2017 was a good year for biotechs looking to raise money. Venture capital investment in biotech companies reached the stratospheric levels of $16 billion. And it was not only seasoned companies that benefitted—the largesse extended to companies raising their first rounds, which accounted for nearly a third of risk capital entering the sector. Click here to original publication. Fonte: NATURE BIOTECHNOLOGY VOLUME 36 NUMBER 4 APRIL 2018…
Top drugs and companies by sales in 2017
A look at the best-selling drugs in 2017 shows the top spots dominated by monoclonal antibodies which, despite their age and looming competition, are still pulling in billions of dollars (FIG. 1a). The success of these huge franchises is partly down to the ability of manufacturers to continue to raise prices in the United States, even on these long-established assets, while… Click here to original publication. Fonte: 232 | APRIL 2018 | VOLUME 17 www.nature.com/nrd…
Upcoming market catalysts in Q2 2018
Potential market catalysts in the second quarter of 2018 include top-line clinical trial results for NEOD001 (developed by Prothena) for light-chain (AL) amyloidosis and ALXN1210 (developed by Alexion) for paroxysmal nocturnal haemoglobinuria (PNH), as well as an FDA advisory committee meeting on volanesorsen (developed by Akcea Therapeutics) for familial chylomicronaemia syndrome (FCS)… Click here to original publication. Fonte: NATURE REVIEWS | DRUG DISCOVERY VOLUME 17 | APRIL 2018 | 233 …
First off-the-shelf mesenchymal stem cell therapy nears European approval
Almost as soon as TiGenix gained the European regulator’s recommendation for its allogeneic adipose-derived mesenchymal stem cell (MSC) therapy to treat complex perianal fistulas in Crohn’s disease, its partner Takeda moved to strike a deal. On December 15 the European Medicines Agency (EMA)… Click here to original publication. Fonte: 212 VOLUME 36 NUMBER 3 MARCH 2018 NATURE BIOTECHNOLOGY…
Migraine drug race turns its final corner, FDA decisions in sight
Over the next six months, the US Food and Drug Administration (FDA) is widely anticipated to approve a trio of monoclonal antibody (mAb) drugs for preventing migraine, in what could be a new era in the disease’s treatment… Click here to original publication. Fonte: NATURE BIOTECHNOLOGY VOLUME 36 NUMBER 3 MARCH 2018 207…
The market for chimeric antigen receptor T cell therapies
2017 was a landmark year in oncology. The FDA approved the first two CD19-targeted chimeric antigen receptor (CAR) T cell therapies: tisagenlecleucel-T (Kymriah; Novartis) and axicabtagene ciloleucel (Yescarta; Kite Pharma/Gilead Sciences). These therapies have remarkable efficacy in some blood cancers and herald a paradigm shift in oncology treatment… Click here to original publication. NATURE REVIEWS | DRUG DISCOVERY VOLUME 17 | MARCH 2018 | 161…
Alnylam prepares to land first RNAi drug approval
Drug developers have been fishing in the bumpy RNA interference (RNAi) waters for years without a catch in sight. They probably won’t have to wait much longer. In December 2017, RNAi pioneer Alnylam Pharmaceuticals… Click here to original publication. Fonte: 156 | MARCH 2018 | VOLUME 17 www.nature.com/nrd…
Gene therapies for hemophilia hit the mark in clinical trials
Although the first gene therapy proposals are almost 50 years old1, the past year has been a coming of age for this field. In March, after approval by the European Medicines Agency the previous year, the first patient was treated with a commercial gene therapy to correct an inherited immunodeficiency by modifying blood stem cells ex vivo. In August, a genetically modified cell product was approved for the first time by the US Food and Drug Administration (FDA), comprising engineered…
Fresh from the biotech pipeline—2017
The mid-December approval of a gene therapy packaged inside an adeno-associated virus (AAV) vector to treat patients with certain inherited retinal diseases capped several decades of effort to bring in vivo gene therapy to patients. It was also the cherry on top of an impressive year… Click here to original publication. Fonte: NATURE BIOTECHNOLOGY VOLUME 36 NUMBER 2 FEBRUARY 2018 131…
FDA warns public of dangers of DIY gene therapy
People are experimenting on themselves with unlicensed gene therapies and publicizing the events widely, prompting the US Food and Drug Administration (FDA) to issue a statement on November 21 cautioning against do-it-yourself (DIY) gene therapies… Click here to original publication. Fonte: NATURE BIOTECHNOLOGY VOLUME 36 NUMBER 2 FEBRUARY 2018…