Vertex Pharmaceuticals Inc. and Entrada Therapeutics Inc. have entered into a global collaboration focused on discovering and developing intracellular Endosomal Escape Vehicle (EEV) therapeutics for myotonic dystrophy type 1 (DM1).
An uptick in positive clinical data reported in recent months has helped nudge the BioWorld Biopharmaceutical Index (BBI) to its highest point this year. BBI is up by 15.58%, significantly higher than the 1.36% increase recorded at the end of August, just as statistically significant clinical results began to roll in from a number of biopharma companies.
CRISPR-based cell therapies continued to gain steam Sept. 27 with the announcements of a potentially valuable big pharma collaboration and an ambitious global regulatory push.
Verve Therapeutics Inc. has packed a lot into the past few weeks. The latest is a four-year research deal with Vertex Pharmaceuticals Inc. to find and develop an in vivo gene editing program for an undisclosed liver disease. Vertex will pick up the tab for program costs as Verve does the preclinical R&D. Verve is getting an up-front $60 million from Vertex, along with a $35 million equity investment.
At the European Hematology Association's annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
At the European Hematology Association’s annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
While full details of the underlying science are due to be published in a peer reviewed journal, Kither Biotech Srl has announced its closure of an €18.5 million (US$20.4 million) series B financing with which it aims to translate its approach to rare respiratory diseases into the clinic.
Jonathan Moore, who was employee No. 36 when he came to Vertex Inc. in 1990 and ended up spending the next 28 years at the company, realized he wasn’t done with his work in ATP-binding cassette (ABC) transporters and has now formed Rectify Pharmaceuticals Inc. The new company just closed on a $100 million series A financing to fund development of a pipeline of therapies to restore ABC transporter function for treating genetic diseases.
Facing inevitable challenges ahead beyond peak performance for its wildly successful cystic fibrosis franchise, Vertex Pharmaceuticals Inc. is continuing to look to the future, announcing Aug. 24 it licensed rights to CRISPR gene-editing technology from Arbor Biotechnologies Inc. Co-founded by Feng Zhang and David Walt, Arbor's tech could figure into new Vertex cell therapies for diabetes, hemoglobinopathies and other diseases.
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.
