Keyword: Vertex Pharmaceuticals
The data, from two patients with severe blood disorders, are "promising” for what could be a one-and-done treatment, Cantor Fitzgerald analysts wrote.
Carmen Bozic, M.D., Vertex's new head of global medicines development and medical affairs, will take over the CMO post April 1.
Looking to harness the body’s protein degradation processes, Kymera has raised more than $150 million for its pipeline of small molecules, led by a program targeting IRAK4, a protein involved in disease-signaling pathways.
Vertex Pharmaceuticals is jumping into diabetes with a $950 million acquisition of Semma Therapeutics and its stem cell-based diabetes treatments.
Though Editas and Allergan are not the first to test a CRISPR-based medicine in humans, their treatment is the first that edits DNA within the body.
Vertex taps CMO Kewalramani as newest CEO; Verb Surgical brings SurgiQuest founder on board; Disarm names Enzyvant head Shih to leadership role.
The question over who is really responsible for the costly creation of drugs has arisen in the U.K.
The outlay will give Vertex owenership of Exonics and expand its deal with CRISPR, setting it up to use gene editing to treat DMD and DM1.
The early-stage deal features more than $1 billion in milestones tied to the success of six programs.
Though biopharmas seem to be using their R&D dollars more effectively, they are only converting 24% of new molecular entities to approved drugs.