The first bispecific antibody to win regulatory approval is about to make a comeback 10 years after being taken off the market in Europe for commercial reasons. Catumaxomab, then called Removab, and now reborn with the brand name Korjuny, received a positive opinion for the treatment of malignant ascites from the EMA’s Committee for Medicinal Products for Human Use (CHMP,) at its monthly meeting Oct. 14 to 17.
Aviadobio Ltd. has entered a potential $2.18 billion license and commercialization agreement for its frontotemporal dementia gene therapy, AVB-101, with Astellas Pharma Inc. Astellas is making a $20 million equity investment in London-based Aviadobio and will pay up to $30 million up front in advance of deciding whether or not to exercise the exclusive option to worldwide rights.
Researchers in the U.K. have succeeded in reverse engineering the defective cryptic splicing that drives amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) to enable precisely targeted delivery of transgenes and therapeutic protein expression in diseased neurons. The technique is compatible with conventional adeno-associated viral vectors that are approved for gene therapy, and can readily be adapted for different transgenes. ALS, FTD and other neurogenerative diseases are underpinned by loss of function of the RNA-binding protein TDP-43 (transactive response DNA-binding protein 43), that normally functions as a key regulator of splicing, protecting the transcriptome from toxic cryptic exons.
Mabylon AG has been awarded three grants totaling more than CHF1.3 million (US$1.5 million) from Innosuisse Swiss Innovation Agency, Target ALS and the ALS Association.
Argenx SE’s ARGX-119 is a monoclonal antibody (MAb) targeting the muscle-specific kinase (MuSK) Frizzled (Fz)-like domain, and has entered early clinical development for the treatment of neuromuscular diseases.
It has been previously demonstrated that the activation of spleen tyrosine kinase (SYK) contributes to processes that are central to the pathogenesis of neurodegenerative diseases, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD).
NRG Therapeutics Ltd. has been awarded a grant from Target ALS Foundation to support its discovery program for a treatment for amyotrophic lateral sclerosis (ALS).
Acurastem Inc. has secured $4 million in grant funding from the California Institute for Regenerative Medicine (CIRM) to facilitate the development of its UNC13A program toward clinical trials for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
The big advantage of cell culture to model diseases is its throughput. “You can play the disease over and over again in the dish,” Clive Svendsen told the audience at the International Society of Stem Cell Research (ISSCR) Annual Meeting held in Hamburg last week. That high throughput, however, is not particularly useful if the cell lines themselves do not accurately model the disease. Cancer cell lines are used in many cell culture experiments far beyond cancer for their ability to grow. But they are “highly abnormal,” Bill Skarnes told the audience at an innovation showcase, as well as quite unstable. “I don’t think the [HEK-293] cell line is the same in your lab as it is in the lab next door,” Skarnes said.
