Gene therapy has come a long way since the initial treatments of the early 1990s, but the insertion of genes can't help diseases with toxic gain-of-function mutations. Apic Bio Inc. is looking to overcome that limitation with its gene therapy platform, Thrive, which includes a second component expressing an miRNA to silence toxic gain-of-function mutations from a single vector.
Living up to its name, C4 Therapeutics Inc. exploded onto the scene before the J.P Morgan Healthcare conference in 2016 with a $73 million series A and a Roche Holding AG deal worth more than $750 million in hand. (See BioWorld Today, Jan. 7, 2016.)
Coming out of stealth mode, Ribon Therapeutics Inc. secured a $65 million series B financing round, adding to the $43 million series A the company raised in November 2015, to advance its pipeline of drugs targeting monoPARPs, a subset of 12 members of the poly ADP-ribose polymerase (PARP) family.
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.
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).
