Vertex Pharmaceuticals Inc. brought in $4.16 billion in product revenues in 2019, bolstered in large part by the late October approval of Trikafta, earning $420 million in the few weeks that remained in the year.
Delegates convening in San Francisco Monday for the 38th Annual J.P. Morgan Healthcare Conference and other related biotechnology conferences running at the same time will certainly be in a better frame of mind than just 12 months ago. Back then, the sector had just come off a terrible fourth quarter, with investors shying away from biopharma company equities big time.
Proteostasis Therapeutics Inc. CEO Meenu Chhabra pointed to a “new reality in cystic fibrosis [CF] research” faced by her firm and others: the challenge of enrolling the best patients.
It has been a productive year for cell and gene therapy companies, and every week the sector never fails to generate exciting news flow to keep investors engaged. Given these ongoing developments, it is not surprising that companies involved in the area continue to attract financing and strategic partners.
DUBLIN – Crispr Therapeutics AG has delivered what appears, so far at least, to be a safe, functional cure for the first patient enrolled in each of its phase I/II trials of lead CRISPR/Cas9 gene editing therapy CTX-001, in beta-thalassemia and in sickle cell disease.