mechanisms by which mutations in the ATM gene can lead to cancer drug resistance and how this can be counteracted by changes in other genes. The findings, reported on 8 January 2019 in Nature Communications, show how cells respond to DNA damage as well as highlight potential therapeutic targets for the genetic disease, ataxia-telangiectasia (A-T).

A partnership between German and US companies aims to develop gene therapies for neurodegenerative diseases that would cross the blood-brain barrier and deliver therapeutic agents to the brain.

The collaboration is between Sirion Biotech GmbH, which specialises in viral vector technologies for gene and cell therapies, and Denali Therapeutics Inc, a developer of treatments for diseases of the central nervous system.

Neem Biotech Ltd, which is researching treatments for bacterial infections, has received £50,000 in public funding to advance its studies into treatments for bacterial infections in wounds. The company is investigating potential therapies targeting biofilms which are formed by bacteria as a protection against the human immune system and antibiotics.

The German biotech company Immunic AG is to execute a reverse merger with Vital Therapies Inc of the US in the first step towards an anticipated share listing on Nasdaq. The two companies have agreed to an all-share transaction that will create a new company, Immunic Inc, the majority of whose shares will be held by Immunic AG.

AstraZeneca Plc has shaken up its pharma R&D structure, creating two units with parallel commercial operations whose heads will both report directly to the chief executive, Pascal Soriot

An oncology unit will be led by José Baselga, previously physician-in-chief at the Memorial Sloan Kettering Cancer Center in New York, US, while a unit for biopharmaceuticals, covering cardiovascular, renal, metabolic and respiratory medicines, will be headed by Mene Pangalos, a current AstraZeneca executive.

Regeneron Pharmaceuticals Inc and Sanofi SA have scaled back their collaboration in immuno-oncology to co-develop just two bispecific compounds, leaving other projects to be pursued independently by the two companies.

The restructuring affects a partnership dating from 2015 which has already yielded one approved product, Libtayo (cemiplimab), for the treatment of advanced cutaneous squamous cell carcinoma. Libtayo was approved by the US Food and Drug Administration in September 2018.

AstraZeneca Plc has reached out to a UK biotech, Aptamer Group Ltd, to explore the use of aptamer technology in developing candidate drugs for kidney disease. Specifically, the two companies will explore the feasibility of developing aptamer drug conjugates to deliver therapeutic molecules to the kidney.

Hookipa Pharma Inc is to receive a milestone payment from Gilead Sciences Inc for successfully designing and delivering 14 research grade vectors for a potential treatment for infections from the human immunodeficiency virus (HIV). The size of the payment was not disclosed, but it comes just seven months after a licencing deal between the two companies took effect.