Sickle cell disease
Vertex Pharmaceuticals commits $45 million upfront to leverage Enlaza Therapeutics’ War-Lock platform to create drug conjugates and T cell engagers for autoimmune diseases and gentler conditioning for sickle cell/beta thalassemia gene-editing therapy Casgevy.
Inclacumab, which Pfizer obtained in its 2022 acquisition of Global Blood Therapeutics, failed to significantly lower pain episodes in patients with sickle cell disease over the 48-week treatment period.
CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy Casgevy. While Vertex handles the market, CRISPR has been focused on its clinical program.
At J.P. Morgan, most biopharma executives expressed a neutral stance on the incoming administration, but just days later, President Trump issued multiple executive orders that concern the industry.
Along with its gene editing therapy Casgevy, Vertex is offering fertility preservation support for its patients—a program that the HHS claims violates anti-kickback statutes.
The company had said in October said that it was looking to license out its ex vivo editor renizgamglogene autogedtemcel to focus its resources on its in vivo platform.
Vertex unveiled long-term durability data for Casgevy, while Beam presented Phase I/II findings for its investigational base editor BEAM-101, building up to a BLA by late 2026.
The payment scheme will tie gene therapy payments to improvements in health outcomes—and could potentially boost the uptake of these sickle cell disease treatments.
Bluebird has just two quarters until it’s out of cash. Executives are looking for financing to extend that runway to a projected breakeven point before the end of 2025, with analysts worried they won’t make it.
BEAM-101 seems to be competitive with approved sickle cell treatments, William Blair analysts said in a note to investors, but a patient death underscores the need for less-toxic preconditioning treatments.
PRESS RELEASES