Tag Archive for: sickle cell disease

While Pfizer has ended one of its two Phase III studies for inclacumab in sickle cell disease, the company is still eyeing an approval for the antibody in the inherited blood disorder by 2026.

Bluebird CFO Chris Krawtschuk in a statement said that the debt financing agreement with Hercules “underscores the value bluebird offers as a standalone gene therapy leader,” while also extending the biotech’s runway and “bolstering our ability to bring transformative treatments to patients and their families.”

While gene therapies for treating sickle cell disease are promising, they will only be cost effective in the U.S. if priced below $2 million, according to a new modeling analysis.

A report back on ASH 2023, where innovation was on show everywhere.

Patients treated with the investigational sickle cell therapy saw an increase in hemoglobin levels and improved red blood cell parameters.

These factors, along with bluebird bio’s failure to receive a priority review voucher, all hurt the company’s stock price following Lyfgenia’s approval.

The agency approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology.

Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020, Britain’s Medicines and Healthcare products Regulatory Agency (MHRA) said.

The regulator’s advisory committee on Tuesday found Vertex Pharmaceuticals and CRISPR Therapeutics’ off-target analysis for its sickle cell disease candidate exa-cel to be sufficient.

The agency’s staff reviewers said it was not clear if the limited donor cells used for assessment were adequate to evaluate the potential safety risks of the therapy.