© PIERRE GUERIN S.A.S.

Mid last year, the company Pierre Guerin has opened a new location in Germany, Hildesheim.

© Ian Wilson - Flickr, wikimedia commons

British drug major GlaxoSmithKline  (GSK) has passed its orphan autologous cell and gene therapy pipeline including its EU-approved ADA-SCID treatment Strimvelis to British/US Orchard Therapeutics. GSK acquired a 19,9% equity stake in Orchard and a board membership.

Oral once-daily WTX101 selectively reduced copper levels preventing neurological effects of currently available therapies. © Wilson Therapeutics AB

A public offer from orphan drug major Alexion Pharmaceuticals Inc. has been accepted by Wilson Therapeutics‘ Board of Directors. Alexion’s SEK232/share bid would total in an acqusition price of SEK6.564bn or €638.8m.

Location of neurons affected by spinal muscular atrophy in the spinal cord. © Angelito7, GNU licence

Swiss drug major Novartis has secured a potential stake in the multibillion dollar market of treatments for the orphan genetic muscle disease spinal muscular atrophy (SMA). The company acquired AveXis Inc, whose SMA gene therapy AVXS-101 is in Phase III testing.

Healthy and pathologically folded amyloid beta proteins absorb infrared light differently. In Alzheimer's patients, there is a frequency shift below the threshold level compared to healthy subjects. This is detected by the new blood test.
© K. Gerwert, A. Nabers / University Bochum

A newly developed blood test indicates Alzheimer’s disease on average eight years before the clinical diagnosis. 

Poor animal study design and reporting thwarts the ethical review of proposed human drug trials.
© nosheep/Pixabay

Human drug trials are compromised by poor reporting and potential bias of animal research. German researchers analysed more than 700 preclinical animal studies and investigator brochures provided by three medical centres. They call to establish documentation standards for IBs/preclinical dossiers.

Mode of action of OSE-172 ("Effi-Dem"). © Ose Immunotherapeutics

French  Ose Immunotherapeutics SA has licenced global commercialisation rights of its preclinical programme OSE-172 to Boehringer Ingelheim which hopes to complement its immunoncology portfolio with a tumour microenvironment modifier that reactivates effector T cell responses. 

CAR T cells attacking cancer. 
© Prasad S. Adusumilli, MD, FACS/Memorial Sloan Kettering Cancer Center

French allogenic CAR-T cell therapy developer Cellectis is set to launch an underwritten public offering of $175m of its American Depositary Shares (ADS), each representing one ordinary share of Cellectis. 

Staphylococcus aureus F98Y DHFR complexed with iclaprim © http://www.ebi.ac.uk/pdbe/entry/pdb/3FR

Motif Bio has initiated a NDA rolling submission for iclaprim, a Gram-positive-targeted investigational antibiotic for the treatment of acute bacterial skin and skin structure infections (ABSSSI) in high-risk MRSA (Methicillin-resistant S. aureus) patients. 

Shire's antibody prevents formation of bradykinin by binding kallikrein. © British Society of Immunology

Japanese drug giant Takeda Pharmaceutical Co. Ltd. has confirmed its intention to make a bid for orphan drug leader and neurology specialist Shire plc. The take-over offer has to be made until 25 April.