There are many proteins in humans, bacteria and viruses that would make great targets to shut down a pathway to treat a disease or stop an infection, but, for a variety of reasons, designing drugs to target those proteins has been difficult.

Transthyretin (TTR) amyloidosis (ATTR), a rare disease caused by the misfolding and aggregation of the TTR protein, has reached an inflection point with two drugs approved to treat the disease this year and multiple drugs in development.

SAN DIEGO – Imbruvica (ibrutinib) was first approved for second-line chronic lymphocytic leukemia (CLL) in 2014 by Janssen Biotech Inc. and Pharmacyclics Inc. based on overall response rate from a phase Ib/II study and then, later that year, with data from the phase III Resonate study where Imbruvica improved progression-free survival and overall survival compared to Arzerra (ofatumumab, Novartis AG).

Beigene Ltd. clearly liked what it saw in the bispecific antibody platform being developed by Zymeworks Inc. The Beijing-based company is hooking up with Zymeworks in a pair of deals that could end up grossing Zymeworks more than $1 billion.

Biological drugs – monoclonal antibodies, peptides, recombinant proteins and the like – now account for more than 30 percent of all FDA approvals, according to a new study from the Tufts Center for the Study of Drug Development.

Biological drugs – monoclonal antibodies, peptides, recombinant proteins and the like – now account for more than 30 percent of all FDA approvals according to a new study from the Tufts Center for the Study of Drug Development.

SAN DIEGO – At Neurosciences 2018, researchers highlighted mouse experiments and human biomarker studies further straightening the connection between dementia and the cardiovascular system.

SAN DIEGO – As gene therapy advances in the eye and hematopoietic stem cells, researchers are going after the harder-to-treat diseases of the central nervous system (CNS). At the Neurosciences 2018 conference, preclinical data showed the right vector and delivery technique can improve symptoms in mouse models of amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD) and Batten disease as the treatments advance to the clinic.

Medical cannabis companies developing pharmaceuticals have largely focused on using purified cannabinoids as monotherapies to treat neurological diseases. This year, for example, Cambridge, U.K.-based GW Pharma plc gained FDA approval for Epidiolex (cannabidiol) to treat two rare pediatric epilepsies, Dravet syndrome and Lennox-Gastaut syndrome, and others are on GW's heels, developing drugs for epilepsy and pain indications.