Fulcrum Therapeutics' promising treatment for Sickle Cell Disease and their strong financial position, despite setbacks.

With the addition of Casgevy, Children’s Hospital Los Angeles now offers two gene therapies for sickle cell, the first being Lyfgenia.

President Trump signed an executive order on the day of his inauguration that created doubts about the future of a program ...

Immvention Therapeutix is partnering with Novo Nordisk to develop oral treatments for sickle cell disease (SCD) and other ...

Discover the latest addition to Children's Hospital Los Angeles' 10 cell and gene therapy treatments, including Casgevy for sickle cell disease ...

In 2023, Vertex earned approval for Casgevy, a gene-editing treatment for two rare blood-related conditions with very few treatment options. Vertex Pharmaceuticals didn't create Casgevy itself ...

The company is also making progress with Casgevy, its gene therapy treatment. While the launch is in its early stages, with over 50 treatment centers activated globally and more than 50 patients ...

Secondly, McKesson’s dedicated business unit, InspiroGene, partnered with Vertex Pharmaceuticals to expand distribution options for CASGEVY, the first CRISPR-based gene-editing therapy approved ...

Vertex and partner CRISPR Therapeutics’ CRSP one-shot gene therapy Casgevy was approved for two blood disorders, sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), in ...

For example, the FDA-approved CRISPR-Cas9-based therapy Casgevy, designed to treat sickle cell disease, provides life-changing potential but comes at a staggering one-time cost of $2.2 million.