It’s back to the drawing broad for Vertex Pharmaceuticals Inc. and its efforts to develop treatments for the rare disease alpha-1-antritrypsin deficiency (AATD), after its VX-864 produced disappointing results in a phase II trial.
After cutting a deal worth up to $1.1 billion with Crispr Therapeutics AG, it took Vertex Pharmaceuticals Inc. only two days to come up with another collaboration that could top the $1 billion mark, this time with Obsidian Therapeutics Inc.
In biotech and biopharma’s third-largest ever up-front development and commercialization deal, Crispr Therapeutics AG will receive an initial $900 million in an amended deal with Vertex Pharmaceuticals Inc. to lead the development, manufacturing and commercialization of gene editing therapy CTX-001 for sickle cell disease and transfusion-dependent beta-thalassemia.
Skyhawk Therapeutics Inc., one of the early leaders in developing small-molecule drugs that target RNA, closed out the year with yet another big pharma partnering deal, an alliance with Vertex Pharmaceuticals Inc., in which it is getting $40 million up front plus up to $2.2 billion more in milestones and royalties on product sales.
The dramatic increase in COVID-19 infections in Europe and the U.S., combined with information overload surrounding the lead up to the Nov. 3 contentious U.S. presidential election, was more than enough to send investors to the sidelines in October.
The CDC estimates that sickle cell disease affects well over 100,000 Americans, with the disease occurring most often in African Americans. September has been designated as National Sickle Cell Awareness month designed to focus attention on the ongoing research in this field and the need for new treatments.
While its mRNA COVID-19 vaccination effort gets the most attention these days, Moderna Inc. is also moving in other directions as the company will lead mRNA discovery programs drawn from its early stage pipeline in new collaborations with Vertex Pharmaceuticals Inc. and Italy’s Chiesi Farmaceutici SpA. These two deals highlight the “huge opportunity for mRNA beyond SARS-CoV-2 and vaccines,” Piper Sandler analysts wrote Sept. 16.
Second-quarter earnings reports, flowing fast in the last days of July, delivered a bevy of contrasting results, from A to V. Stymied by the pandemic, Aimmune Therapeutics Inc. booked exactly zero net sales during the quarter for its expected blockbuster peanut allergy treatment, Palforzia. Vertex Pharmaceuticals Inc. and Seattle Genetics Inc. beat top-line expectations, driven primarily by strong sales of new products.
Weeks after raising an oversubscribed $60 million series A, Affinia Therapeutics Inc., of Waltham, Mass., is collaborating with Vertex Pharmaceuticals Inc. in a far larger deal, one potentially worth more than $1.6 billion.
LONDON – Amphista Ltd. becomes the latest to join the growing band of proteolysis targeting chimera (Protac) specialists, arriving on the scene with a $7.5 million series A and what it says is a new approach to targeted protein degradation (TPD).
